The trial is funded through Abu Dhabi’s Healthcare Research and Innovation FundClinical development activities will commence at Cleveland Clinic Abu Dhabi in 2026MERTK-related retinitis pigmentosa affects an estimated 60,000 patients worldwide RESEARCH TRIANGLE PARK, N.C., Jan. 27, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today an ...

Core Insights - Opus Genetics is advancing its gene therapy pipeline aimed at treating inherited retinal diseases (IRDs) with significant milestones achieved in 2025 and upcoming catalysts in 2026 [1][3] Pipeline Progress - In 2026, Opus is set to announce clinical data from its BEST1 program and move its LCA5 program into pivotal testing, targeting FDA approval [2] - The company has multiple programs that may qualify for Rare Pediatric Disease designation, potentially leading to Priority Review Vouchers (PRV) for non-dilutive capital [2] Corporate Achievements - 2025 was marked by strong execution, with two gene therapies advancing in clinical trials and an FDA application submitted for a partnered commercial product, indicating a large market opportunity [3] - The company secured approximately $23 million in gross proceeds through a registered direct offering, raising its total cash position to over $50 million, which is expected to fund operations into the second half of 2027 [10] Clinical Trials and Data - The OPGx-BEST1 program is currently in a Phase 1/2 trial (BIRD-1) with initial data expected in mid-2026, and it is potentially eligible for multiple regulatory designations [8][11] - OPGx-LCA5 is advancing into a pivotal Phase 3 trial, with positive data reported in pediatric participants and durable improvements in adult cohorts [12] Financial Outlook - As of September 30, 2025, Opus Genetics had cash and cash equivalents of $30.8 million, with a total cash position exceeding $50 million after recent fundraising [10]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies aimed at restoring vision and preventing blindness in patients with inherited retinal diseases [3] Company Overview - The company is developing durable, one-time treatments that target the underlying genetic causes of severe retinal disorders [3] - Opus Genetics has a pipeline that includes seven AAV-based programs, with key candidates such as OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration [3] - Additional candidates in development target RHO, RDH12, and MERTK mutations [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is an approved therapy for pharmacologically induced mydriasis, with further indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3] Upcoming Events - George Magrath, M.D., the CEO of Opus Genetics, will present at the J.P. Morgan 2026 Healthcare Conference on January 15, 2026, at 8:15 a.m. PT [1]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies aimed at restoring vision and preventing blindness in patients with inherited retinal diseases [3] Company Overview - The company is developing durable, one-time treatments that target the underlying genetic causes of severe retinal disorders [3] - Opus Genetics has a pipeline that includes seven AAV-based programs, with key candidates such as OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration [3] - Additional candidates in development target RHO, RDH12, and MERTK mutations [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is an approved therapy for pharmacologically induced mydriasis, with further indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3] Upcoming Events - George Magrath, M.D., the CEO of Opus Genetics, will present at the J.P. Morgan 2026 Healthcare Conference on January 15, 2026, at 8:15 a.m. PT [1]

Core Insights - Opus Genetics has received a positive recommendation from the Independent Data Monitoring Committee (IDMC) to continue its Phase 1/2 BEST1 clinical trial (BIRD-1) for the gene therapy OPGx-BEST1 aimed at treating Best disease [1][2][3] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs) [7] - The company is advancing a pipeline that includes seven AAV-based programs, with OPGx-BEST1 targeting BEST1-related retinal degeneration [7] Clinical Trial Details - The BIRD-1 trial is a multi-center, adaptive, open-label, dose-exploring study evaluating the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) [5] - Treatment involves a single subretinal injection in one eye of each participant, with two dosing cohorts being explored [5] Disease Background - Best disease, or vitelliform macular dystrophy, is a rare inherited retinal condition caused by mutations in the BEST1 gene, leading to progressive vision loss and potential blindness [6] - Approximately 9,000 patients in the United States are affected by BEST1-related inherited retinal diseases [6]

Core Insights - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies aimed at restoring vision and preventing blindness in patients with inherited retinal diseases (IRDs) [4] Company Overview - The company is developing durable, one-time treatments that target the underlying genetic causes of severe retinal disorders [4] - Opus Genetics has a pipeline that includes seven AAV-based programs, with key candidates being OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration [4] - Additional candidates in development target RHO, RDH12, and MERTK [4] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for pharmacologically induced mydriasis, with further indications in late-stage development for presbyopia and low-light visual disturbances following keratorefractive surgery [4] - Opus Genetics is headquartered in Research Triangle Park, NC [4] Upcoming Events - Management will participate in the BTIG 5th Annual Ophthalmology Day on December 1, 2025, with Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, in a fireside chat at 1:00 p.m. ET [2] - George Magrath, M.D., Chief Executive Officer, will participate in the Piper Sandler 37th Annual Healthcare Conference on December 2, 2025, at 10:00 a.m. ET in New York, NY [2] - A link to the live and archived webcast for the Piper Sandler conference will be available on the company's website under the Investors section [3]

Core Insights - Opus Genetics has reported positive clinical data from its OPGx-LCA5 Phase 1/2 trial, indicating potential for restoring cone-mediated vision in patients with inherited retinal diseases [1][6] - The company successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA, which may facilitate an accelerated regulatory pathway for OPGx-LCA5 [1][6] Pipeline Updates - The OPGx-LCA5 program is focused on gene therapy for Leber Congenital Amaurosis (LCA) and has shown significant improvements in visual function in both pediatric and adult participants [3][6] - Recruitment is ongoing for the OPGx-BEST1 gene therapy program, targeting BEST1-related inherited retinal diseases [4][5] - The company plans to submit a Supplemental New Drug Application for Phentolamine Ophthalmic Solution 0.75% for presbyopia treatment by the end of 2025 [5][8] Financial Results - As of September 30, 2025, Opus Genetics reported cash and cash equivalents of $30.8 million, with an additional $23.0 million raised through a direct equity offering [10] - License and collaborations revenue for Q3 2025 was $3.1 million, a decrease from $3.9 million in Q3 2024, primarily due to lower research and development service reimbursements [11] - General and Administrative expenses increased to $5.0 million in Q3 2025 from $2.9 million in Q3 2024, attributed to higher legal, payroll, and professional service costs [12] - Research and Development expenses decreased to $6.4 million in Q3 2025 from $9.0 million in Q3 2024, reflecting lower costs in clinical research and manufacturing [13] Net Loss - The net loss for Q3 2025 was $17.5 million, or $(0.25) per share, compared to a net loss of $7.5 million, or $(0.29) per share, in Q3 2024 [15][20]

Core Insights - Opus Genetics has successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA regarding its gene therapy candidate OPGx-LCA5 for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene [1][2][8] - The FDA provided constructive feedback on Opus's registration strategy, including Chemistry, Manufacturing and Controls (CMC), and pivotal trial design, acknowledging the significant unmet medical need for individuals with LCA5-related blindness [2][3] - The company plans to advance its ongoing trial using an adaptive design that includes a Phase 3 portion, which will avoid the need for a separate registrational trial [3][4] Clinical Development - Six late-stage participants have been treated in the Phase 1/2 trial of OPGx-LCA5, with all experiencing clinically meaningful improvements in vision [3][9] - The Phase 3 trial will include a run-in period to evaluate the natural history of each participant, serving as their own control [5][4] - Efficacy and safety will be assessed using various measures, including visual acuity and microperimetry, with dosing anticipated in the second half of 2026 [5][8] Financial Position - Opus Genetics has secured recent financing of $23 million, which will support the advancement of its LCA5 program and fund current operating plans into the second half of 2027 [6][8] Regulatory Environment - The FDA's introduction of the Rare Disease Evidence Principles (RDEP) review process aims to facilitate the approval of drugs for rare diseases, and Opus believes its LCA5 program meets the eligibility criteria for this process [7][8] - OPGx-LCA5 has the potential to be the first gene therapy and one-time treatment for LCA type 5, addressing a significant unmet medical need [8][11] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small-molecule therapies for other ophthalmic disorders [12] - The company's pipeline includes AAV-based gene therapies targeting conditions such as LCA and bestrophinopathy, with OPGx-LCA5 currently in a Phase 1/2 clinical trial [12][9]

Core Viewpoint - Opus Genetics has announced a registered direct offering to raise approximately $23 million to fund its gene therapy clinical programs for inherited retinal diseases and other ophthalmic disorders [1][2]. Group 1: Financing Details - The financing was led by Perceptive Advisors and Balyasny Asset Management, with participation from Nantahala Capital [1]. - The offering includes the sale of 3,827,751 shares of common stock at a price of $2.09 per share and pre-funded warrants to purchase up to 7,177,033 shares at a purchase price of $2.0899 per warrant [3]. - The offering is expected to close on November 7, 2025, subject to customary closing conditions [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to advance the LCA5 and BEST-1 gene therapy clinical programs, as well as for working capital and general corporate purposes [2]. - The company expects its cash resources to fund operations into the second half of 2027, excluding potential proceeds from callable warrants or future milestone payments [2]. Group 3: Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [6]. - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA) and bestrophinopathy, with lead candidates OPGx-LCA5 and OPGx-BEST1 currently in clinical trials [6].

Core Insights - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) [8] - The company’s gene therapy OPGx-LCA5 is currently in a Phase 1/2 trial aimed at restoring vision for individuals with a rare genetic form of blindness caused by mutations in the LCA5 gene [2][8] - The recent feature on Good Morning America highlights the potential of gene therapy to change lives, bringing national attention to the advancements in treating inherited blindness [3][5] Company Overview - Opus Genetics is based in Research Triangle Park, NC, and is developing AAV-based gene therapies targeting various inherited retinal diseases, including Leber congenital amaurosis (LCA) and bestrophinopathy [8] - The company’s pipeline includes lead candidates OPGx-LCA5 and OPGx-BEST1, with ongoing clinical trials for both therapies [8] - In addition to gene therapies, Opus Genetics is advancing a partnered therapy, Phentolamine Ophthalmic Solution 0.75%, which is being studied in Phase 3 programs for presbyopia and other visual disturbances [8]