Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company advancing gene therapies for inherited retinal diseases (IRDs), announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OPGx-BEST1. The therapy is designed to treat bestrophin-1 (BEST1)-related IRD, also known as Best disease, a rare inherited condition that can lead to progressive vision loss and, in severe cases, blindness.
With FDA clearance, Opus plans to initiate a Phase 1/2 clinical trial in the second half of 2025. The multi-center, open-label study will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD. The trial will also measure biological activity through visual function and retinal structure endpoints.
“The FDA’s clearance of our BEST1 IND is a significant step forward for the IRD community and for our mission at Opus Genetics focused on restoring vision for patients,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”
OPGx-BEST1 is built on Opus Genetics’ proprietary AAV-based gene therapy platform, which delivers a functional copy of the BEST1 gene directly to retinal pigment epithelium (RPE) cells. Preclinical studies have shown that the therapy restores BEST1 protein expression and improves retinal function, providing a strong foundation for its transition into human trials.
This trial marks the third clinical program in Opus Genetics’ expanding pipeline, reinforcing its commitment to bringing gene therapies to patients with rare inherited eye diseases.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. The Company’s pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its lead gene therapy candidates are OPGx-LCA5, which is in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, a partnered therapy currently approved in one indication and being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com .
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