Opus Genetics Gets FDA Clearance for Gene Therapy Trial in Best Disease

Opus Genetics Received FDA Approval for the IND Application

Announcement

Opus Genetics leading clinical-stage biopharmaceutical company initiating gene therapies for inherited retinal disease (IRD) treatment and small molecule therapies for different ophthalmic disorders, received FDA clearance for an investigational new drug (IND) application for OPGx-BEST1, a gene therapy for bestrophin-1 (BEST1)-related IRD treatment. Vitelliform macular dystrophy, or best disease, is a rare inherited retinal condition affecting macular degeneration caused by mutations in the BEST1 gene, which causes severe vision loss in a few cases, like blindness.

Opus, with its robust application, has managed to achieve a new milestone and has performed well in the vast healthcare market. Most of the patients and their families affected by the type of retinal condition have reached a major population. The BEST1 IND application’s approval has paved the way for a clinical trial of a potential solution or alternative to IRD treatment. This allows Opus to focus on one direction, moving towards an effective clinical trial.

Opus’s Plans and the Potency of the IND Application

With this IND approval, Opus Genetics intends to conduct a phase 1/2 clinical trial in 2025's second half. The open-label and center-focused study will examine the tolerability, preliminary, and safety efficacy criteria of an individual subretinal injection of OPGx-BEST1 in genetically proven BEST1-based IRD patients. The trial will discover biological activity via anatomical and functional aspects, involving a shift in retinal structure and visual function.

OPGx-BEST1 accelerates opus genetics exclusivity. AAV-related gene therapy platform introduced to serve a functional copy of the BEST1 gene to the retinal pigment epithelium (RPE) cells in areas where defective genes are found to be active. The program stands on expanded preclinical work elaborating restoration of the BEST1 protein indication and enhancing retinal function in relevant disease models.

Views and Statements from Company Leaders

MD, Chief Executive Officer of Opus Genetics, George Magrath, said, “The FDA’s approval for our BEST1 IND is a valuable stepping stone for the IRD individuals, and our plan at Opus Genetics is to focus on restoring vision for the patients in need. The BEST1-based IRDs don’t have any approved treatments yet, which leaves patients and their families unaware about the future of their condition. The OPGx-BEST1 trial is going to be our third running clinical program, mirroring the intensity of our portfolio and our promise to initiate advancement in various therapies for diverse patients in need of urgent treatment.”