Core Viewpoint - Opus Genetics has formed a strategic partnership with the Global RDH12 Alliance to advance the development of OPGx-RDH12, a gene therapy targeting RDH12 mutations associated with Leber congenital amaurosis (RDH12-LCA) [1][2][3] Group 1: Collaboration Details - The Global RDH12 Alliance will contribute up to $1.6 million towards the OPGx-RDH12 program, which includes a risk-sharing structure and performance-based milestones [3] - The collaboration aims to co-develop the OPGx-RDH12 program, focusing on clinical and regulatory strategies, with the goal of filing an Investigational New Drug (IND) application with the FDA by late 2025 [3] Group 2: Background on RDH12-LCA - RDH12-LCA is an ultra-rare form of childhood blindness affecting several thousand people globally, characterized by early visual decline and structural retinal changes by age two [5] - The OPGx-RDH12 therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the RDH12 gene directly to retinal photoreceptors, with preclinical studies showing restoration of RDH12 activity [5] Group 3: Organizational Insights - The RDH12 Fund for Sight is a U.S.-based non-profit organization focused on supporting families affected by RDH12-LCA and funding research for treatments [7] - Eyes on the Future is a UK-based non-profit organization dedicated to research into rare eye diseases, specifically targeting RDH12 inherited retinal dystrophies [8] - Opus Genetics is a clinical-stage biopharmaceutical company developing gene and small molecule therapies for vision-threatening eye diseases, with a pipeline that includes therapies for various inherited retinal diseases [9]
Opus Genetics and the Global RDH12 Alliance Partner to Advance RDH12 Gene Therapy for Inherited Childhood Blindness