Tag: Opus Genetics

Opus Genetics in Alignment with FDA for Phase 3 LCA5 Gene Therapy Clinical Trial

Opus Genetics has reached alignment with the US Food and Drug Administration (FDA) on the Phase 3 clinical trial design for OPGx-LCA5, the company’s emerging LCA5 gene therapy. If OPGx-LCA5 is safe and meets its primary outcome measure in the trial at six months, Opus will likely seek FDA approval by submitting a Biologics License Application or BLA. Treatment durability data at 12 months will also be considered by the FDA during its review.

Leber congenital amaurosis 5 (LCA5) is one of the rarest and most severe forms of LCA affecting approximately 5,000 globally.

The Phase 3 trial investigators plan to treat both eyes in eight patients. The trial includes a pre-treatment run-in period in which the natural history (i.e., the natural disease course) for each planned participant will be evaluated. Seven of the eight patients are currently enrolled in the run-in study. Investigators plan to begin dosing patients in the fourth quarter of 2026.

Investigators will use microperimetry, which measures retinal sensitivity at different loci (points) in the retina, as the primary outcome measure in the Phase 3 clinical trial. A mean improvement of at least seven decibels (dB) in retinal sensitivity across the central 16 test loci is considered clinically meaningful.

Opus Genetics previously reported meaningful vision improvements in three adult and three pediatric patients (aged 16-17) in its Phase 1/2 LCA5 gene therapy clinical trial at the University of Pennsylvania. Some patients saw objects for the first time after treatment. Others had meaningful improvements in visual acuity.

OPGx-LCA5 is administered by an injection underneath the retina. The gene therapy uses an engineered adeno-associated virus (AAV) to deliver health copies of the LCA5 gene into retinal cells. Researchers believe that retinal gene therapies can work for many years, perhaps the lifetime of the patient.

Opus Genetics is a Hope in Focus partner.

LCA5 Gene Therapy Provides Vision Improvements in Clinical Trial

Nothing is more hopeful or gratifying for the retinal disease community than an emerging or FDA-approved therapy that provides vision to people with advanced retinal disease. Such is the case with Opus Genetics’ gene therapy for Leber congenital amaurosis 5 (LCA5) in an early-stage clinical trial.

In March 2024, the company announced vision improvements for the first three adult patients in its Phase 1/2 LCA5 gene therapy clinical trial. Some patients, who had been almost totally blind since birth, can now see and identify objects for the first time. The company has also reported positive safety data for the trial thus far.

Though LCA5 patients have severe vision loss at birth, they have some surviving retinal structure that researchers believe can be harnessed for improved vision using gene therapy.

Known as OPGx-001, the gene therapy uses a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the LCA5 gene to patients’ retinas, augmenting the mutated copies that cause vision loss. The therapy is administered through a one-time injection underneath the retina. Researchers believe gene therapies will be effective for many years, perhaps for the patient’s lifetime.

Opus plans to administer the next highest dose of its LCA5 gene therapy to the next cohort of adult patients in mid-2024. The company also has plans to dose patients as young as 13 years old sometime in the future.

The Phase 1/2 clinical trial is led by Tomas S. Aleman, M.D., at the Center for Advanced Retinal and Ocular Therapeutics (CAROT), Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania.

Courtney Coates, Hope in Focus’s Director of Outreach and Development, stated, “We are thrilled that patients in this trial are having early success with the low-dose treatment. We look forward to hearing more as the next cohort is enrolled for the mid-dose.”

The LCA5 gene therapy clinical trial is the first launched by Opus, a company founded in 2021 by the RD Fund, the venture arm of the Foundation Fighting Blindness, which is investing in companies near or in early-stage clinical trials for their retinal degenerative disease treatments.