Announcement of the Duchenne Muscular Dystrophy Collaborative Research Fellowship

CureDuchenne, Muscular Dystrophy Association and Parent Project Muscular Dystrophy announce a collaborative project to focus on re-dosing gene therapy in Duchenne muscular dystrophy.

CureDuchenne, Muscular Dystrophy Association and Parent Project Muscular Dystrophy announce a collaborative project to focus on re-dosing gene therapy in Duchenne muscular dystrophy.

New York, June 15, 2023 (GLOBE NEWSWIRE) — June 15, 2023 — CureDuchenne, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD), today announced a grant for collaborative clinical trials to test the reuse of the FDA drug approved Vyvgart (developed by argenx), for its potential ability to reduce antibodies against adeno-associated virus (AAV) in patients with Duchenne muscular dystrophy (DMD). The three organizations each provided $100,000 to sponsor a one-year clinical study led by researcher Barry Byrne, MD, Ph.D., Chief Medical Advisor, MDA and director of the Powell Gene Therapy Center at the University of Florida to evaluate Safety and efficacy of efgartigimod alfa-fcab (Vyvgart) to lower AAV antibodies by withholding DMD patients from gene therapy. Vyvgart is an approved drug currently used to treat autoimmune diseases by reducing overall levels of circulating IgG antibodies. It has been shown to be effective in reducing the pathogenic acetylcholine receptor antibodies present in myasthenia gravis (MG). The study will examine whether Vyvgart has the ability to reduce AAV antibodies to levels compatible with the safe and effective delivery of gene therapies in patients with DMD. The positive results of this study may be applicable to the translation of AAV gene therapies for other neuromuscular diseases.

“We are grateful to the Muscular Dystrophy Association, Parent Project Muscular Dystrophy and CureDuchenne for this funding, which allows us to test new strategies to ensure gene therapy is as inclusive as possible,” said Barry Byrne, MD, Ph.D.. , MDA Chief Medical Advisor, and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. “We are just starting to realize the impact of gene therapies that can treat the root cause of genetic diseases. Disease-modifying therapies are no longer a thing of the future. Our focus now is to figure out how to maximize and achieve better the greater impact with the technology in our hands.”

Gene replacement therapy for DMD has long been an attractive therapeutic prospect to target the root cause of the disease, with the goal of restoring dystrophin protein function. Several companies including Sarepta, Solid Biosciences, Pfizer and Regenxbio have undertaken clinical trials with truncated dystrophin constructs for gene therapy. This co-funded clinical trial aims to suppress anti-AAV antibodies in a subset of DMD patients who are currently ineligible for gene therapy due to pre-existing AAV antibodies from natural infections. The study will also explore the potential for reduction in DMD patients of anti-AAV antibodies acquired from previous participation in gene therapy trials to enable future re-dosing efforts.

This study is an open-label, single-center, multi-arm Phase II study taking place at the Powell Center for Rare Disease Research and Therapy, located in Gainsville, Florida. A total of 12 DMD patients will participate in the study, six with high anti-AAV titers before gene therapy and six with high titers after gene therapy. The primary outcome of the study will be to evaluate the ability of Vyvgart to lower anti-AAV capsid antibody before and after AAV exposure. The secondary outcome of the study will be to determine the safety and tolerability of Vyvgart in boys with DMD.

We are delighted to partner with these organizations to support this important study, led by Dr. Byrne, said Debra Miller, founder and CEO of CureDuchenne. As we pursue our mission to bring transformative treatments to more individuals with Duchenne, we must overcome the limitations of adeno-associated virus (AAV)-delivered gene therapy. We hope this study supports a clinical strategy to effectively deliver gene therapy to individuals with pre-existing AAV neutralizing antibodies, who are currently excluded from treatment, and to help pave the way towards re-dosing individuals who have already received a therapy. gene provided by AAV.

Our three organizations (MDA, CureDuchenne and PPMD) have come together in recognizing the importance of developing strategies for as many DMD patients as possible to be eligible for gene therapy, said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA extension. If an approved drug could reduce AAV antibodies to acceptable levels, it would be an efficient way to open the door for patients who are currently ineligible for gene therapy. This approach could also provide retreatment options for patients who are starting to see a reduction in the drug’s effect, even though the hurdle would be higher there.

We are excited to partner with CureDuchenne and MDA to fund meaningful research that could impact many people living with Duchenne, said Eric Camino, PhD, PPMD ​​Vice President of Research and Clinical Innovation. AAV-delivered gene therapy represents a potentially transformative therapy for patients, yet some individuals are prevented from accessing these AAV-delivered therapies due to pre-existing antibodies. This research could create a pathway to broadening who can access AAV-mediated gene therapy; lowering antibodies to AAV over a period of time could allow dosing of individuals with pre-existing antibodies.

Learn about Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare, progressive genetic muscle disease caused by a lack of a critical membrane-stabilizing protein called dystrophin, which causes muscle weakness and wasting. Gene replacement strategies using AAV technology have shown to be a very promising approach to restore dystrophin protein function and are currently being pursued by several companies. The onset of DMD symptoms is in early childhood, usually between the ages of 2 and 3. The disease mainly affects boys, but in rare cases it can affect girls. In Europe and North America, the prevalence of DMD is approximately 6 individuals in 100,000. Doctors often diagnose the disease between the ages of 3 and 6, when children show the first signs of significant muscle weakness, such as a delayed ability to sit, stand, or walk, and difficulty learning to speak. Over time, DMD becomes fatal as muscle wasting disrupts lung and heart function. There is no cure for DMD, however, life spans have been extended and quality of life improved for many through physical therapy and medications to address some symptoms.

About CureDuchenne
Twenty years ago, CureDuchenne was created with one goal: to find and fund a cure for Duchenne muscular dystrophy. Today, CureDuchenne is recognized as a global leader in research, patient care and innovation to improve and extend the lives of people living with Duchenne. CureDuchenne’s innovative venture philanthropy model has advanced transformative treatments for Duchenne muscular dystrophy, including 17 projects that have progressed to human clinical trials and multiple projects to push the limits of first-generation exon-skipping and therapy gene. In addition, CureDuchenne helped fund the first FDA-approved Duchenne drug early on, pioneered the first and only Duchenne Occupational and Physical Therapist Certification Program, and created an innovative biobank and data registry, accelerating research towards a cure. For more information on how to help raise awareness and funds needed for research, visit Follow CureDuchenne on Facebook, Instagram, YouTube, LinkedIn and Twitter.

About the Muscular Dystrophy Association

The Muscular Dystrophy Association (MDA) is the number 1 voluntary healthcare organization in the United States for people with muscular dystrophy, ALS, and related neuromuscular diseases. For more than 70 years, MDA has been at the forefront of advances in DMD and has invested nearly $227 million in research for DMD and Becker muscular dystrophy (BMD). As the largest source of funding for neuromuscular disease research outside the federal government, MDA has collectively invested more than $1 billion in the laboratories of 7,000 scientists, helping to build the entire field of neuromuscular disease biology and pioneering technologies such as the identification of disease-causing genes, gene therapy, antisense oligonucleotides and, more recently, gene editing. Visit Grants at a Glance to view profiles of recently funded MDA investigators and the MDA Gene Therapy Support Network (GTx) for information, support and guidance on recently approved gene therapies. To learn more, visit and follow MDA on Instagram, Facebook, Twitter, TikTok, LinkedIn and YouTube.

About Parent Project Muscular dystrophy

Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive non-profit organization in the United States focused on finding a cure for Duchenne. Our mission is to end Duchenne. We demand optimal standards of care and strive to ensure that every family has access to expert healthcare professionals, state-of-the-art treatments and a supportive community. We invest deeply in treatments for this generation of Duchenne patients and in research that benefits future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and secured five FDA approvals.

Everything we do and everything we’ve done since our founding in 1994 helps people with Duchenne live longer, stronger lives. We will not stop until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at and follow PPMD ​​on Facebook, Twitter, Instagram and YouTube.


CONTACT: Melissa Donahue, Director, Communications Muscular Dystrophy Association [email protected]

#CureDuchenne #Muscular #Dystrophy #Association #Parent #Project #Muscular #Dystrophy #Announce #Collaborative #Project #Focus #Gene #Therapy #Redose #Duchenne #Muscular #Dystrophy

By Admin

Leave a Reply

Your email address will not be published. Required fields are marked *