Opus Genetics, Inc.

Opus Genetics Appoints Dr. Andrew Murphy as Chief Scientific Officer and Jamie Lynne Hart as Executive Director, Executive Operations

Opus Genetics appointed Andrew Murphy, Ph.D., as Chief Scientific Officer and Jamie Lynne Hart as Executive Director, Executive Operations. Dr. Murphy brings 18+ years of gene therapy and AAV vector development experience, most recently as SVP of Research at Kriya Therapeutics; Hart spent 15+ years in biotech operations including at Apellis and Ocular Therapeutix. The hires support the company's seven-program AAV gene therapy pipeline, led by OPGx-LCA5 and OPGx-BEST1, as it advances toward clinical milestones.

•3d ago

Opus Genetics Announces FDA Clearance of IND Application for OPGx-RDH12 Gene Therapy for Inherited Childhood Blindness

The FDA cleared Opus Genetics' IND application for OPGx-RDH12, an AAV-based gene therapy for RDH12-associated Leber congenital amaurosis, a rare inherited retinal disease causing childhood blindness. The clearance lets the company advance its third gene therapy program into the clinic, with a Phase 1/2 trial planned to begin this quarter. The program is supported by the Global RDH12 Alliance, which committed up to $1.6 million in reimbursed development costs under a July 2025 venture-philanthropy collaboration. RDH12-LCA affects an estimated 30,900 people globally; Opus's broader pipeline spans seven AAV-based programs.

•11d ago

Opus Genetics Announces FDA Approval of Phentolamine Ophthalmic Solution 0.75% for the Treatment of Presbyopia

The FDA approved Opus Genetics' sNDA for phentolamine ophthalmic solution 0.75% (RYZUMVI) for treatment of presbyopia in adults, the product's second approved indication. Approval was backed by pivotal Phase 3 VEGA-2 and VEGA-3 trials, which met primary and all key secondary endpoints with no treatment-related serious adverse events. Viatris holds exclusive U.S. commercialization rights under a global licensing agreement, and the approval triggers a milestone payment to Opus Genetics (amount undisclosed).

•16d ago

Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026

Opus Genetics presented positive interim clinical data for its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026. OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable low-dose Cohort 1 participants in the Phase 1/2 BIRD-1 trial; the high-dose Cohort 2 has completed enrollment with 11 participants. OPGx-LCA5 demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults and encouraging six-month results in pediatric participants, supporting advancement into Phase 3. Phase 3 dosing for OPGx-LCA5 is expected in the fourth quarter of 2026, with primary endpoint assessing retinal sensitivity across 16 central retinal test locations.

•17d ago

Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027

Opus Genetics reported positive 3- and 6-month Cohort 1 data from its Phase 1/2 BIRD-1 trial of OPGx-BEST1 in BEST1-related retinal diseases, with all five participants showing clinically meaningful visual function improvement and no serious adverse events or dose-limiting toxicities. A successful August 2026 FDA Type C meeting produced alignment on a potential pivotal endpoint of at least 3 decibels of microperimetry improvement in five or more prespecified loci, paired with a patient-reported outcome, with Phase 3 participant dosing expected to begin in 2027. The higher-dose Cohort 2 has been over-enrolled to eight participants, with dosing expected to complete in Q4 2026 and topline three-month data expected in Q2 2027; 6-month data for the three BVMD participants is also expected in Q2 2027. New epidemiology from Triangle Insights Group estimates roughly 23,600 symptomatic U.S. BEST1 patients and about 45,400 globally -- a larger addressable population than previously estimated -- and management says cash runway extends into 2029, funding multiple clinical inflection points.

•1mo ago

Opus Genetics Announces Financial Results for Second Quarter 2026 and Provides Corporate Update

Opus Genetics reported Q2 revenue of $0.8M, down from $2.9M YoY, with a net loss of $8.1M ($0.14 per share). The company ended the quarter with $88.8M in cash, providing a runway into 2029 to support multiple clinical programs and potential PRV opportunities. Key catalysts include OPGx-BEST1 Cohort 1 topline data expected in the second week of September 2026 and the initiation of dosing for the OPGx-LCA5 registrational trial in Q4 2026.

•2mo ago

Opus Genetics Completes Patient Enrollment in Registrational Phase 3 Trial of OPGx-LCA5 for LCA5-Associated Inherited Retinal Disease

Opus Genetics completed patient enrollment in its registrational Phase 3 trial of OPGx-LCA5 for LCA5-associated inherited retinal disease, marking a key de-risking event. The study incorporates innovative evidence-generation approaches aligned with the FDA's Rare Disease Evidence Principles program, with topline efficacy data expected by the end of 2027. Dosing is set to begin in the fourth quarter of 2026, and the therapy holds potential for a Biologics License Application and a Rare Pediatric Disease Priority Review Voucher.

•2mo ago

Opus Genetics Provides BEST1 Program Update with Detailed Timeline for OPGx-BEST1 Results

Opus Genetics confirmed that enrollment for Cohort 1 of its OPGx-BEST1 Phase 1/2 trial is complete, with five participants treated at a dose of 1.5E9 vg/eye. The company expects to announce three-month topline data in the second week of September 2026, contingent on participants completing scheduled assessments. Data will be presented at the EURETINA Congress in October 2026, with an IDMC review determining whether to advance to Cohort 2 or expand into a pivotal trial based on safety and subretinal fluid reduction.

•3mo ago

Opus Genetics Announces FDA Alignment on Phase 3 Registrational Trial Design for OPGx-LCA5 in LCA5-Associated Inherited Retinal Disease

Opus Genetics reached alignment with the FDA on the Phase 3 design for OPGx-LCA5, allowing for a BLA submission based on 6-month efficacy data with 12-month durability data to follow during review. The registrational trial plans to enroll eight participants using a six-month run-in period as a natural history control, with seven patients already enrolled and dosing anticipated to begin in the fourth quarter of 2026. The trial is designed with greater than 90% statistical power to detect a mean improvement of at least 7 decibels in retinal sensitivity, supported by Phase 1/2 data showing an approximate 10.5 dB improvement.

•3mo ago

Opus Genetics Added to the Russell 3000®, Russell 2000® and Russell Microcap® Indexes

Opus Genetics will be added to the Russell 3000, Russell 2000 and Russell Microcap Indexes effective June 29, 2026, as part of the quarterly reconstitution. CEO George Magrath noted that inclusion validates the company's progress in advancing gene therapies for inherited retinal diseases and enhances visibility within the investment community. Approximately $12.2 trillion in assets are benchmarked against the Russell US indexes, which are widely used by investment managers and institutional investors.

•4mo ago

Opus Genetics Highlights Five Gene Therapy Programs Targeting Significant Unmet Need in Patients with Inherited Retinal Diseases

Opus Genetics hosted an R&D Science Forum highlighting its pipeline of five gene therapy programs targeting inherited retinal diseases, with cash runway expected to extend into 2029. Management detailed the clinical development strategy, expecting OPGx-RDH12 and OPGx-LCA5 dosing to begin in Q4 2026, followed by OPGx-MERTK in Q1 2027 and OPGx-RHO in the second half of 2027. The company noted continued progress in ongoing trials for OPGx-LCA5 and OPGx-BEST1, with topline data from the first cohort of the BEST1 trial anticipated in September 2026.

•4mo ago

Opus Genetics Presents Clinical and Preclinical Data at ARVO 2026 Demonstrating Continued Pipeline Advancement in Inherited Retinal Diseases

Opus Genetics presented positive clinical and preclinical data at ARVO 2026, showing restoration of cone-mediated vision in pediatric LCA5 patients and early visual acuity gains in BEST1 trials. The data also demonstrated a mutation-independent AAV approach with potential to treat genetically diverse retinal diseases using a single therapy. These findings support the company's broader pipeline and regulatory readiness, with topline data from Cohort 1 of the OPGx-BEST1 study expected in September 2026.

•5mo ago

Opus Genetics Completes Enrollment in Cohort 1 of Phase 1/2 OPGx-BEST1 Gene Therapy Study

Opus Genetics completed enrollment in Cohort 1 of its Phase 1/2 study of OPGx-BEST1, enrolling five participants across both dominant and recessive forms of BEST disease. Four participants have been dosed with the subretinal gene therapy, and the fifth is scheduled for dosing this month; topline data from the full cohort is expected in September 2026. The company presented baseline demographics and early tolerability data from the sentinel participant at the ARVO Annual Meeting, highlighting biological activity signals.

•5mo ago

Opus Genetics Announces FDA Acceptance of OPGx-LCA5 into Rare Disease Evidence Principles (RDEP) Program

Opus Genetics announced that the FDA accepted its OPGx-LCA5 program into the Rare Disease Evidence Principles (RDEP) initiative. This program supports therapies for ultra-rare diseases affecting fewer than 1,000 patients in the U.S. and enables early collaboration on trial design and evidence generation. The designation supports Opus Genetics' alignment on a pivotal Phase 3 program for OPGx-LCA5, complementing its existing RMAT and Orphan Drug designations.

•5mo ago