Towards Healthcare

Global RDH12 Alliance and Opus Genetics Join Forces to Advance Gene Therapy for Childhood Vision Loss

The Global RDH12 Alliance and Opus Genetics have partnered to develop a gene therapy for childhood blindness caused by RDH12 mutations. The collaboration, supported by $1.6 million in funding, aims to fast-track clinical trials and file an IND with the FDA by year-end, bringing hope to families affected by Leber congenital amaurosis (RDH12-LCA).

Author: Towards Healthcare Published Date: 28 July 2025
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Global RDH12 Alliance and Opus Genetics Entered a Partnership Agreement

Gene therapy research for childhood vision loss supported by RDH12 and Opus

Announcement

Global RDH12 alliance and Opus Genetics Inc., a biopharmaceutical company accelerating gene therapies for the treatment of small-molecule therapies and inherited retinal disease (IRDs) for other ophthalmic disorders, have entered a partnership for advancing Opus’s gene therapy program for loss of vision patients caused due to retinol dehydrogenase 12 (rdh12) gene mutations. A discussion connects the main advocacy teams who are dedicated to RDH12-based IRDs, also consisting of ‘Eyes on the Future’ in the UK and ‘RDH12 Fund for Sight’ in the US. It is a strategic collaboration that has a high-aim collective vision of achieving multiple targets.

The partnership will elevate the progress of the OPGx-rdh12, an opus gene therapy program that focuses on the RDH12 gene mutation for providing effective treatment of Leber congenital amaurosis (RDH12-LCA). The LCA is a rare IRD that causes quick blindness and vision loss, and is visible in early childhood. The rdh12 mutation patients encounter retinal structural changes and early visual acuity loss from two years of age. The funding is a countable effort and encouragement to this partnership to introduce suitable and effective treatment for this disturbing condition, affecting the childhood of many.

Agreement

The agreement states that the partnership will enable $1.6 million for the development of the OPGx-rh12 program. Further, the collaboration involves the sharing of risk and profit-based milestones. The two parties will be embarking on co-development of the OPGx-rdh12 program, also consisting of regulatory and clinical strategy, with the motive of registering an investigational new drug (IND) application with the US Food and Drug Administration (FDA) by the year-end.

Views and Statements

Board member of the RDH12 Fund for Sight and father of a 19-year-old living with a similar condition, Phd, Mathew Pletcher said, “The funding for RDH12 for Sight since decade has encouraged and strengthened our goal to introduce treatment to the RDH12-LCA community. The partnership is a valuable step. With the help of an experienced perspective on this condition and extensive resources with Opus’s gene therapy expertise, we can leverage this transformative therapy straight from the laboratory and into the clinic.”

CEO of Opus Genetics, George Magrath, said, “Opus is thankful to be able to work directly with the patient team. This partnership is more like a financial arrangement. Both parties respect each other’s insights, connections, and experiences as complex to a progressive co-development of this gene therapy.”

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