US clinical-stage biopharmaceutical company Opus Genetics Inc (NASDAQ:IRD) on Wednesday announced a strategic partnership with the Global RDH12 Alliance to accelerate development of OPGx-RDH12, a gene therapy candidate targeting RDH12-associated Leber congenital amaurosis (RDH12-LCA), a rare inherited retinal disease that leads to early childhood vision loss.
The Global RDH12 Alliance comprises non-profit advocacy groups including RDH12 Fund for Sight (US) and Eyes on the Future (UK), both focused on advancing treatment for RDH12-related conditions. Under the agreement, the Alliance will contribute up to USD1.6m toward development efforts.
The partnership features a risk-sharing structure and performance-based milestones, with both parties collaborating on clinical and regulatory strategies. A key objective is to submit an Investigational New Drug (IND) application to the US Food and Drug Administration by late 2025.
RDH12-LCA is an ultra-rare condition affecting a few thousand individuals globally, with symptoms typically manifesting before the age of two. OPGx-RDH12 utilises an adeno-associated virus vector to deliver a functional copy of the RDH12 gene to retinal photoreceptors.
Preclinical studies of OPGx-RDH12 have demonstrated restored RDH12 activity and functional improvements in cell and mouse models.
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