Institut Charcot, an initiative of ARSLA, ReMedys Foundation and Avrion Therapeutics have signed a memorandum of understanding setting out a collaboration to advance AVR-001, an advanced preclinical gene therapy candidate for certain familial forms of amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1.

AVR-001 arises from research led by Dr. Bernard Schneider at EPFL, is jointly developed by ReMedys and Avrion, and will be included in Institut Charcot's integrated translational pipeline. The collaboration brings together ARSLA's patient-community perspective, Institut Charcot's scientific network, ReMedys' translational development expertise, and Avrion's gene therapy capabilities. Together, the partners aim to move the AVR-001 program towards clinical evaluation.

For Institut Charcot, the collaboration adds an advanced preclinical program to its growing pipeline. For Avrion, it reinforces the company's development focus on AVR-001. For ReMedys, it provides an additional demonstration of its effective model for carrying academic science through structured development.

An advanced precision medicine candidate
AVR-001 targets SOD1, a protein implicated in certain familial forms of ALS. Mutated SOD1 can misfold and accumulate in cells, contributing to the progressive degeneration of motor neurons.

The AAV9-based gene therapy candidate is designed to selectively reduce SOD1 production in motor neurons and astrocytes, two cell types central to ALS pathology. Its cell-focused design is intended to support sustained activity in the parts of the central nervous system most directly involved in the disease.

The program has generated a substantial preclinical package supported by the ReMedys Foundation, including SOD1 down-regulation in cell models, therapeutic activity in SOD1 ALS mouse models, and target engagement, biodistribution and initial safety evaluation. The next steps will consist of evaluating efficacy and safety in patients with ALS.

A partnership with Institut Charcot to accelerate AVR-001's development
Launched by ARSLA in October 2025, Institut Charcot is building France's first integrated translational pipeline dedicated to ALS and motor-neuron diseases, from the discovery of new therapeutic approaches to their evaluation in patients. As part of the partnership, Institut Charcot will provide AVR-001 with its scientific expertise, translational network and resources, combined with the power of ARSLA, France's leading ALS patient association to accelerate the drug candidate's development and pave the way for a first-in-human clinical trial expected in 2028.