January 5, 2021

REGENXBIO Announces New Gene Therapy Program for Duchenne Muscular Dystrophy

PPMD is incredibly excited to share REGENXBIO’s announcement of their new program for Duchenne muscular dystrophy. REGENXBIO is seeking to treat Duchenne through a gene therapy approach delivering a novel microdystrophin transgene. Their gene therapy…

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December 18, 2020

Sarepta Community Bulletin: COVID-19 Vaccination and Gene Therapy

Sarepta Therapeutics has shared a community bulletin addressing questions about COVID-19 vaccination and gene therapy. This update confirms information presented during PPMD’s recent webinar, which will likely be consistent across all clinical trials. For more…

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December 10, 2020

The Duchenne Registry Celebrates One Year of Data Collection in our Smartphone App!

This year has been one for the record books for many reasons, but here at PPMD we are excited to celebrate one year of data collection in The Duchenne Registry’s new smartphone app platform! Our…

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December 7, 2020

Sarepta Announces Positive Clinical Results from MOMENTUM Phase 2 Clinical Trial of SRP-5051

Sarepta has announced positive results from the ongoing MOMENTUM study of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. This is the first clinical data from SRP-5051, an…

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December 3, 2020

WATCH: SRP-9001 Gene Therapy Path Forward (Webinar Recording)

Parent Project Muscular Dystrophy and Sarepta Therapeutics hosted a webinar on November 24, 2020 for a discussion about the SRP-9001 gene therapy development program. If you missed the live event, the recording can be found…

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November 25, 2020

Antisense Therapeutics: Introduction to the Duchenne Community

PPMD is thrilled to welcome Antisense Therapeutics to the Duchenne community. Antisense is developing a new potential therapy, ATL1102, using an antisense oligonucleotide (ASO) inhibitor aimed at treating muscle inflammation and associated muscle wasting. Although…

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November 22, 2020

Lilly and Precision BioSciences Announce Genome Editing Research Collaboration and License Agreement

PPMD is excited to welcome Lilly back to the Duchenne space! With Lilly’s experience in Duchenne, we are thrilled to see their return to the field and look forward to learning more as this early…

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November 17, 2020

ReveraGen Receives $3.3 Million NIH Grant for NDA Preparations for Vamorolone in Duchenne

The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a $3.3 million grant to ReveraGen Inc. to support the advancement of vamorolone towards FDA submission and…

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November 6, 2020

Sarepta to Commence Dosing of Next Study with Commercial Process Material for SRP-9001 Gene Therapy

We are encouraged by the result of Sarepta’s Type C meeting with the FDA. Type C meetings are a “catch all” category and include any topics concerning the development and review of a product. This…

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October 28, 2020

WATCH: Catabasis Phase 3 PolarisDMD Trial Results (Webinar Recording)

We were disheartened to learn earlier this week that the results from the Catabasis Phase 3 trial for edasalonexent (PolarisDMD) did not meet its primary or secondary endpoints. On October 27, 2020, PPMD was joined by…

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