Coave Therapeutics to Collaborate with World-Renowned Bordeaux Institute of Neurodegenerative Diseases to Develop Gene Therapy Programs Targeting Protein Degradation in Neurodegenerative Diseases

Paris, France, September 14, 2022 – Coave Therapeutics (“Coave”), a clinical-stage biotechnology company focused on developing breakthrough gene therapies for CNS (central nervous system) and ocular diseases, announces that it has entered into a collaboration with the Institute of Neurodegenerative Diseases (IMN), a joint research unit associating the University of Bordeaux and the National Center for Scientific Research (CNRS), to develop gene therapy programs targeting protein degradation in neurodegenerative diseases. The collaboration will explore the development of gene therapy products targeting transcription factor EB (TFEB) for the treatment of alpha-synucleinopathies, such as multiple system atrophy (MSA) and idiopathic Parkinson’s disease (PD).

TFEB is a master regulator of the lysosomal pathway of autophagy, a central cellular pathway controlling the breakdown of toxic protein aggregates. Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent accumulation of toxic protein aggregates1and therefore prevent neurodegeneration

A recent article2, authored by eminent IMN scientists, including Erwan Bézard, research director of IMN and INSERM and member of the scientific advisory board (SAB) of Coave, and Dr. Andrea Ballabio, MD, scientific director world renowned, Telethon Institute of Genetics and Medicine (TIGEM) and also a member of SAB de Coave, has demonstrated the effective delivery of AAV TFEB-based gene therapy in MSA and PD disease models. In addition, the results demonstrated reduction of α-synuclein aggregates, prevention of dopaminergic neuron destruction, and recovery of the clinical phenotype. Dr. Ballabio, who discovered the role of TFEB as a master regulator of lysosomal biogenesis and autophagy3.4will be an advisor to this collaboration.

As part of this collaboration, Coave will use its AAV-Ligand Conjugate platform (ALIGATER) to design, develop and manufacture coAAV viral vectors carrying the TFEB gene for targeted delivery to deep brain structures. The IMN will be responsible for carrying out a joint design live studies to assess the effect of gene therapy products in animal models of MSA and PD. The collaboration aims to generate more live proof-of-concept data and enable the selection of therapeutic candidates to enter enabling IND studies.

Erwan Bézard, Research Director, IMN, said: “Targeting the lysosomal autophagy pathway using coAAV-based gene therapy is a unique approach to treat neurodegenerative diseases, such as Parkinson’s disease or multiple system atrophy. Through the partnership with Coave, IMN scientists have an important opportunity to confirm their scientific findings in the clinic. We look forward to leveraging our collective strengths to best develop gene therapy programs for neurodegenerative diseases with the potential to improve patient outcomes.

We are excited to collaborate with IMN to develop TFEB-carrying coAAVs and explore these gene therapy constructs for the treatment of neurodegenerative diseases. TFEB is an exciting target and we look forward to working with IMN and Andrea Ballabio to evaluate the effect of our new gene therapies from our ALIGATER platform for the treatment of MSA and PD, with the potential to develop other programs.” added Rodolphe Clerval, CEO, Coave Therapeutics.


  1. Martini-Stoica et al. The autophagy-lysosomal pathway in neurodegeneration: a TFEB perspective. Neuroscience Trends. 2016 Apr;39(4):221-234.
  2. Arotcarena et al. Overexpression of the transcription factor EB prevents neurodegeneration in experimental synucleinopathies, JCI Preview 2019
  3. Sardiello et al. A network of genes regulating biogenesis and lysosomal function.

Science, 2019 325: 473-7.

Settembre et al. TFEB links autophagy to lysosomal biogenesis. Science 2011 332:1429-33. 2011.

About the Institute for Neurodegenerative Diseases (IMN)

The Institute of Neurodegenerative Diseases (IMN) is a joint research unit associating the University of Bordeaux and the National Center for Scientific Research (CNRS). The IMN, created in January 2011, was founded by Erwan Bézard to develop new therapeutic approaches to neurodegenerative diseases by facilitating translational research from the laboratory to the bedside.

About Coave Therapeutics

Coave Therapeutics is a clinical-stage biotechnology company focused on developing breakthrough gene therapies for CNS (central nervous system) and ocular diseases.

Coave Therapeutics’ next-generation AAV-Ligand conjugate (“ALIGATER”) platform enables targeted delivery and enhanced gene transduction to improve the efficacy of advanced gene therapies for rare diseases.

The company is advancing a pipeline of novel therapies targeting CNS and eye diseases where targeted gene therapy using chemically modified AAVs (coAAVs) has the potential to be most effective.

Coave Therapeutics, headquartered in Paris (France), is backed by leading international life sciences and strategic investors Seroba Life Sciences, Théa Open Innovation, eureKARE, Fund+, Omnes Capital, V-Bio Ventures, Kurma Partners, Idinvest, GO Capital and Sham Innovation Santé/Turenne.

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