Gene therapy developed at PTBTG in partnership with Institut Charcot
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AVR-001 arises from research at the EPFL Bertarelli Platform for gene therapy (PTBTG) and is jointly developed by ReMedys Foundation and Avrion Therapeutics. The gene therapy program will now enter Institut Charcot’s integrated translational pipeline, with the aim of advancing it toward clinical evaluation.
AVR-001, an AAV9-based gene therapy targeting mutated SOD1, a protein implicated in familial forms of amyotrophic lateral sclerosis (ALS, also known as ‘maladie de Charcot’), originates from research led by the Bertarelli Platform for Gene Therapy at EPFL and has been jointly developed with the support of the ReMedys Foundation and Avrion Therapeutics. The program has demonstrated SOD1 downregulation, therapeutic activity in SOD1 ALS mouse models, and encouraging preclinical target-engagement and biodistribution data. Through a new partnership with Institut Charcot, AVR-001 will join its integrated ALS translational pipeline, combining scientific expertise, translational resources, and patient-community support. The collaboration aims to accelerate the program toward clinical development and a first-in-human clinical trial expected in 2028.