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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July 25, 2018

Sarepta Therapeutics Announces Clinical Hold on Micro-Dystrophin Gene Therapy Trial

This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research…

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June 19, 2018

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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June 18, 2018

Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001

PPMD is excited to learn that Solid Biosciences Inc. announced that the FDA has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001. In…

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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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December 19, 2017

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration…

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December 13, 2017

Understanding Gene Therapy: The Screening Process

With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials. While the inclusion criteria of each trial will be different based…

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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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