Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

Excerpt from:
Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - KULR-TV

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent … – KULR-TV | November 8th, 2021

TAZEWELL, Va. (WVVA) - Gabe is a seventeen year old who has battled and beat HLH, however recently he was diagnosed with leukemia and is in need of a blood stem cell and bone marrow transplant. Community members of Gabe's family are holding a bake sale, pumpkin contest and raffles to fund Gabe's cost of medical bills and travel expenses.

A DKMS drive was set up along with the bake sale at the Crab Orchard Veterinarians Services today to help find donors to give the gift of life to families just like Gabe's. All of the proceeds of the bake sale, contest and raffles will be given to Gabe's family.

'It's been a very stressful time for us but it's also been a very blessed time for us and I'm grateful for the outpouring from my community. My church family has been real big. My pastor and his wife and everybody involved at City on a Hill, I love you guys.

"I'm just grateful for everybody at Crab Orchard Vet Services and Honaker Animal Health for doing this for us and stepping up in a big way."

The process of becoming a potential donor for DKMS is simple. You get a swab kit and swab the inside of your cheeks. You fill out a paper with your contact info and from there you are registered into a database to see if you are a match with someone who needs stem cells or bone marrow.

"It means that we can not only help a family in the community but one of our own. Clifford works for our sister office in Honaker and it just means a lot to be able to reach out and help a family."

If you are interested in becoming a potential donor you can go to the DKMS website found here to get a donor kit: Register as a Stem Cell and Bone Marrow Donor | DKMS | DKMS

Read the original:
Community members rally behind teen in need of transplant - WVVA TV

Bone Marrow Stem Cells Comments Off on Community members rally behind teen in need of transplant – WVVA TV | October 28th, 2021

Our bone marrow harbors thousandsof rare hematopoietic stem cells tiny shape shifters that can morph into red or white blood cells. But conditions like sickle cell anemia or immune deficiency can damage these cells, and treatments for blood cancers may destroy them altogether.

To rebuild bone marrow, researchers have perfected the art of transplanting stem cells either those belonging to the patient or ones that have been donated.

You only have so many of these cells, says Jennifer Gillette, PhD, associate professor inThe University of New Mexico Department of Pathology. These are cells that are with us our entire life. They stay turned off when youre healthy and things are moving along. But in a stress condition, these are cells that get turned on and rev up and then go quiescent again.

Gillette has been devising ways to trick donors bone marrow into releasing more stem cells into the bloodstream so they can be harvested and transfused.

Ina new paper published onlinein the journalStem Cell Reports, Gillette and her colleagues describe manipulating a protein called CD82 that sits on the surface of each stem cell and helps them migrate into and out of the bone marrow.

Gillettes team found that when CD82 disruption is coupled with existing medications used to stimulate stem cell release into the bloodstream, the process is significantly amplified, causing more stem cells to find their way into circulation.

One of those medications AMD3100 costs $10,000 for a single dose, she says, and it is hoped that treatment with antibodies might be less expensive.

Patients diagnosed with leukemia or lymphoma may be treated with chemotherapy and/or radiation, which destroy cancerous cells in the bone marrow along with stem cells.

It wipes out everything, Gillette says. For leukemia or blood cancers youre trying to get rid of that malignant population and replace it with healthy cells.

Patients may bank some of their stem cells beforehand for autologous transplants, or they could receive allograft transplants from suitable donors.

There are some patients that just dont mobilize well, she says. Were looking for any way that we can enhance cell release, because the number and quality of cells going in really enhance the response of the patients.

Gillette has applied for a provisional patent to repurpose the antibodies originally developed to study CD82 functioning as a clinical treatment. The next step is to see exactly how that antibody is working, she says.

Originally posted here:
UNM Scientist Jennifer Gillette Tricks the Bone Marrow ...

Bone Marrow Stem Cells Comments Off on UNM Scientist Jennifer Gillette Tricks the Bone Marrow … | October 28th, 2021

-- Biohaven initiates a clinical trial of the novel antibody recruiting molecule BHV-1100 to assess safety, tolerability, and exploratory clinical activity in Multiple Myeloma

Published: Oct. 27, 2021 at 7:30 AM EDT|Updated: 18 hours ago

NEW HAVEN, Conn., Oct. 27, 2021 /PRNewswire/ --Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), announced the enrollment of the first patient in a Phase 1a/1b trial in Multiple Myeloma using the ARM, BHV-1100, in combination with autologous cytokine induced memory-like (CIML) natural killer (NK) cells and immunoglobulin (Ig) to target and kill multiple myeloma cells expressing the cell surface protein CD38 (Figure 1). BHV-1100 is the lead clinical asset from Biohaven's ARM Platform, developed from a strategic alliance with PeptiDream Inc. This clinical trial will assess the safety and tolerability, as well as exploratory efficacy endpoints, in newly diagnosed multiple myeloma patients who have tested positive for minimal residual disease (MRD+) in first remission prior to autologous stem cell transplant (ASCT).

NK cells are part of the innate immune system, which is designed to recognize and destroy "non-self" or diseased cells in the body. However, tumor cells can evade detection by immune effector cells, allowing the tumor to advance. BHV-1100 targets a cell-surface protein, CD38, that is heavily overexpressed on multiple myeloma and binds to it, recruiting primed autologous cytokine induced memory-like (CIML) natural killer (NK) cells to destroy the tumor.

Charlie Conway, Ph.D., Chief Scientific Officer at Biohaven commented, "While many recent advances have been made to benefit multiple myeloma patients, most patients will unfortunately still relapse. We are excited to investigate BHV-1100 for its ability to recruit autologous CIML NK cells to the site of the tumor. Based on preclinical data from Biohaven Labs, we anticipate that our CD38 targeting ARM-enabled NK cells will kill CD38-positive multiple myeloma cells, and recruit other immune effector cells to assist in reducing the tumor burden."

Biohaven has initiated enrollment in the clinical trial and plans to enroll 25 patients for this single-center, open-label study (ClinicalTrials.gov Identifier: NCT04634435; https://clinicaltrials.gov/ct2/show/NCT04634435). The study will enroll newly diagnosed multiple myeloma patients who have minimal residual disease (MRD+) in first remission prior to an autologous stem cell transplant (ASCT).

David Spiegel M.D., PhD, inventor of the ARM technology and Professor of Chemistry and Pharmacology at Yale University, commented, "This is an important milestone in the development of the ARM therapeutic platform taking a novel technology from 'benchtop to bedside'. It also highlights Biohaven's commitment to benefit patients in need."

About ARMs: Antibody Recruiting Molecules

ARMs,antibody recruiting molecules, are engineered with modular components that are readily interchangeable, giving the platform tremendous flexibility and rapid development timelines. ARM compounds are being developed at Biohaven Labs to redirect a patient's own antibodies for therapeutic effect with multiple benefits over traditional monoclonal antibody therapies, including the potential for oral dosing. For BHV

1100, the ARM platform is being used to provide antigen targeting to NK cell-based therapies without genetic engineering. This NK cell targeting approach is also being investigated with allogeneic, or 'off-the-shelf', immune cell-based therapies.

About Multiple Myeloma Multiple myeloma is a type of blood cancer of the plasma cell that develops in the bone marrow, the soft tissue inside our bones. Healthy plasma cells produce antibodies, which are critical for the immune system's ability to recognize disease-causing entities, such as bacteria, viruses and tumor cells. In multiple myeloma, however, genetic abnormalities in a single plasma cell cause it to divide uncontrollably. This leads to the over-production of a single (monoclonal) antibody protein, referred to as an "M protein". Also, these cancerous cells divide to the point of crowding out normal, healthy cells that reside in the bone marrow. Many patients are diagnosed due to symptoms such as bone pain or fractures, kidney failure (thirst, dehydration, confusion), nerve pain, fever, and weakness. The American Cancer Society estimates that approximately 34,920 new cases will be diagnosed, and 12,410 deaths will occur in 2021 from multiple myeloma.

About BiohavenBiohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders and areas of unmet need. Biohaven's neuro-innovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer's disease, and spinocerebellar ataxia; and MPO inhibition for amyotrophic lateral sclerosis. More information about Biohaven is available atwww.biohavenpharma.com.

About PeptiDream PeptiDream Inc. is a public(Tokyo Stock Exchange 1st Section 4587) biopharmaceutical company founded in 2006 employing their proprietary Peptide Discovery Platform System (PDPS), a state-of-the-art highly versatile discovery platform which enables the production of highly diverse (trillions) non-standard peptide libraries with high efficiency, for the identification of highly potent and selective hit candidates, which then can be developed into peptide-based, small molecule-based, or peptide-drug-conjugate-based therapeutics. PeptiDream aspires to be a world leader in drug discovery and development to address unmet medical needs and improve the quality of life of patients worldwide. Further information regarding PeptiDream can be found at: http://www.peptidream.com.

Forward-looking Statement This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven's management about BHV-1100 as a treatment for multiple myeloma. Forward-looking statements include those related to: Biohaven's ability to effectively develop and commercialize BHV-1100, delays or problems in the supply or manufacture of BHV-1100, complying with applicableU.S.regulatory requirements, the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, the timing of planned interactions and filings with the FDA, the timing and outcome of expected regulatory filings, the potential commercialization of Biohaven's product candidates, the potential for Biohaven's product candidates to be firstin class or best in class therapies and the effectiveness and safety of Biohaven's product candidates. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of Biohaven's Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission onMarch 1, 2021, and Biohaven's subsequent filings with the Securities and Exchange Commission. The forward-looking statements are made as of this date and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

NURTEC and NURTEC ODT are registered trademarks of Biohaven Pharmaceutical Ireland DAC.Neuroinnovation is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

ARM is a trademark of Kleo Pharmaceuticals, Inc.

Biohaven ContactDr. Vlad CoricChief Executive OfficerVlad.Coric@biohavenpharma.com

Media ContactMike BeyerSam Brown Inc.mikebeyer@sambrown.com312-961-2502

View original content to download multimedia:

SOURCE Biohaven Pharmaceutical Holding Company Ltd.

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

Read the original:
Biohaven Enrolls Phase 1a/1b Clinical Trial of BHV-1100, Lead Asset from its ARM (Antibody Recruiting Molecule) Platform, in Combination with NK Cell...

Bone Marrow Stem Cells Comments Off on Biohaven Enrolls Phase 1a/1b Clinical Trial of BHV-1100, Lead Asset from its ARM (Antibody Recruiting Molecule) Platform, in Combination with NK Cell… | October 28th, 2021

DUBLIN--(BUSINESS WIRE)--The "Global Cell Therapy Market Size, Share & Trends Analysis Report by Use-type, by Therapy Type (Autologous, Allogenic), by Region (North America, Europe, Asia Pacific, Latin America, MEA), and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market size is expected to reach USD 23.0 billion by 2028 and is expected to expand at a CAGR of 14.5% from 2021 to 2028.

The emergence of new technologies to support the development of advanced cellular therapies has aided in market growth. Companies are leveraging new technologies not only for the expansion of their product portfolio but also for establishing out-licensing or co-development agreements with other entities to support their product development programs.

Cell-based therapies hold great potential for replacing, repairing, restoring, or regenerating damaged tissues, and organs. Researchers are making huge investments in the development of such effective and safe treatments as an alternative to conventional treatment strategies which can be further attributed to the market growth.

Out of all therapeutic areas, oncology has the highest number of ongoing clinical trials. T cells, CD34+ and/or CD133+ stem cells, mesenchymal stem/stromal cells are predominantly employed for clinical investigation.

The majority of biopharmaceutical entities have been affected by the COVID-19 pandemic, while several cellular therapy development companies have witnessed a strongly negative impact, which can be attributed to complications in logistics as well as the manufacturing models employed in this industry. In addition, substantial and stable funding is imperative to ensure successful commercial translation of cell-based therapeutics, a factor that was negatively affected in 2020, further affecting the market growth.

A survey conducted recently among executives of more than 15 European and U.S. cellular therapy companies indicated that disruption caused by the pandemic was significant, which demanded market entities to create strategies to sustain themselves and plan the next wave of innovative therapies.

Key issues faced by companies operating in the market include on-time delivery of therapies to patients at required clinical sites. In addition, the administration of these therapeutics poses several post-pandemic challenges. Hospitals are hesitant in offering services, owing to concerns over transmission of SARS-CoV-2, particularly to vulnerable individuals. Moreover, patients have not been able to visit cellular therapy centers either, owing to the lockdowns and travel bans.

Cell Therapy Market Report Highlights

Market Dynamics

Market driver analysis

Market restraint analysis

Market opportunity analysis

COVID-19 Impact Analysis

Challenges analysis

Opportunities analysis

Challenges in manufacturing cell therapies against COVID-19

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/n1u89x

More here:
Global Cell Therapy Market Research Report 2021: Opportunities with the Approval of Kymriah and Yescarta Across Various Countries -...

Bone Marrow Stem Cells Comments Off on Global Cell Therapy Market Research Report 2021: Opportunities with the Approval of Kymriah and Yescarta Across Various Countries -… | October 28th, 2021

Did you know that every three minutes someone receives a diagnosis of a blood cancer? According to the National Foundation for Cancer Research, blood cancers account for nearly 10% of new cancer diagnoses in the United States annually. That means that approximately 178,520 people will receive a diagnosis this year most commonly with leukemia, lymphoma or myeloma. And although survival rates have increased dramatically over the past 20 years, theres still a long way to go when it comes to this patient population.

In this special issue of CURE, youll read about some of the research going on in blood cancer right now, including new treatment options for acute myeloid leukemia and a novel therapy using BK virus-specific T cells to treat BK virus-associated hemorrhagic cystitis (a painful side effect of stem cell transplants).

Dive into the issue for two patient stories, one about a three-time acute lymphoblastic leukemia survivor who now works as a nurse on the bone marrow transplant floor at Barnes-Jewish Hospital in St. Louis. This year also marks the 20th anniversary of the Food and Drug Administrations approval of Gleevec (imatinib mesylate), the tyrosine kinase inhibitor that saved Mel Mann. Who is Mel Mann? He is the longest-living survivor who was treated with the drug as a part of a clinical trial for chronic myeloid leukemia.

And because cancer is not a disease one should have to face alone, our cover story looks at the responsibilities and experiences of caregivers to patients with blood cancer. Not only will you read about what theyve gone through, but youll also find extra resources and tips for navigating the patient-caregiver relationship.

As always, thank you for reading.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

More here:
New Treatments and Tips for Dealing With Blood Cancer - Curetoday.com

Bone Marrow Stem Cells Comments Off on New Treatments and Tips for Dealing With Blood Cancer – Curetoday.com | October 28th, 2021

Oncology drugs are therapeutics used to treat cancer, a group of diseases caused by uncontrolled growth and division of abnormal cells. Oncology drugs include a range of different types of medications, such as chemotherapy agents, targeted therapies, immunotherapies, and hormone therapies. There are also different types of drugs available to help treat the side effects from oncology drugs.

If you have cancer, its likely youll have a few options when it comes to treatment. Cancer treatment is constantly evolving and progressing. You now have more drug options to choose from than ever before.

A doctor may recommend that you treat your cancer with one or more of the following types of oncology drugs:

Chemotherapy drugs are chemical agents that work by destroying fast-growing cells in the body. Cancer cells grow and divide more rapidly than other cells. The goal of chemotherapy is to lower the total number of cancer cells in your body and reduce the chances that the cancer will spread to other parts of the body (metastasize).

There are at least 61 chemotherapy drugs approved in the United States. Your doctor may decide to treat your cancer with a a single chemotherapy drug or a combination of chemotherapy drugs.

Hormone therapy is used to treat cancers that rely on hormones to grow. This may include certain types of breast cancer and prostate cancer. Breast cancers may be fed by estrogen or progesterone, while prostate cancers often depend on the hormone androgen.

Some examples of hormone therapies include:

Targeted therapies are part of a new approach known as personalized medicine or precision medicine. Targeted therapies are able to seek out and kill cancerous cells without harming the normal cells in your body. These drugs work by blocking the molecular pathways that are critical to tumor growth.

To see if youre eligible for a particular targeted therapy, a doctor will first perform genetic or biomarker testing. The results of this testing will allow your doctor to make an informed decision about which drug is more likely to work for your type of cancer, based on the genetic mutations or other molecular characteristics of your tumor.

Examples of targeted oncology drugs include:

Immunotherapy is a type of targeted therapy that uses the bodys own immune system to attack cancer cells. Cancer cells sometimes have strategic ways of hiding from your immune system, but immunotherapies work by blocking these mechanisms.

Immunotherapies are approved to treat a variety of cancers. Examples include:

To counter the side effects of oncology drugs, your oncologist may prescribe additional medications. Examples include:

Oncology drugs have many benefits. Depending on your individual cancer, these drugs may:

On the other hand, oncology drugs often have side effects and risks. Chemotherapy, for example, can attack some of the normal cells in your body especially blood cells, skin, hair, and the cells lining the intestine and mouth. This can cause serious side effects that can negatively impact your quality of life.

Common side effects of chemotherapy include:

Targeted therapies and immunotherapies often have fewer side effects compared to chemotherapy since they only attack cancer cells while sparing healthy cells from harm, but they can still cause side effects. These may include:

Hormone therapies can block your bodys ability to make hormones, and can interfere with how hormones act in your body. Some common side effects of hormone therapies include:

Apart from oncology drugs, there are other treatments available for cancer, including:

Other therapies such as acupuncture, meditation, and herbal supplements may help with side effects of cancer treatment, but are ineffective at treating the cancer itself.

Yes, you can have a say in your cancer treatment, including making the decision to delay treatment or not have treatment at all. You can also seek a second opinion from another qualified doctor. Dont hesitate to ask your doctor for resources so you can do your own research on available treatments before you make a decision.

Ask your doctor about clinical trials taking place in your area. The National Institutes of Health (NIH) maintains a large database of clinical trials at clinicaltrials.gov. You can also search the National Cancer Institute (NCI)s online tool or contact them for help at 800-4-CANCER.

There are over 100 types of cancer, according to the National Cancer Institute (NCI). Your oncologist will formulate a recommended treatment plan based on the type of cancer you have, the stage or grade of the cancer, the characteristics of your tumor, your age, overall health, and several other considerations.

To determine the best treatment for you, your oncologist may perform molecular testing on your tumor among other imaging and blood tests. He or she will also likely consult with medical guidelines, such as those set forth by the National Comprehensive Cancer Network (NCCN).

To treat cancer, your doctor may recommend one type of oncology drug or a combination of drugs alongside surgery, radiation, or other types of treatment.

Before deciding to move forward with a cancer drug, learn as much as you can about the recommended treatment. Ask your doctor about benefits and risks of your recommended treatment plan and work together to make an informed decision.

Excerpt from:
Types of Oncology Drugs - Healthline

Bone Marrow Stem Cells Comments Off on Types of Oncology Drugs – Healthline | October 28th, 2021

DUARTE, Calif.--(BUSINESS WIRE)--As a 16-year-old high school sophomore, Julian Castaeda was focused on running track specifically, trying to run a mile in under five minutes. He was also planning to attend two camps that summer that would help him prepare for the rigors of college.

Despite being diagnosed with precursor B cell acute lymphoblastic leukemia at age 10 and receiving chemotherapy on and off for three and a half years, Castaeda had been in remission for two years. He had moved on from that difficult experience.

But in March 2017, Castaeda and his mother, Erica Palacios, again received devastating news the leukemia had returned. Castaeda received chemotherapy for a few months, but the cancer kept proliferating. Castaeda would need a hematopoietic stem cell transplant (more commonly referred to as a bone marrow transplant, or BMT) this time to put his cancer back into remission.

It was heartbreaking. I knew at that point that all my plans for sophomore year would be gone, Castaeda recalled.

But Castaeda was determined to get his life back. This was possible thanks to Johannes Eppler, 27, of Breisach, Germany, who joined the bone marrow registry via DKMS, an international nonprofit that is dedicated to the fight against blood cancers and blood disorders, including the recruitment of bone marrow donors. Castaeda received a bone marrow transplant on Aug. 2, 2017, putting the cancer into remission.

He has a big heart, Palacios said about Eppler. Hes an angel. He saved my son. I am thankful that people are willing to [donate].

Castaeda, who grew up in Bakersfield, California, and was treated by City of Hopes Joseph Rosenthal, M.D., M.H.C.M., the Barron Hilton Chair in Pediatrics, is now 20 years old and a junior at California State University Northridge. He also founded Bags of Love Foundation, a nonprofit that has delivered more than 200 care packages to young cancer patients in treatment and has provided $11,000 in scholarships to survivors.

On Friday, Oct. 15, Castaeda will meet his donor for the first time virtually during City of Hopes BMT Reunion. City of Hope, a pioneer and leader in BMT, has hosted a Celebration of Life for bone marrow, stem cell and cord blood transplant recipients, their families and donors for more than 40 years. The celebration honors children and adult cancer survivors, including those who have received autologous transplants, which use a patients own stem cells, and those who received an allogeneic procedure, which require a bone marrow or stem cell donation from a related or unrelated donor.

What began with a birthday cake and a single candle representing a patients first year free from cancer has grown into an annual extravaganza that draws thousands of cancer survivors, donors and families from around the world, as well as the doctors, nurses and staff who help them through the lifesaving therapy.

Each year, patient-donor meetings are the events emotional highlight. Many recipients, though overwhelmed with curiosity and the need to express their gratitude, can only dream of meeting the stranger who saved their lives. City of Hope is making that dream come true for Castaeda, as well as Dona Garrish, a Fullerton, California resident and retired school teacher. Her donor was Michael Fischer, 35, of Wlkau, Germany.

Garrish, 75, received her transplant on March 22, 2017, after it was delayed several times due to infections and other complications that prevented her from going through with the treatment. Garrish, who was diagnosed with acute myeloid leukemia, felt a strong connection to Fischer from the first time a City of Hope employee told her a German male, whom she had never met, was a perfect match for her. She refers to him as her gift from God and her angel on Earth.

He unknowingly encouraged me to fight harder and not to become discouraged, as someday I wanted to meet him and thank him, she added. Garrish recalled watching two patients meeting their donors at the 2017 BMT Reunion. The reunions were held in front of City of Hope Helford Clinical Research Hospital, where Garrish was recovering from her transplant.

While tethered to her IV pole, Garrish looked down from the hospitals sixth floor and said, Thats what I want to do.

City of Hope nurses, doctors and staff were constantly there supporting me every step of the way, even when I couldnt take a single step, said Garrish, who was treated by City of Hopes Liana Nikolaenko, M.D. The timing was urgent, my battle was rough and long, but I live, breathe and enjoy life today because of City of Hope.

Other event highlights include videos of grateful patients wearing the signature BMT buttons that display the number of years since their transplants, comedy by City of Hope BMT patient Sean Kent and a dance/song performed by BMT nurses, known as the Marrowettes. There will be special guest appearances by a Los Angeles Dodger and Katharina Harf, executive chairwoman of DKMS U.S., to congratulate patients, their donors and the BMT program.

During our annual BMT reunion, we express our most heartfelt thanks to the many selfless individuals who each year donate their bone marrow or stem cells to save a persons life, said Stephen J. Forman, M.D., director of City of Hopes Hematologic Malignancies Research Institute and former chair of its Department of Hematology & Hematopoietic Cell Transplantation. Whether the donor is a patients family member or a person she or he has never met, we are all extremely grateful that these donors took the time to donate and gave someone a second chance at life.

About City of Hopes BMT program

City of Hopes BMT program has performed more than 17,000 transplants, making it one of the largest and most successful programs in the nation. The institution has the largest BMT program in California, performing over 700 transplants annually, and is among the top three hospitals in the nation in terms of total transplants performed.

Over the years, City of Hope has also helped pioneer several BMT innovations. In addition to being one of the first institutions to perform BMTs in older adults, it was one of the first programs to show that BMTs could be safely performed for patients with HIV. City of Hope has had growing success with nonrelated matched donors and, most recently, half matched family donors.

City of Hopes BMT program is the only one in the nation that has had one-year survival above the expected rate for 15 consecutive years, based on analysis by the Center for International Blood and Marrow Transplant Research.

City of Hope was also one of the first programs to develop a treatment for prevention of cytomegalovirus (CMV), a common and potentially deadly infection after transplant, which has nearly eliminated the threat of CMV for BMT patients. The institution successfully conducted clinical trials of a CMV vaccine developed at City of Hope. As a pioneer in the development of CAR T cells to treat cancer, City of Hope is also testing how this form of cancer immunotherapy can help patients have a more successful transplant.

In addition, Be The Match at City of Hope last year added more than 13,000 new volunteers willing to save a life when they match a patient who needs a bone marrow transplant. In total, nearly 300,000 potential donors have signed up via City of Hope, motivated by a patient at the cancer center. Be The Match encourages healthy individuals between the ages of 18 and 40 to take the first step of registering by texting COHSAVES to 61474. To learn more about the donation process, visit Be The Match at City of Hopes website.

The public can register to view the event here.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

Read more here:
College Student and Retired Teacher to Thank Stem Cell Donors They've Never Met for Saving Their Lives During City of Hope's 45th Bone Marrow...

Bone Marrow Stem Cells Comments Off on College Student and Retired Teacher to Thank Stem Cell Donors They’ve Never Met for Saving Their Lives During City of Hope’s 45th Bone Marrow… | October 16th, 2021

Stem Cell Therapy Market Research Report by Cell Source (Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs), by Type (Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy), by Therapeutic Application, by End-User, by Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2026 - Cumulative Impact of COVID-19

New York, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region - Global Forecast to 2026 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06175517/?utm_source=GNW

The Global Stem Cell Therapy Market size was estimated at USD 202.87 million in 2020 and expected to reach USD 240.88 million in 2021, at a CAGR 19.07% to reach USD 578.27 million by 2026.

Market Statistics:The report provides market sizing and forecast across five major currencies - USD, EUR GBP, JPY, and AUD. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2019 are considered historical years, 2020 as the base year, 2021 as the estimated year, and years from 2022 to 2026 are considered the forecast period.

Market Segmentation & Coverage:This research report categorizes the Stem Cell Therapy to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Cell Source, the market was studied across Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs.

Based on Type, the market was studied across Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy.

Based on Therapeutic Application, the market was studied across Cardiovascular Diseases Surgeries, Inflammatory & Autoimmune Diseases, Musculoskeletal Disorders, Neurological Disorders, Other Therapeutic Applications, and Wounds & Injuries.

Based on End-User, the market was studied across Academic and Research Centers, Ambulatory Surgical Centers (ASCs), and Hospitals & Clinics.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, and Thailand. The Europe, Middle East & Africa is further studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Stem Cell Therapy Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Stem Cell Therapy Market, including Advanced Cell Technology, Inc., AlloSource, Inc., Anterogen Co., Ltd., Bioheart Inc., BioTime, Inc., BrainStorm Cell Therapeutics Inc., Celgene Corporation, Cellartis AB, CellGenix GmbH, Cellular Engineering Technologies Inc., Gamida Cell Ltd, Gilead Sciences, Inc., Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Lonza Group AG, Medipost Co., Ltd., Nuvasive, Inc., Osiris Therapeutics, Inc., Pharmicell Co., Ltd., Pluristem Therapeutics Inc., PromoCell GmbH, RTI Surgical, Inc., STEMCELL Technologies, Inc., Takeda Pharmaceutical Company Limited, Vericel Corporation, and VistaGen Therapeutics, Inc..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Stem Cell Therapy Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Stem Cell Therapy Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Stem Cell Therapy Market?4. What is the competitive strategic window for opportunities in the Global Stem Cell Therapy Market?5. What are the technology trends and regulatory frameworks in the Global Stem Cell Therapy Market?6. What is the market share of the leading vendors in the Global Stem Cell Therapy Market?7. What modes and strategic moves are considered suitable for entering the Global Stem Cell Therapy Market?Read the full report: https://www.reportlinker.com/p06175517/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Story continues

Go here to read the rest:
Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region - Global Forecast to 2026 -...

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region – Global Forecast to 2026 -… | October 16th, 2021

250 Pages Rheumatoid Arthritis Stem Cell Therapy Market Survey by Fact MR, A Leading Business and Competitive Intelligence Provider

Rheumatoid arthritis stem cell therapy has been demonstrated to induce profound healing activity, halt arthritic conditions, and in many cases, reverse and regenerate joint tissue. Today, bone marrow transplant, adipose or fat-derived stem cells, and allogeneic mesenchymal stem cells (human umbilical cord tissue) are used for rheumatoid arthritis stem cell therapy.

The Market Research Survey by Fact.MR, highlights the key reasons behind increasing demand and sales of Rheumatoid Arthritis Stem Cell Therapy.Rheumatoid Arthritis Stem Cell Therapy market driversand constraints, threats and opportunities, regional segmentation and opportunity assessment, end-use/application prospects review are addressed in the Rheumatoid Arthritis Stem Cell Therapy market survey report. The survey report provides a comprehensive analysis of Rheumatoid Arthritis Stem Cell Therapy market key trends and insights on Rheumatoid Arthritis Stem Cell Therapy market size and share.

To Get In-depth Insights Request for Brochure here https://www.factmr.com/connectus/sample?flag=B&rep_id=1001

Rheumatoid Arthritis Stem Cell Therapy Market: Segmentation

Tentatively, the global rheumatoid arthritis stem cell therapy market can be segmented on the basis of treatment type, application, end user and geography.

Based on treatment type, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Based on application, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Based on distribution channel, the global rheumatoid arthritis stem cell therapy market can be segmented into:

Key questions answered in Rheumatoid Arthritis Stem Cell Therapy Market Survey Report:

Request Customized Report as Per Your Requirements https://www.factmr.com/connectus/sample?flag=RC&rep_id=1001

Rheumatoid Arthritis Stem Cell Therapy Market: Key Players

The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain.

Essential Takeaways from the this Market Report

Enquire Before Buying Here https://www.factmr.com/connectus/sample?flag=EB&rep_id=1001

The Report Covers Exhaustive Analysis On:

Explore Fact.MRs Coverage on theHealthcareDomain:

Hemostasis Valves MarketAccording to the latest research by Fact.MR, the Hemostasis Valves Market is set to witness steady growth during 2021-2031. The increasing advancement in the hemostasis valves industry will offer lucrative opportunities and an increasing number of interventional procedures is propelling the market growth.

Cosmetic Dentistry MarketThe global cosmetic dentistry market is estimated to witness significant growth during the forecast period of 2021-2031. This growth is factored in due to the rising consumer awareness and well-defined grooming interest in the aesthetical value of oral care.

Creatinine Testing MarketCreatinine testing involves the measurement of the level of creatinine, which is a product of muscle creatine catabolism and is thus a reliable indicator of kidney function by an indication that a rise in the level of creatinine may indicate Acute Kidney Injury or Chronic Kidney Disease.

About Fact.MR

Market research and consulting agency with a difference! Thats why 80% of Fortune 1,000 companies trust us for making their most critical decisions. We have offices in US and Dublin, whereas our global headquarter is in Dubai. While our experienced consultants employ the latest technologies to extract hard-to-find insights, we believe our USP is the trust clients have on our expertise. Spanning a wide range from automotive & industry 4.0 to healthcare & retail, our coverage is expansive, but we ensure even the most niche categories are analyzed. Reach out to us with your goals, and well be an able research partner.

Contact:US Sales Office:11140 Rockville PikeSuite 400Rockville, MD 20852United StatesTel: +1 (628) 251-1583E-Mail:[emailprotected]

Corporate Headquarter:Unit No: AU-01-H Gold Tower (AU),Plot No: JLT-PH1-I3A,Jumeirah Lakes Towers,Dubai, United Arab Emirates

The post Rheumatoid Arthritis Stem Cell Therapy Market By Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant, Adipose Tissue Stem Cells) and By Application (Rheumatoid Arthritis Stem Cell Therapy) Forecast to 2021-2031 appeared first on The Swiss Times.

Read more:
Rheumatoid Arthritis Stem Cell Therapy Market By Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant, Adipose Tissue Stem Cells) and By...

Bone Marrow Stem Cells Comments Off on Rheumatoid Arthritis Stem Cell Therapy Market By Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant, Adipose Tissue Stem Cells) and By… | October 16th, 2021

By Sam Boughedda

Investing.com Shares of biotechnology firm Jasper Therapeutics (NASDAQ:JSPR) soared 112% after Oppenheimer analyst Jay Olson initiated coverage of the company with a buy-equivalent outperform rating and $21 price target.

In a research note released after the close on Tuesday, Olson told investors that he sees Jasper as an "emerging leader" in developing novel targeted conditioning agents for hematopoietic stem cell transplantation.

The company's lead candidate, JSP191, is used to remove hematopoietic stem cells from bone marrow before a transplant.

Olsen believes Jasper Therapeutics is well-positioned to conceivably change what he describes as the "decades-old standard" hematopoietic stem cell transplantation for various diseases "either as a standalone conditioning agent or as the backbone of the conditioning regimen."

At the beginning of October, biopharmaceutical firm Amgen (NASDAQ:AMGN) disclosed a 7.4% stake in Jasper.

Related Articles

Jasper Therapeutics Stock Soars after Oppenheimer Calls it a Buy

Tech stocks lift Nasdaq, banks weigh on S&P 500, Dow

Plug Power Up on Morgan Stanley Upgrade, Deals for Clean Fuel

Read more here:
Jasper Therapeutics Stock Soars after Oppenheimer Calls it a Buy - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Stock Soars after Oppenheimer Calls it a Buy – Yahoo Finance | October 16th, 2021

By Alycea Wood and Kamran Zamanian, Ph.D., iData Research Inc.

When choosing a treatment option for orthopedic procedures, biomaterials have become widely popular. Biomaterials are biomedical materials that can be safely implanted or injected into the body and are, more often than not, a form of biologically active tissue themselves.1 Their prevalence in orthopedic procedures is largely attributed to their ability to mimic the structure or properties of osseous tissue. Many products can offer a number of beneficial properties, such as promoting bone growth within the body (osteoinduction), promoting bone growth on the biomaterials scaffold (osteoconduction), or inducing the differentiation of stem cells into osseous tissue (osteogenesis).2,3 The orthopedic biomaterials market includes bone graft substitutes, growth factors, cellular allografts, cell therapy, hyaluronic acid viscosupplementation, and even cartilage repair devices. The U.S. orthopedic biomaterials market saw a dramatic dip and subsequent rebound in market value in 2020 and 2021 as a result of the COVID-19 pandemic. After recovery, the market is projected to see a consistently steady growth in value within the next few years. This growth is expected to be seen across all market segments apart from cellular allograft devices (Figure 1).4

Figure 1: Orthopedic biomaterials market growth trends by market segment, U.S., 20192028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

Cellular allografts may consist of either allograft bone (donated bone tissue) in conjunction with adipose-derived adult stem cells or viable cells within a cortical cancellous bone matrix.4,5 In both scenarios, the devices provide osteoconduction, osteoinduction, and osteogenesis to the site of implantation. Historically, this market had seen promising growth because of the optimal environment for bone growth they can provide.6 The cellular allograft market is projected to see a much slower rate of growth in market value in the next few years despite its market potential due to increased constraints on the market itself. These include, but are not limited to, direct federal restriction on product research, cost of product development, and product recalls.4

There is a strong interest in the scientific community in embryonic stem cell (ESC) research, which is largely due to ESCs high differentiability when compared to adult stem cell (ASC) lines.7 The development of new cellular allograft products, and the resulting growth in the market, is dependent on continued research into realizing the full medical potential of stem cell use. In 2019, the Trump administration eliminated federal funding of research relying on ESC tissue and instituted the National Institutes of Health (NIH) Human Fetal Tissue Research Ethics Advisory Board. This negatively impacted a large number of studies in progress while restricting the ability of new projects to commence.8,9,10 While the board was in effect, it rejected all but one application for funding.11 In April 2021, the Biden administration removed both the board and the restrictions on current projects, allowing federally funded research using ESC to continue.12 This was not the first instance where restrictions were placed and then removed on ESC research. In March 2009, President Obama signed an executive order to overturn the Bush administrations restriction on ESC research.13

The repeated restrictions on ESC research have a number of long-term ramifications in the development and implementation of new, effective cellular allograft treatments. Scientists may need to divert their research efforts away from stem cells and into less turbulent fields, and the progress of product development slows down as studies have funding pulled; this may contribute to increased hesitancy by end users to use stem cell products. Reduced research efforts, funding, and faith in stem cell products will continue to limit the growth of the cellular allograft market.

Cellular allografts tend to be notably more expensive than others within the broader cell-based biomaterials market. When compared to the cell therapy market, which uses either concentrated platelet-rich plasma (PRP) or bone marrow aspirate concentrate (BMAC) in its treatment, the cellular allograft average selling price (ASP) sits over three times higher (Figure 2).3

Figure 2: The average selling price (ASP) of the cellular allograft & cell therapy markets, U.S., 20182028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

The ASP of the cellular allograft market is so high because of the prohibitively expensive cost of developing new products. During the development process, reliable efficacy of a new product is uncertain, and using protein markers to help distinguish stem cell types can be very challenging.4,14 The increased cost of product development acts as a significant barrier to parties looking to enter the U.S. cellular allograft market. The result is fewer products entering and rejuvenating the market, and existing products sit at prohibitively high prices as they have low direct competition.4 The high cost of cellular allograft products hinders new entrants from introducing products and prevents end users from being able to afford existing ones. A broader consequence of this is end users turning to more affordable orthopedic biomaterial types to reduce procedural costs.

Any product recalls within the U.S. orthopedic biomaterials market, especially within cell-based therapies, will negatively impact the use of cellular allografts. This impact is amplified when a recall occurs within the market segment itself, which was seen in the cellular allograft market as recently as June 2021. On June 2, 2021, Aziyo Biologics recalled its product FiberCel following a number of patients contracting tuberculosis.15 Recalls deter the use of cell-based products through increased distrust in the safety of the products themselves, potential public backlash against the specific product itself or, in the market more broadly, reduced reimbursement from health insurance providers as well as the introduction of more restrictive FDA protocols. This is another reason why effective, safe, cell-based products are necessary for the cellular allograft market to move forward.

Conclusion

Federal research restrictions, high development costs, and product recalls all negatively impact the growth of the cellular allograft market in the United States. These constraints contribute to the projected low growth rate in market value in the coming years despite the potential uses for stem cell therapies. To shift the tide back toward growth, the cellular allograft space will need consistent research progress through large-scale studies, more affordable product development, and strict enforcement of sanitization protocols for existing products to prevent future product recalls. The large therapeutic potential of stem cell therapy has been discussed extensively in scientific and popular literature, but it may take a while to realize it.

References

About The Authors:

Alycea Wood is a research analyst at iData Research. She develops and composes syndicated research projects regarding the medical device industry, and published the U.S. Orthopedic Biomaterials report series.

Kamran Zamanian, Ph.D., is CEO and founding partner of iData Research. He has spent over 20 years working in the market research industry with a dedication to the study of medical devices used in the health of patients all over the globe.

About iData Research

For 16 years, iData Research has been a strong advocate for data-driven decision-making within the global medical device, dental, and pharmaceutical industries. By providing custom research and consulting solutions, iData empowers its clients to trust the source of data and make important strategic decisions with confidence.

Read the rest here:
The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online

Bone Marrow Stem Cells Comments Off on The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market – Med Device Online | October 16th, 2021

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

T-Cell Immune Response to COVID-19 Vaccines and Natural Infections

Much of the discussions and news reports about immune responses to vaccines and COVID-19 revolve around antibody levels. Much less has been said about T-cells, which provide longer-term protection. Researchers atGladstone Institutesconducteda detailed T-cell survey before and after COVID-19 immunization, which they published ineLife. They concluded that the Pfizer-BioNTech and Moderna mRNA vaccines create long-term populations of T-cells that recognize multiple SARS-COV-2 virus variants. They also found key differences in the T-cell responses in people who had COVID-19 infections before vaccination compared to people who had never been infected.

Overall, our data support the idea that vaccines are eliciting a very robust T-cell response in healthy individuals, said Nadia Roan, senior author of the study and Gladstone Associate Investigator. But they also suggest there may be some ways to improve them further, by getting more of the vaccine-elicited T-cells to park themselves in the respiratory tract.

Antibodies produced by B-cells quickly recognize viruses, target them, and prevent infection by destroying the viruses. T-cells, however, identify and destroy cells that are already infected. Antibodies are better at stopping initial infection, but T-cells typically last longer after an initial infection or vaccine. At that point they are better at fighting off disease in its early stages, which prevents severe symptoms. But T-cells are very diverse and difficult to analyze. Some subsets respond differently to infected cells and behave differently, while others have different functions within the overall T-cell immune response.

One key finding was that in people who had not been previously infected, the T-cell responses become stronger in quantity and quality after the second dose of the vaccine. But in people who had previously had COVID-19, there was not much of a change between the first and second vaccine dose.

Blood Biomarkers Provide Warning Signs of Dementia

Investigators at theGerman Center for Neurodegenerative Diseases (DZNE)identifiedmolecules in the blood that potentially warn of impending dementia. The research study included several university hospitals across Germany. The biomarkers were based on measuring levels of microRNAs. They say that the technique isnt ready yet for practical use, but they hope to develop a simple blood test. MicroRNAs have regulatory properties, influencing protein production and metabolism. In tests in humans, mice and cell cultures, they found three microRNAs whose levels were linked to mental performance. The three microRNAs also influence neuro-inflammation and neuroplasticity, including the ability of neurons to establish connections with each other.

Stem Cell Population Essential for Bone Regeneration

Researchers at theUniversity of Tsukuba, Japan,identifieda subpopulation of mesenchymal stem cells that play a major role in bone healing. The stem cells are found in the bone marrow and express the marker CD73. When a bone fracture heals, it moves through a series of stages, including clotted blood forming at the fracture. This clot is replaced by fibrous tissue and cartilage, then by a hard bony callus. The bone is then remodeled, with regular bone replacing the hard callus. They found that the generation of the callus is critically dependent on recruiting MSCs from the surrounding tissue and bone marrow. They observed the CD73-positive MSCs migrating toward the fracture site and forming new cartilage and bone cells. When they grafted CD73-positive MSCs into the fracture, they noted enhanced healing processes.

Antiviral Molecule Prevents SARS-CoV-2 from Entering Cells

Scientists atWashington University School of Medicinein St. Louisdevelopeda compound that prevents the SARS-CoV-2 virus, which causes COVID-19, from entering cells. The compound is called MM3122 and has been studied in cell cultures and in mice. MM3122 targets a key human protein called transmembrane serine protease 2 (TMPRSS2), which coronaviruses use to enter and infect human cells. Once the virus attached onto a cell in the epithelia of the airway, the TMPRSS2 protein cuts the viral spike protein, which activates the spike protein to mediate fusion of the viral and cellular membranesstarting the infection process. In cell cultures, MM3122 protected cells from viral damage better than remdesivir,Gilead Sciences antiviral against COVID-19; and an acute safety assay in mice demonstrated that large doses of MM3122 given for seven days did not cause noticeable issues. The compound also was effective against SARS-CoV, the virus behind SARS, and MERS-CoV, the coronavirus that causes MERS. The researchers are now working with researchers at the NIH to test it in animal models of COVID-19. They are also working on an oral version of the injectable compound.

Specific Personality Traits Might Signal Pending Alzheimers

Researchers atFlorida State Universityfoundthat specific changes in the brain linked with Alzheimers disease are often visible earlier in people with personality traits associated with the disease. The research focused on two traits: neuroticism, or a predisposition for negative emotions, and conscientiousness, linked to a tendency to be careful, organized, goal-directed and responsible. They found that people with amyloid and tau deposits, proteins linked to Alzheimers disease in the brain, were identified in participants who scored higher in neuroticism levels and lower in conscientiousness. The study suggests that personality traits might help protect against Alzheimers and other brain diseases by delaying or preventing the neuropathology for people strong in conscientiousness and low in neuroticism.

Why We Overeat

Astudyfrom theUniversity of Washington School of Medicine/UW Medicinereported on the function of glutamatergic neurons in mice. These neurons communicate to the lateral habenula, a brain region associated with the pathophysiology of depression, and the ventral tegmental area, which is involved in motivation, reward and addiction. They found that when mice are eating, the neurons in the lateral habenula are more responsive than the neurons in the ventral tegmental area. They suggest that these neurons might play a bigger role in guiding feeding. In addition, they studied the influence of the leptin and ghrelin hormones, which are believed to regulate behavior via the mesolimbic dopamine system, part of the reward pathway. The research adds additional insight into satiety and why people or at least mice overeat.

We found these cells are not a monolithic group, and that different flavors of these cells do different things, said Garret Stuber, a joint UW professor of anesthesiology and pain medicine and pharmacology, the papers senior author.

Read the rest here:
Research Roundup: T-Cell Immune Response to COVID-19 Vaccines and More - BioSpace

Bone Marrow Stem Cells Comments Off on Research Roundup: T-Cell Immune Response to COVID-19 Vaccines and More – BioSpace | October 16th, 2021

Nine accepted abstracts demonstrate broad potential of the companys HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

Story continues

Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

Read more:
Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress – Yahoo Finance | October 16th, 2021

TEL AVIV, Israel, Oct. 13, 2021 /PRNewswire/ --BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company's view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin's lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient's own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient's stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a late clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company's lead program, Motixafortide (BL-8040), is a cancer therapy platform that was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation, as well as reporting positive results from a pre-planned pharmacoeconomic study, and is currently in preparations for an NDA submission. Motixafortide was also successfully evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a clinical trial collaboration agreement with MSD (BioLineRx owns all rights to Motixafortide), and is currently being studied in combination with LIBTAYO and chemotherapy as a first-line PDAC therapy.

BioLineRx is also developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being investigated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for additional financing; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; risks related to the COVID-19 pandemic; and statements as to the impact of the political and security situation in Israel on BioLineRx's business. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on February 23, 2021. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564[emailprotected]

or

Moran MeirLifeSci Advisors, LLC+972-54-476-4945[emailprotected]

SOURCE BioLineRx Ltd.

See the article here:
BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -...

Bone Marrow Stem Cells Comments Off on BioLineRx Announces Positive Results from Pharmacoeconomic Study Positioning Motixafortide as Potential Standard of Care in Stem Cell Mobilization -… | October 16th, 2021

NEW YORK, Oct. 14, 2021 /PRNewswire/ --BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, will present findings from a multicenter, open label clinical trial of NurOwn in progressive multiple sclerosis. The study, "Phase 2 Safety and Efficacy Study of Intrathecal MSC-NTF cells in Progressive Multiple Sclerosis," will be delivered in an oral presentation today at the fully digital37thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

The Phase 2 clinical trial was designed to evaluate intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS. The study achieved the primary endpoint of safety and tolerability. It demonstrated a reduction of neuroinflammatory biomarkers and an increase in neuroprotective biomarkers in the cerebrospinal fluid (CSF) and consistent improvement across MS functional outcome measures, including measures of walking, upper extremity function, vision and cognition.

"We were pleased that this study demonstrated safety, preliminary evidence of efficacy and relevant biomarker outcomes in patients with progressive multiple sclerosis, in an area of high unmet need," said Jeffrey Cohen, M.D., Director of Experimental Therapeutics at the Cleveland Clinic Mellen Center for MS and principal investigator for the trial. "These results should be confirmed in a randomized placebo-controlled trial."

The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses from the National Multiple Sclerosis Society Fast-Forward Program. It was conducted at four U.S. MS centers of excellence:

"We very much appreciate the tremendous collaboration among many premier organizations, for their generous sharing of expertise, support and data, which enabled the important balance between scientific rigor and ethical treatment of progressive MS participants in the trial," said Ralph Kern, M.D., MHSc., President and Chief Medical Officer, Brainstorm Cell Therapeutics. "We are holding discussions with key MS experts, and seeking guidance from the FDA to determine next steps for the development of NurOwn in progressive MS."

"The National MS Society is pleased to support the biomarker portion of this study through our commercial funding program Fast Forward," said Mark Allegretta, Ph.D., Vice President, Research. "We're encouraged to see evidence that the biomarker analysis showed proof of concept for detecting neuroprotection and reduced inflammation."

About the trial

The Phase 2 open-label studyevaluated the safety and efficacy of intrathecal administration of autologous MSC-NTF cells in patients with primary or secondary progressive MS. The primary study endpoint was safety and tolerability. Secondary efficacy endpoints included: timed 25-foot walk (T25FW); 9-Hole Peg Test (9-HPT); Low Contrast Letter Acuity (LCLA); Symbol Digit Modalities Test (SDMT); 12 item MS Walking Scale (MSWS-12); as well as cerebrospinal fluid (CSF) and blood biomarkers. Clinical efficacy outcomes were compared with matched (n=48) participants in the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry, Tanuja Chitnis, MD Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases, and 255 patient randomized double blind placebo controlled NN-102 SPRINT-MS Study, courtesy NIH/NINDS, PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic, CTR: NCT01982942. Baseline characteristics from these two cohorts were similar allowing for comparison of efficacy results, comparisons with SPRINT-MS were with the placebo arm of this study.

Mean age of participants was 47 years, 56% were female, and mean baseline EDSS score was 5.4. 18 participants were treated, 16 (80%) received all 3 treatments and completed the entire study; 2 study discontinuations were due to procedure-related adverse events. No deaths or treatment-related adverse events due to worsening of MS were observed.

In responder analyses, 14% and 13% of MSC-NTF treated participants showed at least a 25% improvement in T25FW and 9-HPT (combined hands) respectively, compared to 5% and 0% in matched CLIMB patients and 9% and 3% in SPRINT. Twenty-seven percent (27%) showed at least an 8-letter improvement in LCLA (binocular, 2.5% threshold) and 67% showed at least a 3-point improvement in SDMT, compared to 6% and 18% in CLIMB and 13% and 35% in SPRINT, respectively.

Mean improvements of +0.10 ft/sec in T25FW and -0.23 sec in 9-HPT (combined hands), were observed in MSC-NTF treated participants, compared to a mean worsening of -0.07 ft/sec and +0.49 sec in CLIMB and -0.06 ft/sec and +0.28 sec in SPRINT, respectively. MSC-NTF treated participants showed a mean improvement of +3.3 letters in LCLA (binocular, 2.5% threshold) and 3.8 points in SDMT, compared to a mean worsening of -1.07 letters in LCLA (binocular, 2.5% threshold) and mean improvement of +0.10 in SDMT, in CLIMB and -0.6 and -0.1 in SPRINT. In addition the MSFC-4 Composite Z-score of T25W, 9-HPT, SDMT and LCLA showed a 0.18 point improvement in MSC-NTF treated participants, while CLIMB and SPRINT showed decreases of -0.02 and -0.05.

Furthermore, 38% of treated patients showed at least a 10-point improvement in the MSWS-12 a patient reported outcome that evaluates the impact of MS on walking function, whereas this outcome was not evaluated in CLIMB or SPRINT.

CSF biomarkers obtained at 3 consecutive time points, showed increases in neuroprotective molecules (VEGF, HGF, NCAM-1,Follistatin, Fetuin-A) and decreases in neuroinflammatory biomarkers (MCP-1, SDF-1, sCD27 and Osteopontin).

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Investor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 (646) 791-9729egoldstein@lifesciadvisors.com

Media:Mariesa Kemble kemblem@mac.com

View original content:

SOURCE Brainstorm Cell Therapeutics Inc

Link:
Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in...

Bone Marrow Stem Cells Comments Off on Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in… | October 16th, 2021

GRAND RAPIDS, Mich. As day turned to darkness, a crowd gathered at Monroe North on Saturday in Grand Rapids for the annual Light the Night celebration.

Its an inspirational community event, said Anne Bradley, Light the Night campaign development manager.

People eventually made their way to the street and showed support in each stride for blood cancer patients, their families and the countless others impacted by the disease.

Among the participants was Colette Smiley. Her white light gave a hint as to why she showed up.

I am a leukemia survivor, said Smiley. It completely alters your life.

In 2014, doctors diagnosed Smiley with Acute Myeloid Leukemia, or AML. Its an often fatal cancer that forms in a persons blood and bone marrow.

Smiley credits her treatment plan and a strangers stem cells for helping her enter remission within weeks of her diagnosis.

I come here to celebrate, to celebrate each re-birthday that I have, but to also thank LLS for [what] they do each and every day and each and every year for blood cancer patients, said Smiley.

According to the Leukemia and Lymphoma Society, blood cancers impact an estimated 1.5 million people in the United States, with a person diagnosed once every three minutes.

Leukemia is the most common cancer diagnosed in children and adolescents younger than 20 years old. It accounts for 25.1 percent of all cancer cases in the age group.

Symptoms of blood cancers often only appear in advanced stages, but can include ones similar to a severe cold or flu. Survival rates at one point hovered around 3 percent, but now, its up to 95 percent.

Its our goal and our mission to end blood cancer and to improve the quality of life for patients and their families, said Bradley.

All proceeds from Light the Night benefit LLS, which says 80 cents of every dollars goes directly to their mission.

Attendees, though, found a deeper value in the night.

We all live different stories, said Smiley. We all work in different places and our families are different and our backgrounds are different, but something brought us together that we certainly didnt wish for, blood cancer, but its brought us together and we can support each other now.

Light the Night hopes to collect $1 million in Michigan.

To donate, click here.

Follow FOX 17: Facebook - Twitter - Instagram - YouTube

Continue reading here:
'Light the Night' returns to Grand Rapids, raising awareness, donations for blood cancers - Fox17

Bone Marrow Stem Cells Comments Off on ‘Light the Night’ returns to Grand Rapids, raising awareness, donations for blood cancers – Fox17 | October 16th, 2021

A genetic syndrome that affects bones

Osteogenesis Imperfecta (OI) is a hereditary disorder occurring in 1:10,000 births and characterised by osteopenia (bone loss) and skeletal fragility (fractures). Secondary features include short stature, skeletal deformities, blue sclera and dentinogenesis imperfect. (1) There is a large clinical variability in OI, and severity ranges from mild to lethal, based on radiological characteristics. Genetically, OI is a collagen-related syndrome. Type I collagen is a heterotrimeric helical structure synthesized by bone-forming cells (osteoblasts), and it constitutes the most abundant protein of the skeletal organic matrix. (2) Synthesis of type I collagen is a complex process. (3) Collagen molecules are cross-linked into fibrils (which confer tensile strength to the bones). Those are then mineralised by hydroxy-apatites (which provides compressive strength) and assembled into fibres.

Dominant mutations in either the COL1A1 or the COLA1A2 genes are responsible for up to 90% of all OI cases. These mutations (more than 1,000 of which have been identified) lead to impairment of collagen structure and production, which in either quantitative or qualitative bone extracellular matrix (ECM) defects. Mutations affecting ECM structure have serious health consequences because the skeleton protects visceral organs and the central nervous system and provides structural support. Bones also store fat in the yellow bone marrow found within the medullary cavity, whilst the red marrow located at the end of long bones is the site of haematopoiesis. In addition, the ECM constitutes a reservoir of phosphate, calcium, and growth factors, and is involved in trapping dangerous molecules.

Stem cell therapy for OI aims to improve bone quality by harnessing the ability of mesenchymal stem cells (MSC) to differentiate into osteoblasts, with the rationale that donor cells would engraft into bones, produce normal collagen and function as a cell replacement. Stem cells have, therefore, been proposed for the treatment of OI (4) and, in particular, prenatal foetal stem cell therapy (foetal stem cells injected into a foetus, i.e. foetal-to-foetal) approach, which offers a promising route to effective treatment. (5) Human foetal stem cells are more primitive than stem cells isolated from adult tissues and present advantageous characteristics compared to their adult counterparts, i.e. they possess a higher level of plasticity, differentiate more readily into specific lineages, grow faster, senesce later, express higher levels of adhesion molecules, and are smaller in size. (6,7) Prenatal cell therapy capitalises on the small size of the foetus and its immunological naivete. In addition, stem cells delivered in utero benefit from the expansion of endogenous stem cells and may prevent organ injury before irreversible damage. (8)

However, human foetal stem cells used are isolated from either foetal blood drawn by cardiac puncture, either during termination of pregnancy or during ongoing pregnancy, albeit using an invasive procedure associated with a high risk of morbidity and mortality for both the foetus and the mother (9). Foetal cells can also be isolated from the first-trimester liver (following termination of pregnancy) and such cells are currently used in The Boost Brittle Bones Before Birth (BOOSTB4) clinical trial, which aims to investigate the safety and efficacy of transplanting foetal derived MSCs prenatally and/or in early postnatal life to treat severe Osteogenesis Imperfecta (OI) (10). Alternatively, foetal stem cells can be isolated during ongoing pregnancy from the amniotic fluid, either during mid-trimester amniocentesis or at birth (11,12) or from the chorionic villi of the placenta during first-trimester chorionic villi sampling (13).

We have demonstrated that human fetal stem cells isolated from first trimester blood possess superior osteogenic differentiation potential compared to adult stem cells isolated from bone marrow and to fetal stem cells isolated from first trimester liver. We showed that in utero transplantation of these cells in an experimental model of severe OI resulted in a drastic 75% decrease in fracture rate incidence and skeletal brittleness, and improvement of bone strength and quality.(14) A similar outcome was obtained using placenta-derived foetal stem cells (15) and amniotic fluid stem cells following perinatal transplantation into experimental models. (16,17)

Understanding the mechanisms of action of donor cells will enable the engineering of donor cells with superior efficacy to stimulate bone formation and strengthen the skeleton. Despite their potential to differentiate down the osteogenic lineage, there is little evidence that donor cells contribute to regenerating bones through direct differentiation, due to the very low level of donor cell engraftment reported in all our studies. When placed in an osteogenic microenvironment in vitro, foetal stem cells readily differentiate into osteoblasts and produce wild type collagen molecules. However, there are insufficient proofs that collagen molecules of donor cell origin contribute to the formation of the host bone ECM to confer superior resistance to fracture.

It is now well accepted that stem cells can influence the behaviour of target cells through the release of paracrine factors and, therefore, contribute to tissue regeneration indirectly. We have indeed recently shown that donor stem cells stimulate the differentiation of resident osteoblasts, which were unable to fully mature in the absence of stem cell treatment. (16,17) We are now focusing our efforts on understanding the precise molecular mechanisms by which donor cells improve skeletal health to counteract bone fragility caused by various OI-causative mutations.

References

Please note: This is a commercial profile

2019. This work is licensed under aCC BY 4.0 license.

Editor's Recommended Articles

Read the original here:
Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope - Open Access Government

Bone Marrow Stem Cells Comments Off on Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope – Open Access Government | October 5th, 2021

SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E [emailprotected]

Join the Pharmaxis mailing listhere

Follow us on LinkedInand Twitter

About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

About Myelofibrosis

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E [emailprotected]

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E [emailprotected]

SOURCE Pharmaxis Limited

http://www.pharmaxis.com.au

View original post here:
Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - PRNewswire

Bone Marrow Stem Cells Comments Off on Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial – PRNewswire | October 5th, 2021

Myles Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day. That day came in November 2020.

INDIANAPOLIS A 13-year-old boy living with sickle cell disease has been given a second chance at life, thanks to his mother.

Myles Glass has been through more in his young life than most adults. For the past few years, Glass has spent his days in and out of Riley Hospital for Children.

"[I] kind of have to look on the bright side of things. Being in the hospital, I meet new nurses and kids who go through what I go through. It's kind of hard to go through that at my age," Glass said.

He was diagnosed with sickle cell disease as a newborn. According to the Centers for Disease Control and Prevention, African Americans make up the largest number of people with the disease in the U.S.

Sickle cell disease is an inherited condition that impacts red blood cells and causes pain, infections and extreme fatigue. These symptoms keep Glass from doing things he loves.

"For him, it's kind of like we have to have him in a bubble," said his mother, Melissa Sanders.

Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day.

"[I would] hope that one day, I can do what kids do, like playing football and basketball," Glass said.

That day came in November 2020 when his mother donated bone marrow for a stem cell transplant, curing him of sickle cell disease.

"I was able to give him a second life with being a donor so that he can somewhat be a normal kid," Sanders said.

Riley Hospital for Children Dr. Seethal Jacob, who has been working with Glass and his family, said one baby every two minutes is born with sickle cell disease. She also said studies show there is a clear disparity for funding for this disease.

"There's been a lot of neglect when it comes to the disease itself. I think it's important to pay attention to the population it affects. I think that likely tells the story why sickle cell disease has been a neglected disease for so long," Jacob said.

Despite his challenges, Glass is staying positive and making strides in his physical therapy at Riley Hospital for Children.

"He's already been through harder things than most people will ever go through. I think anything else in life is going to be a piece of cake," said his physical therapist, Sarah Johnson.

"This gives me a glimpse of hope that even though you may have been diagnosed with this disease, it's not the end of the world," Sanders said.

For Glass, this is just the beginning. He hopes his story encourages other people living with sickle cell disease to keep moving forward.

"I know it's hard now, but you'll get through it. You'll be able to do what kids do your own age," Glass said.

Click here for more information on sickle cell disease and treatment options.

What other people are reading:

Go here to read the rest:
Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life - WTHR

Bone Marrow Stem Cells Comments Off on Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life – WTHR | October 5th, 2021