RESEARCH TRIANGLE PARK, N.C., Feb. 02, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that members of its leadership team will participate in several leading ophthalmology and industry conferences. Collaborative Community on Ophthalmic Innovation (CCOI) Topic: Opus Genetics leadership ...

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 ...

PresentationSusmita Roy Good morning, everyone. My name is Susmita Roy, and I'm an associate on the JPMorgan Healthcare Investment Banking team. On behalf of JPMorgan, I would like to thank everyone for attending the Healthcare Conference this year. And I'm pleased and honored to introduce Opus Genetics for the company presentation today. A bit about Opus Genetics. It is a clinical stage biopharmaceutical company developing gene therapies for inherited retinal diseases, trading under the ticker symbol IRD. ...

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 - Biotech stocks, particularly those in clinical stages, carry high risks due to dependency on trial outcomes and significant financial burn [1] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company valued at $1.4 billion, focusing on gene therapies for inherited retinal diseases [2] - The company aims to develop long-lasting medicines that address genetic causes of eye disorders rather than just treating symptoms [4] Stock Performance - Opus stock has increased by 60.3% year-to-date, outperforming the broader market, with expectations for further growth contingent on clinical success and regulatory outcomes [3] Clinical Developments - The company’s pipeline includes OPGx-LCA5, a gene therapy for Leber Congenital Amaurosis (LCA5), which has shown promising early clinical data with significant vision improvements in pediatric participants [4] - A successful FDA Regenerative Medicine Advanced Therapy (RMAT) meeting for OPGx-LCA5 has been completed, potentially leading to an accelerated regulatory pathway [5] Financial Position - Opus Genetics reported a net loss of $17.5 million in Q3 and ended the quarter with $30.8 million in cash, later raising about $23 million through an equity offering, increasing total liquidity to over $50 million [5] - The management plans to use the raised funds to support operations into the second half of 2027, indicating confidence in achieving clinical milestones before needing additional funding [5]

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 Viewpoint - Opus Genetics, Inc. is gaining attention as a promising investment opportunity due to its innovative gene therapy programs aimed at treating inherited retinal diseases, particularly with the initiation of coverage by Piper Sandler with an "Overweight" rating and a price target of $7 [2]. Financial Performance - For Q3 2025, Opus Genetics reported a net loss of $17.5 million, an increase from $7.5 million in the same quarter of the previous year, primarily due to changes in the fair value of warrants [3]. - The company completed a $23 million equity offering to support its clinical trials, including the ongoing Phase 1/2 trial for OPGx-BEST1 [4]. Product Development - The lead gene therapy program, OPGx-LCA5, aims to treat Leber congenital amaurosis type 5 (LCA5), a condition with no current treatment options, and has shown significant improvements in vision for pediatric patients in Phase 1/2 trials [2][3]. - Opus Genetics successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA, which may facilitate an accelerated regulatory pathway for OPGx-LCA5 approval [4]. Market Position - The company is focused on developing gene therapies to restore vision in patients with inherited retinal diseases, positioning itself as a key player in this niche market [5].

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]