Opus Genetics and the Global RDH12 Alliance Partner to Advance RDH12 Gene Therapy for Inherited Childhood Blindness
Opus Genetics announced a partnership with the Global RDH12 Alliance to advance Opus’ gene therapy program for patients with vision loss due to retinol dehydrogenase 12 (RDH12) gene mutations. The Alliance serves as a collaborative platform uniting advocacy groups dedicated to RDH12-related IRDs, including: (i) “RDH12 Fund for Sight” in the US, and (ii) “Eyes on the Future” in the UK.
According to Opus, this collaboration will accelerate development of OPGx-RDH12, Opus’ gene therapy program targeting the RDH12 gene mutation for the potential treatment of Leber congenital amaurosis (RDH12-LCA). Leber congenital amaurosis (LCA) is a rare IRD that causes progressive vision loss and blindness, often beginning in early childhood. Patients with RDH12 mutations often have early visual acuity loss with retinal structural changes by two years of age.
Under the agreement, the Alliance will provide up to $1.6 million toward the development of the OPGx-RDH12 program. The partnership also includes a risk-sharing structure and performance-based milestones. Together, the parties will co-develop the OPGx-RDH12 program, including the clinical and regulatory strategy, with the goal of filing an investigational new drug (IND) application with the FDA by late 2025.
“Since founding the RDH12 Fund for Sight more than a decade ago, our goal has always been to bring a treatment to the RDH12-LCA community,” said Mathew Pletcher, PhD, Board member of the RDH12 Fund for Sight and father to a 19-year-old living with the condition. “This partnership represents a significant step forward. By combining our patient community’s unique, first-hand perspectives on RDH12-LCA and resources with Opus’ gene therapy expertise, we can accelerate the transition of this promising therapy out of the laboratory and into the clinic.”
About RDH12-LCA and OPGx-RDH12
RDH12-LCA is an ultra-rare form of childhood blindness affecting several thousand people globally. Mutations in the RDH12 gene impair protein function in the retina, leading to early visual decline, often with structural retinal changes by age two, and rapid progression during the second decade of life. OPGx-RDH12 uses an adeno-associated virus (AAV) vector to deliver a functional copy of the RDH12 gene directly to photoreceptors in the retina. “Preclinical studies in cell and mouse models have shown restoration of RDH12 activity and functional improvements,” said Professor Jean Bennett, MD, PhD, Opus Genetics Scientific Advisor and Board of Directors member.