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A new partnership between Andelyn Biosciences and South Korea's Celliaz is pushing forward CLZ002, an AAV-based gene therapy for retinitis pigmentosa (RP). Unlike most gene therapies, CLZ002 doesn't target a specific mutation — it works by restoring the retina's own regenerative capacity, potentially helping patients across a wide range of genetic causes. A Phase 1/2 clinical trial is on the horizon.
Andelyn Biosciences and South Korean biotech Celliaz have announced a strategic partnership to develop CLZ002, an investigational AAV-based gene therapy for retinitis pigmentosa (RP) — a group of inherited retinal diseases that progressively rob patients of their vision. What makes CLZ002 stand out is its gene-agnostic approach: rather than targeting one of the 90+ mutations that can cause RP, it aims to unlock the retina's own regenerative potential by blocking PROX1, a protein that suppresses the regenerative activity of Müller glia cells.
Andelyn will provide end-to-end development and manufacturing services using its proprietary AAV Curator® Suspension Platform, leveraging an AAV2 capsid for delivery. The goal is to advance CLZ002 through IND-enabling studies and into a planned Phase 1/2 clinical trial.
Key Takeaways:
Why it matters: With over 3 million people affected by RP worldwide and very limited treatment options, a therapy that works across genetic subtypes could be a game-changer for a patient population that has long had few options.
