Andelyn partners with Celliaz to advance gene therapy for retinitis pigmentosa

Andelyn Biosciences (Andelyn), a leading US-based patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), and Celliaz Co., Ltd., a South Korean biotechnology company developing next-generation regenerative therapeutics for retinal degenerative diseases, today announced a strategic partnership to advance CLZ002, an investigational adeno-associated virus (AAV)-based gene therapy for retinitis pigmentosa (RP).

RP is a group of inherited retinal diseases that affect more than 3 million people worldwide and cause progressive vision loss. Treatment options for patients remain limited. Celliaz's approach to treating the disease is blocking PROX1, which suppresses the regenerative activity of Müller glia after transferring into Müller glia from neighboring retinal cells. Leveraging an AAV2 capsid for the gene therapy, Andelyn is providing end-to-end development and manufacturing services for Celliaz using its proprietary AAV Curator^® Suspension Platform.

RP remains an area of significant unmet need, and Celliaz's approach is compelling. Andelyn supports client INDs with deep AAV expertise including ophthalmology and rare disease programs, and this relationship with Celliaz adds to our work supporting multiple other South Korean programs. We are proud to bring that experience, together with our Curator^® Platform, to help Celliaz bring CLZ002 to the clinic."

Matt Niloff, Chief Commercial Officer, Andelyn Biosciences

"RP can result from mutations in more than 90 genes, making it challenging to develop gene-specific therapies that benefit a broad patient population," said Kyung Hwa Kang, PhD, Chief Executive Officer of Celliaz. "CLZ002 represents a fundamentally different approach. Rather than targeting individual disease-causing mutations, our therapy aims to restore the retina's intrinsic regenerative capacity by preventing PROX1 transfer into Müller glia. These retinal cells retain latent regenerative potential. This gene-agnostic strategy could potentially benefit patients with RP caused by a wide range of genetic mutations. To translate this innovative approach into a clinical therapy, we needed a partner with strong scientific expertise and reliable manufacturing capabilities. Andelyn's extensive experience in ophthalmic and rare disease AAV programs, its Curator® Suspension Platform, and its integrated plasmid and viral vector manufacturing capabilities make it an ideal partner. We believe this partnership will be instrumental in advancing CLZ002 through IND-enabling development and toward our planned Phase 1/2 clinical trial."

This partnership reflects Andelyn's broader mission to advance cell and gene therapies from development through clinical and commercial readiness. With its concept-to-commercial capabilities, comprehensive and phase-appropriate quality system, and patient-focused approach, Andelyn helps organizations bring critical therapies to patients worldwide.