CLZ002

CLZ002 is an investigational, gene-agnostic adeno-associated virus (AAV)-based gene therapy being developed by Celliaz for the treatment of retinitis pigmentosa (RP) and other retinal degenerative diseases. The therapy utilizes an AAV2 vector to deliver a gene encoding a human anti-PROX1 single-chain variable fragment fused to the Fc region of human IgG1κ (AAV2-anti-PROX1-scFvFc). Following a single intravitreal injection, the retina expresses a PROX1-neutralizing antibody that blocks the intercellular transfer of PROX1, a homeoprotein that acts as a "brake" on retinal regeneration by suppressing the regenerative activity of Müller glia. By preventing PROX1 transfer, CLZ002 aims to restore the retina's intrinsic regenerative capacity and promote Müller glia-mediated retinal repair. CLZ002 has received Orphan Drug Designation (ODD) from both the FDA and EMA, and is undergoing preclinical development with plans for IND-enabling studies and a Phase 1/2 clinical trial.