StemCell Therapy

The European Medicines Agency has validated BioMarins application to market its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.

As such, the company said it expects the agencys review of the therapy in January next year under accelerated assessment.

The EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 for valoctocogene roxaparvovec and recently granted BioMarin's request for accelerated assessment of the MAA, potentially shortening the review period.

The submission is based on an interim analysis of study participants treated in an ongoing Phase III study with material from the to-be-commercialised process and updated three-year Phase I/II data.

It marks the first marketing application to be filed in Europe for a gene therapy product for any type of hemophilia.

BioMarin also announced the filing of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the treatment, with the review expected to being in February.

"We are pleased that the agency has recognised the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A," said Hank Fuchs, president, Global Research and Development at BioMarin.

"We continue to move thoughtfully and urgently through the regulatory review process to deliver a treatment that we believe has the potential to make a meaningful difference to people with hemophilia A.

See more here:
BioMarin's haemophilia gene therapy moves forward in the EU - PharmaTimes

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the worlds costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartiss AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January, Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a Russian roulette.

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

This may feel like youre blindly passing it out, but it may be the best we can do, said Alan Regenberg, who is on the faculty at Johns Hopkins Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2, he said.

According to Rucinski, the parties will continue their discussion in January to see what can be improved in the design of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogens Spinraza.

Not all of the SMA community are opposed to Novartis program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who receives the treatment, he said.

Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler

Originally posted here:
Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States. Meredith Rizzo/NPR hide caption

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

"My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out," Gray says. "That's when they found out that I was having my first crisis."

It was Gray's first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

"Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And it's a deep pain. I can't touch it and make it better," says Gray. "Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Gray's journey through this medical experiment, which is being watched closely for some of the first hints that changing a person's genes with CRISPR could provide a powerful new way to treat many diseases.

"This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line," says Dr. Francis Collins, director of the National Institutes of Health.

"To be able to take this new technology and give people a chance for a new life is a dream come true," Collins says. "And here we are."

Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that it's far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

"We have to be cautious. It's too early to celebrate," Frangoul says. "But we are very encouraged so far."

Collins agrees.

"That first person is an absolute groundbreaker. She's out on the frontier," Collins says. "Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation."

This is the story of Gray's journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe. Meredith Rizzo/NPR hide caption

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

It's July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

"Nice to meet y'all," she says.

Gray is just days away from her birthday, which she'll be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

"It's the right time to get healed," says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company. Meredith Rizzo/NPR hide caption

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

"It's horrible," Gray says. "When you can't walk or, you know, lift up a spoon to feed yourself, it gets real hard."

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells don't carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

"I didn't feel normal. I couldn't do the regular things that every other kid could do. So I had to be labeled as the sick one."

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Gray's heart and other parts of her body. Gray knows that many patients with sickle cell don't live beyond middle age.

"It's horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen," she says. "Who wouldn't worry?"

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says. Meredith Rizzo/NPR hide caption

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says.

Gray married and had children. But she hasn't been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

"It's scary. And it affected my oldest son, you know, because he's older. So he understands. He started acting out in school. And his teacher told me, 'I believe Jemarius is acting out because he really believes you're going to die,' " Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; there's no cure for most patients.

"It was just my religion that kind of kept me going," Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

"I was excited," Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

"CRISPR technology has a lot of potential use in the future," Frangoul says.

To try to treat Gray's sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. It's a protein that fetuses make in the womb to get oxygen from their mothers' blood.

"Once a baby is born, a switch will flip on. It's a gene that tells the ... bone marrow cells that produce red cells to stop making fetal hemoglobin," says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain. Ed Reschke/Getty Images hide caption

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

"We are trying to introduce enough ... fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where it's supposed to," Frangoul says.

Then on July 2, after extracting Gray's cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

"They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath," Gray says. "So that was a little scary, tough moment for me."

After that moment passed, Gray says, she cried. But her tears were "happy tears," she adds.

"It was amazing and just kind of overwhelming," she says, "after all that I had went through, to finally get what I came for."

The cells won't cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the disease's complications.

"This opens the door for many patients to potentially be treated and to have their disease modified to become mild," Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her "supercells."

"They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family," she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer. Meredith Rizzo/NPR hide caption

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Concerns about risk

Other doctors and scientists are excited about the research. But they're cautious too.

"This is an exciting moment in medicine," says Laurie Zoloth, a bioethicist at the University of Chicago. "Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases."

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

"This a brand-new technology. It seems to work really well in animals and really well in culture dishes," she says. "It's completely unknown how it works in actual human beings. So there are a lot of unknowns. It might make you sicker."

Zoloth is especially concerned because the research involves African Americans, who have been mistreated in past medical studies.

Frangoul acknowledges that there are risks with experimental treatments. But he says the research is going very slowly with close oversight by the Food and Drug Administration and others.

"We are very cautious about how we do this trial in a very systematic way to monitor the patients carefully for any complications related to the therapy," Frangoul says.

Gray says she understands the risks of being the first patient and that the study could be just a first step that benefits only other patients, years from now. But she can't help but hope it works for her.

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville. Meredith Rizzo/NPR hide caption

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville.

She imagines a day when she may "wake up and not be in pain" and "be tired because I've done something not just tired for no reason." Perhaps she could play more with her kids, she says, and look forward to watching them grow up.

"That means the world to me," Gray says.

It could be many weeks or even months before the first clues emerge about whether the edited cells are safe and might be working.

"This gives me hope if it gives me nothing else," she says in July.

Heading home at last

About two months later, Gray has recovered enough to leave the hospital. She has been living in a nearby apartment for several weeks.

Enough time has passed since Gray received the cells for any concerns about immediate side effects from the cells to have largely passed. And her gene-edited cells have started working well enough for her immune system to have resumed functioning.

So Gray is packing. She will finally go home to see her children in Mississippi for the first time in months. Gray's husband is there to drive her home.

"I'm excited," she says. "I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss. Meredith Rizzo/NPR hide caption

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss.

Gray is wearing bright red glittery eye shadow. It matches her red tank top, which repeats "I am important" across the front.

She unzips a suitcase and starts pulling clothes from the closet.

"My goodness. Did I really bring all this?" she says with a laugh.

Before Gray can finish packing and depart, she has to stop by the hospital again.

"Are you excited about seeing the kids?" Frangoul says as he greets her. "Are they going to have a big welcome sign for you in Mississippi?"

Turns out that Gray has decided to make her homecoming a surprise.

"I'm just going to show up tomorrow. Like, 'Mama's home,' " she says, and laughs.

After examining Gray, Frangoul tells her that she will need to come back to Nashville once a month for checkups and blood tests to see if her genetically modified cells are producing fetal hemoglobin and giving her healthier red blood cells.

"We are very hopeful that this will work for Victoria, but we don't know that yet," Frangoul says.

Gray will also keep detailed diaries about her health, including how much pain she's experiencing, how much pain medication she needs and whether she needs any blood transfusions.

"Victoria is a pioneer in this. And we are very excited. This is a big moment for Victoria and for this pivotal trial," Frangoul says. "If we can show that this therapy is safe and effective, it can potentially change the lives of many patients."

Gray hopes so too.

Read more from the original source:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Health News - NPR

I want to congratulate our team for their success in developing SB-525 through to this important milestone where we have handed over the IND to Pfizer for Phase 3 development, said Sandy Macrae, CEO of Sangamo. We are thrilled to be in a partnership where both parties have cooperated to accelerate study timelines, resulting in completion of the IND transfer ahead of schedule. Pfizer and Sangamo are united in our common interest to help patients with Hemophilia A and will do everything that we can to safely and expeditiously advance this promising gene therapy candidate for patients in need.

The SB-525 collaboration was established in May 2017. Under the terms of the collaboration agreement, Sangamo has been responsible for Phase 1/2 clinical development. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525. Sangamo is eligible to receive total potential milestone payments of up to $300 million for the development and commercialization of SB-525, and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will, additionally, receive tiered royalties starting in the low teens and up to 20% of annual net sales of SB-525.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Sangamo Forward Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of United States securities law. These forward-looking statements include, but are not limited to, the therapeutic potential of SB-525; the enrollment of clinical trials and global registration and commercialization and the expected timing for milestones the expected benefits of Sangamos collaboration with Pfizer; the anticipated capabilities of Sangamos technologies; and other statements that are not historical fact. These statements are based upon Sangamos current expectations and speak only as of the date hereof. Sangamos actual results may differ materially and adversely from those expressed in any forward-looking statements. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to dependence on the success of clinical trials; the uncertain regulatory approval process; the costly research and development process, including the uncertain timing of clinical trials; whether interim, preliminary or initial data from ongoing clinical trials will be representative of the final results from such clinical trials; whether the final results from ongoing clinical trials will validate and support the safety and efficacy of product candidates; the risk that clinical trial data are subject to differing interpretations by regulatory authorities; the potential inability of Sangamo and its partners to advance product candidates into registrational studies; Sangamos reliance on itself, partners and other third-parties to meet clinical and manufacturing obligations; Sangamos ability to maintain strategic partnerships; competing drugs and product candidates that may be superior to Sangamos product candidates; and the potential for technological developments by Sangamo's competitors that will obviate Sangamo's gene therapy technology. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamos operations. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 1, 2019 and Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 that it filed on or about November 6, 2019. Except as required by law, we assume no obligation, and we disclaim any intent, to update these statements to reflect actual results.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191223005185/en/

Original post:
Sangamo Announces Early Completion of Transfer to Pfizer of SB-525 Hemophilia A Gene Therapy IND and an Earned $25 Million Milestone Payment -...

The New Jersey Institute of Technology will collaborate with the New Jersey Innovation Institute, an NJIT corporation, to offer a professional science masters degree program and professional graduate certificate in the rapidly expanding field of cell and gene therapy.

Students be able to begin taking the 30-credit masters degree in spring 2020 as part of NJITs Department of Chemistry and Environmental Sciences pharmaceutical chemistry masters program.

NJIT says this is a four-course certificate program for anyone who is seeking training in cutting-edge technologies required for processing and commercializing of new therapies.

Hands-on training in the fields newest approaches and technologies will be supported at NJITs labs and at The New Jersey Innovation Institutes Cell and Gene Therapy Development Center. NJII says the center will upgrade the knowledge and skills of bio-pharmaceutical professionals in the processing of new, breakthrough classes of biologic therapies.

Both programs were developed to meet growing demands from the bio-pharmaceutical industry for trained scientists and engineers at the forefront of the coming wave of breakthrough gene and cell therapies, including advanced gene delivery technologies to immunotherapies such as CAR-T cancer therapy.

Original post:
NJIT to start programs in cell and gene therapy - NJBIZ

Whats the best way to measure the real rate of progress in personalized cell therapies, gene therapies, and other advanced therapies?

Ive been tracking the ever-growing flow of reports about these therapies in scientific journals and press releases for 15 years, ever since I co-led the passage of Californias $3 billion Stem Cell Research and Cures Act in 2004.

But to truly gauge who will benefit from todays innovations, Ive learned I also need to study the stream of business and technology announcements that runs in parallel. That might seem more mundane but to veterans of advanced therapies, making the science work actually signals success for these gene-, tissue-, and cell-based advanced therapies.

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The reason is simple. My experience working with advanced therapies has taught me, time and again, that true next-generation medicine requires the industrialization of personalization. That sounds like an oxymoron, but it isnt. To create individualized therapeutics in a sustainable way, we need to deliver even if it seems counterintuitive mass customization.

Breakthroughs such as CAR-T cell therapies are inspiring. They are also unsustainably expensive, difficult to manufacture, and complicated to deliver. We can change this by creating a more focused cross-collaborative production and delivery ecosystem.

The Food and Drug Administration anticipates that it will approve 10 to 20 advanced therapies a year beginning in 2025. It also expects to receive up to 200 clinical trial applications for cell and gene therapies per year, starting now. The more than 1,000 advanced therapy clinical trials now underway worldwide could enroll almost 60,000 patients, according to the Alliance for Regenerative Medicine. That pace wont be possible without new systems and networks that reduce cost, simplify manufacturing, and streamline delivery.

I can see some of these on the horizon when I read the biotech and pharma partnerships reported in BioSpace and BioCentury. Of the 100 most recent, almost 10% were dedicated to cell- and gene-therapy companies and organizations. These partnership announcements are typically viewed as opportunities to highlight new business deals or contract wins. But they are also daily snapshots of the infrastructure of an evolving next-generation health care system forming from within. Here are just a few examples from 2019:

Its encouraging to see biopharma manufacturing, logistics, transport, and other partners in the cell- and gene-therapy ecosystem coming together in new ways to ensure the successful and reliable delivery of advanced therapies for individual patients. But much more evolution is needed to provide sustainable patient access to advanced therapies.

We need even more industry collaboration to overhaul and connect existing health care systems, so production and delivery of cell- and gene-based therapies can be more automated and affordable. According to estimates from credible industry colleagues and leaders, end-to-end automation can shave costs by at least 20% to 30%, and at the same time greatly improve predictability and patient safety.

We must also make this new world simpler for health care providers. Doctors and nurses must not only understand how advanced therapies work medically, but be able to order and deliver them safely with a minimum of delay or hassle. As noted in the New Yorker, CAR-T requires bringing a manufacturing lens to medicine. Supporting health care providers means creating true collaboration between digital technology providers, hospitals, logistics providers, biotech and pharma companies, and manufacturing, like the Boston initiative I described earlier.

Standardization is often decried as cookie-cutter medicine. In this space, however, it is the wave of the future.

While patient biology is unique, and each patients cells may produce a one-of-a-kind manufacturing batch, essential parts of the production and delivery process should be as predictable and easy as possible. One key place to start is in-process drug labeling. When patients cells become the raw material for advanced therapies, these labels become more complex and more necessary: When a patient is about to receive a cell therapy infusion, its essential that the name on the bag of genetically re-engineered cells is his or hers. The Standards Coordinating Body, an FDA-funded but independent nonprofit, is now leading an industry-wide labeling initiative for cell and gene therapies.

There are other clear signs that the advanced therapies field gets it when it comes to infrastructure needs, such as the inclusion of digital health and handling of patient data as categories of focus in the federal Cures 2.0 initiative currently circulating in Washington. But much remains to be done.

In centers caring for individuals with cancer and rare diseases, thousands of patients are today receiving advanced therapies that are transforming their lives. We need to make that possible for many, many more by working together to industrialize and personalize in parallel.

Amy DuRoss is the CEO and co-founder of Vineti, a digital technology company that provides next-generation software platforms for advanced therapies. Before that she was managing director for new business creation for GE Ventures, chief business officer at Navigenics, the co-founder and executive director of Proposition 71, Californias $3 billion stem cell research initiative that passed in 2004, and chief of staff at the resulting California Institute for Regenerative Medicine.

Original post:
Making advanced therapies takes industrializing personalization - STAT

Sanofi was locked in a bidding war right up to the final moments of closing its $2.5 billion buyout of Synthorx, as it rushed to complete a deal this or another to bolster CEO Paul Hudsons new R&D vision before wrapping the year. By Synthorxs account, what began as routine partnership talks took a sharp turn into two weeks of intense negotiations in which the San Diego biotech was able to almost double the offer.

By moving swiftly and aggressively, Sanofi fended off three other suitors to pocket a slate of next-gen IL-2 drugs for cancer and autoimmune diseases as well as a synthetic biology platform. The pharma giant now takes over a pipeline whose most advanced asset it still in Phase I/II befitting an organization that now vows to get in early enough to change a treatment paradigm.

The initial meetings with Synthorx took place at all the usual places: ESMO 2018, JP Morgan and AACR 2019. Soon Sanofi R&D chief John Reed stepped in, but the smaller player continued to explore options with other companies at ASCO and BIO over the summer.

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UPDATED: Sarepta cements its DMD throne with $1B+ gene therapy deal with mighty Roche - Endpoints News

I never thought Id see the day when words like process, scale, and automation would make news in the biopharma industry. Yet as the race heats up to bring more first-of-their-kind gene therapies to market, breakthroughs in manufacturing are often the key or break down the barrier to delivering these therapies to patients.

In my career, which has largely focused on drug manufacturing, Ive been lucky to be directly involved in the approval of six new medicines. My current work, as head of technical operations at Spark Therapeutics, is offering the biggest challenge: bringing Luxturna, the first gene therapy for a genetic disease, to patients and families in the U.S. Getting here has been no small task.

With no precedent to guide us, we had to forge new clinical, regulatory, and manufacturing pathways. Working through the unknown meant developing a robust set of assays to test various aspects of the gene therapy product just so we could better understand it. We also built, from scratch, the only in-house manufacturing facility for a licensed gene therapy that is approved by both the U.S. Food and Drug Administration and the European Medicines Agency. This facility is located on the 13th floor of a high-rise in West Philadelphia.

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Gene therapy, as others in this space know, is not a one-size-fits-all approach. That means there isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, as there is for well-established therapeutic categories. And at least for now, every gene therapy is different. Each relies on a different delivery mechanism (vector) to transport functional copies of a gene into the patient.

Even if one day we have a platform that is flexible enough to accommodate multiple vector types, well still need to consider the fact that individual therapies require different dosing and modes of administration, both dependent on the patients cells and disease. While we certainly seek to standardize processes through enhanced analytics, automation, and even artificial intelligence, manufacturing each therapy will still require custom processes.

And time is of the essence, because patients and their families are waiting for these therapies. Given that many of these diseases have limited or no treatment options, regulatory authorities are rightly granting expedited approval pathways for investigational gene therapies. The tight timelines in these pathways narrow the window for manufacturing teams to plan and implement strategies to create gene therapies at scale for commercial use.

Here are three aspects I see as unique to the gene therapy manufacturing process:

Get comfortable with the uncomfortable. Given the shortened clinical development timelines and limited precedent to guide them, gene therapy manufacturers must make decisions about investing in Phase 3 manufacturing processes far in advance of knowing the clinical outcome of their therapy. Its important to trust your expertise and invest in well informed good risk. We saw the success of this at Spark with the first gene therapy, which is helping create a clearer road map for future ones.

Develop capability for capacity. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

Spark, for example, is optimizing the way it produces viral vectors, shifting from adherent cell lines, which attach cells to the sides of roller bottles, to a suspension process that is more efficient and scalable. In this process, bioreactors grow cells unattached, in a liquid or suspended environment. This alternate way of manufacturing uses well-established unit operations commonly employed in the biotechnology industry, making efficiency at scale more easily achievable. Less manual manipulation provides for more process consistency and higher success rates. Each of these elements aids in our ability to scale more easily.

Dont let perfection be the enemy of progress. Versions of this phrase have been attributed to Voltaire, Shakespeare, and Winston Churchill, among others, but the point here is that when it comes to manufacturing, the process is never perfect and can always be better. Our gene therapy manufacturing processes are constantly evolving based on what we learn from them and from new best practices. What matters most today is that we can manufacture gene therapies safely and effectively. The speed will continue to improve.

Manufacturers are accustomed to setting up highly repeatable processes for making and delivering medicines. But when it comes to gene therapies, we understand that the ingenuity for manufacturing needs to be as unique and cutting-edge as the therapies themselves.

While its exciting to see gene therapy manufacturing in the limelight today, I hope that the progress we are making will soon make these challenges old news.

Diane Blumenthal is the head of technical operations at Spark Therapeutics, where her responsibilities include manufacturing, quality control, and more.

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Manufacturing: the next breakthrough in gene therapy - STAT - STAT

A new method allows researchers to develop adeno-associated virus (AVV) commonly used as the vehicle for gene therapies that accurately target and deliver genes to specific cells in the body.

This new technology may be suitable to target dopaminergic neurons that are damaged in Parkinsons disease.

We believe that the new synthetic [lab-made] virus we succeeded in creating would be very well suited for gene therapy for Parkinsons disease, for example, and we have high hopes that these virus vectors will be able to be put into clinical use, Tomas Bjrklund, PhD,Lund University, Sweden, said in a press release.

Bjrklund is lead author of the studyA systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism, which was published in the journal Proceedings of the National Academy of Sciences.

The adeno-associated virus (AAV)is a common, naturally-occurring virus, which has been shown to work as an effective gene therapy delivery vehicle for genetic diseases, such asspinal muscular atrophy. In gene therapy, scientists deliver a working version of a faulty gene using a harmless AAV that was modified and inactivated in the lab. This way the virus functions only as a delivery vehicle and does not have the capacity to damage tissues and cause disease.

While AAVs have a natural ability to penetrate any cell of the body and infect as many cells as possible, their usefulness as a potential therapy requires the capacity to specifically deliver a working gene to a particular cell type, such as dopamine producing-nerve cells. Those are the ones hose responsible for releasing the neurotransmitter dopamine and that are gradually lost during Parkinsons disease.

A team of Swedish researchers have developed a new method called barcoded rational AAV vector evolution, or BRAVE that combines powerful computational analysis with the latest gene and sequencing technology to produce AAVs that can specifically target neurons.

To make AAVs neuron specific, the team selected 131 proteins known to specifically interact with synapses (the junctions between two nerve cells that allow them to communicate).

They then divided the proteins into small sequences, called peptides, and created a large library where each peptide could be identified by a specific pool of genetic barcodes (a short sequence of DNA that is unique and easily identified).

The peptide is then displayed on the surface of the AAV capsid, allowing researchers to test the simultaneous delivery of many cell-specific AAVs in a single experiment.

The team then injected these AAVs into the forebrain of adult rats and observed that around 13% of the peptides successfully homed to the brain. Moreover, 4% of the peptides were transported effectively through axons (long neuronal projections that conduct electrical impulses) toward the nerve cells body.

Researchers then selected 23 of these unique AAV capsids and injected them into rats striatum, a brain region involved in voluntary movement control and affected in Parkinsons disease. Twenty-one of the new AAV capsids had an improved transport capacity within nerve cells than in standard AAVs.

One particular capsid, called MNM008, showed a high affinity for rat dopaminergic neurons. Researchers then tested whether this viral vector also could target human dopaminergic neurons.

The team transplanted neurons generated from human embryonic stem cells into rats striatum. Six months later, they injected either MNM008 or a control AAV capsid and found that MNM008 was able to target these specific cells and be transported into dopaminergic neuronal cell bodies through axons.

Thanks to this technology, we can study millions of new virus variants in cell culture and animal models simultaneously. From this, we can subsequently create a computer simulation that constructs the most suitable virus shell for the chosen application in this case, the dopamine-producing nerve cells for the treatment of Parkinsons disease, Bjrklund said.

Overall, researchers believe the BRAVE method opens up the design and development of synthetic AAV vectors expressing capsid structures with unique properties and broad potential for clinical applications and brain connectivity studies.

The team has established a collaboration with a biotech company, Dyno Therapeutics, to use the BRAVE method in the design of new AAVs.

Together with researchers at Harvard University, we have established a new biotechnology company in Boston, Dyno Therapeutics, to further develop the virus engineering technology, using artificial intelligence, for future treatments, Bjrklund said.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 208

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today

XconomyBoston

Ring Therapeutics, a Flagship Pioneering spinout, launched Thursday with ambitious plans to expand the universe of vectors available for gene therapy delivery.

Gene therapy, treatments intended to treat disease by inserting a gene instead of using drugs or surgery, has had a banner year, with the second ever such therapy approved this year in the US.

Ring want to use itsresearch into viruses that exist in the human body without apparent negative effects to provide more and better options to fuel the rise of gene therapy treatments.

For the past two years, Flagship Pioneering partner and Rings founding CEO Avak Kahvejian says the company has been exploring the human commensal viromebasically, a group of viruses that exist within humans without negative effectsfor its potential to address limitations of the vectors currently used.

The sector relies heavily on adeno-associated viruses (AAVs), which naturally infect humans but arent known to cause disease, to deliver the DNA. Previous exposure, however, can spark an immune response.

A lot of the workhouses in gene therapy have either been pathogenic viruses or viruses that have been taken from other species or viruses that are highly immunogenic, or all of the above, Kahvejian tells Xconomy. That leads to a certain number of limitations, despite the successes and advances weve made to date.

A number of issues stymie widespread use of AAVs, Kahvejian says, including the fact that 10 percent to 20 percent of people have at one time or another been infected with such a virus, thereby building up an immune response to it. Another concern is where such gene therapies end up, because viruses tend to gravitate toward certain types of tissues, and to go elsewhere, require special tweaking.

The Cambridge, MA-based startup believes the viruses it has found are unlikely to cause an immune response or prove pathogenic, given their ubiquity in the body.

Like extrachromosomal DNAa new discovery at least one company is exploring for its potential as a target in cancer treatmentsthe viral sequencing Ring is studying are circular pieces of DNA that exist outside the 23 chromosomes of the human genome.

Ring says it has found thousands of these viruses that coexist with our immune system. It aims to use those to develop vectors that can facilitate gene replacement throughout the bodymultiple times, if necessary. While gene therapy is thought of as a one-time fix, cell turnover means whatever the fix engendered by the inserted gene could falter over time, necessitating a re-up.

Kahvejian wouldnt share a timeline for Rings plan to develop re-dosable, tissue-targeted treatments.

Were looking at the unique features and activities of these viruses in different tissues to establish the various vectors were going to pursue, he said.

Flagship, which pursues scientific questions in-house and builds and funds companies around the answershas put $50 million toward Ring, which has about 30 employees.

Rings president is Rahul Singhvi, an operating partner at Flagship. Most recently he was chief operating officer of Takedas global vaccine business unit. Its head of R&D is Roger Hajjar, who has led gene therapy trials in patients with heart failure.

Ring is the second startup Flagship has spun out this month. Cellarity launched last week.

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

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Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy

Gene therapy is providing unprecedented hope for growing numbers of patients and families. This game changer in medicine restores vision in babies born with congenital blindness, reconstitutes defences against infection in inherited immunodeficiencies and offers the perspective of curing the devastating neuromuscular disease, spinal muscular atrophy.

Gene therapy is also removing the need for repeat blood transfusions in adolescents with the inherited blood disorder, beta-thalassemia. Meanwhile, in oncology, CAR-T therapies, involving genetic modifications of a patients own immune cells, are proving life-saving for children or adults with certain types of blood cancers.

All these revolutionary treatments are now approved by regulatory agencies in Europe or the US. Unfortunately, they carry astronomical price tags which prevent their effective delivery to patients. As one case in point, Bluebird Bios Zynteglo for treating beta-thalassemia, has a list price of 1.57 million.

Can high prices be justified?

Gene therapy manufacturers defend their prices by pointing to high development and manufacturing costs, small markets, and unique therapeutic effectiveness as compared to the current standard of care. However, R&D costs are kept secret, and higher numbers of patients eligible for a given therapy do not translate into lower prices.

Indeed, several arguments the manufacturers put forward are dubious or even far-fetched. As of today, claims that a single administration of a gene therapy product will ensure a lifelong cure are simply not supported by the scientific evidence.

Likewise, value-based pricing is often misconceived. As stated by the US Institute for Clinical and Economic Review in its 2017 white paper on gene therapy, the established value of a treatment reflects the maximum price society might be prepared to pay for it - but should not dictate the price that is actually paid. In an ideal world, actual prices should provide market-consistent returns for shareholders and sufficient incentive to innovate.

The EU, a pioneer in gene therapy

European scientists, institutions and charities have been central to the development of gene therapy. The world's first successful clinical trial was reported in 2000 by Alain Fischer and his team at Necker Hospital in Paris, while the first authorisation of a gene therapy product in a regulated market was granted by the European Medicines Agency in 2012.

According to the Cordis database of EU-supported research, 86 gene therapy projects for rare diseases had funding from the European Commission during the FP7 (2007-2013) and Horizon 2020 (2014-2020) research programmes. One can estimate that overall more than 1 billion has been invested in this area by the EU Commission, member states and not-for-profit organisations.

To ensure European patients benefit from these achievements and investments, it is essential to ensure reasonable pricing of gene therapies. Laudable efforts are currently being made by the World Health Organization to increase transparency, and by some member states to join forces in negotiating prices, but such initiatives are unlikely to solve the current crisis as they do not address its root, namely that the sole objective of most gene therapy companies is to maximise the return on investment and shareholder value.

A way forward: enforcing the corporate social responsibility of gene therapy manufacturers

As I recently argued with Alain Fischer and the economist Mathias Dewatripont in the journal Nature Medicine (November 25, 2019), now is the time to reflect on how to enforce the corporate social responsibility of gene therapy companies.

Among the measures we would like to see considered are the insertion of clauses into technology transfer agreements made between academic organisations receiving grants from the European Commission and for-profit companies to make reasonable pricing compulsory.

We also propose to make reimbursement of gene therapies by EU healthcare payers conditional on the companies which are commercialising these products being certified for their corporate social responsibility. This is in line with several commitments made recently by pharma companies. For example, in August 2019, the CEOs of US-based pharma companies signed the Business Roundtable Statement, affirming their commitment to generate value for all their stakeholders not just their shareholders.

Also in August, Novartis announced it had joined the Value Balancing Alliance, a body whose goal is to increase transparency around business decisions, work with external bodies to develop accounting frameworks, and shift priority from profit maximisation to optimising value creation.

Earlier this year, the pharmaceutical company Chiesi was certified as a Benefit Corporation, meaning its legally defined goals include positive social impact in addition to profit.

Of course, the effective implementation of such commitments and their translation into reasonable pricing policies will require both incentives and regulatory controls. The starting point should be a renewed multi-stakeholder conversation with industry, investors, regulators, payers and, of course, patients.

Professor Michel Goldman is Co-director of the I3h Institute at the Universit Libre de Bruxelles and former Executive Director of the EU Innovative Medicines Initiative.

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Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business

In January 2019, then-FDA commissioner Scott Gottlieb ushered in the new year with a bold prediction: The agency, he said, would be approving between 10 and 20 gene and cell therapies per year by 2025. At the time, there were a whopping 800 such therapies in the biopharma pipeline and the FDA was aiming to hire 50 new clinical reviewers to handle the development of the products.

That momentum will no doubt start to pick up in 2020, as several companies in late-stage development of their gene and cell therapies achieve key milestones or FDA approval. Among the companies expected to make major strides in gene and cell therapies next year are Biomarin, with valoctocogene roxaparvovec to treat hemophilia A, Sarepta and its gene therapy for Duchenne muscular dystrophy, plus multiple players developing CAR-T treatments for cancer, including Bristol-Myers Squibb and Gilead.

But with such explosive growth comes challenges. Gene and cell therapies require enormous up-front investing in complex manufacturing processes, as well asinnovative approaches to securing insurance coverage for products that come with eye-popping price tagssuch as Novartis $2 million gene therapy Zolgensma to treat spinal muscular atrophy. Those are just a few of the obstacles that will be front-and-center in 2020 as more gene and cell therapies make their way towardthe finish line.

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Pharma companies will face challenges figuring out how to incorporate gene and cell therapies into their overall business, said Michael Choy, partner and managing director at Boston Consulting Group, in an interview with FiercePharma. They dont fit well into the normal paradigms of budgeting and decision-making. They require a different pace of evolution and specialized expertise. For now, companies are shoe-horning gene therapies into their current model, but over the long-term there will have to be changes.

That will become increasingly clear in 2020 as both Big Pharma and small up-and-comers move towardthe clinic with their gene and cell therapies. John Zaia, M.D., director of the Center for Gene Therapy at City of Hope, predicts there will be at least three gene and cell therapy FDA approvals in 2020. He also expects to see momentum among companies seeking to improve on the technology to address unmet needs in medicine.

For example, Zaia believes off-the-shelf CAR-T cancer treatments will show promise in early studiesand will be met with enthusiasm in the cancer community, he told FiercePharma in an email. The first generation of FDA-approved CAR-T treatments, Novartis Kymriah and Gileads Yescarta, take several weeks to make because they require removing T cells from patients and engineering them to recognize and attack the patients'cancers. Several companies are advancing off-the-shelf CAR-T treatments, including Precision BioSciences, which has been building out a manufacturing plant equipped to make 10,000 doses per year.

RELATED: Biotech building facility to make genome-edited, off-the-shelf CAR-T therapies

Gene therapies for inherited diseases will make strides in 2020, too, Zaia predicts. City of Hope is one of the participants in a phase 1 study of CSL Behrings gene therapy to treat adults with sickle cell disease. CSL will be racing against several companies working on the disease, including Bluebird Bio, which is testing its beta thalassemia gene therapy Zynteglo in sickle cell. There is a big push from many research centers to cure sickle cell diseaseand early results with the use of gene therapy look very promising, Zaia said. Years of research is finally coming to realization.

With such robust R&D underway in gene and cell therapies, its no surprise several players are stepping up their investments in manufacturing. In October, Sanofi said it would retrofit a vaccine plant in France so it couldbe used for gene therapy manufacturing. Pfizer shelled out $19 million for a North Carolina facility that will serve as its manufacturing hub for gene therapies. Even Harvard University is getting into the game, working with a consortium of contract manufacturers to build a $50 million facility dedicated to making cell therapies and viral vectors for gene therapies.

But how will the healthcare system pay for all of these complex therapies? Its a question that will continue to dog the industry, BCGs Choy said. Theres a lot of interest in outcomes-based payments and payments over time, but the issue is theyre very difficult to implementbecause the infrastructure to track outcomes over time doesnt really exist, he said.

Still, payers and pharma companies are hinting at their willingness to put that infrastructure in place. Pfizer, which is developing DMD and hemophilia gene therapies, said recently its brainstorming with payers on innovative strategies for reimbursement. Novartis and Spark have already pioneered payment strategies that deviate from the standard pay-everything-up-front system. Novartis has some pay-for-performance contracts in place for the $475,000 Kymriah. And in September, Cigna agreed to cover Novartis Zolgensma and Sparks Luxturna on a per-month, per-member schedule.

RELATED: Novartis, Spark gene therapies win a boost with soup-to-nuts Cigna coverage

Despite the many challenges in cell and gene therapy, some players are showing theres likely to be a robust market for these innovative treatments. In its first quarter on the market, Zolgensma brought in $160 million in salesfar surpassing analysts expectations.

The promise of huge returns on gene and cell therapies will likely drive acquisitions in 2020, Choy predicted. These treatments are so transformative for patients, and as the clinical proof of effectiveness continues to grow, youre going to see a lot more deal-making in this area, he said.

Buyers will likely show a willingness to invest in early-stage gene and cell therapies, especially if they come with technology platforms that allow for the development of many follow-up products, Choy added. For these types of therapies, the lifecycles will be much shorter than they are for traditional pharmaceuticals, particularly for rare diseases, he said. If you administer a one-time therapy, that revenue peaks quite quickly and then drops off. So to have a sustainable revenue from a gene therapy business, you need to replace that, which requires managing a pipeline.

Judging from recent events in the burgeoning gene and cell therapy industry, the news flow in 2020 will be generated not just by the industrys largest players, but also by its upstarts. In December, Ferring Pharmaceuticals spinout FerGene turned heads with data showing that its gene therapy to treat non-muscle invasive bladder cancer eliminated tumors in more than half of participants in a phase 3 trial. And Gileads Kite Pharma just applied for FDA approval for its mantle cell lymphoma CAR-T, KTE-X19, based on a 93% overall response rate in a phase 2 trial.

There were 75 gene therapy clinical trials initiated in 2018, nearly doubling the trial starts of 2016momentum thats likely to continue next year, BCG said in a recent report. The scientific foundation is in place, BCG analysts concluded, but there is still much to do to deliver the full benefit of gene therapy to patients."

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Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma

RESEARCH TRIANGLE PARK AskBio, a gene therapy company based in RTP that recently raised $225 million from investors, will pay up to $240 million in upfront and milestone payments for a license to use gene therapy technology from Massachusetts-based Selecta Biosciences as part of a regime to treat Pompe disease.

The license is for ImmTOR, what Selecta describes as a immune tolerance platform. The technology is addressing barriers to repeat administration of gene therapies.

Pompe disease affects between 5,000 and 10,000 people a year, affectingventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills, AskBio says.

There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selectas ImmTOR technology, said Sheila Mikhail, CEO and co-founder of AskBio, in a statement. The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.

The companies initially announced a partnership in August.

AskBio gets $235 million in gene therapy support

RTP-based AskBio expands gene therapy target list with acquisition of Scottish biotech

AskBio acquires nano drug delivery tech company RoverMed (+ video: how process works)

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Triangle-based AskBio to pay up to $240M for rights to use gene therapy technology - WRAL Tech Wire

Takedahas presented early data on the prevalence of and a possible solution for one of gene therapys main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies.

The findings, presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting Dec. 710 in Orlando, Florida, may inform the development of investigational gene therapies forhemophilia A and B.

Takedas gene therapy pipeline for hemophilia includes TAK-754 for hemophilia A, which is currently in a Phase 1 clinical study, and TAK-748 for hemophilia B, still in pre-clinical development.

Gene therapy involves the use of a modified viral vector, which does not cause an infection, to deliver a copy of the gene that provides instructions for making the clotting factor missing in hemophilia patients. The goal is to allow patients to produce their own clotting factor at normal levels, and in a durable manner, to limit the need for regular infusions of factor concentrates.

Most gene therapies being developed for hemophilia use protein shells, or capsids, based on adeno-associated virus (AAV), particularly AAV5 and AAV8, for packing and delivering a working copy of the clotting factor gene. Takedas gene therapy candidates for hemophilia A and B both use recombinant (lab-made) versions of AAV8.

The vector delivers the gene into a patients liver cells, where most clotting factors are produced naturally.

One of the major challenges with this approach is the fact that some patients have been exposed in the past to naturally-occurring AAVs and have become immune to these vectors.

While natural exposure to AAVs does not result in any known disease, people develop antibodies (called neutralizing antibodies, or NAbs) and cell-mediated immune responses that recognize and attack AAV capsids. That blocks gene therapy delivery and compromises its safety and effectiveness. These antibodies are known as anti-AAV.

The presence of neutralizing antibodies against AAVs is one of the major limitations for the successful use of gene therapies, and one of the reasons why patients are excluded from gene therapy trials.

At the ASH meeting, one of the posters presented by Takeda, titled Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia, reported a study of the prevalence of pre-existing natural immunity against AAVs in adults with hemophilia A and B.

The study enrolled 194 patients with hemophilia A and 48 with hemophilia B, in the U.S. and Europe (NCT03185897). Results showed that approximately 50% of them have neutralizing antibodies to AAV2 (the most common in natural infections), to AAV5 or to AAV8. (Notably, 40% of patients carried antibodies against all three vector types.)

Such patients probably will not respond to AAV-based gene therapies and will be excluded from trials. These data will add to our appreciation of preexisting AAV immunity that prevent patient participation in gene therapy trials, the abstract concluded.

Another study conducted by Takeda focused on a potential strategy to overcome this problem.

The data were presented in a poster titled AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies.

Researchers developed an immune adsorption column (IAC) specifially designed to remove anti-AAV8 antibodies from patients plasma using apheresis. In this process, blood is drawn from the patient and separated in plasma and its other components, outside the patients body. The plasma is then run through a platform which could be the IAC column to remove anti-AAV8 antibodies. After this process, the plasma is given back to the patient.

The column under development has a coat of AAV8 capsids that serve as bait to specifically fish out AAV8-targeted antibodies.

Early laboratory tests showed that the column effectively eliminated anti-AAV8 antibodies from human plasma samples, a result further supported by animal studies.

IAC is an enabler for treatment of patients with pre-existing immunity against AAV8 and would also facilitate re-administration. IAC is intended to be applied in combination with Takedas AAV8 based hemophilia programs, researchers wrote.

As we continue to advance our hemophilia A and hemophilia B investigational gene therapy programs, Takeda is also investigating approaches to overcome the challenges of current AAV gene therapies that could potentially be applied to hemophilia and other rare monogenic [a single gene] diseases, Dan Curran, MD, head of Rare Diseases Therapeutic Area Unit at Takeda, said in a press release.

Developing new gene therapy approaches including those capable of treating pre-existing immunity to AAV, enabling re-dosing, lowering doses, enhancing biodistribution and developing alternative gene delivery vehicles are critical to one day providing functional cures to patients, Curran said.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 121

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Takeda Presents Data for Hemophilia A and B Gene Therapy Optimization - Hemophilia News Today

As a new mother, she didnt know to look for blue-tinged lips. She could just tell her babys color was off. On a chest X-ray, the clean, white-against-dark curves of his ribs were obscured, clouded by fluid. Pneumonia. That tipped Ray Ballards physicians off: He had a form of severe combined immunodeficiency SCID, for short a genetic mutation that hampered the growth of crucial immune cells, leaving him utterly vulnerable to infection.

The best fix was a transplant of his mothers bone marrow. The attitude was that in three to six months, you should be able to go back to normal life, recalled his mom, Barb Ballard.

That was true at least sort of. He got two more booster transplants before he hit 10. An antibiotic left him with hearing loss, and a virus with digestive tract damage. His lack of B cells meant he needed regular injections of other peoples antibodies, and his T cell counts were never ideal. But he was healthy enough to go to public school, to move through the hallways high-fiving half the guys, to slowly inhale and take aim during rifle team practice.

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His T cells had to be working well enough that he wasnt coming down with everything that walked into the classroom, Ballard said.

Then, when Ray was around 18, his immunity began to wane. For him, it came in the form of a norovirus he couldnt shake. For others with the same rare disease, it appears as pneumonia or gastrointestinal trouble or an unexpected T cell decline. Over the last 10 years, the trend has become increasingly clear: The bone marrow transplants that kept certain babies with SCID alive sometimes stop working after years or decades of providing fairly reliable immune defenses.

Now, to patient advocates, this has become an urgent lesson in the language people use to talk about treatment and not just for SCID. They see their communitys experience as a cautionary tale for anyone developing or receiving a therapy thats marketed as potentially curative.

Theres an expectation and a hope: When they hear about bone marrow transplants, it sounds like a lifetime deal, a forever fix, said John Boyle, president and CEO of the Immune Deficiency Foundation. Weve discovered, as a result of this issue, that bone marrow transplant ended up not being the forever fix we thought it was.

Experts have known for years that some of these transplants wouldnt provide full immune protection over the course of a SCID patients entire life. They say clinicians should have avoided the word cure. But even scientific papers that hinted at such complications called the treatment curative. Just this year, an Immune Deficiency Foundation employee was given the unenviable task of sifting through the organizations thousands of pages of online material, scrubbing out every cure that popped up. It was only there a handful of times sometimes in quotes from clinicians, Boyle said but it was there and it needed to be removed.

The language patients hear can sometimes even change their outcomes. Weve heard of cases where, years later, they realized their immune system isnt as healthy as they thought, but nobody was tracking that because they hadnt maintained a relationship with the physician, or the physician didnt maintain a relationship with them, explained Ballard. The word cure, it gives them a false sense of security.

At a time when seemingly every biotech is promoting the idea of one-and-done therapies and setting prices accordingly these advocates hope companies, too, will be more wary. One of the things Im trying to make them very aware of is the need for lifelong follow-up, said Heather Smith, who runs the SCID Angels for Life foundation. For her, its personal: This summer, her son took part in a clinical trial for a gene therapy in the hope that it would provide the immune protection that his decades-old bone marrow transplant no longer could. My son will be followed for 15 years, she said. But what about after that?

Part of the issue with bone marrow transplants from one person to another is the natural genetic variation between us, particularly in the proteins that help our bodies distinguish its own cells from foreign ones. Receiving cells from someone whose proteins dont match yours could cause a civil war within you. Thats why bone marrow transplants began back in the 1950s with identical twins: Sharing those genes meant increasing the likelihood of harmony between the body and the graft.

But the vast majority of people dont have a protein-matched sibling, let alone an identical twin. So researchers set about figuring out how to transplant bone marrow from a parent to a child in spite of only sharing half of their genes and from a matched unrelated donor to a stranger. Like cooks intent on refining recipes to their taste, the doctors who adapted the technique for SCID often did so slightly differently from one another. Over the past 35 years, those idiosyncrasies have hardened into habits. Right now, everybody transplants their patients their way, said Dr. Sung-Yun Pai, an immune deficiency researcher and co-director of the gene therapy program at Boston Childrens Hospital.

Perhaps the most vociferous controversy has been about whether to use chemotherapy to wipe out the existing stem cells within a recipients bone marrow to make room for the donors. The doctors who do use chemo before a transplant might prescribe different doses; others forego it entirely.

The arguments were sound on both sides. On the one hand, the toxic drugs could clean out the niches within our bone and increase the chances that the donors cells take root. On the other, these chemicals could hamper growth, brain development, and fertility, could make an infant who was already sick even sicker, and could increase the likelihood of certain cancers later in life. Its like being exposed to a bunch of X-rays and sunlight, or other DNA-damaging agents, Pai explained.

Because SCID is so rare the most common subtype is thought to occur in 1 out of every 50,000 to 100,000 newborns and because every hospital was doing transplants slightly differently, it was hard for physicians to systematically study what was working best. But even early on, they could tell that some of the infants whod gotten no chemo were developing incomplete immune systems. They didnt produce their own B cells, for instance, and so needed regular injections of antibodies collected from other peoples blood.

In healthy infants, stem cells migrate from the crevices of the skeleton to an organ in the chest called the thymus, where theyre trained to become T cells. In these infants, the T cell counts grew after transplant but it wasnt necessarily because the sludge was securely taking hold in the niches of their bones. Rather, immunologists say, the donors progenitor cells were only transient. Some were able to head toward the thymus for schooling. Some graduated and started fighting off infections. But as those populations were depleted with age, there werent robust reserves of stem cells in the bone marrow that could arrive to produce more. To Pai, its like trying to fill a kindergarten class in a neighborhood where no ones having babies.

You and I continue to have a slow trickle of new T cells coming out, said Dr. Harry Malech, a senior investigator at the National Institutes of Health, who sits on the board of a gene therapy company, Orchard Therapeutics (ORTX), but does not receive any financial compensation. Instead of a torrent becoming slower, in these patients it goes from a trickle to practically nothing.

Thats why immunity starts to wane in kids like Ray Ballard. To many immunologists, it isnt a surprise, though they still arent sure why chemo-less transplants last longer for some of these kids than others. They can also understand how some families and clinicians might have viewed this treatment as a lifetime fix.

As Malech put it, If I said to you, Your child, instead of dying in infancy, will likely get to adulthood, go to school, have a normal life, you might think the word cure in your mind.

Even for parents who knew the protection might not last forever, the failure of a long-ago bone marrow transplant puts them in a bind. If they do nothing, their child will once again be vulnerable to any passing infection, which could prove fatal. They can try another round of the same procedure, though booster transplants sometimes come with added complications. Or they can try getting their child into a research trial for gene therapy, which comes with the risks of any experimental treatment.

Some feel an irrational guilt when the bone marrow they donated to their child stops functioning. Its your cells, and if it doesnt work, you failed them, said Ballard, who lives in Clifton, Va., about a 40-minute drive from Washington, D.C. Her son Ray had already had three transplants as a child. When his immune system started to fail again in early adulthood, gene therapy at the NIH seemed like the only reasonable choice.

That would involve researchers removing cells from his bone marrow, using an engineered virus as a kind of molecular syringe to slip in a healthy copy of the gene in which he had a defect, and then threading these corrected cells back into his veins a bone marrow transplant to himself. But preparing a virus can be tricky, and there were delays.

Meanwhile, Rays condition was getting worse. His norovirus was preventing him from absorbing much nutrition, and as Ballard put it, his bone structure was just crumbling at that point. His doctors told her he had the skeleton of an 85-year-old.

He died this past February, at 25 years old. One friend got his birth and death dates tattooed onto her shoulder. Another painted a portrait of him for Ballard, in which his arms are crossed, his lips pressed together in a wry smile.

At Boston Childrens, Pai is now helping to lead a randomized trial to better understand what dose of chemo works best for SCID patients receiving transplants. Over the last decade or so, she, Malech, and many other clinicians have also teamed up to track the long-term results of immune deficient patients whove received someone elses bone marrow.

Pai is hopeful that knowing about the phenomenon of waning immunity will give gene therapies a better shot at becoming a durable fix. They probably have a better chance of achieving a one-time, lifelong cure, but its never wrong to be humble, she said. Only after decades more and hundreds or thousands of patients will we know for sure.

Patient advocates point out that even then, these patients will still have the capacity of passing on their SCID-causing gene to future generations, and so the word cure is overly optimistic. Thats why I like the word remission, said Smith. That still gives you the hope. If you were given a cancer diagnosis, you wouldnt go through treatment and then just forget about it for the rest of your life.

As Boyle put it, Weve seen the promise and then weve seen the reality. Everyone who is looking at a transformational therapy should be optimistic, but also realistic, and not assume that this is truly one and done. (Boyles foundation has received financial support from Orchard Therapeutics, which is developing a gene therapy for a form of SCID.)

To Amy Saada, of South Windsor, Conn., that isnt theoretical. Her son Adam is now 12, and the immunity from the bone marrow transplant he got as a baby is wearing off. He isnt yet sick, but his parents know they need to decide between gene therapy or another transplant soon. She has a very clear memory of how long and uncertain the recovery from treatment felt. In some ways, she wishes she didnt know quite as much as she does; that way, she would feel less trepidation about what lies ahead.

Your heart kind of sinks, she said. Youve already been through it once, and it was hell. Its harder the second time.

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Waning treatment is a warning for all 'one-and-done' therapies - STAT - STAT

Neveah Taouk, 4

At last, when Mary was seven and Neveah three, new developments in whole-genome sequencing enabled specialists to identify the disorder. The diagnosis gave the Taouks information but not hope. They knew what the problem was, but there was no treatment and no cure.

Desperate, Charlie contacted specialists around the world. I must have spoken to at least fifty people scientists, doctors, professors, he says. Most of them had never heard of the condition.

His search eventually led to Dr Wendy Gold, a specialist in rare genetic disorders in children, based at the University of Sydney and the Childrens Hospital at Westmead. We arranged to talk, says Charlie. To be honest, I wasnt expecting much. But then she said, Have you heard of gene therapy?

Gene therapy is a new and rapidly evolving field of research. One of the therapys forms involves adding new genes to a patients cells to replace missing or malfunctioning genes. The new genes are typically delivered to the appropriate cells in the body using a benign virus as a carrier. Gene therapy is already being used to treat diseases including spinal muscular atrophy. It could also be a promising treatment for Parkinsons disease. Dr Gold believed there was a chance it could help the Taouk girls.

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The gene therapy research that could save a family of four - News - The University of Sydney

Indian Council of Medical Research (ICMR) is setting up a task force on gene therapy research to encourage research in the emerging field.

The research body among other things has proposed forming the task force to explore gene editing based therapeutic approaches to treat illnesses.

In a call for research proposals, ICMR has stressed that many inherited disorders are not treated by current available drugs or traditional therapies.

Gene Therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

While the western world has made considerable strides with regards to gene therapy over the past 30 years, ICMR stated that drugs like Luxuturna for Retinitis Pigmentosa, a condition which leads to breakdown of retinal cells in the eye, and leads to low vision, or Yescarta which is a cell therapy for cancer, are currently in clinical trial phase.

However for the vast majority of inherited diseases, appropriate targeted therapies are yet unavailable despite the large load of genetic disease in our population. To address this particular gap, ICMR is inviting proposals to fund gene therapy research projects, the research body has said in its circular.

ICMR has narrowed down on genetic diseases affecting the brain and muscles, eye disorders affecting the retina and cornea, heart diseases and blood disorders like Thalassemia, Sickle Cell Disease and Haemophilia. It has also stressed on diseases like Cancer, Diabetes and Lung diseases. The strategies proposed shuold have a possibility of translation into future human trials, the circular states.

In recently released guidelines on gene therapy ICMR stated, India has large burden of genetic disorders and unmet medical needs and gene therapy can prove to be a turning point in treatment of such disorders. However, it also brings along with it unique technical risks and ethical challenges. Creation of babies using germline gene editing by a Chinese scientist recently, attracted global criticism and fuelled a debate on ethical concerns regarding applications of gene therapy technologies. This also brought to forefront the requirement of stringent guidelines and regulations to prevent misuse and premature commercialization.

It further said, Many countries around the world have developed rules and guidelines to regulate gene therapy trials. Taking cognizance of situation, it was felt necessary to frame national guidelines and regulations to direct scientists and clinicians including industry regarding the procedures and requirements to be followed for performing gene therapy in India.

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ICMR sets up a task force on gene therapy research - BusinessLine

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) today announced that members of the management team will present at the following upcoming investor conferences in January:

To access the live webcasts of bluebird bios presentations, please visit the Events & Presentations page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com. Replays of the webcasts will be available on the bluebird bio website for 90 days following the events.

About bluebird bio, Inc.bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

bluebird bio is a trademark of bluebird bio, Inc.

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the advancement of, and anticipated development and commercialization plans for, the Companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of our product candidates will not continue or be repeated in our ongoing or planned clinical trials; risks that the current or planned clinical trials of our product candidates will be insufficient to support future regulatory submissions or to support marketing approval in the U.S. and EU; and the risk that our product candidates will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio Announces Investor Events in January - Business Wire

Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular atrophy. SMA is a neuromuscular disorder that, in severe cases, can lead to infant death. The genetic correction is currently used to treat affected newborns, but as symptoms for some types of SMA may appear before birth, an earlier treatment would be potentially more effective.

In a study published December 4 in Molecular Therapy, researchers were able to fix a mutation in the survival motor neuron 1 (SMN1) genewhich causes SMA in humansin mice modelling the disease, while they were still inside their mothers uterus. The treated mice lived longer and had fewer symptoms than untreated animals.

Tippi MacKenzie, a fetal and pediatric surgeon at the University of California, San Francisco, who did not participate in this study, says it is an important paper because it is the first time fetal gene therapy has succeeded in SMA mice. Before you even think about doing something in patients, you have to first do it in the disease model of the mouse . . . so this group has supplied a very important piece to the literature, she adds.

SMN1encodes an essential protein for the maintenance of motor neurons, which are nerve cells in the brain and spinal cord responsible for controlling muscle movement. The result in children with mutations in the gene is the loss of motor neurons, leading to muscle weakness and associated complications. SMA affects one out of every 6,000 to 10,000 babies.

Correcting the SMN1 sequence is a potentially efficient treatment for those born with SMA. Zolgensma, the recently approved medication for this disorder, consists of an intravenous administration of an adeno-associated virus that ferries a functional copy of the SMN1 gene to the brain.

To see if the same fix could be accomplished before birth, the research team tested two different injection methods: one into the placenta (intraplacental or IP) and the other into one of the brain lateral ventricles (intracerebroventricular or ICV). The latter proved to be more effective. By injecting the viral vector into the fetuss brain, the virus will go directly into the cerebrospinal fluid, and it will transduce motor neurons in the spinal cord with a very high efficiency, compared to the IP [injection], says Afrooz Rashnonejad. who participated in this study while working at Ege University in Izmir, Turkey, but has recently moved to Nationwide Childrens Hospital in Columbus, Ohio.

Rashnonejad and her colleagues then monitored the injected mice that were carried to term. Those treated with the vector carrying a functional copy of SMN1 lived a median lifespan of 63 or 105 days (depending on the type of cassette carrying the gene), much longer than untreated SMA mice, which did not survive more than 14 days, but still less than wildtype pups, which had a median lifespan of 405 days. The treated mice were also heavier than untreated mice, but smaller than healthy mice.

The investigators also observed differences at the cellular and molecular levels. SMN protein levels were completely recovered in the brain and spinal cord, and the number of motor neurons was higher in treated animals.

I was just very impressed by what theyve done, says Simon Waddington, a gene therapy researcher at University College London who did not participate in this work, but was one of the reviewers of the paper. He adds that he and other colleagues had previously tried fetal gene therapy on SMA mice, but had failed as it is a technically difficult experiment. So it was really nice to see this group actually did a really good job.

This is the first time viral vectors have been used to successfully boost gene expression in SMA mice before birth. Interventions to edit the genome in utero have been previously used in mice that model other severe genetic diseases. Last year, for instance, Waddington and colleagues used fetal gene therapy to treat mice affected by Gaucher disease, a neurodegenerative disorder that can be fatal for newborns. Other successful attempts include intrauterine gene editing for mice affected by -thalassemia, an inherited blood disorder, and mice suffering a monogenic lung disease that normally results in newborn death.

MacKenzie says that, in a recent national meeting on in utero gene therapy, it was discussed how to move forward with a clinical application to the FDA. We are definitively moving towards that direction, but we dont have a particular application yet, because its still not clear which disease should be the first.

SMA makes a lot of sense because its so severe, MacKenzie adds. But at the same time, the results that are coming out at conferences, she observes, suggest that newborn babies receiving Zolgensma are doing pretty well, better than anybody could have imagined. So its not clear that you have to go before birth. A good candidate, she explains, would be a very rare type of SMA, where the baby dies before birth.

Waddington says that researchers might have to wait for neonatal gene therapy to become standard for certain diseases before using fetal gene therapy in humans. Once we actually understand how efficient this is, and if we come to the point where we discover that the earlier that you go the more effective it is . . . in a human setting, then we may be able to do fetal gene therapy. I think that we are looking at more than five years away before thats even likely to happen, he hypothesizes.

A. Rashnonejad et al., Fetal gene therapy using a single injection of recombinant AAV9 rescued SMA phenotype in mice,Molecular Therapy, 27:212333, 2019.

Alejandra Manjarrez is a freelance science journalist. Email her atalejandra.manjarrezc@gmail.com.

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Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy - The Scientist

Gene therapy, once dismissed as too dangerous, has made a comeback, with two products approved in the U.S. since December 2017 and hundreds more in the pipeline. STATs latest report takes a deep dive into a crucial component of these new treatments: the viral vectors used to deliver gene therapies to cells and organs.

As dozens of new gene therapies near the market, we spoke with academic experts, pioneers in the field, and executives with 18 companies, large and small, to identify the most important challenges surrounding the engineering of better vectors, their safety, effectiveness, efficiency, production, and cost and how key players are thinking about overcoming those hurdles.

These engineered viruses are difficult to manufacture, particularly at the massive scale needed for some indications. Scientists are working hard to bring down the cost and speed up the process of making viral vectors, so that all the patients that could benefit from gene therapy will have access to it.

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Beyond the introduction, this report has four major components:

The basics of viral vectors and the history of their development;

Major challenges in the development, manufacturing, and testing of viral vectors, and possible solutions;

A close look at the status of gene therapies in 10 disease categories that are advancing through preclinical studies or are being tested in early-stage clinical trials;

And perspective on the U.S. Food and Drug Administrations approach to regulating viral vectors.

The report The STAT guide to viral vectors, the linchpin of gene therapy is intended for anyone with a strong interest in gene therapy, including biotech executives, investors, scientists, lawyers, policymakers, and patients and families interested in learning more. Our aim is to make the problems, stakes, and possibilities clear to everyone.

To buy the full report, please click here.

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New STAT report explores viral vectors, the linchpin of gene therapy - STAT - STAT