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Gene therapy trials for sickle cell disease halted after two patients develop cancer – Science Magazine

There are new cancer concerns swirling around a gene therapy approach designed to preventthe sickling of blood cells (above).

By Jocelyn KaiserFeb. 16, 2021 , 6:15 PM

A company has stopped its clinical studies of a promising gene therapy for the blood disorder sickle cell disease after two people who participated developed leukemia-like cancer. Bluebird bio is now investigating whether a virus it uses to deliver a therapeutic gene caused the cancers, reviving old concerns about the risks of this approach.

Its also possible the cancers stemmed from chemotherapy the patients received to prepare their bodies for the genes delivery. This is really a sad development whatever the cause, says Donald Kohn of the University of California, Los Angeles, who has led gene therapy trials for sickle cell and other diseases.

In the bluebird bio trials, scientists remove a patients blood stem cells and treat them in a dish with a modified virus related to HIV. It carries DNA encoding the oxygen-carrying protein hemoglobin and is intended to compensate for the patients defective gene for this molecule. After this step, called ex vivo because a patients cells are treated outside the body, doctors infuse the cells back into the person. Fourteen people who have received the latest version of the bluebird bio therapy are now virtually free of the pain crises their sickled red blood cells once caused.

But today came thenewsthat a patient treated 5 years ago in one of the studies has developed acute myeloid leukemia (AML). Another has myelodysplastic syndrome (MDS), which can develop into AML. A previous patient in the same studydeveloped MDS in 2018, but tests showedit had likely resulted from the DNA-damaging chemotherapy that wipes out a patients blood cells to make room for treated blood cells.

Still, the gene therapy could play a more direct role. In past small clinical trials, several boys with an inherited immune disorder who received similar ex vivo gene therapy developed leukemia. In those cases, a mouse virus ferrying a curative gene into cells landed its genetic cargo in a location that turned on a cancer gene. Researchers then switched to a potentially safer delivery system, a lentivirus that also inserts the genes it carries into the hosts DNA but in sites less likely to trigger a cancer gene. A 2019 report that a monkey treated with a lentiviral gene therapy had developed a leukemia-like condition suggested, however, that thecancer risk had not been eliminated.

Bluebird biotold investors todaythat although its scientists have found the virus inserted DNA into the chromosomes of the leukemia cells of the treated sickle cell patient with AML, they dont yet know its location. Theyll look to see whether the viral DNA landed near a known cancer-promoting gene, perhaps driving its activity. The company says these tests should take a matter of weeks.

Meanwhile, bluebird bio has also halted sales in Europe of an approved treatment that uses the same vector to treat the blood disorder beta-thalassemia. The companys stock price plunged 38% today.

Another sickle cell disease clinical trial that uses the CRISPR gene-editing tool to turn on a fetal form of hemoglobinreported promising results last year. That treatment doesnt rely on a virus to deliver CRISPR; instead, it uses a zap of electricity to get CRISPR editing molecules into cells in a dish. However, CRISPR itself can make off-target effects and rearrange chromosomes, and whether that can trigger cancer may not be known for several years.

The bluebird bio news comes on the heels of a December 2020 report thata patient in a gene therapy trial for hemophilia had developed a liver tumor. The company, uniQure, planned to explore the possible role of its vector, an adeno-associated virus (AAV). Even though AAVs are supposed to be safer than lentiviruses for gene therapy because they are not designed to insert their cargo into a cells genome, animal studies have found they sometimes can.

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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.


Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.


Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT

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Sensorion and Institut Pasteur announce new gene therapy collaboration –

The new gene therapy target,GJB2 coding for the Connexin 26 protein, has been added to Sensorions development portfolio: with the target the third candidate to emerge from the R&D collaboration with Institut Pasteur. It represents the largest gene therapy opportunity for the French biotech to date.

The GJB2 program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the US alone.

Sensorion, a French clinical-stage biotech based in Montpellier, was founded in 2009 to develop novel therapies to restore, treat and prevent hearing loss disorders.

The GJB2 program draws on new research from Institut Pasteur which shows that the same genes that underly congenital deafness are also involved in severe forms of presbycusis (age-related hearing loss). These forms of presbycusis appearing to be monogenic types of hearing loss that can be potentially treated by gene therapy.

Although the types ofGJB2mutations in children and adults may differ, Sensorion says gene therapy could potentially provide a solution to both.

Mutations inGJB2are believed to alter a gap junction protein widely expressed in the inner ear, disturbing intercellular exchanges of molecules and leading to hearing loss that is severe-to-profound in a majority of cases.

Institut Pasteur research now shows three pathologies related to GJB2 mutations: congenital deafness;age-related hearing loss in adults; and progressive forms of hearing loss in children. Sensorion will prioritize the latter two forms, saying it is the first company to address these needs and offering the potential of large market opportunities.

The emergence of a new gene therapy target candidate validates our conviction that long-term solutions for restoring hereditary hearing loss will arise from an in-depth analysis of the "genetic landscape" of hearing loss," saidNawal Ouzren, CEO of Sensorion.

"It was clear that mutations in the GJB2 gene are important in severe to profound childhood hearing loss. However, the new discovery made by our collaborators at Institut Pasteur shows that alteration of this gene in adults offers new opportunities for Sensorion. It marks significant potential expansion of our pipeline and supports our goal of becoming a global leader in the field of gene therapies for hearing loss disorders.

Sensorions collaboration with Institut Pasteur initiated in 2019 has already led to gene therapy candidate programs in two other indications. Its USHER-CT gene therapy development program aims to restore inner ear function for patients suffering from Usher Syndrome Type 1 by providing a healthy copy of the USH1G gene coding for the SANS protein.

Meanwhile, the OTOF-GT gene therapy development program seeks to restore hearing in people with Otoferlin deficiency, one of the most common forms of congenital deafness.

Both of these have been proved in concept in preclinical studies.

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Outlook on the Gene Therapy Global Market to 2028 – Growing Incidence of Cancer and Target Diseases are Driving Growth – -…

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market Share, Size, Trends, Industry Analysis Report, By Vector Type; By Approach; By Therapeutic Area; By Route of Administration; By Regions; Segment Forecast, 2021-2028" report has been added to's offering.

The global gene therapy market expected to reach USD 5.02 billion by 2028, according to the study. This report gives a detailed insight into current market dynamics and provides analysis on future market growth.

The rising prevalence of chronic disorders including heredity and oncology diseases, rise in the number of clinical trials in the concerned arena, and increase in product approvals from regulatory agencies are few factors boosting the market growth. Moreover, innovations in the gene delivery systems also expedited the demand for such products.

The market is fragmented based on the therapy area, vector type, approach, route of administration, and region. In terms of therapeutic area, the market is segmented into autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases, and others.

Based on the vector type, the industry is further bifurcated into viral and non-viral vectors. Based on the approach, the industry is further bifurcated into gene augmentation, oncolytic viral therapy, immunotherapy, and others. Based on the route of administration, the market is further bifurcated into intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal, and others.

List of Key Players

Segment Highlights

Key Topics Covered:

1. Introduction

2. Executive Summary

3. Research Methodology

4. Gene Therapy Market Insights

4.1. Gene Therapy - Industry snapshot

4.2. Gene Therapy Market Dynamics

4.2.1. Drivers and Opportunities Growing incidence of cancer and target diseases Research and funding in gene therapy

4.2.2. Restraints and Challenges High treatment cost

4.3. Porter's Five Forces Analysis

4.3.1. Bargaining Power of Suppliers (Moderate)

4.3.2. Threats of New Entrants: (Low)

4.3.3. Bargaining Power of Buyers (Moderate)

4.3.4. Threat of Substitute (Moderate)

4.3.5. Rivalry among existing firms (High)

4.4. PESTLE Analysis

4.5. Gene Therapy Market Industry trends

5. Gene Therapy Market Assessment by Therapeutic Area

6. Global Gene Therapy Market, by Vector Type

7. Global Gene Therapy Market, by Approach

8. Gene Therapy Market Assessment by Route of Administration

9. Gene Therapy Market Assessment by Geography

9.1. Key findings

9.2. Introduction

9.3. Gene Therapy Market - North America

9.4. Gene Therapy Market - Europe

9.5. Gene Therapy Market - Asia-Pacific

9.6. Gene Therapy Market - Middle East & Africa

9.7. Gene Therapy Market - Latin America

10. Competitive Landscape

10.1. Expansion and Acquisition Analysis

10.1.1. Expansion

10.1.2. Acquisition

10.2. Partnerships/Collaborations/Agreements/Exhibitions

11. Company Profiles

For more information about this report visit

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Outlook on the Gene Therapy Global Market to 2028 - Growing Incidence of Cancer and Target Diseases are Driving Growth - -...

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Global Gene Therapy Market Outlook to 2030 – by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of…

Dublin, Feb. 15, 2021 (GLOBE NEWSWIRE) -- The "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" report has been added to's offering.

Over time, several gene therapies have been developed for the treatment of both simple and complex genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma, ZyntegloT and Collategene) to date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods and genome editing and manipulation technologies, have enabled the development of novel therapy development tools/platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

Story continues

The report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Key Questions Answered

Who are the leading industry players engaged in the development of gene therapies?

How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?

Which types of vectors are most commonly used for effective delivery of gene therapies?

What are the key regulatory requirements for gene therapy approval, across various geographies?

Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?

What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?

What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?

Who are the key CMOs/CDMOs engaged in supplying viral/plasmid vectors for gene therapy development?

What are the key value drivers of the merger and acquisition activity in the gene therapy industry?

Who are the key stakeholders that have actively made investments in the gene therapy domain?

Which are the most active trial sites related to this domain?

How is the current and future market opportunity likely to be distributed across key market segments?

Key Topics Covered:
























A Selection of Companies Mentioned Include:

For more information about this report visit

About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of...

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe…

COLUMBUS, Ohio, Feb. 16, 2021 /PRNewswire/ --Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.

"FDA's decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease."

Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company's Biologics License Application (BLA).

The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

"Infantile Krabbe is a progressive and devastating leukodystrophy," said Jessie Barnum, M.D., AssistantProfessor,Department of Pediatrics,Division of Blood and Marrow Transplantation and Cellular Therapies and Principal Investigator of the FBX-101 trial at UMPC. "FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course."

"The FBX-101 preclinical data brings a new wave of hope to the Krabbe community," said Anna Grantham, Director of Leukodystrophy Care Network Programs at Hunter's Hope. "These FDA designations for FBX-101 underscore a beautiful and collective effort to accelerate the timelines of bringing this potential therapy to patients who urgently need them."

"To see a promising new treatment for Krabbe receive these designations so quickly brings us one step closer to what everyone in our disease community is ultimately working towards: an FDA-approved treatmentfor Krabbe disease to reach the beside of all patients impacted by this disease," said Stacy Pike-Langenfeld, Director of Programs and Administration at The Legacy of Angels Foundation. "Our mission has always been to promote research to develop and enhance treatments for Krabbe disease, so it's very encouraging to see that Forge and FBX-101 have made so much progress in such a short amount of time."

Patients and families can learn more about clinical trials for FBX-101 by visiting

About Krabbe diseaseKrabbe disease is a rare, inherited leukodystrophy affecting approximately 1:12,500 - 100,000 people in the U.S.A. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing problems, cognitive, vision and hearing loss. Early onset or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101Forge is developing FBX-101 to treat patients with infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) gene therapy that is delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease in animal models, and to drastically improve the lifespan of treated animals. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies.

About Forge BiologicsForge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, "The Hearth", to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit

Patient, Pediatrician, Genetic Counselors & Family InquiriesDr. Maria EscolarChief Medical OfficerForge Biologics

Media Inquiries:Dan SalvoDirector of Communications and Community DevelopmentForge Biologics

Investor Relations and Business DevelopmentChristina PerryVice President, Finance and OperationsForge Biologics

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe...

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