exon skipping

Entrada Therapeutics Shares New Data on ENTR-601-44 Exon Skipping Therapy

Entrada Therapeutics has shared new data from a preclinical study evaluating ENTR-601-44, an exon-skipping therapy set to target individuals with Duchenne amenable to exon 44 skipping. We are pleased to hear new data on this…

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Astellas Gene Therapies Announces plans to discontinue development of Duchenne gene therapy programs

Last night, based on recent preclinical data, Astellas Gene Therapies announced that the research and development of gene therapy programs AT702, AT751 and AT753 for Duchenne Muscular Dystrophy have been terminated. • AT702 was designed…

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January 20, 2022

Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne

Dyne Therapeutics recently shared that the FDA issued a clinical hold on their Investigational New Drug (IND) application for the Phase I/II study of their exon skipping therapy utilizing a conjugated monoclonal antibody DYNE-251. For…

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September 29, 2021

Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a Duchenne Clinical Trial of WVE-N531 Starting in Canada and the UK

This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy…

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August 12, 2021

NS Pharma Shares Community Letter

August 12, 2020, marked the approval of Viltepso, the 4th FDA approved therapy for Duchenne. NS Pharma has posted the following community letter marking the occasion. Today we recognize the one-year anniversary of this approval….

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NS Pharma Announces VILTEPSO® Long-Term Trial Data to be Presented at PPMD’s 2021 Virtual Annual Conference

NS Pharma has announced that they will be presenting new, long-term efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) at PPMD’s 2021 Virtual Annual Conference next month….

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WATCH: Viltepso – A Treatment Option for Patients Amenable to Exon 53 Skipping (Webinar Recording)

NS Pharma joined PPMD for a webinar on April 22, 2021 to provide an update about VILTEPSO™, a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. Part…

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February 25, 2021

FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen)

PPMD is excited to learn that the FDA has granted accelerated approval to AMONDYS 45™ (casimersen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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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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