Opus Genetics has received FDA clearance for its IND application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease (IRD), marking a major milestone for the company and the IRD community.

This IND approval signifies a significant step forward in developing treatments for a rare condition that causes progressive vision loss, with no current approved therapies available.

The therapy uses Opus's proprietary AAV-based platform to deliver a functional copy of the BEST1 gene directly to retinal pigment epithelium (RPE) cells, aiming to restore protein expression and improve retinal function.

Preclinical studies in 2024 demonstrated that a unilateral subretinal injection of OPGx-BEST1 was well tolerated in canines, with no significant toxicity at low and mid doses, although high doses showed some retinal signs of toxicity.

The targeted condition, Best disease, caused by mutations in the BEST1 gene, leads to progressive vision loss or blindness, affecting approximately 9,000 patients in the U.S., with no approved treatments currently available.

A primary focus of this therapy is Vitelliform macular dystrophy (VMD), including its early-onset form known as Best disease, which results in worsening vision over time.

Opus plans to initiate a Phase 1/2 clinical trial in the second half of 2025, involving genetically confirmed patients, to evaluate safety, tolerability, and early signs of efficacy through functional and anatomical measures.

The upcoming multicenter, open-label trial will involve a single subretinal injection of OPGx-BEST1 and will assess outcomes such as visual function and retinal structure changes.

This therapy builds on promising preclinical results and aims to restore the function of the BEST1 gene in retinal cells, potentially offering a groundbreaking treatment for affected patients.

This development marks Opus's third ongoing clinical program, underscoring its robust pipeline focused on gene therapies for retinal diseases.

Opus's pipeline also includes other gene therapies like OPGx-LCA5 for Leber congenital amaurosis and partnered products such as Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and in Phase 3 trials for presbyopia and visual disturbances.

George Magrath, CEO of Opus Genetics, emphasized the importance of the FDA clearance and highlighted the lack of approved treatments for BEST1-related IRDs, reaffirming the company's commitment to addressing this urgent unmet medical need.