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Stem Cell Banking Market to witness an impressive growth during the forecast period 2020 2025 – Express Journal

By daniellenierenberg

In the latest Stem Cell Banking market report, factors that are positively impacting the industry progression as well as the major threats & challenges existing in this domain are expounded. To unveil all the possible opportunities for business expansion, the study scrutinizes the regulatory and macroeconomic framework across the various geographies. It also delves into the competitive dynamics and evaluates how it will evolve during the forecast period. Further, it suggests strategies for dealing with the impact of the COVID-19.

Key highlights from COVID-19 impact analysis:

A gist of the regional landscape:

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Other highlights from the Stem Cell Banking market report:

Market Status:The complete details on Stem Cell Banking Market situation, principal regions, distribution channels, pricing structures are blanketed.

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Strategic Analysis Covered in TOC: - Key Topics Covered

Initially, the document offers an outline of the global market with a complete take a look at key drivers, constraints, challenges, traits and product types sold by using the employer. The file studies the Stem Cell Banking market capacity of key packages with the identity of forecast opportunities. The local evaluation with a focus on specific international locations and area of interest markets is presented. The pinnacle organization profiles with key-word market size and proportion estimation, revenue strategies, products, and other factors are studied.

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Clinical trials with stem cells to treat effects of COVID-19 in the lungs advance – Granma English

By daniellenierenberg

Photo composition: Claudia Garca Martnez

Dr. Odalis Mara de la Guardia Pea, an expert immunologist, describes as "encouraging" preliminary findings obtained at the conclusion of the first phase of clinical trials evaluating the use of stem cells in patients facing lung damage caused by COVID-19.

The study, begun during the month of May at the Cuban Institute of Hematology and Immunology (IHI), was undertaken with a view toward eliminating or reducing interstitial inflammatory or fibrotic lung lesions following the infection.

The doctor, also an infectious disease specialist and head of External Services at the IHI, explains that the research will have significant impact "if, as we hope, stem cell therapy produces positive results in these patients with pulmonary alterations post-COVID-19.

"If the treatment is effective, it will be generalized across the entire country, improving the quality of life and respiratory capacity of these patients," she stated with the enthusiasm of someone devoted to the most important mission in the world: saving lives.


De la Guardia Pea commented that, although SARS-COV-2 has a variety of dissimilar effects (cardiovascular, renal, cerebral, vascular, in distal or lower limbs, and others); the "target" organ in the case of COVID-19 is the lung, in which patients experience the most serious impact, both during the disease and once they have recovered, a pattern being studied internationally.

"We have detected cases, specifically in Cuban patients, who have presented this kind of affectation, especially those who have suffered symptoms over a longer period. Among those visited for the study, there were cases of important pulmonary alterations, which is the most frequent, but perhaps not the most serious," the specialist continued.


"These recruitment consultations were atypical, as they were done in the field, visiting the homes of recovered patients," the doctor explained, adding that potential volunteers needed to meet several criteria for inclusion in the clinical trials.

Those selected were between 18 and 70 years of age, of both sexes, who had contracted COVID-19 thirty days prior to the trial treatment, testing negative on a PCR at the time of recruitment, and exhibited respiratory symptoms since the beginning of the disease.

Specifically sought for the trials were patients who experienced a more torpid evolution of the disease, those who were hospitalized for more than 20 days, requiring oxygen, assisted ventilation, or the use of some aerosol as treatment, upon reaching serious or critical condition.

"More than 130 homes were visited over almost three months, from May to June; and 141 patients were interviewed, of which about 50 were studied. Twenty patients were included in the trial, which was the determined number," the doctor reported.


"During the investigation, several long term effects of COVID-19 were noted, although the most frequent involved the lungs. In some cases, indications of pulmonary fibrosis were detected, a condition that cannot be completely corrected, and can only be treated to increase lung capacity and improve quality of life," the doctor explained.

"The study is still in progress. The first phase has been completed, but there is some time remaining before final evaluation of the patients. What we can say is that, thus far, we are very happy with the results we have observed, they are encouraging," she emphasized.


-Could you recount some stories that particularly impacted you?

-The first day I went out to recruit volunteers, I arrived at the home of a patient who, when she opened the door, exhibited obvious difficulty breathing, evident in plain sight.

We conducted the interview and learned that she experienced this difficulty on a daily basis, five weeks after being diagnosed with COVID-19 and 15 days after a negative PCR test.

This case was significant because we became aware of the lingering effects some patients face, who after having the disease, being discharged and completing all treatment, can have symptoms for a long time.

On another occasion, a patient received us effusively, grateful that he would continue to be treated, that he would receive some follow-up. This attitude was very common in many cases, confirming for us that the patients we visited were still feeling unwell, despite having recovered and been discharged from the hospital.


"You can be infected and be asymptomatic, or develop the most severe symptoms of the disease and die. This is random, no one understands or can control it," the specialist warns, emphasizing the importance of being fully conscious of taking care of ourselves, since anyone can develop an aggressive case of COVID-19.

"I agree with everything Professor Durn says every day at nine o'clock in the morning, about how measures must be maintained and complied with: the use of facemasks, hand washing, shoe disinfection (with doormats soaked in 0.5% hypochlorite at the entrance to common areas), social distancing, and collective discipline.

"The population must take care; success in containing the pandemic lies in individual responsibility," she concluded.


-When the patient is included in the study, treatment begins by injecting the granulocyte colony stimulating factor, Ior Leukocim, a product manufactured at Cubas Center for Molecular Immunology, to achieve the mobilization of stem cells from the bone marrow to the bloodstream.

-Subsequently, the patient's blood is extracted and mononuclear cells are separated and concentrated.

-This pool of cells includes hematopoietic and non-hematopoietic stem cells, which have immune-regulatory properties and promote the disappearance of lesions and the reconstitution of lung tissue.

-The cells are infused intravenously.

-The patient is evaluated one month following treatment and again at six months, to determine the clinical efficacy of the stem cell therapy.

Source: Granma interview with Consuelo Macas Abraham, director of the National Institute of Hematology and Immunology.

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This Dublin-based startup raises 30M to develop promising therapies for leukemia – Silicon Canals

By daniellenierenberg

The global blood and bone marrow cancer treatment market was valued at $38.8B (approx 32.8B) in 2018 and is reportedly expected to reach $74.9B (approx 63.4B) by 2027, expanding at a CAGR of 7.7% from 2019 to 2027.

Blood cancer begins in the bone marrow which is the integral source of stem cells, which are later differentiated into different types of blood cells in the human body. Researchers have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe.

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Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer.

In the recent development, blood and bone marrow cancer treatment developer Priothera Limited, has raised 30M in its Series A round of funding led by Fountain Healthcare Partners with participation from co-lead investor HealthCap and funds managed by Tekla Capital Management, LLC, as well as EarlyBird Venture Capital.

According to the medtech startup, the raised funds will be used to progress the clinical development of mocravimod a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogenic hematopoietic stem cell transplantation (HSCT) for treating AML.

Priothera expects to generate further randomised clinical data in high-risk AML patients with these funds.

Dublin-based Priothera was founded in 2020 by Drs. Florent Gros and Dhaval Patel. Joining the founding team include experienced industry executive, Dr. Christoph Bucher, Dr. Simone Seiter, and CFO Brice Suire.

The company claims to be leading the way in developing orally applied sphingosine 1 phosphate (S1P) receptor modulators for haematological malignancies. S1P receptor modulators have been suggested to largely reduce egress of T cell subsets from lymphatic tissues allowing for dual inhibition of graft-versus-host-disease (GvHD) and enhancing graft-versus-leukemia benefits in patients receiving allogenic stem cell transplant.

Allogenic stem cell transplant is the only potentially curative approach for AML patients but has unacceptably high mortality with current treatments, says Florent Gros, co-founder, and CEO of Priothera.

Florent Gros further adds, We are excited about mocravimod which has a unique mechanism of action and clinical proof of concept demonstrating its ability to improve survival outcomes for this devastating disease.

Acute myeloid leukemia (AML) is an aggressive and highly proliferative form of cancer where the bone marrow generates abnormal myeloblasts (a type of white blood cell). According to the company, AML is the most common form of leukemia in adults and can metastasise quickly if left untreated. This can typically lead to death within a few months of diagnosis.

Priothera has acquired rights to a drug called mocravimod from Japans Kyorin Pharmaceutical for the treatment of acute myeloid leukaemia.

According to the company, Mocravimod has already been extensively tested in multiple immunologic indications and has shown a survival benefit in an early clinical study evaluating acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL) patients undergoing hematopoietic stem cell transplantation (HSCT).

Priothera is developing mocravimod in AML with the aim of enhancing the curative potential of Hematopoietic Stem Cell Transplantation (HSCT). The company claims that promising early clinical results have revealed that mocravimod has the potential to rebalance the patients immune system by decoupling Graft-versus-Host Disease (GvHD) from Graft-versus-Leukemia (GvL), preventing the first and preserving the latter.

Following the closing of the financing, people who have joined the Board of Directors include Florent Gros (Priotheras co-founder and CEO), Dr. Dhaval Patel (Priotheras co-founder and CSO at UCB), Dr. Manus Rogan (Fountain Healthcare Partners co-founder and MD), Dr. Marten Steen (partner at HealthCap), Dr. Henry Skinner (senior vice president at Tekla Capital Management, LLC) and Lionel Carnot (partner at EarlyBird Venture Capital).

Image credits: Jarun Ontakrai/ShutterStock

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Direct Biologics Granted Expanded Access by FDA for ExoFlo in the Treatment of COVID-19 – PRNewswire

By daniellenierenberg

AUSTIN, Texas, Oct. 13, 2020 /PRNewswire/ --Direct Biologics, LLC, announced today that the FDA has granted expanded access for ExoFlo in the treatment of patients with COVID-19 associated acute respiratory distress syndrome (ARDS).

While Direct Biologics is already enrolling patients in EXIT COVID-19, its national multi-center, Phase II, placebo controlled, randomized clinical trial, the new expanded access protocol will make ExoFlo available to a broader group of patients with severe COVID-19, many of whom would not meet acceptance criteria for EXIT COVID-19, often under conditions of "compassionate use."

Co-Founder and Chief Executive Officer, Mark Adams, states, "The FDA's approval of expanded access for ExoFlo signifies a critical milestone in the development of advanced treatment for COVID-19 associated illnesses including ARDS. We are excited to be able to provide our product to patients with ARDS associated with COVID-19 in critical need of treatment."

"Amid a potential surge in new COVID-19 cases moving into the fall and winter seasons, this approval could not have come at a better time," notes Joe Schmidt, Co-Founder and President. "Our team is working hard to advance our Phase II EXIT COVID-19 trial to offer additional treatment options."

"Approval of our expanded access protocol offers an option for doctors to administer ExoFlo as a treatment to reverse disease progression, extending hope to COVID-19 patients who are not responding favorably to standard of care," states Chief Medical Officer, Vik Sengupta, MD. "We at Direct Biologics are grateful for every opportunity to help these additional patients in need."

Also in attendance was Congressman Michael McCaul who commented, "I am thrilled to hear a local company from Austin, Texas, has been approved for Expanded Access IND by the FDA. Their product, ExoFlo, will help treat COVID-19 patients who are at risk of severe respiratory infection, which often leads to life-threatening circumstances. Direct Biologics, with support of the FDA, will bring high-class treatments and services to Americans who need them the most."

This approval comes on the heels of multiple approvals for single patient Emergency Investigational New Drug (eIND) applications granted in September and October. Emergency INDs are a mechanism by which physicians can obtain rapid approval to administer medication to a single patient through a direct appeal to the FDA.

About ExoFlo ExoFlo is an investigational new drug that has not been approved or licensed by FDA. It is an extracellular vesicle product isolated from human bone marrow mesenchymal stem or stromal cells (MSCs). ExoFlo provides natural bioactive signals that have been shown to modulate inflammation and direct cellular communication.

About Direct Biologics Direct Biologics, LLC, is headquartered in Austin, Texas, with a recently expanded R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in St. Louis, Missouri. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust line of extracellular vesicle-based biological products. The Company was created to expand the science of regenerative healing by delivering cutting-edge biologic technologies. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving, next generation segment of the biotherapeutics industry. Direct Biologics is dedicated to pursuing additional clinical applications of its extracellular vesicle biologic products through the FDA's investigational new drug application process. For more information visit


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Lymphoma: Higher rates of survival on the horizon – The Straits Times

By daniellenierenberg

To keep you healthy from day to day, your body has its own in-built, germ-fighting network.

This network is also known as the lymphatic system, made up of different vessels and organs in your body, from the lymph nodes to the bone marrow. Lymphoma is the name of the cancer that affects this system.

There are many types of lymphoma but Diffuse Large B-Cell Lymphoma (DLBCL) is the most common aggressive type of non-Hodgkin lymphoma, affecting the B-lymphocytes that produce antibodies which help your body fight infections.

According to the World Health Organizations guidance on classifying tumours, DLBCL accounts for 30 to 40 per cent of newly diagnosed cases of non-Hodgkin lymphoma globally.1

In Singapore, Dr Daryl Tan Chen Lung, who specialises in haematology and practises at Mount Elizabeth Hospitals, estimates that lymphoma is the fifth most common cancer in Singapore.2

Unlike other cancer patients, DLBCL patients hardly have any contributing factors to the development of the condition not even hereditary ones though people with existing HIV infections are more prone to getting DLBCL.

As a result of this, and also because lymphoma is less common than other cancers like breast or lung cancer, regular medical check-ups do not test for its genetic indicators. Instead, patients seek medical help only when they spot early symptoms, says Dr Tan. A growing awareness of lymphoma World Lymphoma Awareness Day takes place on Sept 15 each year and better diagnostics measures have led to much earlier detection of the cancer today, compared to a decade ago.

Dr Tan says that early symptoms of DLBCL include lumps in the neck or groin. Sometimes, other organs like the spleen and bone marrow can be affected. However, some patients show no obvious symptoms beyond abdominal pain, fever and loss of weight. It is thus especially important to seek medical attention should you display any of the common symptoms of lymphoma including swollen lymph nodes in the neck, armpit or groin, a persistent fever, excessive night sweats and unexplained weight loss.

Dr Tan adds that the median age of DLBCL patients in Singapore is 60 to 65, though a very small number of patients in their 20s are diagnosed with a subtype of the cancer known as primary mediastinal B-cell lymphoma.

While DLBCL is an aggressive cancer, the good news is that it is highly treatable in the early stages.

Current therapies for treating first-line DLBCL include chemotherapy, chemo-immunotherapy a combination use of chemotherapy and immunotherapy and stem cell transplant in certain groups of patients. About six in 10 of these patients will respond to their treatment3 and not suffer a relapse within the next five years.

This has a huge impact on younger patients who are then able to resume their daily life and go back to work, for example.

Dr Tan remembers a female patient in her early 20s whose career was just taking off. She was admitted with fever and breathlessness. After performing scans on her, it was discovered that there was fluid surrounding her heart and lungs, and she had a tumour in her chest the size of a rugby ball.

Fortunately, because the cure rate for DLBCL is high, the patients condition has since gone into remission.

A combination of chemo-immunotherapy drugs is commonly recommended as a first-line treatment in DLBCL. Radiation therapy or stem cell transplant can also be included as part of the treatment.

However, about four in 10 patients may not eventually respond to these therapies or suffer from a relapse.3 When this happens, treatment options are limited. Some patients can undergo a stem cell transplant, but about half of them are not eligible.4 This group includes older patients with compromised immune systems.

While affected patients can consider continuing with chemotherapy and chemo-immunotherapy, there are new promising treatment options on the horizon, including targeted therapies such as CAR T-cell therapy. These targeted therapies involve using the patients own immune cells also known as T cells or molecules that bind a chemotherapy agent to an antibody to fight cancerous blood cells.

Beyond that, there are also treatment options known as combination therapy. Previously, patients suffering a relapse have had to rely on existing, first-line treatment, which may or may not be effective in combating the cancer. Combination therapy uses the collective effect of different medications to target different areas of cancerous B-cells and destroy them.

Says Dr Tan: [The medical community is] seeing progress in DLBCL immunotherapy. The current cure rate is about 70 per cent. Hopefully, we can increase the number to 80 per cent or more. But we mustnt rest on our laurels as we hope to focus on the 20 per cent of patients who still dont respond to any treatments.

For more information on lymphoma and treatment options, please speak to your healthcare specialist.

1. Lyon, France. World Health Organization Classification of Tumors of Haematopoietic and Lymphoid Tissues. IARC Press; 2008.2. SingHealth. (n.d). Lymphoma. Retrieved on 30 Sept 2020. ( Maurer, MJ et al. Event-free survival at 24 months is a robust end point for disease-related outcome in diffuse large B-cell lymphoma treated with immunochemotherapy. J Clin Oncol. 2014; 32: 1066-73.4. Gisselbrecht C, Van Den Neste E. How I manage patients with relapsed/refractory diffuse large B cell lymphoma. Br J Haematol. 2018;182:633643.

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Becoming a donor easier than you think – Randfontein Herald

By daniellenierenberg

When thinking about donating bone marrow, most will break out in a cold sweat.

The thought of needles, prodding and poking is enough to put anyone off from becoming a donor but Ndinae Muligwe, Sustainability and Donor Recruitment Coordinator for the South African Bone Marrow Registry (SABMR) explained that it is a less complicated and relatively painless process.

The SABMR was established in 1991 and is a non-profit organisation that conducts searches to find matching bone marrow donors for critically ill children and adults in South Africa who cannot find a match in their own families.

Bone marrow transplants help to treat and even sometimes cure illnesses like leukaemia, Non-Hodgkin lymphoma, bone marrow failure, and some genetic blood and immune-system disorders.

Ndinae explained that the likelihood of a donor finding a match is about one in 100 000. What is more concerning is that there are currently only around 74 000 local donors on the South African Bone Marrow Registry.

Although they do form part of the World Marrow Donor Association that represents about 38 million donors, there are not enough donors for the South African demographic.

Ethnicity plays a role when it comes to who is able to donate, and at the moment the numbers do not match the ethnic groups represented in South Africa. You are more likely to find a match within your own ethnic group.

But how do you become a donor and what is the process involved?

Ndinae said it is as easy as registering on the website. Of course there are some questionnaires to fill in and you will have to meet the criteria and be healthy.

The donating age has recently been lowered from 18 to 16 years of age, and applicants must be between 16 and 45 to register as a potential donor.

If you are eligible you will then be contacted by the SABMR to do a cheek swab free of charge.

Peripheral blood stem cell (PBSC) collection is the most likely way of collecting stem cells. These cells are found in your bone marrow and also the blood stream. A five-day course of growth factor or Granulocyte-Colony Stimulating Factors is given prior to the donation to encourage the stem cells to move from your marrow to your blood.

At the time of donation a needle is placed in one arm. The blood is then passed through a machine that collects the stem cells, and the remaining blood is returned to your body similar to donating blood platelets.

You do not have to pay for anything to make a tissue or blood donation of your bone marrow stem cells, the SABMR covers the cost of testing and collection. more information.

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Proposition 14: With Just Handful of Cures, California Stem Cell Agency’s Fate Is In Hands of Voters – KQED

By daniellenierenberg

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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Cytovia Therapeutics partners with Inserm to develop selective CD38 NK therapeutics and offer new treatment options for Multiple Myeloma patients |…

By daniellenierenberg

DetailsCategory: DNA RNA and CellsPublished on Thursday, 08 October 2020 15:15Hits: 339

NEW YORK, NY, USA and PARIS, France I October 08, 2020 I Cytovia Therapeutics ("Cytovia"), an emerging biopharmaceutical company, announces today that it has entered a research and licensing agreement with Inserm to develop NK engager bi-specific antibodies and iPSC CAR NK cell therapy targeting CD38, a key marker of multiple myeloma. The licensing agreement has been negotiated and signed by Inserm Transfert, the private subsidiary of Inserm, on behalf of Inserm (the French National Institute of Health and Medical Research) and its academic partners. Cytovia is licensing Inserm's CD38 antibody and Chimeric Antigen Receptor (CAR) patent and applying its proprietary NK engager bispecific antibody and iPSC CAR NK technology platforms. The research agreement will include evaluation of the therapeutic candidates at Hpital Saint-Louis Research Institute (Inserm Unit 976) under the leadership of Professors Armand Bensussan and Jean-Christophe Bories.

Dr Daniel Teper, Cytovia's Chairman and CEO commented: "We are delighted to partner with one of the top centers of excellence in the world for research and treatment in hematology. CD38 is a validated target and Natural Killer cells have significant cytotoxicity to Myeloma cells. We are looking forward to bringing promising new options to address the unmet needs of patients with Multiple Myeloma and aim for a cure."

Professor Armand Bensussan, Director of The Immuno-Oncology Research Institute at Hpital Saint-Louis added: "We have demonstrated the selectivity of our novel CD38 antibody in killing myeloma cells but not normal cells such as NK, T, and B cells. The activation of NK cells through NKp46 may enhance the efficacy of the bispecific antibody in patients not responsive to CD38 monoclonal antibody therapy. CD38 CAR NK is a promising approach forrelapsed/refractory patients and an alternative to CAR T therapies."

About Multiple MyelomaMultiple Myeloma is a currently incurable cancer, affecting a type of white blood cell known as plasma cells. It leads to an accumulation of tumor cells in the bone marrow, rapidly outnumbering healthy blood cells. Instead of producing beneficial antibodies, cancerous cells release abnormal proteins causing several complications. While symptoms are not always present, the majority of patients are diagnosed due to symptoms such as bone pain or fracture, low red blood cell counts, fatigue, high calcium levels, kidney problems, and infections. According to the World Cancer Research Fund, Multiple Myeloma is the second most common blood cancer, with nearly 160,000 new annual cases worldwide, including close to 50,000 in Europe. 32,000 in the US, and 30,000 in Eastern Asia. Over 95% of cases are diagnosed late, with a 5-year survival rate of 51%. Initial treatment comprises of a combination of different therapies, including biological and targeted therapies, corticosteroids, and chemotherapy, with the option for bone marrow transplants for eligible patients. Immunotherapy and cell therapy are the most promising new treatment option for Multiple Myeloma, with the potential for long term cancer remission.

About CAR NK cellsChimeric Antigen Receptors (CAR) are fusion proteins that combine an extracellular antigen recognition domain with an intracellular co-stimulatory signaling domain. Natural Killer (NK) cells are modified genetically to allow insertion of a CAR. CAR-NK cell therapy has demonstrated initial clinical relevance without the limitations of CAR-T, such as Cytokine Release Syndrome, neurotoxicity or Graft vs Host Disease (GVHD). Induced Pluripotent Stem Cells (iPSC) - derived CAR-NKs are naturally allogeneic, available off-the-shelf and may be able to be administered on an outpatient basis. Recent innovative developments with the iPSC, an innovative technology, allow large quantities of homogeneous genetically modified CAR NK cells to be produced from a master cell bank, and thus hold promise to expand access to cell therapy for many patients.

About CytoviaCytovia Therapeutics Inc is an emerging biotechnology company that aims to accelerate patient access to transformational immunotherapies, addressing several of the most challenging unmet medical needs in cancer and severe acute infectious diseases. Cytovia focuses on Natural Killer (NK) cell biology and is leveraging multiple advanced patented technologies, including an induced pluripotent stem cell (iPSC) platform for CAR (Chimeric Antigen Receptors) NK cell therapy, next-generation precision gene-editing to enhance targeting of NK cells, and NK engager multi-functional antibodies. Our initial product portfolio focuses on both hematological malignancies such as multiple myeloma and solid tumors including hepatocellular carcinoma and glioblastoma. The company partners with the University of California San Francisco (UCSF), the New York Stem Cell Foundation (NYSCF), the Hebrew University of Jerusalem, and CytoImmune Therapeutics. Learn more at

About InsermFounded in 1964, the French National Institute of Health and Medical Research (Inserm) is a public science and technology institute, jointly supervised by the French Ministry of National Education, Higher Education and Research, and the Ministry of Social Affairs, Health and Womens Rights. Inserm is the only French public research institute to focus entirely on human health and position itself on the pathway from the research laboratory to the patients bedside. The mission of its scientists is to study all diseases, from the most common to the rarest. With an initial 2020 budget of 927.28 million, Inserm supports nearly 350 laboratories throughout France, with a team of nearly 14,000 researchers, engineers, technicians, and post-doctoral students.

SOURCE: Cytovia Therapeutics

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Knowledge is key: What you need to know about the most common childhood cancer in SA – News24

By daniellenierenberg

There are only a few specialised childhood cancer centres in Southern Africa.

Leukaemia and lymphoma are two of the most prevalent cancers in children in South Africa with between 800 and 1000 children diagnosed annually. Tragically, it is estimated that half of the children with cancer in this country are never diagnosed.

Dr Marion Morkel, Chief Medical Officer at Sanlam, believes that we all need to educate ourselves so we can recognise the symptoms of cancer.

Below, Dr Morkel explains what can be done in the fight against leukaemia and lymphoma.

Knowledge is key

You must be aware of the symptoms related to leukaemia and lymphoma so that you can notify your health professional should you see these symptoms in your child.


Leukaemia is the most common childhood cancer accounting for 25% of all cases in South Africa.

Symptoms include:


Lymphoma primarily originates from the lymph nodes and can often appear like any other illness that triggers an inflammatory response.

Symptoms to look out for include:

While other childhood illnesses can present in the same manner as leukaemia and lymphoma, health professionals have been trained to look out for symptoms that persist after routine treatment and will conduct tests to rule out the possibility of these childhood blood-related cancers.

Parents are encouraged to consult their doctor if there are any concerns about their childs health.

ALSO READ|Should I be worried if my child has pain in his tummy?

Register to become a blood stem cell (bone marrow) donor

The Sunflower Fund is a non-profit organisation that fights blood diseases through a blood stem cell transplant which replaces a persons defective stem cells with healthy ones and can be a potentially life-saving treatment for more than 70 different diseases.

Kim Webster, Head of Communications at The Sunflower Fund advises that finding a matching donor for a stem cell transplant is not as easy as finding a blood type match.

There is only a 1:100 000 chance of a patient finding their life-saving match with siblings only having a 25% chance of a match.

You can register to become a donor online via

Submitted to Parent24 by Atmosphere


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Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis of Key Vendors with Their Size, Share and Year-over-Year Growth 2027 – Press Release -…

By daniellenierenberg

The study methodologies used to examine Hematopoietic Stem Cell Transplantation (HSCT) Market for the forecast period, 2020 - 2027 further classifies the industry by type, geography, end-use and end-user to add more precision and bring to light factors responsible for augmenting business development.

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 10/08/2020 -- Reports and Data has recently published a research report on the Global Hematopoietic Stem Cell Transplantation (HSCT) Market, expanding its extensive database. The research study offers a detailed assessment of the current and emerging market trends and offers a holistic overview of the sector considering product portfolio, types, segmentation, applications, and supply chain analysis. It also provides a comprehensive analysis of the international market, growth trends, development patterns, competitive landscape, demand and supply dynamics, and gross margins.

Additionally, the report examines the impact of the COVID-19 crisis on the Hematopoietic Stem Cell Transplantation (HSCT) market and its key segments. The report assesses the changing market dynamics, demands, and trends of the Hematopoietic Stem Cell Transplantation (HSCT) industry with regards to the COVID-19 pandemic. The report furthermore presents a present and future impact analysis of the crisis on the Hematopoietic Stem Cell Transplantation (HSCT) market and offers a futuristic outlook with regards to trends and demands patterns in a post-COVID-19 scenario.

Get a Sample of the Report @

The Global Hematopoietic Stem Cell Transplantation (HSCT) Market research report provides a complete outlook on the challenges existing in the industry and also discusses the emerging threats, constraints, and limitations. The report is an investigative study that offers an extensive breakdown of the market dynamics such as drivers, growth prospects, product portfolio, technological advancements, and an extensive analysis of the key competitors of the market. The global Hematopoietic Stem Cell Transplantation (HSCT) market is further segmented into types, applications, technology, end-users, industry verticals, and key geographies across the world. Furthermore, the report offers a forecast estimation of the valuation of the Hematopoietic Stem Cell Transplantation (HSCT) market.

Leading companies profiled in the report are China Cord Blood Corp, Pluristem Therapeutics Inc., CBR Systems Inc CellGenix Technologie Transfer GmbH, Cryo-Save AG Kite Pharma Inc., Regen Biopharma Inc., ViaCord Inc., BiolineRx, Cynata Therapeutics, Cesca Therapeutics Inc, Lonza Group Ltd, TiGenix N.V., Bluebird Bio, Cellular Dynamics International, and Escape Therapeutics Inc., among others.

Type Outlook (Revenue in USD Million; 2017-2027)


Indication Outlook (Revenue in USD Million; 2017-2027)

LeukemiaLymphatic disorderMyelomaOther non-malignant disorders

Application Outlook (Revenue in USD Million; 2017-2027)

Peripheral blood cellsBone marrowUmbilical cord blood

The report presents a detailed study of the Hematopoietic Stem Cell Transplantation (HSCT) industry through data gathered by thorough primary and secondary research. The data formulated is verified and validated by industry experts and professionals. Additionally, the report utilizes advanced analytical tools such as SWOT analysis, Porter's Five Forces analysis along with feasibility analysis and investment return analysis. The report provides a comprehensive analysis of the prominent players of the market with a detailed analysis of their company overview, product portfolio, production and manufacturing capacity, technological and product developments, and revenue estimations. The report further examines key statistical data and facts pertaining to the Global Hematopoietic Stem Cell Transplantation (HSCT) market. The report further aims to provide a competitive advantage to the readers, clients, consumers, and market professionals engaged in the industry.

Browse Full Report @

Key Geographies Mapped in the Report are:

North America (U.S, Canada, and Rest of North America)Europe (Germany, France, Italy, and Rest of Europe)Asia Pacific (China, Japan, India, South Korea, and Rest of Asia-Pacific)Latin America (Brazil, Argentina, and Rest of Latin America)Middle East & Africa (South Africa, Saudi Arabia, U.A.E, and Rest of MEA

The report offers:

An in-depth overview of the Hematopoietic Stem Cell Transplantation (HSCT) market landscapeAssessment of the global industry trends for the historical period of 2017-2018, the current year 2019-2020, and a forecast estimation for the period 2020-2027Overview of the company profiles and product portfoliosR&D advancements and technological developments in the Hematopoietic Stem Cell Transplantation (HSCT) industry.Market dynamics, trends, opportunities, and risksStudy of the market in terms of revenue and product consumption patterns.

Additionally, the report offers historical analysis and forecast analysis for the global Hematopoietic Stem Cell Transplantation (HSCT) market.

Historical Years: 2017-2018

Base Year: 2019

Estimated Year: 2020

Forecast Years: 2020-2027

The report answers radical questions about the global Hematopoietic Stem Cell Transplantation (HSCT) market. It aims to offer a competitive edge to the reader by providing insightful data about strategic alliances such as mergers and acquisitions, joint ventures, collaborations, partnerships, agreements, government deals, and product launches.

Pre Book- Hematopoietic Stem Cell Transplantation (HSCT) Market Research Report:

Thank you for reading our report. For further inquiries or queries regarding customization, kindly connect with us.

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Southfield woman meets boy she saved with bone marrow donation – C&G Newspapers

By daniellenierenberg

Grady Smith, 10, and Southfield resident Jessica Carroll were able to meet for the first time via Zoom in September at the DKMS Gala. Carroll was the bone marrow donor that helped save the boys life.

Photo provided by the Smith family

SOUTHFIELD If you were to take one look at 10-year-old Grady Smith, youd see a young boy who enjoys sports and school.

But the young Salem, New Hampshire, boy has been through more in 10 years than some people have in 50.

Grady was diagnosed with adrenoleukodystrophy, or ALD, back in 2018.

According to Boston Childrens Hospital, ALD is a rare genetic condition that causes the buildup of very long chain fatty acids in the brain. When the fatty acids accumulate, they destroy the protective myelin sheath around nerve cells, responsible for brain function. Without the myelin sheath, the nerves can no longer relay information to and from the brain.

Every single thing I read said, terminal, slow deterioration to death, one to five years, horrible, horrible death, Jillian Smith said. We just died. I havent been the same person since that day. It just changes you for the rest of your life.

With a diagnosis, Grady and his parents looked for options on how to help. Grady had a lesion with a Loes score which is a way of rating severity of 10. Scores range from 0-34.

His parents werent sure Grady would qualify for a bone marrow transplant because they usually only perform transplants for boys with scores of 9 and under. Grady was in luck, however, as Boston Childrens Hospital decided to move forward anyway.

The next move was to find a match for the boy, but that process could take weeks, months or even years. In Gradys case, it took just a few weeks.

Southfield resident Jessica Carroll registered as a potential bone marrow/blood stem cell donor with DKMS, a German bone marrow donor file, in 2014, but she didnt think much would come of it.

Four years later she got a call from the nonprofit organization letting her know that she was a match for a young boy. After some research, Carroll was totally on board with donating.

It was great knowing during that donation that this little bit that I went through was potentially saving somebodys life, Carroll said. Thats all I really cared about, was that I was helping somebody.

Grady was able to get his transplant in 2018.

According to his mother, Grady hasnt had any progression and has even made some recovery. Hes back in line with his academics and is playing sports again.

Grady has auditory processing issues, which make it hard for him to comprehend language and sound. His mother said he relies on reading lips to communicate.

Theres still a lot to it. It stopped the monster thats how we look at it but its not just so cut and dry, Jillian said. Hes a very rare outcome with his Loes score and with just how well hes doing. Hes just a really, really good boy. He works really hard to help bring awareness.

Carroll and the Smiths have talked via text, and they were able to meet virtually for the first time in September at the DKMS Gala.

For the Smiths and Carroll, the meeting was emotional. Grady was finally able to put a face to his donor, and vice versa for Carroll.

It was of course emotional, Carroll said. Being able to hear everything they went through, though, definitely made me so happy that I had chosen to register.

The Smiths and Carroll still talk periodically throughout the year, and Sept. 20 was the two-year anniversary of the transplant.

They are hoping to be able to meet in person soon, and the DKMS team wants to bring them to next years gala to help make that happen. However, they hope it will be sooner.

Throughout this journey with Grady, the Smiths have advocated, learned and spoken more about ALD.

Prior to Gradys birth, Massachusetts wasnt testing for ALD in newborns, but it has since started. New Hampshire wasnt either, but the Smiths got the state to add ALD to the newborn screening panel.

The next goal is to get more states to add the ALD screening. The family has also spoken at conferences to share Gradys story and have become big proponents of what DKMS has been able to do for not only their family, but families around the world.

I think a big thing, too, that we really want to get out there is bone marrow transplant or stem cell transplant, how easy it is, Jillian said. All people need to do is go on DKMSs website, and they can get a packet sent out to them. They just swab their cheeks, send it in and they could be saving anyones life, someone just like Grady.

According to the DKMS website, the organization is dedicated to the fight against blood cancer and blood disorders by creating awareness, recruiting bone marrow donors to provide a second chance at life, raising funds to match donor registration costs and supporting the improvement of blood cancer therapies by our own research.

Those looking for more information or wanting to register can visit or call (212) 209-6700.

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Explainer: What is Crispr and why did it win the Nobel prize? – Chemistry World

By daniellenierenberg

Emmanuelle Charpentier and Jennifer Doudna have scooped the 2020 Nobel prize in chemistry for the development of a method for genome editing. Specifically, theyve been awarded the prize for their discovery of the CrisprCas9 genome editing technique that allows scientists to make precise alterations to the genetic code of living organisms. CrisprCas9 is a powerful tool that could revolutionise many aspects of our lives, from medical treatments to the way we produce food. Its also seen its fair share of controversy in recent years. Here, we take a deeper look at these genetic scissors and why theyve won the Nobel prize.

Since Charpentier and Doudna began investigating the CrisprCas9 system in 2011, the field has exploded. Due to the relative simplicity and affordability of Crispr systems, researchers around the world have been able to apply the tools to all manner of different problems. Today there are entire journals, conferences and companies dedicated to the technique.

The ability to cut any DNA molecule at a chosen site has huge potential from treating genetic illnesses to creating disease-resistant crops. Trials have even shown how Crispr-delivered genetic modifications can spread through populations of mosquitoes and stop malaria infections such gene-drives offer a way to eliminate the disease altogether. And in the face of the Covid-19 pandemic, researchers have found ways to use Crispr in rapid coronavirus diagnostic tests and have also proposed using it to attack the viruss genome.

As Claes Gustafsson, chair of the Nobel committee for chemistry, said at the award announcement, There is enormous power in this genetic tool, which affects us all.

Crispr technology has even been used to make more delicious beer.

The whole Crispr gene editing tool has been adapted from the immune system of bacteria. The term Crispr comes from clustered regularly interspaced short palindromic repeats, which refers to distinct genetic sequences found in the genomes of bacteria. Each Crispr sequence is transcribed into RNA sequences that will target the DNA of a virus. These sequences also include cas (Crispr-associated) genes that code for DNA-cutting Cas enzymes. Together, the guide RNA and Cas enzyme form a complex that hunts out viral DNA and chops it up.

In Crispr gene editing, scientists repurpose this system by designing a guide RNA sequence of around 20 nucleobases that matches up to a DNA sequence they wish to target in a cells genome. This RNA sequence is paired with the Cas9 enzyme that will cut the DNA strand at the targeted site. The whole DNA sequence coding for both these components of the Crispr-Cas9 tool can be delivered to the target cell via a plasmid.

The tool can therefore be used to edit a cells genome with incredible precision for example, it can cut out a dysfunctional gene associated with a hereditary illness. And if the healthy version of the gene is also delivered to the cell, the cells own repair system will then incorporate the healthy strands at the site where it has been cleaved.

In 2011, when investigating the bacteria Streptococcus pyogenes, Charpentier discovered a molecule called tracrRNA that forms a key part of the CrisprCas system in bacteria.

Meanwhile, Doudna had been investigated the function of the cas genes, and learned that the Cas proteins they code for are involved in cutting up DNA as part of the bacterial immune system against pathogenic viruses.

That year Charpentier teamed up with Doudna to investigate the system further. Together they revealed how the Cas9 protein, CrisprRNA and tracrRNA worked together to snip DNA strands into two parts. They then simplified the system by combining the CrisprRNA and tracrRNA into a single molecule guide RNA making it easier to use, and showed how this could be used to cut any DNA strand at a site of their choosing, opening the door to using the tool in all manner of genome editing experiments.

While previous tools for genetic editing existed before Crispr-Cas9, the new tools are much simpler and cheaper. This has led to the huge expansion of the field by making gene editing accessible for scientists all around the globe.

For years Crispr has been at the centre of a long-running patent dispute. Shortly after Doudna and Charpentiers discovery, Feng Zhangs team the Broad Institute in Cambridge, US, patented a way to use the technique in eukaryotic cells. There have been protracted court battles between Doudnas group at the University of California in Berkeley, US, and the Broad team over who holds the key piece of intellectual property. In the meantime, numerous groups and companies have been granted patents for many new Crispr-related technologies, meaning that as time goes on, the original patents at the centre of the dispute are becoming less relevant.

Another area of controversy surrounds the potential consequences of using genome editing tools at all. As the genome is so complex, we cant always know what will happen when we edit genes. Some genes have multiple and often unknown functions editing them to correct for one problem could end up creating new unforeseen ones. This is particularly important when it comes to editing germline cells (those that can be passed on to an organisms children), because the modified genes can be inherited by future generations.

As a relatively new technique, we also know that Crispr itself isnt perfect. Some studies have shown off-target cuts, where the tool has snipped DNA strands at additional locations to the desired site. This clearly can have harmful consequences, and so many researchers are looking into ways to improve the technique and make it more suitable for medical uses.

With these concerns in mind, scientists worldwide including Doudna and Charpentier have called for a moratorium on editing human germline cells, until we can know more about the consequences. Such calls intensified after the rogue Chinese scientist He Jiankui edited human embryos that were then brought to term in 2018. He is now serving a three year prison sentence for conducting the study.

Several clinical trials have already begun on Crispr-based therapies, with promising reports emerging this year. In February, the first study to look at a cancer treatment using Crispr-edited immune cells reported that the modified cells were safe, with no serious side-effects in the three patients studied. While the efficacy of the treatment on the cancers was minimal, it may help to inform future Crispr-based T-cell treatments.

One month later, a patient with hereditary blindness became the first person ever to have a CrisprCas9 therapy directly administered into their body. And in June, the Swiss gene-editing company Crispr Therapeutics announced that two patients with beta thalassaemia and one with sickle cell disease would no longer require blood transfusions after their bone marrow stem cells were edited using Crispr techniques.

Earlier this week, Doudna launched a new company, Scribe Therapeutics, to begin work on treatments for amyotrophic lateral sclerosis.

Other Crispr-based technologies are coming closer to commercial reality. For example, the US genome engineering company eGenesis is developing ways to use the technique to edit pigs genes so that their organs might be transplanted safely into humans. In the agricultural sector, many companies are working on ways to use Crispr to speed up the selection process for crops with desirable traits such as disease-resistance or improved flavour.

At the fundamental level, researchers are working on ways to improve the system itself. By using alternative Cas proteins, some groups hope to make the tool more effective and easier to use in certain settings. Doudnas group recently reported on a CasX protein that is smaller than Cas9 and potentially easier to introduce into target cells.

Delivering DNA into cells and tissues is an important part of gene therapy, even more so for Crispr-Cas9 approaches because plasmids carrying this system are very large. This research paper describes a non-viral vector for delivering plasmid DNA carrying Crispr-Cas9 into tumour spheroids, which are good in vitro models for tissues but also challenging transfecting targets.

1 S J Zamolo, T Darbre and J-L Reymond, Transfecting tissue models with CRISPR/Cas9 plasmid DNA using peptide dendrimers, Chem. Commun., 2020, DOI: 10.1039/d0cc04750c

Regulating the function of Crispr-Cas9 is on the agenda for many researchers because the ability to restrict it in a spatial and temporal manner opens the door to precisely manipulating genomes and minimising any side effects. By introducing photolabile groups into the system, these researchers have shown how they can regulate Cas9 activity with light

2 Y Wang et al, Photocontrol of CRISPR/Cas9 function by site-specific chemical modification of guide RNA,Chem. Sci., 2020, DOI: 10.1039/d0sc04343e

It seems that Crispr-Cas systems arent just handy for gene editing. This paper describes how the Crispr-Cas system was used to assemble a multi-enzyme cascade containing five distinct enzymes. The team behind the work hope it could be the beginnings of a general method for building complex scaffolded biocatalytic pathways

3 S Lim et al, CRISPR/Cas-directed programmable assembly of multi-enzyme complexes, Chem. Commun., 2020, 56, 4950 (DOI: 10.1039/d0cc01174f)

And to round things off, here are some reviews on how Crispr-Cas9 works, the delivery processes for therapeutic nanoparticles and the physiological obstacles for those process

4 Y Xu, R Liu and Z Dai, Key considerations in designing CRISPR/Cas9-carrying nanoparticles for therapeutic genome editing, Nanoscale, 2020, DOI: 10.1039/d0nr05452f

5 Y Gong et al,Lipid and polymer mediated CRISPR/Cas9 gene editing, J. Mater. Chem. B, 2020,8, 4369 (DOI: 10.1039/d0tb00207k)

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First Man Cured of AIDS Dies From Cancer – The Keystone Newspaper

By daniellenierenberg

By Dylan Adams News Editor

Timothy Ray Brown, the first known person to be cured of HIV, died on Sept. 29 at age 54 after battling cancer.

Timothy Ray Brown, a figurehead in the AIDS and HIV community, passed away surrounded by friends after a five-month battle with leukemia, stated Tim Hoeffgen, Browns partner.

Brown received a positive HIV diagnosis in 1995 while studying in Berlin.

In 2006, Brown was diagnosed with acute myeloid leukemia, which is a cancer that builds in the bone marrow and blood interfering with blood cell production. After bouts of infections from several rough rounds of chemotherapy, Browns leukemia came out of remission.

Due to leukemia in his bones, Brown required a stem cell transplant, a process that allows healthy stem cells to be introduced into a host to stimulate the immune system and healthy bone marrow growth. At the time, the survival rates for stem cell transplant were around fifty percent.

Doctors found a match to Browns genetic type, a donor with the CCR5 Delta 32 mutation, a protein that acts as a doorway to stop the HIV from infecting new cells. Three months after Brown stopped taking his HIV medication, doctors found he no longer had HIV in his blood.

After another round of stem cell treatment in February of 2008, Brown went through several near-death complications, almost going blind and becoming paralyzed but slowly recovering. His body was still successfully fighting off HIV.

In July 2012, the Timothy Ray Brown Foundation was created during the World AIDS Conference in Washington, DC. This foundation was built for Brown to show his support and work with medical institutions and scientists to develop a unifying cure and vaccination against HIV.

Brown would often donate large amounts of blood and tissue samples to researchers in the hope of progressing closer towards an HIV cure. According to his partner, Hoeffgen, Tims lifework was to tell his story about his HIV cure and become an ambassador of hope to those in need.

Doctors have since used Brown as a blueprint to work on a potential cure and vaccine for HIV. Most notably for the second person to ever be cured of HIV the London Patient, Adam Castillejo who went through similar stem cell transplants in 2019 before coming forward to the public.

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NMDP/Be The Match partners with M Health Fairview and Duke University cryopreservation labs to launch Be The Match BioBank – PRNewswire

By daniellenierenberg

MINNEAPOLIS, Oct. 6, 2020 /PRNewswire/ --The National Marrow Donor Program (NMDP)/Be The Match today announced a collaboration with the Minnesota health system M Health Fairview and Marcus Center for Cellular Cures (MC3)/Carolinas Cord Blood Bank at Duke University (Duke) to offer cryopreservation services to transplant centers through the Be The Match BioBank. The collaboration brings together industry-leading expertise in cryopreservation and storage of patient-directed donor blood stem cell products to improve donor availability, collection quality, and ultimately, to provide a more reliable path to transplant for patients.

Through the Be The Match BioBank, blood stem cell donors will be able to donate bone marrow or peripheral blood stem cells (PBSC) for an intended patient on a timeline that is convenient for the donor. The cells are then cryopreserved and stored for the transplant center at no cost to them and shipped to coincide with initiation of the patient's conditioning regimen and optimal treatment timeline.

"We're excited to expand our partnership with Duke University by adding the expertise of physicians and researchers at M Health Fairview University of Minnesota Medical Center to continue to overcome logistical barriers to blood and marrow transplantation that might otherwise disrupt optimal patient care. Through the flexibility offered by the Be The Match BioBank, we believe we can provide transplant centers with a well-matched, available donor more often, and allow the transplant to occur at the best time for the patient," explained Steven Devine, MD, Chief Medical Officer, NMDP/Be The Match, and Associate Scientific Director, CIBMTR (Center for International Blood and Marrow Transplant Research). "The team at the Duke University lab was instrumental in the development of the Be The Match BioBank, as well as supporting donor product cryopreservation during the COVID-19 pandemic to ensure patients can continue to receive the transplants they need."

"We are proud to extend our partnership with the NMDP/Be The Match in a new way. Be The Match BioBank is an innovative way to remove barriers that otherwise may stand in the way of a patient's transplant," said Joanne Kurtzberg, MD, who leads the Marcus Center for Cellular Cures (MC3)/Carolinas Cord Blood Bank at Duke University.

"We are thrilled to be working with the NMDP/Be The Match to offer Be The Match BioBank. Through this partnership, transplant physicians can have confidence a high-quality bone marrow or PBSC product will be available from the donor they requested in the timeframe that works best for their patient," said David McKenna, MD, who leads the Molecular and Cellular Therapeutics program at M Health Fairview.

Be The Match BioBank can be used by any transplant center in the NMDP/Be The Match Network of more than 180 transplant centers worldwide. Blood stem cell donors are informed that the transplant center is requesting cryopreservation and provide consent prior to collection. Donors can also consent to having their donated cells made available to other searching patients in the unlikely event the intended patient is unable to proceed to transplant as planned.

To learn more about Be The Match BioBank, visit

About the National Marrow Donor Program/Be The Match The National Marrow Donor Program/Be The Match is the global leader in providing a cure to patients with life-threatening blood and marrow cancers like leukemia and lymphoma, as well as other diseases. The organization manages the world's largest registry of potential blood stem cell donors and cord blood units. The NMDP/Be The Match partners with a global network to connect patients to their donor match for a transplant, and provides education and support for patients. Through Be The Match BioTherapies, the NMDP/Be The Match partners with cell and gene therapy companies to support the development and delivery of new therapies. The organization conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), in collaboration with Medical College of Wisconsin.

About M Health Fairview M Health Fairview is the newly expanded collaboration betweenthe University of Minnesota, University of Minnesota Physicians,and Fairview Health Services. The healthcare system combines the best of academic and community medicine expanding access to world-class, breakthrough care through its 10 hospitals and 60 clinics.

SOURCE Be The Match

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Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -…

By daniellenierenberg

Gosselies, Belgium, 5 October 2020, 7am CEST BONE THERAPEUTICS(Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, Link Health Pharma Co., Ltd (Link Health) and Shenzhen Pregene Biopharma Company, Ltd (Pregene) today announce the signing of an exclusive license agreement for the manufacturing, clinical development and commercialization of Bone Therapeutics allogeneic, off-the-shelf, bone cell therapy platform ALLOB in China (including Hong Kong and Macau), Taiwan, Singapore, South Korea, and Thailand.

Under the agreement, Bone Therapeutics is eligible to receive up to 55 million in development, regulatory and commercial milestone payments including 10 million in upfront and milestone payments anticipated in the next 24 months. Bone Therapeutics is also entitled to receive tiered double-digit royalties on annual net sales of ALLOB. Bone Therapeutics retains development and commercialization rights to ALLOB in all other geographies outside of those covered by this agreement. As a result, Bone Therapeutics will continue to concentrate on its development and commercialization plans for ALLOB in the US and Europe and novel innovative cell-based products globally.

This collaboration between Bone Therapeutics, Link Health and Pregene expands our geographic reach and demonstrates the global commercial potential of ALLOB,said Miguel Forte, MD, PhD, Chief Executive Officer of Bone Therapeutics. We already have operational experience in Asia with the Phase III clinical trial of our lead product JTA-004 in Hong Kong. We selected Link Health and Pregene to partner with us in Asia as a result of their expertise in advanced therapeutics and cell therapies, their proven track record of development and commercial implementation in Chinese and Asian markets, and Pregenes well established cell therapy manufacturing capacity. Bone Therapeutics will continue to develop the ALLOB cell therapy platform for other markets while exploring additional partnership opportunities in the U.S. and Europe.

The agreement grants Link Health and Pregene exclusive rights to clinically develop and commercialize ALLOB for the treatment of human bone disorders in Greater China, Taiwan, Singapore, South Korea, and Thailand. All rights for China will be transferred to Pregene and Link Health will gain rights for the remaining countries Bone Therapeutics will share its patented proprietary manufacturing expertise for the expansion and differentiation of bone-forming cells and has the option to sell clinical supplies to Link Health and Pregene in preparation for their clinical development of ALLOB.

This collaboration and license agreement for Bone Therapeutics ALLOB provides a strong addition to our pipeline. ALLOB has demonstrated the potential to reduce the recovery time and stimulate bone growth for a variety of bone conditions, and to have a considerable impact on patients lives,said Yan Song, PhD, Chief Executive Officer of Link Health. It is important for Link Health to collaborate with companies that have strong therapeutic product portfolios and entrepreneurial management. This partnership with Bone Therapeutics is a direct result of our shared commitment to appreciate the enormous potential of cell therapy and regenerative medicine.

Pregene now has a flourishing portfolio of CAR-T cell therapy-based cancer treatments. Bone Therapeutics ALLOB provides anallogeneic, off-the-shelf cell therapy that expands our portfolio of cell therapies to include the sizable commercial potential of orthopedics,said Hongjian Li, Co-founder and Chief Executive Officer of Pregene. We expect to be able to leverage our extensive international cell and gene therapy experience to develop Bone Therapeutics ALLOB platform and subsequently launch products in China and Southeast Asian markets.

ALLOB, an allogeneic and off-the-shelf cell therapy product manufactured through a proprietary, scalable production process, consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors. In preclinical studies ALLOB has shown to reduce healing time in a delayed-union fracture model by half, and has demonstrated good tolerability and signs of efficacy in two Phase IIa studies for two separate indications. The Companys randomized, placebo-controlled, double-blind Phase IIb clinical trial in patients with difficult tibial fractures has received approval from regulatory authorities in six of the seven planned European countries to date, and is expected to enroll the first patient later this year.

About Link Health Pharma Co., Ltd

Link Health is a leading Chinese pharmaceutical company based in Guangzhou, Southern China, focusing on the development of innovative drugs for unmet medical needs.

Link Health has created a highly professional team with diverse expertise in drug development, medical affairs and regulatory affairs. Leveraging deep understanding of China market, regulatory environment and strong network with global biopharmaceutical companies, Link Health is well positioned to bring innovative drugs to the market efficiently. The company has a drug development pipeline of 5 clinical stage assets and 1 under NDA reviewing in China.

The company has also established a fully owned subsidiary in Amsterdam, the Netherlands. The Dutch office builds and further strengthen collaborations with global pharma/biotech partners and research institutes.

About Pregene Biopharma Co., Ltd

Shenzhen Pregene Biopharma Co. Ltd is a leading enterprise in the cell and gene therapy field with the core technology for industrialization. The companys core team comes from well-known institutions and companies including the Academy of Military Medical Sciences, the University of Toronto, and the US FDA.

Pregene has established the gene editing platform, viral vector and cell production platform, nanobody selection platform and other small to pilot trial manufacturing system, with total investment over 100 million CNY. It has the laboratories and GMP plants for cell and gene therapy of over 10,000 square meter.

The company focuses on the research and development of cell and gene therapy drugs, and participated in the drafting the national standard Considerations for CAR-T Cell Quality Study and Non-clinical Evaluation issued by the National Institutes for Food and Drug Control in June 2018. The CAR-T cell therapy for the treatment of multiple myeloma have obtained NMPA IND clearance as the Class I new drug, which is the first in China and fastest in the world using the humanized single domain antibody in CAR construct, and phase I clinical trials are now in progress. Other pipelines such as CAR-T, TCR-T and mRNA drugs for tumors, autoimmune diseases and other indications are in the development at different stages. The company has broad development prospects with the abundant backup technologies.

Looking forward to the future, the company will build the core capacity in one-stop solution for cell and gene therapy drugs, and fulfill the Express of innovative medicine development from drug discovery to clinical products.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company is ready to start the phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 /

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94

For US Media and Investor Enquiries:LHA Investor RelationsYvonne BriggsTel: +1 310 691

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Hematopoietic Stem Cell Transplantation (HSCT) Market to eyewitness massive growth by 2026 | Escape Therapeutics Inc., Cryo-Save AG, Regen Biopharma…

By daniellenierenberg

Hematopoietic Stem Cell Transplantation (HSCT) Market Scenario 2020-2026:

The Global Hematopoietic Stem Cell Transplantation (HSCT) market exhibits comprehensive information that is a valuable source of insightful data for business strategists during the decade 2014-2026. On the basis of historical data, Hematopoietic Stem Cell Transplantation (HSCT) market report provides key segments and their sub-segments, revenue and demand & supply data. Considering technological breakthroughs of the market Hematopoietic Stem Cell Transplantation (HSCT) industry is likely to appear as a commendable platform for emerging Hematopoietic Stem Cell Transplantation (HSCT) market investors.

This Hematopoietic Stem Cell Transplantation (HSCT) Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution, etc., these data help the consumer know about the competitors better.

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The complete value chain and downstream and upstream essentials are scrutinized in this report. Essential trends like globalization, growth progress boost fragmentation regulation & ecological concerns. This Market report covers technical data, manufacturing plants analysis, and raw material sources analysis of Hematopoietic Stem Cell Transplantation (HSCT) Industry as well as explains which product has the highest penetration, their profit margins, and R&D status. The report makes future projections based on the analysis of the subdivision of the market which includes the global market size by product category, end-user application, and various regions.

Topmost Leading Manufacturer Covered in this report:Escape Therapeutics Inc., Cryo-Save AG, Regen Biopharma Inc., CBR Systems Inc., ViaCord Inc., Lonza Group Ltd., Pluristem Therapeutics Inc., China Cord Blood Corp.

Product Segment Analysis: Allogeneic, Autologous

Application Segment Analysis:Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

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Regional Analysis For Hematopoietic Stem Cell Transplantation (HSCT)Market

North America(the United States, Canada, and Mexico)Europe(Germany, France, UK, Russia, and Italy)Asia-Pacific(China, Japan, Korea, India, and Southeast Asia)South America(Brazil, Argentina, Colombia, etc.)The Middle East and Africa(Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Market Synopsis:The market research report consists of extensive primary research, as well as an in-depth analysis of the qualitative and quantitative aspects by various industry specialists and professionals, to gain a deeper insight into the market and the overall landscape.

The objectives of the report are:

To analyze and forecast the market size of Hematopoietic Stem Cell Transplantation (HSCT)Industry in theglobal market. To study the global key players, SWOT analysis, value and global market share for leading players. To determine, explain and forecast the market by type, end use, and region. To analyze the market potential and advantage, opportunity and challenge, restraints and risks of global key regions. To find out significant trends and factors driving or restraining the market growth. To analyze the opportunities in the market for stakeholders by identifying the high growth segments. To critically analyze each submarket in terms of individual growth trend and their contribution to the market. To understand competitive developments such as agreements, expansions, new product launches, and possessions in the market. To strategically outline the key players and comprehensively analyze their growth strategies.

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At last, the study gives out details about the major challenges that are going to impact market growth. They also report provides comprehensive details about the business opportunities to key stakeholders to grow their business and raise revenues in the precise verticals. The report will aid the companys existing or intend to join in this market to analyze the various aspects of this domain before investing or expanding their business in the Hematopoietic Stem Cell Transplantation (HSCT) markets.

Contact Us:Grand View Report(UK) +44-208-133-9198(APAC) +91-73789-80300Email : [emailprotected]

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Hemogenyx Pharmaceuticals PLC Announces SAFE-HEMO-CAR-T Effective against AML in vitro – BioSpace

By daniellenierenberg

LONDON, UK / ACCESSWIRE / October 6, 2020 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the biopharmaceutical group developing new therapies and treatments for blood diseases, is pleased to announce the following update on its activities.

As previously announced, Hemogenyx Pharmaceuticals' CDX bi-specific antibody has the potential to treat Acute Myeloid Leukemia ("AML") directly as well as to provide a benign conditioning regimen for blood stem cell replacement therapy. The Company has now carried out extensive work developing treatments for AML and has to date obtained encouraging results.

As announced on 20 February 2020, the Company has constructed and successfully tested in vivo Chimeric Antigen Receptor ("CAR") programmed T cells ("HEMO-CAR-T") for the potential treatment of AML. HEMO-CAR was constructed using the Company's proprietary humanized monoclonal antibody against a target on the surface of AML cells.

It was also announced that the Company was engaging in additional engineering of HEMO-CAR-T cells to increase their safety and versatility. The Company has now introduced and successfully in vitro tested a safety switch within the HEMO-CAR. The aim of this safety switch is to modulate the activity of HEMO-CAR-T cells and to turn them into a "controllable drug" - SAFE-HEMO-CAR-T. The purpose of these efforts is to dramatically improve the safety and potential versatility of HEMO-CAR-T cells for the treatment of AML and/or conditioning of bone marrow transplants, as well as a number of additional potential indications.

Following the successful completion of these in vitro tests, in vivo tests of the efficacy of SAFE-HEMO-CAR-T against AML are being conducted using a model of AML established on the background of Advanced peripheral blood Hematopoietic Chimera (ApbHC) - humanized mice developed by Immugenyx, LLC, a subsidiary of Hemogenyx Pharmaceuticals. If these in vivo tests are successful, the Company will discuss its findings with its partners under the Sponsored Research Agreement with the University of Pennsylvania, announced on 11 August 2020, with a view to considering the inclusion of SAFE-HEMO-CAR-T in the program of pre-clinical trials currently underway there.

Dr Vladislav Sandler, Chief Executive Officer, commented, "We are encouraged by this new data which demonstrates our continuing progress in the development of novel treatments for blood cancers such as AML. The development of SAFE-HEMO-CAR-T further expands the Company's pipeline and advances it into a cutting-edge area of cell-based immune therapy. We are excited to have developed another unique product candidate that should, if successful, provide a new and potentially effective treatment for blood cancers for which survival rates are currently very poor."

About AML and CAR-T

AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 25% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx Pharmaceuticals. The successful development of the new therapy for AML would have a major impact on treatment and survival rates for the disease.

CAR-T therapy is a treatment in which a patient's own T cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T cells from the patient, modifying the isolated T cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been deemed inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 until the release of this announcement.


Hemogenyx Pharmaceuticals plc


Dr Vladislav Sandler, Chief Executive Officer & Co-Founder

Peter Redmond, Director

SP Angel Corporate Finance LLP

Tel: +44 (0)20 3470 0470

Matthew Johnson, Vadim Alexandre, Soltan Tagiev

Peterhouse Capital Limited

Tel: +44 (0)20 7469 0930

Lucy Williams, Duncan Vasey, Charles Goodfellow

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility.

The Company is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. The Company's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation.

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact or visit

SOURCE: Hemogenyx Pharmaceuticals PLC

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AVROBIO and University of Manchester Enter Agreement for MPS II Research – BioSpace

By daniellenierenberg

Massachusetts-based AVROBIO announced today that it has entered an exclusive global license agreement, as well as a collaborative research funding agreement, with The University of Manchester. Together, the university and AVROBIO will look into an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome.

The condition, which impacts an estimated one in 100,000 males worldwide, causes complications throughout the body and brain. Children with severe cases typically show symptoms beginning in their toddler years. At the moment, the standard of care is weekly enzyme replacement therapy, but it does not halt progression of the disease or address cognitive issues that may arise.

We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures, said Geoff MacKay, AVROBIOs president and CEO. We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Prof. Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. Were proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.

The investigational gene therapy, titled AVR-RD-05, includes ex vivo transduction of the patients own hematopoietic stem cells with a therapeutic transgene. The transgene is meant to express functional enzymes that the patient needs to maintain cellular health. When reinfused back into the patient, the modified stem cells are designed to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene.

This is just one company looking toward making an impact in the MPS II realm as of late. REGENXBIO announced at the end of September that it was expanding its RGX-121 program, looking into the treatment of MPS II. RGX-121 is an investigational one-time gene therapy that uses the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system.

An ongoing Phase I/II study is evaluating a single intracisternal administration of RGX-121 in severe instances of MPS II in patients under the age of five. As of Sept. 16, RGX-121 was reported to be well-tolerated in patients and there were no drug-related serious adverse events.

"MPS II is a serious and debilitating lysosomal disease that affects 1 in 100,000 children, and available treatments are inadequate to treat the neurodegenerative manifestations of the disease, said Terri Klein, President and Chief Executive Officer of the National MPS Society. Initiating a natural history study will increase the understanding of neurocognitive effects and key biomarkers of severe MPS II, and is critical to advancing the development of new treatment options. We are grateful for REGENXBIO's dedication to MPS and commitment to share the learnings from this observational study with the community.

REGENXBIO has also announced that the U.S. Food and Drug Administration cleared an Investigational New Drug application. The company plans on initiating a second Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with severe MPS II between the ages of five and 18.

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Congress Votes | News | – The Albany Herald

By daniellenierenberg

WASHINGTON Heres a look at how Georgias members of Congress voted over the previous week.

Along with its roll call votes this week, the House also passed the Cyber Sense Act (H.R. 360) to require the Secretary of Energy to establish a voluntary Cyber Sense program to test the cybersecurity of products and technologies intended for use in the bulk-power system; the Consumer Product Safety Inspection Enhancement Act (H.R. 8134) to support the Consumer Product Safety Commissions capability to protect consumers from unsafe consumer products; the School-Based Allergies and Asthma Management Program Act (H.R. 2468) to increase the preference given in awarding certain allergies and asthma-related grants to states that require certain public schools to have allergies and asthma management programs; and the Effective Suicide Screening and Assessment in the Emergency Department Act (H.R. 4861) to establish a program to improve the identification, assessment and treatment of patients in the emergency department who are at risk of suicide.

DISCLOSING TIES TO UYGHUR LABOR: The House has passed the Uyghur Forced Labor Disclosure Act (H.R. 6270) sponsored by Rep. Jennifer Wexton, D-Va., to require publicly traded companies to disclose whether they have business ties to Chinas Uyghur Autonomous Region in Xinjiang province. Wexton said the requirement would let investors know of a given companys passive complicity or active exploitation of one of the most pressing and ongoing human rights violations of our lifetime. A bill opponent, Rep. Anthony Gonzalez, R-Ohio, said it wrongly tried to have the Securities and Exchange Commission police human rights violations, a role that would be better handled by the Treasury Department. The vote, on Sept. 30, was 253 yeas to 163 nays.

NAYS: Loudermilk R-GA (11th), Allen R-GA (12th), Scott, Austin R-GA (8th), Collins R-GA (9th), Carter R-GA (1st), Woodall R-GA (7th), Ferguson R-GA (3rd), Hice R-GA (10th)

YEAS: Bishop D-GA (2nd), Scott, David D-GA (13th), McBath D-GA (6th), Johnson D-GA (4th)

NOT VOTING: Graves R-GA (14th)

PRESIDENTIAL ELECTION: The House has passed a resolution (H. Res. 1155) sponsored by Rep. Eric Swalwell, D-Calif., reaffirming the Houses commitment to an orderly and peaceful transfer of presidential power after the November election. Swalwell said: The peaceful transition of power is not only a bedrock principle of Americas founding, it is a living ideal that we must exercise and pass down to our children. An opponent, Rep. Matt Gaetz, R-Fla., called the resolution a way for Democrats to attack the president and disguise the fact that they will refuse to accept the election results unless they win. The vote, on Sept. 29, was 397 yeas to 5 nays.

YEAS: Entire delegation, except Collins R-GA (9th), Graves R-GA (14th), who did not vote

DISEASE THERAPIES: The House has passed the Timely ReAuthorization of Necessary Stem-cell Programs Lends Access to Needed Therapies Act (H.R. 4764) sponsored by Rep. Doris O. Matsui, D-Calif. The bill would reauthorize a program for transplanting umbilical cord blood, stem cells, and bone marrow to adults and children suffering from various diseases. The vote, on Sept. 30, was unanimous with 414 yeas.

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YEAS: Entire delegation, except Graves R-GA (14th), who did not vote

FURTHER COVID-19 SPENDING: The House has approved an amendment to the Americas Conservation Enhancement Act (H.R. 925). The amendment would spend $2.2 trillion on new COVID-19 measures, including testing and treatment efforts and unemployment benefits. A supporter, Rep. James P. McGovern, D-Mass., said the spending was needed for families to pay for necessities like food, utilities, and rent during this pandemic. An opponent, Rep. Tom Cole, R-Okla., said the amendment had been hurriedly brought to the floor without minority input or adequate time for review, and that it would not pass the Senate. The vote, on Oct. 1, was 214 yeas to 207 nays.

NOT VOTING: Loudermilk R-GA (11th), Graves R-GA (14th)

YEAS: Bishop D-GA (2nd), Scott, David D-GA (13th), McBath D-GA (6th), Johnson D-GA (4th)

NAYS: Allen R-GA (12th), Scott, Austin R-GA (8th), Collins R-GA (9th), Carter R-GA (1st), Woodall R-GA (7th), Ferguson R-GA (3rd), Hice R-GA (10th)

CONTINUING APPROPRIATIONS: The Senate has passed the Continuing Appropriations Act and Other Extensions Act (H.R. 8337) sponsored by Rep. Nita M. Lowey, D-N.Y., to extend through Dec. 11 funding for health programs, including Medicare, surface transportation, and many other government programs. The vote, on Sept. 30, was 84 yeas to 10 nays.

AFFORDABLE CARE ACT LITIGATION: The Senate has rejected a cloture motion to end debate on a motion to consider a bill (S. 4653) sponsored by Senate Minority Leader Chuck Schumer, D-N.Y., that would block the Justice Department from making arguments in court for cancelling any provision of the 2010 health care reform law (ACA). The vote to end debate on Oct. 1, was 51 yeas to 43 nays, with a three-fifths majority needed for approval.

Congress Votes | News | - The Albany Herald

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EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD – – pharmaphorum

By daniellenierenberg

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

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