- Multi-asset pipeline with significant targeted data readouts and milestones -- Two lead ophthalmic gene therapy programs in clinical trials with new data anticipated in 2026 -- Supplemental New Drug Application (sNDA) submitted for partnered presbyopia treatment - - Corporate presentation at J.P. Morgan Healthcare Conference on Thursday, January 15, at 8:15 a.m. PT - RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company,” “Opus,” or “Opus Genetics” ...

Biotech stocks are not for the faint of heart, as companies in the clinical stage push risk to the extreme. With key clinical data still to come, huge financial burn, and valuation almost entirely dependent on trial outcomes, these companies only offer massive upside if everything goes as planned. Here are two of the riskiest biotech stocks investors are betting on. Biotech Stock #1: Opus Genetics Opus Genetics (IRD) has emerged as one of the boldest and riskiest bets investors are making right now. Val ...

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]

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 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]

Core Insights - Opus Genetics has initiated the first dosing in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [1][4] - The trial aims to evaluate the safety, tolerability, and preliminary efficacy of a one-time subretinal injection of OPGx-BEST1, targeting the underlying genetic cause of Best disease [6][9] 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 [9] - The company is advancing multiple AAV-based programs, including OPGx-LCA5 and OPGx-BEST1, with a pipeline targeting various genetic mutations associated with severe retinal disorders [9] Clinical Trial Details - The OPGx-BEST1 trial is a multi-center, adaptive, open-label study designed to explore the safety and efficacy of the gene therapy in patients with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) [6] - Initial data from the trial is expected in the first quarter of 2026, with the study focusing on functional and anatomical endpoints related to visual function and retinal structure [6] Disease Background - Best disease, caused by mutations in the BEST1 gene, leads to progressive vision loss and affects an estimated 9,000 patients in the United States [2][8] - The BEST1 gene is crucial for producing bestrophin, a protein that regulates chloride ion movement in retinal cells, and its mutations can result in severe retinal conditions [7][8]

Core Insights - Opus Genetics has initiated the dosing of the first participant in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [2][5] - The OPGx-BEST1 therapy is a one-time subretinal injection aimed at restoring function to retinal pigment epithelium cells affected by mutations in the BEST1 gene [3][6] - Initial data from the trial is expected in Q1 2026, which will evaluate the safety, tolerability, and preliminary efficacy of the treatment [7] 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 [10] - The company’s pipeline includes seven AAV-based programs, with OPGx-BEST1 targeting BEST1-related retinal degeneration [10] - The company is based in Research Triangle Park, NC, and is also advancing other therapies for various ocular conditions [10] Clinical Trial Details - The Phase 1/2 trial, known as BIRD-1, is an adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy or Autosomal-Recessive Bestrophinopathy [7] - The trial will explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure [7] - The study is being conducted by a team of experts from the Retina Foundation of the Southwest and Retina Consultants of Texas [4]

Core Insights - Opus Genetics, Inc. reported a quarterly loss of $0.12 per share, better than the Zacks Consensus Estimate of a loss of $0.14, and improved from a loss of $0.29 per share a year ago, resulting in an earnings surprise of +14.29% [1] - The company generated revenues of $3.08 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 11.36%, although this represents a decline from $3.87 million in the same quarter last year [2] - Opus Genetics shares have increased approximately 65.6% year-to-date, significantly outperforming the S&P 500's gain of 16.4% [3] Earnings Outlook - The company's earnings outlook is crucial for investors, as it includes current consensus earnings expectations for upcoming quarters and any recent changes to these expectations [4] - The current consensus EPS estimate for the next quarter is -$0.12 on revenues of $3.75 million, and for the current fiscal year, it is -$0.56 on revenues of $13.67 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Opus Genetics belongs, is currently ranked in the top 34% of over 250 Zacks industries, indicating a favorable outlook compared to lower-ranked industries [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors or through tools like the Zacks Rank [5][6]