Four Duchenne Muscular Dystrophy (DMD) Advocacy Organizations Fund Next Generation Gene Therapy Delivery For Duchenne Muscular Dystrophy

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Ryan's Quest, Michael's Cause, Pietro's Fight & Powers Promise Provide Research Grant for Development of a Potential Novel Adeno-associated Virus for DMD Gene Therapy in Human Patients

“We greatly appreciate the support and commitment from Ryan’s Quest, Michael’s Cause, Pietro’s Fight, and Powers Promise in helping researchers like us to develop a safer and more effective gene therapy for DMD.

Ryan's Quest, a New Jersey based non-profit organization dedicated to finding treatments and a cure for Duchenne muscular dystrophy (DMD) is excited to announce the collaboration of four Duchenne foundations to provide a $200,000 research grant to begin funding a promising Duchenne research project for Dr. Dongsheng Duan at The University of Missouri.

The fundamental problem in Duchenne muscular dystrophy (DMD) is dystrophin gene mutation. If we can replace the mutated dystrophin gene with a good one, it will address the root cause of the disease.

Adeno-associated virus (AAV) is a powerful gene therapy vector. FDA has recently approved two gene therapy drugs that are made of the AAV vector. These AAV drugs are currently been prescribed to children who suffer from a rare blindness disease and infants who suffer from an inherited motor neuron degenerative disease.

The AAV vector is now being tested in Duchenne boys to deliver a miniaturized micro-dystrophin gene to the muscle. In order to treat all muscles in the body, investigators have to inject at least 10^15 vector particles to a single patient. This creates many problems. The entry of the gigantic quantities of the AAV particles to the body increases the odds of adverse reactions. The need for the huge amount of AAV vectors also brings in a huge burden and pressure to manufacture. Innovative strategies are in urgent demand to overcome these hurdles.

Dr. Dongsheng Duan, a professor at the University of Missouri has worked on AAV micro-dystrophin gene therapy for two decades. In this project, Dr. Duan and his team will develop the next-generation AAV vectors. They will utilize a novel human muscle platform to screen for the best candidates and identify new AAVs that can more effectively deliver a gene to the muscle.    

“We greatly appreciate the support and commitment from Ryan’s Quest, Michael’s Cause, Pietro’s Fight, and Powers Promise in helping researchers like us to develop a safer and more effective gene therapy for DMD. Grassroots effort from the Duchenne community is a great inspiration and a critical driving force in our day to day research. We cannot thank you enough for your relentless efforts in raising funding for research.”

“If we can develop a super-AAV that is much more potent than the AAV currently in use in DMD patients, we will be able to achieve the same therapeutic efficacy at a much lower AAV dosage, and hence greatly reduce safety concerns and AAV manufacture burden” said Dr. Duan.

About Duchenne muscular dystrophy
Duchenne is a progressive neuromuscular disorder that causes a loss of motor, pulmonary, and cardiac function, and premature death. It is the most common lethal pediatric genetic disorder, and it affects every 1 in 3,500 live male births and some females. Duchenne is caused by the body's inability to create dystrophin, a large protein found in muscle cells. Duchenne has no FDA approved treatment or cure and is 100% fatal. Children with Duchenne are born normal and decline over time, usually losing their ability to walk around the age of 12 and succumbing to the disease in their early to mid-twenties.

About The Foundations
Ryan’s Quest (NJ), Michael's Cause (NY), Pietro's Fight (NY), and Powers Promise (NJ) are grassroots foundations founded by families affected by Duchenne. The Foundations raise awareness of Duchenne and direct all funding to the best medical and scientific research to find better treatments and a future cure for this disease. These family-founded and operated organizations believe that collaboration between Duchenne patient organizations is essential to speeding potential therapies through the drug development pipeline.

Contact Information for Media, RFP, and Grant Inquiries
David Schultz
President, Ryan’s Quest

Robert Capolongo
President, Michael’s Cause

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David Schultz
Ryan's Quest
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Robert Capolongo
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