GNT 0004 / Genethon, Sarepta Therap - LARVOL DELTA

GNT0004, Genethon's AAV-based gene therapy for Duchenne muscular dystrophy: long-term follow-up of ambulatory boys enrolled in the dose-escalation phase of GNT-016-MDYF. (ASGCT 2025) - "These effects will need to be confirmed in Part 2, the pivotal phase of GNT-016-MDYF. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Infectious Disease • Muscular Dystrophy

GNT0004, Genethon’s AAV8 vector-delivered microdystrophin gene therapy for Duchenne muscular dystrophy: first data from Phase 1/2 part of GNT-016-MDYF all-in-one clinical trial in ambulant boys (ESGCT 2024) - No abstract available

Clinical • Gene therapy • P1/2 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Step by step development of a cell-based potency assay for the gene therapy product G3MDYF/GNT0004 (rAAV8 human Microdystrophin) (ESGCT 2024) - No abstract available

Gene therapy • Gene Therapies

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy (News-Medical) - "Genethon... received...the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004. This trial's goal is to assess the safety and efficacy of an innovative gene therapy designed at Genethon..."

New trial • Duchenne Muscular Dystrophy

Genethon announces expanded collaboration with Sarepta Therapeutics (Eurekalert) - "Genethon...and Sarepta Therapeutics...announced an expansion of their collaboration to develop a gene therapy approach for Duchenne muscular dystrophy...The agreement between Sarepta and Genethon paves the way for a clinical trial to begin in 2020...Under the terms of agreement, Genethon will be responsible for commercializing the product GNT0004 in Europe (excluding the UK) and Sarepta will be responsible for the rest of the world."

Licensing / partnership • New trial