exon skipping

Entrada Reports Positive Topline Results from Phase 1/2 ELEVATE-44-201 Study in Duchenne

Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular…

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Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53

Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals…

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December 8, 2025

Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne

Dyne Therapeutics has announced positive topline results from its Registrational Expansion Cohort (REC) of the Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in people living with Duchenne who are amenable…

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November 19, 2025

Avidity Biosciences Announces U.S. Managed Access Program for del-zota

Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited…

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September 10, 2025

Avidity Biosciences Announces Positive New Data from EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 Studies

Today, Avidity Biosciences announced new data from its EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 studies of del-zota in people living with Duchenne muscular dystrophy amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino…

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FDA Grants Breakthrough Therapy Designation to Avidity’s del-zota

Avidity Biosciences, Inc. today shared that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to del-zota (AOC 1044) for the treatment of Duchenne in individuals amenable to exon 44 skipping. Del-zota…

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Watch: Dyne Therapeutics — The Clinical Impact of the FORCE™ Platform for Duchenne (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a community webinar on the DELIVER clinical trial evaluating DYNE-251. The Dyne team provided the latest updates on the Phase 1/2 global DELIVER study of the company’s investigational therapy…

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PepGen Announces Discontinuation of PGN-EDO51 Development

We are disappointed to share that PepGen Inc. announced the company’s decision to end development of PGN-EDO51, its investigational therapy for the treatment of people with Duchenne amenable to exon 51 skipping. PepGen indicated that…

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Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with…

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Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial

Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon…

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