Coave Therapeutics and the Institute of Neurodegenerative Diseases (IMN) of Bordeaux have entered into collaboration for developing gene therapy programmes that target protein degradation in neurodegenerative disorders.
Established in January 2011, IMN is a joint research unit of the University of Bordeaux and the French National Centre of Scientific Research (CNRS).
The collaboration will focus on developing gene therapy products that target the transcription factor EB (TFEB) to treat alpha-synucleinopathies, including idiopathic Parkinson’s disease (PD) and Multiple System Atrophy (MSA).
TFEB is a master regulator of the lysosomal autophagy pathway, a central cellular pathway that controls the degradation of toxic protein aggregates.
TFEB overexpression through gene therapy shows potential to reduce and prevent the toxic protein aggregates accumulation and thereby prevent neurodegeneration.
Coave Therapeutics CEO Rodolphe Clerval 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.
“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.”
As part of the partnership deal, Coave will leverage its AAV-Ligand Conjugate (ALIGATER) platform for designing, development and production of coAAV viral vectors that carry the TFEB gene for specific delivery to deep brain areas.
Meanwhile, IMN will carry out the in vivo studies that are designed for assessing the effect of gene therapy products in MSA and PD animal models.
The partnership plans to produce additional in vivo proof of concept data as well as allow the possibility of selecting therapeutic candidates for participation in IND enabling studies.