exon skipping

NS Pharma Announces FDA Clearance of IND for Clinical Trial of NS-089/NCNP-02 Exon 44 Skipping Candidate

PPMD is excited to share that NS Pharma has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for NS-089/NCNP-02, a potential exon skipping therapy targeting people with…

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WATCH: Viltepso, An FDA-Approved Treatment for Patients Amenable to Exon 53 Skipping – Webinar Recording

PPMD and NS Pharma hosted a community webinar on March 2, 2023 to discuss VILTEPSO, an FDA-approved treatment for patients with Duchenne amenable to exon 53 skipping. Mathula Thangarajh, MD joined us to discuss the…

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December 20, 2022

Entrada Therapeutics Announces Clinical Hold on IND Application for ENTR-601-44 in Duchenne

Entrada Therapeutics has shared that the FDA issued a clinical hold on the Investigational New Drug (IND) application for its ENTR-601-44 program, an exon-skipping therapy set to target individuals with Duchenne amenable to exon 44…

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September 6, 2022

Dyne Therapeutics Announces First Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251

Dyne has announced that the first participant has been dosed in the Phase 1/2 DELIVER clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon 51. Dyne-251 is an exon skipping product that combines…

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Dyne Therapeutics Announces FDA Clearance of IND Application for DYNE-251

Dyne Therapeutics has announced that the FDA has lifted the clinical hold and cleared its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon…

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NS Pharma Announces Publication of Long-Term Efficacy and Safety Data of VILTEPSO® (Viltolarsen) in Duchenne

NS Pharma, Inc. has announced the publication of long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) in participants with Duchenne. We are pleased to see…

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Nationwide Children’s Announces Restoration of Full-Length Dystrophin Using Dup 2 Gene Therapy Approach

PPMD is very excited to see promising results shared from Nationwide Children’s scAAV9.U7.ACCA gene therapy trial, a Phase 1/2 study aiming to induce exon skipping for individuals with a duplication of exon 2 to restore full-length…

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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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