RESEARCH TRIANGLE PARK, N.C., Dec. 11, 2025 (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, announced today that George Magrath, M.D., Chief Executive Officer, will present at the J.P. Morgan 2026 Healthcare Conference on Thursday, January 15, 2026 at 8:15 a.m. PT. A link to the live and archived webcast may be acc ...

RESEARCH TRIANGLE PARK, N.C., Dec. 11, 2025 (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, announced today that George Magrath, M.D., Chief Executive Officer, will present at the J.P. Morgan 2026 Healthcare Conference on Thursday, January 15, 2026 at 8:15 a.m. PT. A link to the live and archived webcast may be acc ...

RESEARCH TRIANGLE PARK, N.C., Dec. 09, 2025 (GLOBE NEWSWIRE) -- Opus Genetics (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 the Independent Data Monitoring Committee (IDMC) issued a positive recommendation to continue as planned in the Company’s Phase 1/2 BEST1 clinical trial (BIRD-1), which is a multi-center, adaptive, open ...

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]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [3] Company Overview - The company is based in Research Triangle Park, NC, and its pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Opus Genetics' lead gene therapy candidates are OPGx-LCA5, currently in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, targeting BEST1-related retinal degeneration [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and reduced low light vision [3] Upcoming Events - Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, will present a corporate update at Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025, at 12:00 p.m. ET [1]

Core Insights - Opus Genetics, Inc. is set to present positive clinical results from its gene therapy and Phentolamine Ophthalmic Solution programs at various medical and industry conferences in October 2025 [1][2] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [5] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [5] - Lead gene therapy candidates include OPGx-LCA5, currently in a Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1 for BEST1-related retinal degeneration [5] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and visual disturbances [5] Conference Participation - The company will participate in several key conferences, including: - Cell and Gene Meeting on the Mesa - Fierce Biotech Week - American Academy of Optometry Annual Meeting 2025 - Eyecelerator @ American Academy of Ophthalmology (AAO) [3][4] Presentation Details - Presentation on OPGx-LCA5 will include three-month pediatric and 18-month adult clinical data from the Phase 1/2 trial for Leber congenital amaurosis type 5 [6] - A fireside chat titled "Investing with Intention – How Early Vision and Partnership Shaped Opus Genetics" will feature key executives from the company [6] - A poster presentation on the pivotal Phase 3 trial of Phentolamine Ophthalmic Solution will also take place [6]