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Few drugs exist that treat amyotrophic lateral sclerosis, a progressive disease that kills the nerve cells that allow patients to initiate and control muscle movement.

QurAlis, a Cambridge, MA-based startup, has an ambitious plan to develop a number of precision therapies for the disease based on forms of the condition identified by genetic mutation or a biomarker that CEO Kasper Roet (pictured) hopes to could one day, in combination, help most ALS patients.

Now the company has raised $42 million from investors in the US, Europe, and Japanmoney that will fund a move from the LabCentral incubator in Kendall Square to its own office, more than double the companys headcount by years end, and get at least one of its programs into human testing sometime next year.

The company is leveraging stem cell research from company co-founders Kevin Eggan and Clifford Woolf, Harvard University professors whoby harvesting normal skin cells from ALS patients and turning them into cells such as the motor neurons that damages as the disease progresseshave created models with the same DNA and gene mutations as those patients in an effort to identify new therapeutics for known ALS genes.

Mutations in more than 25 human genes have been implicated in ALS, the company says, and its strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient subgroups. Some of those genes are also believed to cause frontotemporal dementia, a common cause of dementia that QurAlis also plans to treat.

One program QurAlis is advancing is intended for patients whose neurons are damaged and killed by the overactivation of certain receptors for glutamate, a key neurotransmitter, in a process known as excitotoxicity.

The company is also working on a treatment intended to return the autophagy process, through which cells recycle unwanted or damage components, to normal functioning. To do so, QurAlis is looking to target the enzyme TBK1, which plays a key role.

Roet, in an interview, said the company views its strategy as analogous to that pursued by Bostons Vertex Pharmaceuticals (NASDAQ: VRTX), which has developed multiple drugs for forms of cystic fibrosis (CF) caused by certain mutations, and late last year received approval for a combination of those drugs for about 90 percent of all CF patients.

We have identified ALS as a disease that we think we understand now, at least for specific subgroups of patients, he said. We understand what is driving the disease and we are able to develop very specific therapies for those patients.

Eggan, Woolf, Roet, and Jonathan Fleming launched QurAlis just over two years ago with seed funding from investors including MP Healthcare Venture Management, the investment arm of Mitsubishi Tanabe Pharma; the investment arm of Amgen (NASDAQ: AMGN); and Alexandria Venture Investments. Mitsubishi Tanabe markets edaravone (Radicava), one of four FDA-approved treatments for ALS. The FDAs 2017 nod for the drug made it the only ALS therapy OKd in the past 20 years.

The Cambridge, MA-based company said the new capital, a Series A financing round, brings the total it has raised to $50.5 million. The investment was led by LS Polaris Innovation Fund, Mission BioCapital, Dutch firm Inkef Capital, and the Dementia Discovery Fund. New investors including Droia Ventures, which operates from Luxembourg and Belgium, Mitsui Global Investment, and Dolby Family Ventures also participated, as did earlier investors including Amgen, MP Healthcare, and Sanford Biosciences.

As part of the deal, LS Polariss Amy Schulman, Inkef Capitals Roel Bulthuis, Dementia Discovery Funds Jonathan Behr, and Droia Ventures Luc Dochez join Mission BioCapitals Johannes Fruehauf on the QurAlis board.

Earlier this year some of the same investors, including Amgen and Dolby Family Ventures, backed a Series A financing for EnClear Therapies, a spinout of QurAlis. That company raised $10 million to advance the development of a dialysis-like medical device designed to filter out harmful proteins in the cerebral spinal fluid of patients with neurodegenerative diseases.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing - Xconomy

Skin Stem Cells Comments Off on QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing – Xconomy | May 13th, 2020

Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003

Mont-Saint Guibert, Belgium, 13 May, 2020: Novadip Biosciences (Novadip or the Company), a clinical-stage biopharmaceutical company leveraging its proprietary tissue regeneration technology platform, today announces positive data from the interim analysis of its Phase I/IIa clinical trial for autologous NVD-003 in adults with non-healing fracture of the lower limb.

NVD-003 is a novel autologous cell-based osteogenic (bone healing/[bone forming]) product that has been generated from Novadips proprietary 3M tissue regeneration platform. This platform is aimed at healing damaged tissues by restoring their natural physiology and consists of a 3-dimensional, scaffold-free extracellular matrix (ECM), utilizing differentiated adipose-derived stem cells (ASCs) to restore the physiology of natural healing. NVD003 presents as a scaffold-free 3D implant to fill critical-size bone defects where healing is compromised.

This phase I/II study is investigating in five European centers the potential of NVD-003 to promote bone union in nine adults with a non-healing fracture of the lower limb. There was 100% manufacturing success for NVD-003 and grafting surgery was completed successfully in all patients without deviating from standard medical practice. To date, with a median of 12 months post-treatment, no NVD003 related safety signal has been reported. Further exploratory analysis performed on data from the first five patients to complete a six month follow up showed a strong positive trend in radiological healing with confirmed bone formation for all patients and radiologically confirmed union for three of the patients.

Prof. Gunnar Anderson (MD, PhD), Professor and Chairman Emeritus of the Department of Orthopedic Surgery at Rush University Medical Center, Chicago, Illinois and Chairman of the Scientific Advisory Board commented: The early results of this study are remarkable both clinically and for the patients and we look forward to replicating these in a larger group in the future. It is hugely encouraging that we may potentially have a future solution for these patients with unmet needs.

Dr. Denis Dufrane (MD, PhD), Chief Executive Officer, Chief Scientific Officer commented: We are encouraged by the data from this interim analysis, which demonstrates the potential of our tissue regeneration 3M3 platform to restore natural healing processes in patients with reputedly difficult to treat bone defects. We look forward to further progressing NVD-003s clinical program in bone non-union and in patients with other similar conditions with no effective treatment option and hope to provide full study results in 2025.

NVD-003 is also in clinical stage for congenital pseudarthrosis of the tibia (CPT), a rare and disabling pediatric condition with very limited treatment options and has demonstrated clinical proof-of-concept in case studies.

Novadips tissue regeneration platform drives several new classes of product candidates with an initial focus on autologous cell therapies for critical size tissue reconstruction. Allogeneic therapeutics are in development for prevalent and complex tissue defects for bone and skin tissue and exosomes/miRNA-based therapeutics are being developed for immediate (off-the-shelf) clinical use.

- End -

Notes to editors

Novadip Biosciences

Novadip Biosciences is a clinical stage biopharmaceutical company leveraging its unique 3D tissue regeneration technology platform to generate multiple product candidates to address hard and soft tissue reconstruction for patients who have limited or no treatment options. The companys proprietary 3M3 platform is a 3-dimensional, extracellular matrix that utilizes adipose-derived stem cells to deliver highly-specific growth factors and miRNAs to mimic the physiology of natural healing and creates a range of products that address specific challenges in tissue regeneration. Novadips initial focus is on critical size bone reconstruction. The company is also applying its 3M3 platform to develop truly novel off-the-shelf/allogeneic therapies to address more prevalent tissue defects and miRNA/exosome products for broader indications. For more information, visit http://www.novadip.com .

For further information, please contact:

Novadip Biosciences

Denis Dufrane

Chief Executive Officer, Chief Scientific Officer

+32 (10) 779 220

info@novadip.com

For media enquiries:

Consilium Strategic Communications

Chris Gardner, Matthew Neal, Angela Gray

+44 (0) 20 3709 5700

novadip@consilium-comms.com

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Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003 - Yahoo Finance UK

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Carlos R. Bachier, MD

Chimeric antigen receptor (CAR) T-cell therapies quickly burst into the spotlight of hematology-oncology disease management because of their potential to illicit deep and durable responses from patients whose disease is relapsed or refractory to multiple previous lines of therapy. Relevant professional meetings and oncology publications exploded with research and news about CAR T cells, and this cellular therapy strategy is now being explored across hematologic and solid malignancies.

CAR T cells are a scientific revolution, Tania Jain, MBBS, assistant professor of oncology at Johns Hopkins University in Baltimore, Maryland, said in an interview with Targeted Therapies in Oncology (TTO). They have brought about a paradigm shift in terms of how were treating patients.

The 2 currently FDA-approved CAR T-cell therapies, axicabtagene ciloleucel (Yescarta) and tisagenlecleucel (Kymriah), are both indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma; additionally, tisagenlecleucel is approved for patients up to 25 years with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).1-3 With a second wave of approvals likely on the horizon for therapies such as lisocabtagene maraleucel (liso-cel) and idecabtagene vicleucel (bb2121), CAR T is gaining traction and will likely play an increasingly prominent role in the future treatment paradigm in oncology.

CAR T-cell therapy administration is largely limited to the inpatient setting at both academic institutions and large accredited cancer centers, making such treatments unavailable to most patients. Other challenges with this type of therapy include its potential to cause serious toxicities resulting in organ damage and death.4

David G. Maloney, MD, PhD

Due to the promising efficacy of these agents, investigators have been working toward viable solutions to bring CAR T-cell therapies to more patients by alleviating difficulties associated with therapy delivery and patient care.

CAR T-cell therapies, both those currently approved and the many being explored in late-phase clinical trials, are produced from autologous T cells obtained from the patient receiving therapy. This personalization has led to tremendous success, yet it is a large part of why CAR T-cell therapy use remains limited to a select group of patients.

Time is an important consideration for patients who have experienced multiple relapses and may be too weakened by numerous lines of prior therapy to wait several weeks for the CAR T-cell manufacturing process. The effects of previous treatments or the disease itself can also present challenges, as manypatients are rendered lymphopenic and may be unable to produce enough T cells for harvesting. Roadblocks may remain for patients who are not limited by these factors; manufacturing success and effectiveness of the CAR T-cell product can be negatively influenced by disease-related dysfunctions of patients T cells.4

A new option, off-the-shelf CAR T-cell products, may help solve these problems. These premade products are manufactured using allogeneic donor cells (instead of autologous cells from the patient), and they present immediate advantages to clinicians, such as immediate availability, opportunity for product standardization, and decreased cost.5

The advantages [include] being able to access the cellular therapy in real time, as opposed to autologous products that havetobe manufactured,Craig S. Sauter, MD, clinical director of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center in New York, New York, explained in an interview withTTO. This is particularly important for patients who are not responding to therapy, which is a current requirement for treatment with CAR T cells, he added.

Findings from a phase I trial (NCT01430390) in patients with relapsed or refractory B-cell malignancies showed that patients with non-Hodgkin lymphoma (NHL) experienced durable responses with an Epstein-Barr virusspecific cytotoxic lymphocyte CAR product derived from cells harvested from third-party donors (rather than from their more precisely matched stem cell donors). All 4 patients with NHL and a single patient with chronic lymphocytic leukemia, who were treated with third-party cells, remained disease free and alive at the time of analysis, with a median follow-up of over 2 years.6

The advantages [of this type of therapy are] that it eliminates the need for apheresis [and] shipping cellular products back and forth. [Instead, clinicians] have a pharmaceutical product on the shelf for access, Sauter, who was an author on the trial, said. Another notable product being investigated in clinical trials is UCART19, an allogeneic engineered anti CD19CAR T-cell product, which is being evaluated in the phase I CALM trial in adult patients with relapsed or refractory B-cell ALL (NCT02746952) and in the phase I PALL trial of pediatric patients with relapsed or refractory CD19-positive B-cell ALL (NCT02808442). Other off-the-shelf agents are described in theTABLE.5

Issues with inpatient CAR T-cell therapy administrationinclude high demands on health care resources and strain on patients and their families. Moving treatment to the outpatient setting has the potential to reduce this strain; however,clinicians taking over care of patients receiving CAR T-cell therapy must be prepared with the proper resources to identify and manage adverse events associated with therapy.

One of the most notable risks to patients receiving CAR T-cell therapy is cytokine release syndrome (CRS), a systemic inflammatory response that is characterized by increased serum levels of inflammatory cytokines, fever, hypotension, hypoxia, and organ dysfunction.4 [CAR T] can also lead to neurological events and can cause confusion and, in some patients, seizures,Carlos R. Bachier, MD, Director of Cellular Research at Sarah Cannon Cancer Center in Nashville, Tennessee, explained in an interview with TTO.

Regardless of the infusion setting, patients require close monitoring in the hours and days following therapy administration. A review byLucrecia Yez, PhD, MS, and colleagues stated that key criteria for treating patients in the outpatient setting include an educated caregiver and necessary infrastructure allowing for outpatient visits plus adequate emergency and intensive care unit (ICU) access. Patients followed as outpatients must be given twice-daily temperature checks for a minimum of 14 days following treatment and preferably extending up to 3 to 4 weeks following infusion. Anysigns of back pain, skin rash, dizziness, chills, shortness of breath, chest pain, tachycardia, or neurological events that may indicate neurotoxicity or signs of CRS must be reported immediately so treatment can begin as quickly as possible.7

Because of the risk of CRS and neurotoxicity, both FDA-approved agents are restricted under the Risk Evaluation and Mitigation Strategy, an FDA-mandated program that builds in caution for use of agents with serious safety concerns.8,9 Therefore, 2 doses of tocilizumab (Actemra), an interleukin (IL)-6 receptor antagonistthat was approved in 2017 for management of CRS associated with CAR T-cell therapy,1,4 should be on hand for each patient before the infusion of CAR T cells. Steroids have also demonstrated efficacy against CRS, but concernssurrounding CAR T-cell suppression with these agents have established them as a second-line choice after tociluzumab.9

Immune effector cellassociated neurotoxicity syndrome (ICANS) is a group of neurologic symptoms associated with treatments such as CAR T-cell therapy. Predisposing factors include younger age, higher tumor burden, high levels of pretreatment inflammation, and history of early or high-grade CRS. Treatments for complications of ICANS vary. Some centers may prescribe prophylactic antiepileptic medications, such as levetiracetam, to prevent seizures in patients with grade 2 or higher neurologic events. AntiIL-6 therapy can be considered in patients with concurrent CRS, but corticosteroids are the preferred regimen in those with neurotoxicity alone.9

In February of this year, the investigational CAR T-cell product liso-cel was granted priority review by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma who had undergone at least 2 prior therapies.10 Investigators believe that liso-cel therapy may have a place in a broad range of patients and in the outpatient setting.11

It turns out liso-cel has a low incidence of [CRS and ICANS], and they occurred relatively late compared with other products, said Bachier. Because of this low incidence, the strategy was to deliver liso-cel in an outpatient setting.

The feasibility of liso-cel administration on an outpatient basiswas evaluatedby Bachier and colleagues, and the results were presented at the 2020 Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood & Marrow Transplant Research, held February 19 to 23, 2020, in Orlando, Florida.12

The authors analyzed data from 3 clinical trials of liso-cel, with a focus on the subset of participants who were treated as outpatients. The included trials were the phase I TRANSCEND-NHL-001 (NCT02631044) and phase II OUTREACH (NCT03744676) trials in patientswhohadundergone at least 2 lines of prior treatment, as well as the PILOT study (NCT03483103), which examined liso-cel as second-line therapy in patients who were ineligible for autologous hematopoietic stem cell transplant because of age, organ function, or ECOG performance score. All 3 studies allowed outpatienttreatment, with some patients receiving their therapy in the nonuniversity setting.

This clinical trial included sites that were not a part of a university but had experience treating patients for stem cell transplant, Bachier said. Some of these sites that participated were notyour traditional university centers that had traditionally been involved in the development of these therapies.

Much caution was required in order to maximize patient safety and treatment efficacy. The approach of doing CAR T-cell therapy, in general, in the outpatient setting requires a robust clinical ability of the centers, said coauthor David G. Maloney, MD, PhD, medical director of Cellular Immunotherapy at the Immunotherapy Integrated Research Center of Fred Hutchinson Cancer Research Center in Seattle, Washington, in an interview. We were able to get people safely to the hospital, and it was rare that you would have to do escalation of care when people were admitted. Most of the time, patients could bemanagedand wereout of the ICU, withrare exceptions. But again, you still have to have the wherewithal to get patients to the ICU pot entially for aggressive care if needed.

Results of the analysis of outpatient data from the 3 trials showed that rates of toxicity and response were similar to those previously reported for the entire patient cohort (both inpatients and outpatients) of the TRANSCEND-NHL-001 trial.

Based on these results, the indication is that you can deliver [liso-cel] in the outpatient setting and the outcomes are good compared with those treated in the inpatient setting, said Bachier. Aside from that, it also showed that liso-cel could be safely administered outside of university programs and in more community-based programs, most of them being aligned [with] or part of stem cell and bone marrow transplant programs.

When planning or setting up a CAR T-cell therapy outpatient program, investigators anticipate possible barriers to successfultreatment. The greatest barrier, according to Bachier, is access to physicians and staff who are knowledgeable and trained to manage toxicities related to CART-cell therapy. These therapies still should not, in my opinion, be delivered [by clinicians in] community centers that do not have the expertise to deliver the therapies safely, he said.

Maloney added that centers should be required to have the ability to triage patients 24/7 and allow for patients to be directly admitted to the hospital if needed. In the case of the analysis of outpatient data from the 3 liso-cel trials however, he said, We found that around 30% to 40% of patients did not actually ever require hospitalization, whichis quite interesting. Most of the 60% to 70% of patients who were hospitalized were admitted for fever, he added.

In addition, sites must gain accreditation and approval, Jain pointed out.

Every center that intends to do CAR T-cell therapy is first approved by each of the companies [that manufacturethese agents], Jain said. The centers also have to be approved by FACT [Foundation for the Accreditation of Cellular Therapy], which is the same organization that approves centers for allogeneic stem cell transplant. These are some of the largest things that a center needs to go through, which takes care of things like developing standard practices and other guidelines to make sure that these [therapies] are used safely and appropriately.

As investigators and oncologists explore the feasibility of moving CAR T-cell therapy into more settings, 2 questions arise: What settings have on this therapy?

What type of training and skills do clinicians need? Like other clinicians, Sauter has concerns about new allogeneic cellular therapies,andhe hopes future research will focus on mitigating these challenges. The concern would be that these are not autologous products and there is the risk of rejection from the host immune system, he said. Strategies to circumnavigate that risk are at the forefront of investigationin off-the-shelf CAR T cells.

The research is not stopping with CAR T-cell therapy,though. Were seeing a lot of new molecules coming in that will be challenging the roles of CAR T cells, [such as] specific antibodies, which may even work in cases of CAR T-cell failure, Maloney said. We are still learning how to make those more effective and safer.

References:

1. FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine releasesyndrome.FDAwebsite.PublishedAugust30,2017.AccessedApril14, 2020. bit.ly/2RC4eQ8

2. FDA approves axicabtagene ciloleucel for large B-cell lymphoma. FDA website. Published October 18, 2017. Accessed April 14, 2020. bit.ly/2yYIQOp

3. FDA approves tisagenlecleucel for adults with relapsed or refractory large B-cell lymphoma. FDA website. Published May 1, 2018. Accessed April 14, 2020. bit.ly/34zPoi8

4. Rafiq S, Hackett CS, Brentjens RJ. Engineering strategies to overcome the current roadblocks in CAR T cell therapy. Nat Rev Clin Oncol. 2020;17(3):147167. doi: 10.1038/s41571-019-0297-y

5. DepilS,DuchateauP,GruppSA,MuftiG,PoirotL.Off-the-shelfallogeneic CAR T cells: development and challenges. Nat Rev Drug Discov. 2020;19(3):185199. doi: 10.1038/s41573-019-0051-2

6. Curran KJ, Sauter CS, Kernan CS, et al. Durable remission following off-theshelf chimeric antigen receptor (CAR) T-cells in patients with relapse/refractory (R/R) B-cell malignancies. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 120. bit.ly/2ufDYCu

7. Yez L, Snchez-Escamilla M, Perales MA. CAR T cell toxicity: current managementandfuturedirections. Hemasphere.2019;3(2):e186.doi:10.1097/ HS9.0000000000000186

8. Risk evaluation and mitigation strategies | REMS. FDA website. Updated August 8, 2019. Accessed April 14, 2020. bit.ly/2ykhLVt

9. JainT,BarM,KansagraAJ,etal.UseofchimericantigenreceptorTcell therapy in clinical practice for relapsed/refractory aggressive B cell non-Hodgkin lymphoma: an expert panel opinion from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant. 2019;25(12):2305-2321. doi: 10.1016/j.bbmt.2019.08.015

10. U.S. Food and Drug Administration (FDA) accepts for Priority Review Bristol-Myers Squibbs Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. News release. Bristol-Myers Squibb; February 12, 2020. Accessed April 15, 2020. bit.ly/37ruQbs

11. Helwick C. Strong activity shown for lisocabtagene maraleucel CAR T-cell therapy in aggressive large B-cell lymphoma. ASCO Post website. Published February 25, 2020. Accessed April 15, 2020. bit.ly/3eoD0pT

12. Bachier CR, Palomba ML, Abramson JA, et al. Outpatient treatment with lisocabtagene maraleucel (liso-cel) in 3 ongoing clinical studies in relapsed/refractory (R/R) large B cell non-Hodgkin lymphoma (NHL), including second-line transplant noneligible (TNE) patients: TRANSCEND NHL 001, OUTREACH, and PILOT. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, Orlando, FL. Abstract 29. bit.ly/37I7DC9

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Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings - Targeted Oncology

Skin Stem Cells Comments Off on Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings – Targeted Oncology | May 13th, 2020

Families stuck at home during lockdown are being warned about a common but dangerous garden weed which can leave children - and adults - with burns and blisters.

Giant Hogweed has been dubbed Britain's most dangerous plant because of the horrific burns it inflicts on anyone who touches it - especially children - its set to thrive in the weeks ahead thanks to recent weather conditions.

The plant grows wild as well as in gardens and is becoming common the in the UK, but when it comes into contact with skin it causes a painful blistering rash.

The recent warm weather and plenty of rain after a mild winter has created the perfect conditions for this hazardous plant to thrive.

The hogweed looks relatively attractive and is part of the carrot family, but contains toxic chemicals.

Giant hogweed, or Heracleum mantegazzianum, is a weed which has dangerous effects on human health.

Growing up to five metres tall, its sap contains toxic chemicals which react with light when in contact with human skin, causing blistering within 48 hours.

Effectively it prevents the skin from protecting itself from sunlight, which can lead to very bad sunburn and scarring.

It's actually pretty and looks a bit like cow parsley. It's got green stem spotted with dark red which varies from 38 cm in diameter. Each dark red spot on the stem surrounds a hair, and large, coarse white hairs occur at the base of the leaf stalk.

It produces white flowers clustered in an umbrella-shaped head that is up to 80 cm in diameter across its flat top.

Colette Jones, Chairwoman of Friends of Close Park where Giant Hogweed was spotted, told The Bolton News : "Children are drawn to them because they grow so tall. They break them off to use them as sticks not realising how dangerous they are."

Exposure can result in blisters, long-lasting scars, and - if it comes in contact with eyes - blindness.

The blisters will form within 48 hours - scars can last for years.

It can also cause cause long-term sunlight sensitivity in people who touch it.

Black or purplish scars may be left on your skin for years after.

Medical professionals say you should cover the affected area, and wash it with soap and water.

The blisters heal very slowly and can develop into phytophotodermatitis, a type of skin rash which flares up in sunlight.

If you feel unwell or have a severe reaction you are advised to see a doctor.

Giant hogweed was among the foreign plants introduced to Britain in the 19th century as an ornamental plant, but it's now widespread throughout the British Isles.

It's invasive, which means that it chokes off other plants and can reduce wildlife in an area.

The plant is native to the Caucasus region and Central Asia.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild.

It is found in most of the UK, along footpaths and riverbanks though it also grows in places like parks, cemeteries and wasteland.

The sap of giant hogweed has chemicals which are toxic to humans and cause photosensitivity. The sap is phototoxic and can cause phytophotodermatitis.

When they touch skin, they effectively remove any protection against the sunlight causing severe skin inflammations.

Children have been hospitalised and suffered third-degree burns to their skin before.

The severe reaction to the plant is caused by the presence of linear derivates of furanocomarin in the plant's leaves, seeds, flowers, stems and roots.

The chemicals enter the cells' nucleus forming bonds with DNA and cause cells to die.

The RHS advises caution when removing the plant - cover arms and legs, and ideally wear a face mask when working on it.

Cut plant debris, contaminated clothing and tools are potentially hazardous too.

Wash any skin that comes in contact with the plant immediately.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild. Giant hogweed clearances are carried out to remove the plant.

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Warning over common garden weed that causes horrific burns - Cambridgeshire Live

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Repeated setbacks aside, little Capricor has suggested it has generated some long-term data to support its pursuit to garner approval for its stem cell therapy for Duchenne muscular dystrophy, although some of the data appeared to underwhelmed investors.

The data from the small, placebo-controlled mid-stage study, HOPE-2, tracked the effects of the companys stem cell therapy CAP-1002, which is designed to temper the inflammation associated with DMD, in 8 boys and young men who are in advanced stages of DMD. The remaining 12 enrolled patients received the placebo.

The main goal of the study was a measure that evaluates shoulder, arm and hand strength in patients who are generally non-ambulant (performance of the upper limb (PUL) 2.0), as suggested by the FDA, Capricor said. It is one of several ways Capricor quantified skeletal muscle improvement in the trial.

The intravenous infusion of CAP-1002, given every 3 months, induced a statistically meaningful improvement of 2.4 points (p=0.05) versus the placebo group, in which patient declines were consistent with natural history data. However, on another measure of upper limb function, the trend was in favor of the Capricor drug, but did not hit statistical significance.

The companys shares $CAPR were down nearly 13% to $6.89 in morning trading.

Click on the image to see the full-sized version

Meanwhile, there were also some encouraging data on cardiac function the genetic condition is characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles.

As reflected above, CAP-1002 elicited an improvement across different measures of cardiac function, although the effect was not always statistically significant. In particular, the drug also caused a reduction in the levels of the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage.

Armed with these data and an RMAT and orphan drug designation from the FDA, Capricor is now hoping to eke out a plan with the FDA for marketing approval.

LA-based Capricor initially set out to test the potential of technology that Eduardo Marbn, CEO Linda Marbns husband, developed at Johns Hopkins. But repeated setbacks clobbered the company, which in 2014 traded north of $14 a share. In 2017, J&J walked away from a collaboration on a stem cell therapy for damaged hearts after it flopped in the clinic.

In late 2018, the company voluntarily halted a DMD clinical trial, following a severe allergic reaction that occurred during infusion. In February 2019, the company said it is exploring strategic alternatives for one or more of its products and cutting 21 jobs to keep financially afloat, but had resumed dosing in its DMD trial.

The first batch of positive data on CAP-1002, which consists of progenitor cells derived from donor hearts and is designed to exude exosomes that initiate muscle repair by suppressing inflammation and driving immunomodulation, came last July when the company announced the drug had generated a positive effect at the interim analysis juncture of HOPE-2. Capricor is now working on to flexing its therapeutic muscle with CAP-1002 to fight the Covid-19 pandemic.

DMD is a rare muscle-wasting disease caused by the absence of dystrophin, a protein that helps keep muscle cells intact. It disproportionately affects boys and affects roughly 6,000 in the United States.

Patients are essentially treated with steroids. Sarepta Therapeutics now has two exon-skipping drugs designed to treat certain subsets of the disease, although the magnitude of their effect is controversial given that approvals were not based on placebo-controlled data. Meanwhile, Sarepta and others are also pursuing one-time cures in the form of gene therapies to replace the missing dystrophin gene in patients.

Social: Linda Marbn, Capricor CEO (Twitter)

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One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data - Endpoints News

Cardiac Stem Cells Comments Off on One year on, Capricor’s stem cell therapy appears to help DMD patients in small study, but investors balk at the data – Endpoints News | May 13th, 2020

The Cell and Gene Therapy Catapult (CGT Catapult) and Kyoto, Japan-based CiRA Foundation are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation.

With the move, the groups are hoping to further the application of iPS cell technologies for the manufacture of regenerative medicine products.

The potential of distinct iPS cell lines for differentiation into specific cell types is usually biased towards some cell line-specificity which, the parties note, is very difficult to predict. As such, in order to select an appropriate iPS cell line for clinical trials it is necessary to differentiate several candidate cell lines, which is time-consuming.

CGT Catapult and CiRA plan to explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.

We are honoured to collaborate with CiRA Foundation, an organisation with world-leading capabilities in iPS cell technology, and to be the first group to utilise CiRAs innovative iPS cell lines outside of Japan, said CGT's chief executive Matthew Durdy

This is a truly exciting project to help further the application and manufacture of iPS cells into cell therapies. We look forward to progressing this promising research together, which has potential benefits for the global advanced therapies industry.

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Cell and Gene Therapy Catapult links with Japan's CiRA Foundation - PharmaTimes

IPS Cell Therapy Comments Off on Cell and Gene Therapy Catapult links with Japan’s CiRA Foundation – PharmaTimes | May 13th, 2020

Washington D.C: Already known for their shape-shifting abilities, stem cells can now add "death-defying" to their list of remarkable qualities, suggests a novel study.

The new study shows how stem cells, which can contribute to creating many parts of the body, not just one organ or body part, are able to postpone their own death in order to respond to an injury that needs their attention.

The study was done in planarians, which are tiny worms used as model organisms to study regeneration because of their ability to recover from any injury using stem cells.

"Planarian stem cells, even when challenged and under a lot of duress, will still respond to an injury by delaying death," said Divya Shiroor, first author and a graduate student in Dr Carolyn Adler's lab, in the College of Veterinary Medicine.

This could have important implications for cancer research and therapies, particularly when examining chemotherapy and surgery options for patients.

"By understanding how injury prompts planarian stem cells to withstand radiation. We hope to identify genes that, if shared with mammals, could perhaps help hone existing therapies," Shiroor said.

Planarians are commonly used in basic research because of their similarities to humans. Like humans, planarians have stem cells, similar organs and similar genes, but are much more adept at responding to injury, thanks to their higher volume of stem cells and lack of a developed immune system, which in humans complicates the healing process.

"This really simplifies the process of understanding the effects of both injury and radiation on stem cells, and allows us to study it directly without being hampered by parallel processes integral to wound healing, such as inflammation, that get simultaneously triggered in mammals," Shiroor said.

By uncovering the mechanisms that govern stem cells after wounding in a system like planarians, researchers could also apply this knowledge when engineering stem cells to respond similarly in the human body.

Labs have many ways to understand how planarians use stem cells to successfully recover and regenerate, but the Adler lab's combination of radiation and injury to identify a novel stem cell response is unique. The researchers plan on digging deeper to understand how the stressed stem cells know that there is an injury and what role other cells may play in their response.

"We have identified a key gene that is required for stem cell persistence after radiation and injury. and we plan on using this as a stepping stone for further exploration," Shiroor said.

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Researchers discover stem cells' 'death-defying' quality that aids healing - ETHealthworld.com

Skin Stem Cells Comments Off on Researchers discover stem cells’ ‘death-defying’ quality that aids healing – ETHealthworld.com | May 12th, 2020

A decade ago, scientists won the nobel prize for connecting the dots between telomeres and aging. Telomeres are the protective caps on the ends of the strands of DNA called chromosomes. Telomeres shorten every time a cell divides. When they become very short, they trigger cell crisis and cell death.

Since this discovery was made, researchers have found new insights into telomeres and their connection to wrinkled skin, as well as how their length might be extended.

Scientists have uncovered new information on telomeres that could help combat the effects of inflammation and aging. Researchers from the University of Pittsburg recently discovered how oxidative stress plays a critical in the link between telomeres and cancer. I dont want to blind you with science (telomeres are a complex area), but every breakthrough into telomeres will ultimately equal a breakthrough in understanding how we age and how we can keep cells including our skin cells healthy.

Telomeres are composed of repeated sequences of DNA. The results from this study suggest that the mechanism by which oxidative stress accelerates telomere shortening is by damaging the DNA precursor molecules, not the telomere itself. This will have a big impact on appreciating how to manage oxidative stress to prevent aging and diseases such as cancer.

At Stanford University School of Medicine, scientists claim they can now lengthen teleomeres. Skin cells with telomeres lengthened by the procedure were able to divide up to 40 more times than untreated cells (source).

However, longer teleomeres is not necessarily associated with a longer life (source). Indeed, those naysayers say that good diet and an exercise regimen will do that. Yet, it does seem that preventing further shortening of the telomeres could be beneficial, especially for aging skin. Telomeres are likened to the tips of shoelaces that stop them unraveling and so you really want to be thinking about how to stabilize your telomeres. Happily, advances in skin care can help you out. There are three approaches to consider: special ingredients that target telomeres, ingredients that prevent oxidative damage (known, of course, as antioxidants) and stem cells.

Target your telomeres

One very interesting ingredient is called astragalus, and although it is rare, we do have a couple of Truth In Aging finds with it. But first, what is it and how does it work? In 2008, a UCLA AIDS Institute study found that a chemical they called TAT2 from the astragalus root, which is frequently used in Chinese herbal therapy, can prevent or slow the progressive shortening of telomeres. It can be found in Prana Reishi Mushroom Shield ($42 in the shop) and ExPrtise Effective Anti-Aging Face Serum ($120 in the shop).

Another ingredient to look for is treprenone, also marketed under the name of Renovage. Its promise is to stabilize telomeres, so at the very least they won't shorten. Maintaining telomere length extends the Hayflick Limit (the rate at which cells turn over before conking out completely) by one third. Youll find treprenone (Renovage) in Your Best Face Correct ($150 in the shop) Your Best Face Boost ($65 in the shop.

Amp up with antioxidants

The research is clear: Preventing oxidative damage is the job of an antioxidant. Free radicals are charged chemical particles of oxygen that enter into destructive chemical bonds with organic substances such as proteins, as explained by Gerald Imber, MD. Antioxidants limit the production of free radicals and therefore help prevent oxidative stress. There are many sources in plants, and vitamins are also antioxidants.

There are 33 antioxidants in Skin 2 Skin Aging Intervention Cream ($73 in the shop), including the universal antioxidant alpha lipoic acid. The powerful antioxidants in Your Best Face Rescue ($145 in the shop) include spin trap, an advanced form of vitamin C and EGCG. Bee venom has some 18 active compounds include antioxidants, peptides with powerful anti-inflammatory actions, and enzymes. It is the key ingredient in LaCrme Beaut Luxurious Bee Venom Rich Face Treatment Cream ($202 in the shop).

Look for plant stem cells

Plant stem cells never age. British scientists found that plant stem cells were much more sensitive to DNA damage than other cells. Once they sense damage, they trigger death of these cells before it spreads and causes more. In addition, they have the ability to stimulate cell renewal and replace specific cells in need of repair.

Ao Skincare Raw Nourish AM Treatment ($65 in the shop) has the powerful antioxidant astaxanthin and plant stem cells.

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New Study Suggests the Consequences of Oxidative Stress on Telomeres - Truth In Aging

Skin Stem Cells Comments Off on New Study Suggests the Consequences of Oxidative Stress on Telomeres – Truth In Aging | May 12th, 2020

TEL AVIV, Israel, May 11, 2020 /PRNewswire/ -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of stem cells, today announced the publication of an article in Bone Marrow Transplantation, a peer-reviewed medical journal )member of the Nature publishing house) covering transplantation of bone marrow in humans and published monthly by the prestigious Nature Research, entitled 'Ex-vivo FAS-ligand to Improve Allograft Safety'. The article is co-authored by researchers at Cellect and its academic partners.

The paper highlights the pre-clinical research and demonstrates that engraftment is robust following transplantation of treated graft, and the graft retains its immune reconstitution and anti-leukemic effects. The Company has initiated a Phase 1/2 study in adults undergoing stem cell transplant for the treatment of hematological malignancies. The primary endpoint of the study is to evaluate the overall incidence, frequency, and severity of adverse events potentially related to ApoGraft at 180-days post-transplant. All patients transplanted through present time using the ApoGraft process were engrafted and time to engraftment was similar to the standard of care. To date, there have not been any safety and tolerability concerns during the study and patient enrollment is continuing. Both, the principal investigator (PI) and independent data safety monitoring board (DSMB) agree that no serious adverse events (SAEs) reported during the course of the study were related to the ApoGraft process.

The data from the pre-clinical research, and published in this paper, was included in the Company's Investigational New Drug (IND) application, which was approved by the U.S. Food and Drug Administration in late 2019. The Company has received all the necessary approvals to initiate the trial with its academic partner, Washington University, and plans to begin patient recruitment once the COVID-19 pandemic is mitigated and clinics can resume normal practices.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (NASDAQ: APOP) has developed a breakthrough technology, for the selection of stem cells from any given tissue, that aims to improve a variety of stem cell-based therapies.

The Company's technology is expected to provide researchers, clinical community and pharma companies with the tools to rapidly isolate stem cells in quantity and quality allowing stem cell-based treatments and procedures in a wide variety of applications in regenerative medicine. The Company's current clinical trial is aimed at bone marrow transplantations in cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellect's expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company's history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company's ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Company's ability to obtain regulatory approvals; the Company's ability to obtain favorable pre-clinical and clinical trial results; the Company's technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company's clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company's ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company's industry; unforeseen scientific difficulties may develop with the Company's technology; the Company's ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Company's ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov, and in the Company's periodic filings with the SEC.

Contact

Cellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

Or

EVC Group LLCMichael Polyviou+732-933-2754mpolyviou@evcgroup.com

View original content:http://www.prnewswire.com/news-releases/cellect-biotechnology-announces-positive-data-demonstrating-robust-engraftment-using-apograft-was-featured-in-bone-marrow-transplantation-primary-data-points-were-submitted-to-the-fda-during-investigational-new-drug-approval-proc-301056409.html

SOURCE Cellect Biotechnology Ltd.

Company Codes: NASDAQ-SMALL:APOP, TelAviv:APOP

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Cellect Biotechnology Announces Positive Data Demonstrating Robust Engraftment Using ApoGraft was Featured in Bone Marrow Transplantation; Primary...

Bone Marrow Stem Cells Comments Off on Cellect Biotechnology Announces Positive Data Demonstrating Robust Engraftment Using ApoGraft was Featured in Bone Marrow Transplantation; Primary… | May 12th, 2020

The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met. Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

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In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition.

This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification. This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation.

A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strongCAGR of 7.0%from 2018 to 2026. In 2017, the market was worthUS$ 1,254.0 Mnand is expected to reachUS$ 2,299.5 Mnby the end of the forecast period in 2026.

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Customization to be the Key Focus of Market Players

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products. The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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PMR Sales Revenue in the Induced Pluripotent Stem Cells Market to Witness Growth at Robust CAGR 7% During 2026 - Cole of Duty

Skin Stem Cells Comments Off on PMR Sales Revenue in the Induced Pluripotent Stem Cells Market to Witness Growth at Robust CAGR 7% During 2026 – Cole of Duty | May 11th, 2020

Megan Dunn takes a long look at the art in the Auckland Hospital collection and finds out that yes, there is art, even in intensive care but the price of seeing it is everything.

I got the pamphlet in the mail back in January. It says after someone close to you dies it is natural to:

The day the pamphlet arrived I splayed it open and looked at the photo inside: a network of hands holding other hands. Cropped close. Just linked hands held in support of one another. Beneath the photo: we grieve as deeply as we love.

I am unable to return to normal services.

Painting from collection donated by the Art Komiti of Aucklands Paremoremo Prison

I had noticed art in waiting rooms before, not art with a capital A, not the kind of art that an art writer such as myself would bother to write about, because art like so much else in our society has its hierarchy. Contemporary art is a high-stakes game; who you write about is as important as what you say. Who cares about the art in the waiting room? Lets abbreviate: who cares?

Me. Ive had cause to think about it because last year Mums cancer returned from outer space. Shed been in remission for 17 years, which might be a national record for multiple myeloma, a cancer of the blood. Ive never really understood what her cancer is, just that she beat it first time round, and, last July, the cancer cell count was up. Then she started chemo.

Im not coping, Im just not coping, Mum called after the first round, in tears.

Have you eaten anything?

No. I dont feel like eating.

It was the dex. The specialist, a blonde in a miniskirt and long black boots, said, Thatll be the dex. Short for dexamethasone, a steroid. Another bitter pill to swallow. At that point Mum had to take up to 10 pills a day. I looked at the specialist. I was angry. Mums not coping. I had flown to Auckland to join Mum for her next haematology appointment.

But what art could I prescribe? Optimism matters, but art isnt always soothing or kind.

Im lucky I have a chance of getting better, Mum said. True. Her prognosis was good.

We went upstairs for her second round of chemo. The nurse in blue gown, gloves and a shower cap wheeled it around in a mauve container, hooked it on a drip, and inserted the port into Mums vein.

Other patients stretched out on beds like La-Z-Boys, skin drained. They too were waiting, but looked like they didnt have a chance of getting better.

I stared into the little office next to Mums chair. Harried papers on the desk; a PC lit up like a bright face, ready to supply details, cheer, whatever counts most. On the wall, an original painting of a phutukawa tree. I call it original but the concept wasnt. The sea peeked out of the background, the sky bluer than the sea. The oils had been used with little mixing, but I didnt mind that painting of the beach in summer rudely lit up by the phutukawa tree, the needles burst open in the leaves, red as fireworks.

The nurses are very nice here, Mum said.

Niceness counts like stem cells it has a tally.

That was quick, I said.

It really doesnt take long.

I wondered how long the painting took the artist. Guessing from the paint application, not long, but longer than it takes a dose of chemo to run into your veins.

I wanted to ask the nurse: who bought that painting? Who is the artist? Who put that there? But I was too shy. Why stop this real raw moment, for an intellectual aside, a detour into the art we look at it when were waiting to get better or to see how much worse it can get?

The painting of the phutukawa tree in summer yields its bouquet. Just be.

***

Mum and I got lost en route to blood work. The hospital has an art collection in its corridors that must be maintained by someone, however irregularly. We passed a Claudia Pond Eyley print black lines of plants, bright colours, a Pacific infusion abandoned along a corridor. A trio of photographs of flowers. Where are we? Is it this the way? They need to give better directions. Then, in a gangway, a pair of large, textured, brown abstracts, neither good nor bad, just out of time, like photos of your parents when they were young, in the fashions of the day. Those sideburns. What chops! Mums perm. Dads flares. A big Pat Hanly painting called Vacation was by the escalator. I meant to come back, retrace my steps, and find all this art again. And I did come back, but by then everything had changed.

I dont want to write what comes next, because I dont want the wait to be over. Waiting is a comfort in its own sad way. Just wait and see.

The sea. Waves lap in and out. I see it whenever I attend my doctors surgery above Courtenay Place. Piha is by the Korean-born photographer Jae Hoon Lee. Its moved around the surgery over the years. Now its in the waiting room above a line of plastic chairs. Whoever bought it must have thought it was soothing and anaesthetic, a balm for a worried soul like mine.

What do you want to see when you go for a smear test, or hold a baby that wont go the fuck to sleep, or wait with your partner for test results, a prognosis, a new vaccine?

I thought Ill wait to write about the art at the doctors surgery or the hospital. Or the dentist, though I have not been to the dentist in ages, no cash, so Ill wait. How much art is in your life? How many fillings? Do you have art at home? If so, what? Where did you get it? At what price?

In the waiting room, I dont blame you if you dont want to look at something difficult and ugly or think about something hard. If youre just after a good view, Arent we all?

At my doctors surgery Pihahangs near a plastic container for pamphlets, ruffled in waves.

For every problem there is a pamphlet.

The pamphlet that arrived in the mail in January is titled Department of Critical Care Medicine Bereavement Follow-up Service.

Piha in situ at Courtenay Medical, Wellington

The waiting room isnt just literal, but it is literal too. I know because the last time I was there I got a hot chocolate from the machine. Warm, syrupy. No art, just a TV on the wall.

At the haematology department, I watched a dad sitting with his young daughter on his lap. Her mind looked far away. She waited with the patience of one who has waited before. Then her mum appeared in a turban. I looked at this young mother, I had no idea what cancer of the blood she had, but I really hoped she had a chance of getting better.

Two in 100 people die, Mum said. Back in December she was waiting for her stem-cell transplant. She would be in hospital for two to three weeks. The transplant would take her immunity to zero that was where the risk of infection crept in but then the white cells would ingraft and her count would go back up. Shed signed the forms, accepting the 2% chance she would die. If theres not a bed on Thursday, it will be next week.

I wondered: Will there be any art in her room? And if so, what will it say?

The phutukawa painting in the office of the haematology department says shush shush, that lulling noise of waves from the beach. Dont worry, relax. Its OK. Look at the view. But the Jae Hoon Lee photograph in my doctors surgery says to me your hurt is timeless, the sea will exist whether we do or not, release your grip, whatever happens next will be surgically safe, emotions are never still, time is an inlet, the sea runs in and out.

***

I always knew that waiting was part of the problem. What if you wait too long? Then you cant get around to what you were going to say because you are:

***

Outside Ward 82, ICU (acute surgery). On the intercom, the number one has been buzzed off, pushed too many times. All other numbers present and correct. My mother is in bed 17, I tell the intercom, after Ive pressed the number one that isnt there and waited. Then I walked through vaguely yellow corridors lined with three framed prints, each composition a rectangle yellow, blue, orange lined with holes down the middle. They reminded me of paeans to the common household sponge. I stop at the hand-gel pump and sanitised. The art at the hospital is sanitised too but Im beyond caring. Too much caring and you move though it and pass out on the other side somewhere in the vicinity of bed 17.

At the end of the corridor past the hand gel, the Chen family have donated a small square print, red with black scribble, in honour of the ICU team. I clocked the gold engraved plaque, their appreciation registered on the wall. The painting not unlike the size of a fire alarm, but there is no glass to break open, the call has already been raised.

I got the call from the registrar on December 22 to say that Mums stem-cell plant was not going as expected and she had been admitted to ICU.

Ive been thinking about what I will do when I get better, Mum said, the night I arrived. She sat propped up in bed, on oxygen. Her face flushed, swollen, but superficially OK.

I sat next to her bed. Oh yes, what do you think?

Im going to come to your book launch.

I smiled. My book of personal essays about art and life, already way behind schedule.

What else? I asked.

I might meet someone new. I might travel.

Where would you like to go? I asked.

She paused. Maybe Africa, she said. I could go on a safari.

I nodded. Mum found it hard to walk up the small pronounced hill to our house in Wellington, sweated easily, mopped her forehead. Then shed wait, slightly panting, for her breath to right itself again, restart.

That night, I slept in the hospital. They say you can stay in my room at the Motutapu Ward, Mum said. There was no art in her room, but a wall-to-ceiling poster of a forest, kauri trees, dense, shady, green.

In the morning I got buzzed back into ICU. Mums arms and legs, twitched, calling out; face red, body puffed up; trying to unpick the PICC line from her arm, in among the beeping and the rising heart monitor, the oxygen exhaling. She doesnt recognise me, is raving to the charge nurse, who held her hand and looked at me and explained, Im just going some gentle reorientation work.

I must have seemed stunned.

Do you want to speak outside the room? the charge nurse asked.

I nodded. In the room next door to bed 17, I wailed, What is happening? Oh, what is happening? The charge nurse held me in place and comforted me, when there was no comfort to give.

Next the intensivist arrived in her blue scrubs, removed her surgical mask, wiped her hands with gel, introduced herself as Kylie. She wanted a family meeting.

The whnau room: the painting was donated by the Art Komiti of Aucklands Paremoremo Prison

The whnau room at ICU contains another Claudia Pond Eyley print on one wall and a multi-panel painting of a New Zealand landscape on the other. The mountains, a lake, smooth and even and still. A perennial view of nature, so calm, so undisturbing to see. I cant blame the hospital for containing so many paintings of the view sky, sand, sea, soothing, stretching, somehow infinite. A vista of comfort, comprehended.

I sat down on one side of the big meeting table, Kylie on the other. What is your understanding of your mothers condition? she asked. I rattled off the facts while an accompanying nurse took notes.

Mum had come in for a stem-cell transplant. She had caught pneumonia at a point in the process when she had no natural immunity. She had developed delirium. Her pre-existing heart condition had been set off: arterial fibrillation. She needed oxygen support to breathe.

This is what the 2% of risk looks like? I asked.

Yes. Kylie explained that they couldnt provide sedatives like morphine as that would risk compromising Mums breathing further. They could put Mum on a ventilator if she deteriorated but that would not be a good sign and would come with an extra level of risk. What helped patients with delirium was familiar voices.

I asked her, Are you holding anything back?

Just that this is very life threatening. Her eyes, an expression, I can now only call grave.

Well, it seemed apt. I liked Kylie. The intensivist understood how much intensity was required.

The job title intensivist seemed funny to me because in high school I was always told I was too intense. We have to find a happy medium, a teenage girlfriend once told me. So I got into art because it seemed like a place where intensity went and didnt have to turn down a notch. But art isnt the only place for intensity. At the intensive care unit (acute) on the eighth floor of Auckland Hospital the intensivists are also at work.

All day the nurse at bed 17 and I spooned jelly or orange juice into her mouth, helped her not frantically unpick her PICC line, I just want to go for a walk! No, no dear, you cant get up. Mum, you just need to rest. Rest. A familiar voice on replay. Youll feel so much better if you rest.

In the night I noticed the print of a lone bugle boy down some New Orleans alley, presumably playing jazz. No plaque. Who bought it? What family? Who was lost?

In ICU the beeping was persistent, insistent. The constant sound of inhaling, exhaling. Poke your tongue out. Ahhhh. Good girl.

Were concerned about the delirium. In ICU, delirium can be intensified, especially in older people, by the strange sounds, lights, faces.

I forgot the ducks! I keep meaning to mention the duck painting, a watercolour, a good one too, of some ducks paddling around their pond, giving no quacks in ICU. The duck pond was donated by another family, with a brass plaque. I should have jotted down the name.

The eyeball is so moveable, up and over, it can even see things that arent there. Mum said her family prayer, over and over, eyes roving. We consecrate to Thee, O Jesus of Love My aunt clutched her hands and said the prayer with her.

One night in ICU I passed a large rectangular collage of brightly coloured red and pink buttons like some budget Damien Hirst pill painting. Never passed it again. Beep, beep.

Her oxygen levels are saturating nicely.

How much?

Fifty-five percent, then down to 48%.

Dont obsess about the blood count.

Look at the patient. Look at the face.

The face her not her.

My daughter in the car on the way to the hospital singing: Pop bang crack goes the Christmas cracker, pop bang, crack goes the Christmas cracker, we will pull it off POP.

Eyes popped, snapped.

Shes been restless.

You need to sleep.

Sleep.

I stop and start, keep typing the next line, then deleting it. I dont want to get to the end of this. I dont want to remember all the family meetings in the whnau room waiting. I dont want to chart the order of those disordered days.

What was I going to say about art?

***

I spent the first weekend after Mums stem-cell transplant in her room at the Motutapu Ward. Motutapu means sacred or sanctuary. Shed just had the big dose of chemo and was quiet, but not yet unwell. My aunt was going to join her on Monday Mums 69th birthday for what would be the worst week of the process. I sat on the bed, Mum on the La-Z-Boy. We said not a whole lot.

At one point, I think I can manage a walk.

We passed the two nurses stations. En route I pointed out the art. We stopped by a faded print of sunflowers, beneath glass, but the artist was no Van Gogh. The first time Id passed the sunflowers, I hadnt rated them at all. But that was before Id read that they were by Chris Corlett, a 17-year-old who died of acute lymphoblastic leukaemia 20 years ago. Sunflowers of Hope does look like it was painted by a teenage boy. Theres something gnarly about them. Large and abundant. Full of life. Except for the leaves Chris paid special attention to the leaves stippled with decay, a bit heavy metal.

Sunflowers of Hope, Chris Cortlett, Auckland Hospital, 2019.

We stood reading the accompanying framed text about the foundation Chris had started to build up a database of 100,000 bone-marrow donors.

Courage, charisma, strength of character, sincerity whatever it is that makes some people inspirational and very special, Chris Corlett had it.

Another Claudia Pond Eyley print near the kitchen and on the door a sign that read: Dont use if youre come from a red room. I was confused by it when Id made Mum a cup of tea earlier and had to ask the nurse, Is this a red room? (Mums room was not a red room.)

I felt the light weight of Mums hand on my arm as we looped around to reception. I showed her my favourite painting, tucked in a corner. Its colours were so bold that from the corner of my eye I first suspected Matisse. Then was embarrassed when I realised the artist is Harriet, aged six, who donated Flowers for the Leukaemia Ward in memory of her father, Ned.

Harriet may not be Matisse but for a six-year-old her vase of flowers is a masterpiece of colour and compression.

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Art in the waiting room - The Spinoff

Skin Stem Cells Comments Off on Art in the waiting room – The Spinoff | May 10th, 2020

World Thalassemia Day is an annual observance day that occurs on May 8th. It is a world-wide campaign to raise awareness about thalassemia and its symptoms. This is done to help the patients living with this genetic disorder. World Thalassemia Day commemorates thalassemia victims and also aims at making thalassemia patients aware about the significance of medical consultation before marriage. This global observance day also tries to debunk myths and misconceptions surrounding the disease. The theme of World Thalassemia Day 2020 is Begin thalassemia prevention from young age, blood test before marriage will make the future generation safe. On this day, here we tell you all about the disease. Also Read - World Thalassemia Day 2019: How to Deal With Thalassemia

It is a genetic blood disorder that significantly reduces your haemoglobin count. Notably, haemoglobin is a protein molecule present in red blood cells. This protein helps RBCs in carrying oxygen and circulating it in the entire body. Also Read - World Thalassemia Day: Risk Factors, Types And Prevention Tips

The signs and symptoms of thalassemia depend on the type of thalassemia you have and its severity. Some common symptoms include fatigue, slow growth, weakness, abdominal swelling, pale skin, dark urine facial bone deformities etc. Usually, either a newborn shows thalassemia symptoms at the time of birth itself or develops it in the first two years of life. Also Read - World Thalassemia Day 2017: Importance of Blood donation and how it helps people with this fatal disease

Thalassemia occurs when the DNA of your body cells responsible for making haemoglobin, undergo mutation. This mutated DNA is passed on to the next generation.

A simple blood test can confirm the disease. Usually, if an expecting mother is known to be suffering from thalassemia, doctors perform a certain tests to find out if the fetus has also inherited the diseases and if yes, what is the severity of the genetic disease. To do that, chorionic villus sampling (testing a tiny sample of placenta) and amniocentesis (examining sample of fluid surrounding foetus) are performed.

In case, you have inherited a minimum number of mutated genes and suffering from mild thalassemia, you do not require treatment. However, in severe case, you may have to go through frequent blood transfusion, chelation therapy, or stem cell transplant.

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World Thalassemia Day 2020: Causes, Symptoms, Diagnosis And Treatment of The Disease - India.com

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The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor

(NASDAQ: AVCO) announced Friday that it is advancing its next-generation immune cell therapy to treat blood cancers using FLASH-CAR technology co-developed with strategic partner Arbele Limited.

The adaptable FLASH-CAR platform can be used to create personalized cell therapy from a patients own cells, as well as off-the-shelf cell therapy from a universal donor.

Currently, the Chimeric Antigen Receptor T (CAR-T) cellular immunotherapy platform is available. It involves a patients own T-cells a type of white blood cell that protects against infections and other diseases including cancer that are turned into personalized cancer-fighting cells.

The T-cells are removed from the patient, reprogrammed in the lab using a viral vector to target cancer cells, and infused back into the patient as a cancer immunotherapy.

But in contrast to these existing therapies, Avalon said its FLASH-CAR platform uses next-generation CAR technology to modify patients T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector.

Similar to T-cells, NK cells are a type of white blood cell, also able to attack cancer cells, but utilize different mechanisms. By using RNA molecules rather than a viral vector, Avalons RNA-based CAR technology is designed to rapidly create personalized CAR therapies in 1 to 2 days compared to the 10- to 14-day bio-manufacturing time necessary to generate currently available CAR-T cellular immunotherapy.

Avalon said its FLASH-CAR technology is also designed to reprogram the immune cells to hone in on multiple crucial cancer cell targets, called tumor antigens, to potentially achieve superior therapeutic effect. Avoiding the use of viral vectors and complicated bio-processing procedures significantly reduces manufacturing costs, resulting in a more affordable and potentially breakthrough therapy for cancer patients.

The FLASH-CAR technology can also be used to generate off-the-shelf,universal cell therapy that has the potential to reach even more patients.

Avalons first FLASH-CAR platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells.

Pre-clinical research on AVA-011, including tumor cytotoxicity studies, has been successfully completed and Avalon said it is immediately entering the process development stage to generate clinical-grade CAR-T and CAR-NK cells for use in human clinical trials.

Avalon and Arbele have jointly filed for US patents for this RNA-based CAR platform cellular therapy and for other applications.

Avalon expects to begin a first-in-human clinical trial with AVA-011 for the treatment of relapsed or refractory B-cell lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma in the first quarter of 2021. The goal is to use AVA-011 as a bridge to bone marrow stem cell transplant therapy, currently the only curative approach for patients with these blood cancers.

Avalon GloboCare is committed to decreasing the time it takes to deliver cellular immunotherapies to cancer patients, as well as lowering the cost of manufacturing by building on our unique RNA-based CAR platform that does not require using a viral vector, said CEO Dr David Jin.

We are accelerating our innovative discovery and development plan, as well as delivering precise clinical execution and leadership in cellular immunotherapy. Our pre-clinical studies are encouraging and we are excited for AVA-011 to enter the clinical development stage, including multi-center clinical trials following completion of process development to generate the cell therapy.

Arbele CEO John Luk added: Through this strategic partnership with Avalon GloboCare, we envision an accelerated scientific and clinical development of the RNA-based FLASH-CAR technology platform with great potential to generate 'off-the-shelf'immune effector cell therapies to treat both hematologic and solid malignancies.

Avalon, based in Freehold, New Jersey, specializes in developing cell-based technologies and is involved in the management of stem-cell banks and clinical laboratories.

The companys stock recently traded up 10% to $1.99a share in New York.

--UPDATES stock price--

Contact the author: [emailprotected]

Follow him on Twitter @PatrickMGraham

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Avalon GloboCare advancing immune cell therapy to treat blood cancers using FLASH-CAR technology - Proactive Investors USA & Canada

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Thalassemia is a type of inherited blood disorder. It is passed from parents to children through genes. This disorder involves lack of oxygen-carrying protein called hemoglobin (an important part of red blood cells). When there is insufficiency of hemoglobin in the body, the red blood cells dont function properly. It also reduces the life of RBC, which means fewer healthy RBC travel in the blood.

RBC carries oxygen to all the cells of the body. Oxygen acts as food, which is used by cells to function. Shortage of healthy RBC means shortage in supply of oxygen to all other cells of the body. This may lead to lethargy in a person. The person may feel tired, weak or short of breath. This condition is termed as Anaemia.

People with thalassemia may suffer from mild or acute Anaemia. Acute Anaemia can be very severe and can lead to damage of major organs. It can even cause death.

Thalassemia major babies are born to parents who are carriers of thalassemia gene. According to rough estimates, each year some 10000 babies are born in India with thalassemia. Best way to prevent or eliminate thalassemia is screening of all pregnant women between 9 to 12 weeks.

Thalassemia is diagnosed through blood tests which include doing a complete blood count (CBC) and special hemoglobin tests. Through a sample of blood, CBC measures the amount of hemoglobin and the different kind of blood cells, such as red blood cells. Hemoglobin tests measure the types of hemoglobin in blood.

Moderate and acute thalassemia is usually diagnosed in childhood. This is because signs and symptoms, such as acute Anemia usually occur at an early age of 2 years. People who have mild form of thalassemia may get diagnosed after a routine blood test, as it will detect if they have anemia.

Here's Dr. Rahul Bhargava, Director and Head, Hematology, Haemato- Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram has to say about the treatments:

Blood Transfusion

Treatment of thalassemia major relies on regular blood transfusion at regular intervals, to keep Hb above 9 gm. percent. It will help prevent form short stature and other skeletal and facial deformities. Recurrent lifelong blood transfusion since 6 months of birth is necessary.

Iron Chelation Therapy

With transfusion comes the problem of iron deposition, as each blood transfusion lead to incremental iron deposition in various tissues like pituitary gland liver and heart leading to early death. So along with transfusion patient also needs iron chelation therapy. It can be either oral (defriprone and defreseirox) or IV desferoxmine. Serum ferritin is one of the surrogate markers of iron overload in thalassemia patients. It needs to be done every 3 months. Gov.s efforts of providing free blood products and iron chelators is bearing fruits as life expectancy has shown an upward trend.

Bone Marrow or Stem Cell Transplant

As it is commonly known, bone marrow or stem cell transplant is the only curative modality for thalassemia. If done at an early age, 80 percent patients can be cured. Source of stem cell could be either brother or sister whose HLA is a complete match. Otherwise fully matched HLA donor can be tried in various international registries. This process is called as match unrelated donor transplant.

Gene Therapy

Gene therapy is gaining lot of traction in field of hemoglobinopathies. It has shown remarkable result with minimum toxicities and sustained haemoglobin production in various trials. There has been no major risk of cancer or other late effects.

We have come a long way and probably this decade will bring the much awaited cheers to thalassaemics. Till then in India, prevention is the only strategy to reduce the burden on already stretched health care system.

Better rate of blood transfusion

Regular Blood screening has significantly impacted reduction of infections due to blood transfusion

Significant improvement in treatment

Bone Marrow Transplant and Stem cell transplantation has led to patients having a good quality of life

Read more| 10 things to keep in mind while travelling with Asthma

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A Washington based biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedthe availability of thepre-print version of the manuscriptdescribing the immunological mechanism by whichleronlimabrestores immune function and impacts disease in COVID-19 patients.

This manuscript,Disruption of the CCL5/RANTES-CCR5 Pathway Restores Immune Homeostasis and Reduces Plasma Viral Load in Critical COVID-19, has been shared with the World Health Organization and is currently under peer review, said CytoDyn, Inc., as of May 6, 2020.

As described in thepre-print, in a cohort of 10critically ill patients, after treatment with leronlimab, these critically ill patients experienced reversed hyperimmune activation and inflammation, as well as reversed immunosuppression, therebyfacilitating a more effective immune responsecorrelated with decreases in SARS-CoV-2 level in blood.

These results demonstrate a novel approach to resolving unchecked inflammation whilerestoring immunologic deficiencies.

This is an important finding since according to various studies, a major driver of severe COVID-19 disease is excessive inflammation.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said in a press statement, We are now most hopeful the entire medical community will understand the potential benefit leronlimab can provide critically ill COVID-19 patients."

"Moreover, this discovery by Dr. Bruce Patterson that leronlimab decreases plasma viral load may have tremendous long-term positive ramifications to bring this pandemic under control. We are grateful that we are able to release this research at such a critical time for patients throughout the world.

Leronlimab (PRO 140) is being used as a treatment for severe COVID-19 under the emergency Investigational New Drug (IND) recently granted by the U.S. Food and Drug Administration (FDA).

Leronlimab is a drug candidate that is aCCR5 antagonist with the potential for multiple therapeutic indications.Leronlimab belongs to a group of HIV drugs calledCCR5antagonists.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions.

Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.

Previously, CytoDyn announced it is seeking a compassionate use designation for leronlimabfor the treatment of COVID-19 patients who are ineligible for participation in its two existing clinical trials. If this request is granted by the FDA, it will significantly expand the pool of patients who would be eligible to receive leronlimabtherapy.

CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor.

Precision Vaccinations publishes developing COVID-19 therapeutic drug news.

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A 7-year-old boy in Marylandwho suffers from sickle cell anemiais on his way to a full recovery after being hospitalized with a case of the coronavirus, according to reports.

Nasir Striggs was first hospitalized at Sinai Hospital in Baltimore in early April. His mother, Deshannon Striggs, brought him in for an examination after she noticed her son was experiencing trouble breathing.

He tested positive for COVID-19 at the hospital. An X-ray revealed he also had pneumonia in both lungs. The child, diagnosed with sickle cell disease, an inherited red blood cell disorder, underwent several blood transfusions at the hospital before his release.

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Nasir Striggs, 7, who has sickle cell anemia, is home from the hospital after recovering from the coronavirus. (Courtesy: Deshannon Striggs)

He had to keep getting stuck by the needle because the needle kept coming out, the mother told WBAL. To watch him go through that, it was really scary.

After undergoing treatment for several days, his condition began to improve, Deshannon said. She said prayers and support, as well as the dedicated care from the hospitals medical team, have helped her sons recovery.

Just keep the faith. Thats the message: keep the faith, she said.

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Deshannon said doctors have been monitoringNasirs conditionvia virtual check-ups since he was discharged from the hospital. Photos she shared with Fox News show the boy at home smiling,his face mask pulled beneath his chin.

Sickle cell disease is usually diagnosed shortly after birth. The genetic disorder results in oxygen-carrying red blood cells taking on a C or sickle shape, instead of round,often getting stuck in small blood vessels and clogging blood flow, according to the Centers for Disease Control and Prevention.

Children with the disease are at an increased risk of infection and other health problems. The only known cure is a bone marrow or stem cell transplant.

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Maryland boy, 7, with sickle cell disease recovers from coronavirus that caused pneumonia in both lungs - News Info Park

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VANCOUVER, Washington, May 08, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, today further clarified the status of the Companys submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for leronlimab as a combination therapy with HAART for highly treatment experienced HIV patients, filed on April 27, 2020 with the FDA. The BLA will not be considered completed until the Company submits to the FDA clinical datasets required to address FDA comments it received in March 2020, as described in the Companys press releases on May 4 and May 6, 2020. CytoDyn expects to submit these clinical datasets on May 11, 2020.

After the BLA submission is deemed completed, FDA makes a filing decision and sets a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation, if granted, means the FDAs goal is to take action on the application within six months of receipt (compared with 10 months under standard review).

About Coronavirus Disease 2019CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) and BLA Submission for the HIV Combination TherapyThe FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

The Company filed its BLA for Leronlimab as a Combination Therapy for Highly Treatment Experienced HIV Patients to the FDA on April 27, 2020. The BLA will not be considered completed until the Company submits required clinical datasets to the FDA. The Company expects to submit the required datasets on May 11, 2020. After the BLA submission is considered completed, FDA will make a filing decision and set a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA and the Company plans to request a priority review for the BLA. A priority review designation means the FDAs goal is to take action on the marketing application within six months of receipt (compared with 10 months under standard review).

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn filed its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients, and plans to submit additional datasets needed to complete the BLA on May 11, 2020. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes that are included elsewhere in this QuarterlyReport on Form 10-Q and our Annual Report on Form 10-K for the fiscal year endedDecember 31, 2019 filed with the U.S. Securities and Exchange Commission, or theSEC, on March 6, 2020, or our 2019 Form 10-K. This discussion containsforward-looking statements based upon current plans, expectations and beliefsthat involve risks and uncertainties. Our actual results may differ materiallyfrom those anticipated in these forward-looking statements as a result ofvarious factors, including, but not limited to, those discussed in the sectionentitled "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Inpreparing this MD&A, we presume that readers have access to and have read theMD&A in our 2019 Form 10-K, pursuant to Instruction 2 to paragraph (b) of Item303 of Regulation S-K. Unless stated otherwise, references in this QuarterlyReport on Form 10-Q to "us," "we," "our," or our "Company" and similar termsrefer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We currently have three clinical-stage ex vivo lentiviralvector ("LVV") programs currently enrolling patients in the US and EU forFanconi Anemia ("FA"), a genetic defect in the bone marrow that reducesproduction of blood cells or promotes the production of faulty blood cells,Leukocyte Adhesion Deficiency-I ("LAD-I"), a genetic disorder that causes theimmune system to malfunction and Pyruvate Kinase Deficiency ("PKD"), a rare redblood cell autosomal recessive disorder that results in chronic non-spherocytichemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-Iprogram are in registration-enabling studies in the US and EU. In addition, inthe US we have a clinical stage in vivo adeno-associated virus ("AAV") programfor Danon disease, a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Finally, we have a pre-clinical stage LVV programfor Infantile Malignant Osteopetrosis ("IMO"), a genetic disorder characterizedby increased bone density and bone mass secondary to impaired bone resorption -this program is anticipated to enter the clinic in 2020. We have globalcommercialization and development rights to all of these product candidatesunder royalty-bearing license agreements. Additional work in the discovery stagefor an FA CRISPR/CAS9 program as well as a gene therapy program for the lesscommon FA subtypes C and G is ongoing.

Recent Developments

On February 20, 2020, we entered into separate, privately negotiated exchangeagreements (the "Exchange Agreements") with certain holders of our outstanding5.75% Convertible Senior Notes due 2021 (the "2021 Convertible Notes") to extendthe maturity date by one year. Pursuant to the Exchange Agreements, we exchangedapproximately $39.35 million aggregate principal amount of the 2021 ConvertibleNotes (which represents approximately 76% of the aggregate outstanding principalamount of the 2021 Convertible Notes) for (a) approximately $39.35 millionaggregate principal amount of 6.25% Convertible Senior Notes due August 2022(the "2022 Convertible Notes") (an exchange ratio equal to 1.00 2022 ConvertibleNote per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay theaccrued and unpaid interest on the exchanged 2021 Convertible Notes from, andincluding, February 1, 2020 to February 20, 2020. The 2022 Convertible Noteswere issued in private placements exempt from registration in reliance onSection 4(a) (2) of the Securities Act of 1933, as amended (the "SecuritiesAct"). Upon completion of the exchange transactions, approximately $12.65million aggregate principal amount of 2021 Convertible Notes remainedoutstanding.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid ("DNA"), which code forproteins that perform a broad range of physiologic functions in all livingorganisms. Although genes are passed on from generation to generation, geneticchanges, also known as mutations, can occur in this process. These changes canresult in the lack of production of proteins or the production of alteredproteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence orsegment of DNA is administered to a patient, most commonly for the purpose oftreating a genetic disease that is caused by genetic mutations. Currentlyavailable therapies for many genetic diseases focus on administration of largeproteins or enzymes and typically address only the symptoms of the disease. Genetherapy aims to address the disease-causing effects of absent or dysfunctionalgenes by delivering functional copies of the gene sequence directly into thepatient's cells, offering the potential for curing the genetic disease, ratherthan simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our genetherapy treatments. Viruses are particularly well suited as delivery vehiclesbecause they are adept at penetrating cells and delivering genetic materialinside a cell. In creating our viral delivery vehicles, the viral (pathogenic)genes are removed and are replaced with a functional form of the missing ormutant gene that is the cause of the patient's genetic disease. The functionalform of a missing or mutant gene is called a therapeutic gene, or the"transgene." The process of inserting the transgene is called "transduction."Once a virus is modified by replacement of the viral genes with a transgene, themodified virus is called a "viral vector." The viral vector delivers thetransgene into the targeted tissue or organ (such as the cells inside apatient's bone marrow). We have two types of viral vectors in development, LVVand AAV. We believe that our LVV and AAV-based programs have the potential tooffer a long-lasting and significant therapeutic benefit to patients.

Gene therapies can be delivered either (1) ex vivo (outside the body), in whichcase the patient's cells are extracted and the vector is delivered to thesecells in a controlled, safe laboratory setting, with the modified cells thenbeing reinserted into the patient, or (2) in vivo (inside the body), in whichcase the vector is injected directly into the patient, either intravenously("IV") or directly into a specific tissue at a targeted site, with the aim ofthe vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greaterregulatory acceptance of gene therapy have created a promising environment toadvance gene therapy products as these products are being designed to restorecell function and improve clinical outcomes, which in many cases includeprevention of death at an early age.

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The chart below shows the current phases of development of Rocket's programs andproduct candidates:

LVV Programs. Rocket's LVV-based programs utilize third-generation,self-inactivating lentiviral vectors to target selected rare diseases.Currently, Rocket is developing LVV programs to treat FA, LAD-I, PKD, and IMO.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from amutation in a single FA gene. An estimated 60 to 70% of cases arise frommutations in the Fanconi-A ("FANCA") gene, which is the focus of our program. FAresults in bone marrow failure, developmental abnormalities, myeloid leukemiaand other malignancies, often during the early years and decades of life. Bonemarrow aplasia, which is bone marrow that no longer produces any or very few redand white blood cells and platelets leading to infections and bleeding, is themost frequent cause of early morbidity and mortality in FA, with a median onsetbefore 10 years of age. Leukemia is the next most common cause of mortality,ultimately occurring in about 20% of patients later in life. Solid organmalignancies, such as head and neck cancers, can also occur, although at lowerrates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem celltransplant ("HSCT"), currently the most frequently utilized therapy for FA, haveresulted in more frequent hematologic correction of the disorder, HSCT isassociated with both acute and long-term risks, including transplant-relatedmortality, graft versus host disease ("GVHD"), a sometimes fatal side effect ofallogeneic transplant characterized by painful ulcers in the GI tract, livertoxicity and skin rashes, as well as increased risk of subsequent cancers. Ourgene therapy program in FA is designed to enable a minimally toxic hematologiccorrection using a patient's own stem cells during the early years of life. Webelieve that the development of a broadly applicable autologous gene therapy canbe transformative for these patients.

Each of our LVV-based programs utilize third-generation, self-inactivatinglentiviral vectors to correct defects in patients' HSCs, which are the cellsfound in bone marrow that are capable of generating blood cells over a patient'slifetime. Defects in the genetic coding of HSCs can result in severe, andpotentially life-threatening anemia, which is when a patient's blood lacksenough properly functioning red blood cells to carry oxygen throughout the body.Stem cell defects can also result in severe and potentially life-threateningdecreases in white blood cells resulting in susceptibility to infections, and inplatelets responsible for blood clotting, which may result in severe andpotentially life-threatening bleeding episodes. Patients with FA have a geneticdefect that prevents the normal repair of genes and chromosomes within bloodcells in the bone marrow, which frequently results in the development of acutemyeloid leukemia ("AML"), a type of blood cancer, as well as bone marrow failureand congenital defects. The average lifespan of an FA patient is estimated to be30 to 40 years. The prevalence of FA in the US and EU is estimated to be about4,000, and given the efficacy seen in non-conditioned patients, the addressableannual market opportunity is now thought to be in the 400 to 500 range.

We currently have one LVV-based program targeting FA, RP-L102. RP-L102 is ourlead lentiviral vector based program that we in-licensed from Centro deInvestigaciones Energticas, Medioambientales y Tecnolgicas ("CIEMAT"), whichis a leading research institute in Madrid, Spain. RP-L102 is currently beingstudied in our sponsored Phase 2 registrational enabling clinical trialstreating FA patients initially at the Center for Definitive and CurativeMedicine at Stanford University School of Medicine ("Stanford") and HospitalInfantil de Nino Jesus ("HNJ") in Spain. The Phase 2 portion of the trial isexpected to enroll ten patients total from the U.S. and EU. Patients willreceive a single IV infusion of RP-L102 that utilizes fresh cells and "ProcessB" which incorporates a modified stem cell enrichment process, transductionenhancers, as well as commercial-grade vector and final drug product.

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Table of ContentsIn October 2019, at the European Society of Cell and Gene Therapy ("ESGCT") 2019Annual Congress, long-term Phase 1/2 clinical data of RP-L102, from the clinicaltrial sponsored by CIEMAT, for FA "Process A", without the use of myeloablativeconditioning was presented demonstrating evidence of increasing and durableengraftment leading to bone marrow restoration exceeding the 10% thresholdagreed to by the FDA and EMA for the ongoing registration-enabling Phase 2trial. In patient 02002, who received what we consider adequate drug product,hemoglobin levels are now similar to those in the first year after birth,suggesting hematologic correction over the long term.

During the third quarter of 2019, we received alignment from the FDA on thetrial design and the primary endpoint. This alignment was similar to thatpreviously received from the European Medicines Agency ("EMA"). Resistance tomitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum timepoint of one year to serve as the primary endpoint for our Phase II study. InDecember 2019, we announced that the first patient of the global Phase 2 studyfor RP-L102 "Process B" for FA received investigational therapy. There will betotal of 10 patients enrolled in the global Phase 2 studies.

In December 2019, we also announced preliminary results from two pediatricpatients treated with "Process B" RP-L102 prior to development of severe bonemarrow failure in our Phase 1 trial of RP-L102 for FA. To evaluate transductionefficiency, an analysis of the proportion of the MMC-resistant colony formingcells was conducted and both patients have thus far exhibited early signs ofengraftment, including increases in blood cell lineages in one patient. Nodrug-related safety or tolerability issues have been reported.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion andmigration, resulting from mutations in the ITGB2 gene encoding for the Beta-2Integrin component, CD18. Deficiencies in CD18 result in an impaired ability forneutrophils (a subset of infection-fighting white blood cells) to leave bloodvessels and enter into tissues where these cells are needed to combatinfections. As is the case with many rare diseases, true estimates of incidenceare difficult; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. SevereLAD-I is notable for recurrent, life-threatening infections and substantialinfant mortality in patients who do not receive an allogeneic HSCT. Mortalityfor severe LAD-I has been reported as 60 to 75% by age two in the absence ofallogeneic HCST.

We currently have one program targeting LAD-I, RP-L201. RP-L201 is a clinicalprogram that we in-licensed from CIEMAT. We have partnered with UCLA to leadU.S. clinical development efforts for the LAD-I program. UCLA and its Eli andEdythe Broad Center of Regenerative Medicine and Stem Cell Research is servingas the lead U.S. clinical research center for the registrational clinical trialfor LAD-I, and HNJ is serving as the lead clinical site in Spain.

The ongoing open-label, single-arm, Phase 1/2 registration enabling clinicaltrial of RP-L201 has dosed one severe LAD-I patient in the U.S. to assess thesafety and tolerability of RP-L201. The first patient was treated with RP-L201in third quarter 2019. This study has received $6.5 million CLIN2 grant awardfrom the California Institute for Regenerative Medicine ("CIRM") to support theclinical development of gene therapy for LAD-I.

In December 2019, we announced initial results from the first pediatric patienttreated with RP-L201, demonstrating early evidence of safety. Analyses ofperipheral vector copy number ("VCN"), and CD18-expressing neutrophils wereperformed through three months after infusion of RP-L201 to evaluate engraftmentand phenotypic correction. The patient exhibited early signs of engraftment withVCN myeloid levels at 1.5 at three months and CD-18 expression of 45%. No safetyor tolerability issues related to RP-L201 administration (or investigationalproduct) had been identified as of that date. The study is expected to enrollnine patients globally.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting frommutations in the pyruvate kinase L/R ("PKLR") gene encoding for a component ofthe red blood cell ("RBC") glycolytic pathway. PKD is characterized by chronicnon-spherocytic hemolytic anemia, a disorder in which RBCs do not assume anormal spherical shape and are broken down, leading to decreased ability tocarry oxygen to cells, with anemia severity that can range from mild(asymptomatic) to severe forms that may result in childhood mortality or arequirement for frequent, lifelong RBC transfusions. The pediatric population isthe most commonly and severely affected subgroup of patients with PKD, and PKDoften results in splenomegaly (abnormal enlargement of the spleen), jaundice andchronic iron overload which is likely the result of both chronic hemolysis andthe RBC transfusions used to treat the disease. The variability in anemiaseverity is believed to arise in part from the large number of diverse mutationsthat may affect the PKLR gene. Estimates of disease incidence have rangedbetween 3.2 and 51 cases per million in the white U.S. and EU population.Industry estimates suggest at least 2,500 cases in the U.S. and EU have alreadybeen diagnosed despite the lack of FDA-approved molecularly targeted therapies.Enrollment is currently ongoing and we anticipate treating the first patient inthe third quarter of 2020.

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Table of ContentsWe currently have one LVV-based program targeting PKD, RP-L301. RP-L301 is aclinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 toinitiate a global Phase 1 study was cleared by the FDA in October 2019. Thisprogram has been granted EMA orphan drug disease designation and FDA orphan drugdisease designation ("ODD").

This global Phase 1 open-label, single-arm, clinical trial is expected to enrollsix adult and pediatric transfusion-dependent PKD patients in the U.S. andEurope. Lucile Packard Children's Hospital Stanford will serve as the lead sitein the U.S. for adult and pediatric patients, and Hospital InfantilUniversitario Nio Jess will serve as the lead site in Europe for pediatricsand Hospital Universitario Fundacin Jimnez Daz will serve as the lead site inEurope for adult patients.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone masssecondary to impaired bone resorption. Normally, small areas of bone areconstantly being broken down by special cells called osteoclasts, then madeagain by cells called osteoblasts. In IMO, the cells that break down bone(osteoclasts) do not work properly, which leads to the bones becoming thickerand not as healthy. Untreated IMO patients may suffer from a compression of thebone-marrow space, which results in bone marrow failure, anemia and increasedinfection risk due to the lack of production of white blood cells. Untreated IMOpatients may also suffer from a compression of cranial nerves, which transmitsignals between vital organs and the brain, resulting in blindness, hearing lossand other neurologic deficits.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is apreclinical program that we in-licensed from Lund University, Sweden. Thisprogram has been granted ODD and Rare Pediatric Disease designation from theFDA. The FDA defines a "rare pediatric disease" as a serious andlife-threatening disease that affects less than 200,000 people in the U.S. thatare aged between birth to 18 years. The Rare Pediatric Disease designationprogram allows for a sponsor who receives an approval for a product topotentially qualify for a voucher that can be redeemed to receive a priorityreview of a subsequent marketing application for a different product. We havepartnered with UCLA to lead U.S. clinical development efforts for the IMOprogram and anticipate that UCLA will serve as the lead U.S. clinical site forIMO. We intend to file an IND for IMO and commence our clinical trial in thefourth quarter of 2020.

Danon disease is a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Danon disease is caused by mutations in the geneencoding lysosome-associated membrane protein 2 ("LAMP-2"), a mediator ofautophagy. This mutation results in the accumulation of autophagic vacuoles,predominantly in cardiac and skeletal muscle. Male patients often require hearttransplantation and typically die in their teens or twenties from progressiveheart failure. Along with severe cardiomyopathy, other Danon disease symptomscan include skeletal muscle weakness, liver disease, and intellectualimpairment. There are no specific therapies available for the treatment of Danondisease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease,which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.and the EU, however new market research is being performed and the prevalence ofpatients may be updated in the future.

In January 2019, we announced the clearance of our IND application by the FDAfor RP-A501, and in February 2019, we were notified by the FDA that we weregranted Fast Track designation for RP-A501. University of California San DiegoHealth is the initial and lead center for our Phase 1 clinical trial.

On May 2, 2019, we presented additional preclinical data at the ASCGT annualmeeting, indicating that high VCN, in Danon disease-relevant organs in both miceand non-human primates ("NHN's"), with high concentrations in heart and livertissue (for NHP, cardiac VCN was approximately 10 times higher on average thanin skeletal muscle and central nervous system), which is consistent withreported results in several studies of heart tissue across different species.There were no treatment-related adverse events or safety issues up to thehighest dose. We have dosed three patients in the RP-A501 phase 1 clinicaltrial. We will continue further enrollment with clinical data read-outs in thefourth quarter of 2020.

As of March 2020, we have dosed three patients in the RP-A501 phase 1 clinicaltrial. This completes the first low dose cohort of the Phase 1 study. Based onthe preliminary safety and efficacy data review of this completed cohort, boththe FDA and IDMC has provided clearance to advance to a higher dose cohort inPhase 1 Trial of RP-A501 for Danon Disease. We will continue further enrollmentwith clinical data read-outs in the second half of 2020.

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In addition to its LVV and AAV programs, we also have a program evaluatingCRISPR/Cas9-based gene editing for FA. This program is currently in thediscovery phase. CRISPR/Cas9-based gene editing is a different method ofcorrecting the defective genes in a patient, where the editing is very specificand targeted to a particular gene sequence. "CRISPR/Cas9" stands for Clustered,Regularly Interspaced Short Palindromic Repeats ("CRISPR") Associated protein-9.The CRISPR/Cas9 technology can be used to make "cuts" in DNA at specific sitesof targeted genes, making it potentially more precise in delivering genetherapies than traditional vector-based delivery approaches. CRISPR/Cas9 canalso be adapted to regulate the activity of an existing gene without modifyingthe actual DNA sequence, which is referred to as gene regulation.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated,rare pediatric diseases through the development and commercialization ofpotentially curative first-in-class gene therapies. To achieve these objectives,we intend to develop into a fully-integrated biotechnology company. In the near-and medium-term, we intend to develop our first-in-class product candidates,which are targeting devastating diseases with substantial unmet need, developproprietary in-house analytics and manufacturing capabilities and continue tocommence registration trials for our currently planned programs. In the mediumand long-term, we expect to submit our first biologics license applications("BLAs"), and establish our gene therapy platform and expand our pipeline totarget additional indications that we believe to be potentially compatible withour gene therapy technologies. In addition, during that time, we believe thatour currently planned programs will become eligible for priority review vouchersfrom the FDA that provide for expedited review. We have assembled a leadershipand research team with expertise in cell and gene therapy, rare disease drugdevelopment and commercialization.

We believe that our competitive advantage lies in our disease-based selectionapproach, a rigorous process with defined criteria to identify target diseases.We believe that this approach to asset development differentiates us as a genetherapy company and potentially provides us with a first-mover advantage.

Financial Overview

Since our inception, we have devoted substantially all of our resources toorganizing and staffing the Company, business planning, raising capital,acquiring or discovering product candidates and securing related intellectualproperty rights, conducting discovery, research and development activities forthe programs and planning for potential commercialization. We do not have anyproducts approved for sale and have not generated revenue from product sales.From inception through March 31, 2020, we raised net cash proceeds ofapproximately $373.1 million from investors through both equity and convertibledebt financing to fund operating activities. As of March 31, 2020, we had cash,cash equivalents and investments of $275.9 million.

Since inception, we have incurred significant operating losses. Our ability togenerate product revenue sufficient to achieve profitability will depend heavilyon the successful development and eventual commercialization of one or more ofthe current or future product candidates and programs. We had net losses of$24.7 million for the three months ended March 31, 2020 and $77.3 million forthe year ended December 31, 2019. As of March 31, 2020, we had an accumulateddeficit of $207.8 million. We expect to continue to incur significant expensesand higher operating losses for the foreseeable future as we advance our currentproduct candidates from discovery through preclinical development and clinicaltrials and seek regulatory approval of our product candidates. In addition, ifwe obtain marketing approval for any of their product candidates, we expect toincur significant commercialization expenses related to product manufacturing,marketing, sales and distribution. Furthermore, we expect to incur additionalcosts as a public company. Accordingly, we will need additional financing tosupport continuing operations and potential acquisitions of licensing or otherrights for product candidates.

Until such a time as we can generate significant revenue from product sales, ifever, we will seek to fund our operations through public or private equity ordebt financings or other sources, which may include collaborations with thirdparties and government programs or grants. Adequate additional financing may notbe available to us on acceptable terms, or at all. We can make no assurancesthat we will be able to raise the cash needed to fund our operations and, if wefail to raise capital when needed, we may have to significantly delay, scaleback or discontinue the development and commercialization of one or more productcandidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with productdevelopment, we are unable to predict the timing or amount of increased expensesor when or if we will be able to achieve or maintain profitability. Even if weare able to generate product sales, we may not become profitable. If we fail tobecome profitable or are unable to sustain profitability on a continuing basis,then we may be unable to continue our operations at planned levels and be forcedto reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including fromproduct sales, and we do not expect to generate any revenue from the sale ofproducts in the near future. If our development efforts for product candidatesare successful and result in regulatory approval or license agreements withthird parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our research and development program ("R&D") expenses consist primarily ofexternal costs incurred for the development of our product candidates. Theseexpenses include:

expenses incurred under agreements with research institutions that conduct

research and development activities including, process development,

preclinical, and clinical activities on Rocket's behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

We recognize external development costs based on contractual payment schedulesaligned with program activities, invoices for work incurred, and milestoneswhich correspond with costs incurred by the third parties. Nonrefundable advancepayments for goods or services to be received in the future for use in researchand development activities are recorded as prepaid expenses.

Our direct research and development expenses are tracked on a program-by-programbasis for product candidates and consist primarily of external costs, such asresearch collaborations and third party manufacturing agreements associated withour preclinical research, process development, manufacturing, and clinicaldevelopment activities. Our direct research and development expenses by programalso include fees incurred under license agreements. Our personnel, non-programand unallocated program expenses include costs associated with activitiesperformed by our internal research and development organization and generallybenefit multiple programs. These costs are not separately allocated by productcandidate and consist primarily of:

Our research and development activities are central to our business model.Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development. As aresult, we expect that research and development expenses will increasesubstantially over the next several years as we increase personnel costs,including stock-based compensation, support ongoing clinical studies, seek toachieve proof-of-concept in one or more product candidates, advance preclinicalprograms to clinical programs, and prepare regulatory filings for productcandidates.

We cannot determine with certainty the duration and costs to complete current orfuture clinical studies of product candidates or if, when, or to what extent wewill generate revenues from the commercialization and sale of any of our productcandidates that obtain regulatory approval. We may never succeed in achievingregulatory approval for any of our product candidates. The duration, costs, andtiming of clinical studies and development of product candidates will depend ona variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any future

clinical studies and other research and development activities that we

undertake;

future clinical trial results;

uncertainties in clinical trial enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

We expect research and development expenses to increase for the foreseeablefuture as we continue to invest in research and development activities relatedto developing product candidates, including investments in manufacturing, as ourprograms advance into later stages of development and as we conduct additionalclinical trials. The process of conducting the necessary clinical research toobtain regulatory approval is costly and time-consuming, and the successfuldevelopment of product candidates is highly uncertain. As a result, we areunable to determine the duration and completion costs of research anddevelopment projects or when and to what extent we will generate revenue fromthe commercialization and sale of any of our product candidates.

Our future research and development expenses will depend on the clinical successof our product candidates, as well as ongoing assessments of the commercialpotential of such product candidates. In addition, we cannot forecast with anydegree of certainty which product candidates may be subject to futurecollaborations, when such arrangements will be secured, if at all, and to whatdegree such arrangements would affect our development plans and capitalrequirements. We expect our research and development expenses to increase infuture periods for the foreseeable future as we seek to complete development ofour product candidates.

The successful development and commercialization of our product candidates ishighly uncertain. This is due to the numerous risks and uncertainties associatedwith product development and commercialization, including the uncertainty of:

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Table of Contents

the scope, progress, outcome and costs of our clinical trials and other

research and development activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt and terms of any marketing approvals.

A change in the outcome of any of these variables with respect to thedevelopment of our product candidates that we may develop could mean asignificant change in the costs and timing associated with the development ofour product candidates. For example, if the FDA or another regulatory authoritywere to require us to conduct clinical trials or other testing beyond those thatwe currently contemplate for the completion of clinical development of any ofour product candidates that we may develop or if we experience significantdelays in enrollment in any of our clinical trials, we could be required toexpend significant additional financial resources and time on the completion ofclinical development of that product candidate.

General and Administrative Expenses

General and administrative ("G&A") expenses consist primarily of salaries andrelated benefit costs for personnel, including stock-based compensation andtravel expenses for our employees in executive, operational, finance, legal,business development, and human resource functions. In addition, othersignificant general and administrative expenses include professional fees forlegal, patents, consulting, investor and public relations, auditing and taxservices as well as other expenses for rent and maintenance of facilities,insurance and other supplies used in general and administrative activities. Weexpect general and administrative expenses to increase for the foreseeablefuture due to anticipated increases in headcount to support the continuedadvancement of our product candidates. We also anticipate that we will incurincreased accounting, audit, legal, regulatory, compliance and director andofficer insurance costs as well as investor and public relations expenses.

Interest Expense

Interest expense is related to the 2021 Convertible Notes, which mature inAugust 2021, and the 2022 Convertible Notes, which mature in August 2022.

Interest Income

Interest income is related to interest earned from investments.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generallyaccepted accounting principles in the U.S. The preparation of our financialstatements and related disclosures requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, costs and expenses, andthe disclosure of contingent assets and liabilities in our financial statements.We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances,the results of which form the basis for making judgments about the carryingvalues of assets and liabilities that are not readily apparent from othersources. We evaluate estimates and assumptions on an ongoing basis. Actualresults may differ from these estimates under different assumptions orconditions.

Our significant accounting policies are described in more detail in our 2019Form 10-K, except as otherwise described below.

Results of Operations

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(MENAFN - iCrowdNewsWire) May 8, 2020

According to the new market research report " Cell Therapy Technologies Market by Product (Consumables, Equipment, Software), Cell Type (Human Stem & Differentiated, Animal), Process Stages (Cell Processing, Distribution, Handling, QC), End User, and Region - Global Forecast to 2023, , published by MarketsandMarkets, The global cell therapy technologies market is projected to reach USD 19.9 billion by 2023 from USD 10.2 billion in 2018, at a CAGR of 14.4% during the forecast period.

Browse in-depth TOC on 'Cell Therapy Technologies Market" 75 - Table30 Figures116 Pages

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Rising government investments for cell-based research, the increasing number of GMP-certified production facilities, and the large number of oncology-oriented cell-based therapy clinical trials are the key factors driving the growth of this market. China, India, Japan, Korea, and Brazil are emerging markets for cell therapy instruments. These markets boast comparatively lenient standards and government regulations as opposed to developed markets in North America and the EU, and thus offer significant growth potential for providers. However, the high cost of cell-based research and the low success rate is expected to restrain market growth to some extent during the forecast period.

Consumables are expected to account for the largest cell therapy technologies market share in 2018 : By product, the cell therapy technologies market is segmented into consumables, equipment, and systems & software. The consumables segment is expected to account for the largest share of the market in 2018. Factors such as increasing investments by companies to develop advanced products as well as government initiatives for enhancing cell-based research are contributing to the growth of the cell therapy consumables market.

Cell processing segment to witness the highest growth during the forecast period :

Based on process, the cell therapy technologies market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control. The cell processing segment is expected to account for the largest market share in 2018 and is projected to witness the highest CAGR during the forecasted period.

Human cells segment accounts for the large share of the cell therapy instruments market, by cell type :

Based on cell type, the market is segmented into human cells and animal cells. In 2018, the human cells segment is expected to account for the largest share of the cell therapy technologies market. The rising adoption of human cells over animal cells for cell therapeutics research, technological advancements, and the rising incidence of diseases such as cancer and cardiac abnormalities are the key factors driving the growth of this segment.

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North America to dominate the cell therapy technologies market during the forecast period : The market is segmented into four major regions, namely, North America, Europe, Asia Pacific, and the Rest of the World (RoW). North America is expected to dominate the market in 2018 owing to the high burden of chronic diseases and increasing R & D activities in the pharmaceutical and biotechnology industries. The Asia Pacific region is expected to register the highest CAGR during the forecast period.

The major players in the western blotting market are Beckman Coulter (US), Becton, Dickinson and Company (US), GE Healthcare (US), Lonza (Switzerland), Merck KGaA (Germany), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Terumo BCT (US), and Thermo Fisher Scientific (US).

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Korean researchers have found a signal transduction system that modulates the treatment of mesenchymal stem cells and immune control functions, opening the way for treating graft-versus-host disease treatment.

Mesenchymal stem cells divide into various cells, have immunomodulatory functions, and are the primary cell sources for stem cell therapy.

Graft-versus-host disease is a fatal disease that leads to death after an allogeneic blood transfusion or bone marrow transplantation. Although there are many clinical trials underway worldwide to treat the symptom, there are no applicable treatments besides alleviating symptoms with high-dose steroids.

The team, led by Professor Shin Dong-myeong of the Department of Biomedical Sciences at Asan Medical Center, discovered that the CREB1 (CAMP responsive element binding protein 1) signaling system activates the treatment and immune control functions of mesenchymal stem cells.

The team administered a therapeutic agent made by upgrading mesenchymal stem cells to graft-versus-host disease mice, and found that it alleviated anorexia symptoms and reduced the weight loss rate by 30 percent while increasing the survival rate by 30 percent.

When developing a cell therapy product, researchers have to cultivate the stem cells in vitro. Thus it is very likely that it will impair stem cell functions due to free radicals generated in the cells. To prevent the deterioration of stem cell function, it is necessary to improve the stem cell function in vitro culture, prevent stem cell oxidation, and increase the antioxidant capacity of the cell itself.

Until now, there was a lack of specific evidence and understanding of how stem cells regulate glutathione, an indicator of antioxidant capacity. Therefore, it was difficult to prevent stem cell dysfunction and oxidation.

Professor Shin's team developed experimental techniques that can monitor and quantify glutathione in real-time and confirmed that the CREB1 signaling system regulated the amount and activity of glutathione.

By activating the CREB1 signaling system, the team found that the process also activated nuclear factor erythroid 2-related factor 2 (NRF2) protein, which maintains the antioxidant capacity of mesenchymal stem cells and the increase of both the expression levels of peroxiredoxin-1 (PRDX1) and glutamate-cysteine ligase modifier subunit (GCLM) protein, which synthesize glutathione and are antioxidant activity indicators.

As a result, the team confirmed that its method was effective in treating the graft-versus-host disease.

"Based on this study, we have secured a technological foundation to advance stem cell treatment by controlling the antioxidant capacity of stem cells," Professor Shin said.

If this technology makes a high-purity and high-quality stem cell treatment, the team expects that it will be a step toward developing a graft-versus-host disease treatment and overcoming various intractable diseases such as nervous system diseases and inflammatory diseases with high medical demand, Shin added.

The results of the study were published in the journal, Science Advances.

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