Opus Genetics Receives FDA Clearance to Launch Clinical Trial for Best1 Gene Therapy

Opus Genetics, a biopharmaceutical company developing gene therapies for inherited retinal diseases, has received authorization from the US Food & Drug Administration (FDA) to launch a Phase 1/2 gene therapy clinical trial for people with Best disease and other inherited retinal diseases caused by mutations in the gene Best1. Opus plans to launch the dose-escalation trial at two sites in the US by the end of 2025. Additional trial details are forthcoming at clinicaltrials.gov.

Opus Genetics was established by the Foundation in 2021. The RD Fund, the Foundation’s venture philanthropy arm, is an investor in the company. The Foundation also provided nearly $2 million in funding for successful, proof-of-concept studies at the University of Pennsylvania for a Best1gene therapy that reversed Best disease in canine models.

Known as OPGx-BEST1, the emerging gene therapy is administered through a one-time subretinal injection. OPGx-BEST1 uses an adeno-associated virus (AAV), which works like a container system, to deliver healthy copies of Best1 into retinal cells to augment the mutated copies.

Best disease is a form of inherited macular degeneration, often leading to severe central vision loss. Lesions comprised of waste material accumulate in a layer of support cells known as the retinal pigment epithelium (RPE) and underneath the retina. The condition also causes micro detachments between the RPE and photoreceptors. The separation leads to loss of photoreceptors and vision.