PRESS RELEASE
27th August 2026
Institut Charcot enters into a collaboration with Avrion Therapeutics and ReMedys Foundation to advance their new gene therapy AVR-001
The collaboration brings an advanced preclinical SOD1 gene therapy program, into Institut Charcot’s growing ALS pipeline, provides a concrete demonstration of ReMedys Foundation’s translational model and strengthens Avrion’s focus on preparing AVR-001 for clinical development.
Paris, Geneva and Lausanne, August 27, 2026 – 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.
“ARSLAs’ goal is to encourage innovative approaches in ALS, and to accelerate their development so they can reach patients faster. Avrion Therapeutics’- ReMedys Foundation therapy uses cutting-edge technology and offers innovative precision medicine: this is exactly the type of project that the Institut Charcot wants to support and accelerate.”
Valérie Goutines-Caramel, President, ARSLA
This collaboration gives an outstanding opportunity to advance an innovative treatment at the forefront of innovation for ALS patients with SOD1 mutations. Through this first international collaboration, we are pursuing our goal, transforming ALS research to accelerate therapeutic development.
Luc Dupuis, Institut Charcot
“AVR-001 is a concrete example of the ReMedys model: helping promising science become a structured development program by supporting critical work and bringing together the right partners at each stage. Having supported the program since its inception, our priorities are now clear-complete manufacturing, prepare for clinical entry and initiate the clinical study, while coordinating the partners and securing the resources needed to move AVR-001 towards patients as efficiently as possible.”
Teresa Giovannini Bugmann, President, and Kostas Kaloulis, Co-founder and Board Member, ReMedys Foundation
“We are honored to join, together with ReMedys Foundation, forces with Institut Charcot as it builds a center of excellence dedicated to confronting the urgent challenge of ALS, a disease for which treatment options remain far too limited,” said Maximilien Murone, CEO of Avrion Therapeutics. “We believe this collaboration can help accelerate the path from innovation to patient impact by advancing promising candidates from Avrion’s pipeline, beginning with our most advanced development candidate, AVR-001.”
Maximilien Murone, Chief Executive Officer, Avrion Therapeutics
About Institut Charcot
Launched in October 2025 by ARSLA, Institut Charcot is an initiative to build France’s first translational pipeline dedicated to ALS and motor-neuron diseases, spanning the discovery of new therapies through to patients.
www.institutcharcot.org
About ARSLA
Since 1985, ARSLA has been the national reference association giving voice to patients living with ALS. It supports more than 4,000 people affected by ALS and their families each year, funds research programs, works to improve care, and raises awareness among the public and policymakers.
www.arsla.org
About Avrion Therapeutics
Avrion Therapeutics is a Swiss biotechnology company founded in 2020 based on more than a decade of research at the Swiss Federal Institute of Technology Lausanne (EPFL)’s Brain Mind Institute. The company is dedicated to developing transformative therapies for ALS and other neurodegenerative diseases, with the ambition to reshape outcomes for patients facing these devastating conditions.
www.avriontx.com
About ReMedys Foundation
ReMedys Foundation is a Swiss not-for-profit foundation established in Geneva in 2013 by biopharmaceutical industry professionals. It builds and manages translational research and development partnerships so that promising therapeutic approaches have a fair chance of reaching patients affected by severe diseases without effective treatment. ReMedys jointly develops AVR-001 with Avrion Therapeutics, has sponsored key preclinical work and coordinates the program’s translational development.
www.remedysfoundation.org
Media and partnership contacts
ARSLA / Institut Charcot
Amélie Balliere
Communications and Fundraising Director
a.balliere@arsla.org
arsla.org
ReMedys Foundation
Patrik Richard
Head of Operations
patrik.richard@remedysfoundation.org
remedysfoundation.org
Avrion Therapeutics
Maximilien Murone
CEO
max.murone@avriontx.com
avriontx.com
Des éléments de réponse aux questions les plus fréquentes qui nous sont posées sur : le diagnostic, le traitement, le suivi de la maladie, la vie quotidienne, la prise en charge et les aides, la recherche, …
Le neurone est une des cellules composant le tissu nerveux avec les cellules gliales. Les neurones constituent l’unité fonctionnelle du système nerveux et les cellules gliales assurent le soutien et la nutrition des cellules nerveuses. Le système nerveux comprend environ cent milliards de neurones. Le neurone est une cellule dite polarisée avec des prolongements qui conduisent l’information et qui véhiculent les substances nécessaires au bon fonctionnement de la cellule. Les ‘dendrites’ sont des prolongements centripètes allant de la périphérie vers le corps cellulaire. Les axones sont centrifuges et vont du corps cellulaire vers la périphérie. La jonction entre l’axone d’une cellule et les dendrites d’une autre cellule est appelée synapse.
Le neurone moteur, également appelé « motoneurone », est une cellule nerveuse spécialisée dans la commande des mouvements. Il existe en fait deux grands types de neurones moteurs, le premier est dit central et se situe dans le cerveau, il achemine le message initial du cerveau jusque dans la moelle épinière. Le second, dit périphérique, débute dans la moelle épinière et achemine le message de la moelle épinière jusqu’aux muscles. Dans les atteintes des neurones moteurs, seules les fonctions motrices sont touchées, il n’existe donc pas de troubles sphinctériens, de troubles de la sensibilité, ni de troubles de l’intelligence.
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