gene therapy

PPMD Awards $175,000 Grant to UCLA to Explore Optimization of CRISPR Technology in Duchenne

Parent Project Muscular Dystrophy (PPMD) is excited to announce that Drs. Melissa Spencer and April Pyle from the David Geffen School of Medicine at UCLA and the Center for Duchenne Muscular Dystrophy at University of…

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PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

PPMD is excited to announce that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment…

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January 24, 2019

PPMD Awards $105,000 Grant to Explore A Mini-Dystrophin Gene Aimed at Protecting Cardiac Function

PPMD is excited to award the University of Missouri School of Medicine a grant for $105,000 to continue development of a minimized dystrophin gene aimed at protecting cardiac function in people with Duchenne. The project is led by Dongsheng…

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December 19, 2018

PPMD Awards $329,000 Grant to Binghamton University to Examine Role of Immune System on Dystrophin Restoration

PPMD is excited to award Dr. Kanneboyina Nagaraju and his team at Binghamton University’s School of Pharmacy and Pharmaceutical Sciences a $329,000 grant to continue his work examining the immune response to the production of…

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September 24, 2018

Sarepta Therapeutics Announces Clinical Hold Lifted for its Micro-dystrophin Gene Therapy Program

We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical…

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August 30, 2018

Gene Repair Technology Restores Dystrophin Expression in Canine Model of Duchenne

We are encouraged to learn that Exonics Therapeutics, Inc., a biotechnology company focused on developing gene editing therapies to treat patients with Duchenne and other neuromuscular diseases, today announced the publication of a preclinical study…

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Positive Preliminary Results from the First Three Children Dosed in Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta Therapeutics announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne. Ryan…

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January 10, 2018

First Duchenne Patient Dosed in Microdystrophin Gene Therapy

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM” On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr….

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January 30, 2017

PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology

PPMD today announced a $250,000 grant to be awarded to Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center. This grant, part of PPMD’s Gene Transfer Initiative, will support Dr. Olson’s ongoing study of CRISPR/Cas9…

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January 6, 2017

PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. Our purpose in awarding this grant is to open and accelerate the field of gene…

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