Collaboration to explore gene therapy products for neurodegenerative disorders

September 14, 2022 - 3 minutes
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Coave Therapeutics is collaborating with the Institute of Neurodegenerative Diseases (IMN) of Bordeaux to develop gene therapy proteins targeting protein degradation in neurodegenerative disorders.

The IMN is a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS) developing gene therapy programs targeting protein degradation in neurodegenerative disorders.

The collaboration will explore the development of gene therapy products targeting the transcription factor EB (TFEB) for the treatment of alpha-synucleinopathies, such as Multiple System Atrophy (MSA) and idiopathic Parkinson’s disease (PD).

Toxic protein aggregates

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

Erwan Bézard an IMN research director, a member pf Coave Therapeutics’ Scientific Advisory Board (SAB) and Andrea Ballabio, scientific director at the Telethon Institute of Genetics and Medicine (TIGEM) also a member of Coave Therapeutics’ SAB, have authored a paper. It demonstrates a reduction of a-synuclein aggregates, prevention of dopaminergic neuron destruction and recovery of clinical phenotype.

Parkinson’s disease

Bézard said: “Targeting the autophagy lysosomal pathway by using coAAV based gene therapy is a unique approach to address neurodegenerative diseases, such as Parkinson’s disease or Multiple System Atrophy.

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“Thanks to the partnership with Coave Therapeutics, IMN scientists have an important opportunity to confirm their scientific findings towards 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.”

Dr Ballabio, who discovered the role of TFEB as a master regulator of lysosomal biogenesis and autophagy will be an advisor to this collaboration.

Rodolphe Clerval, CEO, Coave Therapeutics said: “We are delighted to be collaborating with IMN to develop coAAVs carrying TFEB and explore these gene therapy constructs for the treatment of neurodegenerative diseases.

About Coave Therapeutics’ gene therapies

“TFEB is an exciting target and we look forward to working with IMN and Andrea Ballabio to evaluate the effect of our novel gene therapies from our ALIGATER platform for the treatment of MSA and PD, with the potential to develop further programs.”

Under the collaboration, Coave Therapeutics will use its AAV-Ligand Conjugate (ALIGATER) platform to design, develop and manufacture coAAV viral vectors carrying the TFEB gene for targeted delivery to deep brain structures.

IMN will be responsible for carrying out jointly designed in vivo studies to evaluate the effect of the gene therapy products in animal models of MSA and PD. The collaboration aims to generate further in vivo proof of concept data and enable the selection of therapeutic candidates to enter IND enabling studies.

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