RESEARCH TRIANGLE PARK, N.C., Oct. 14, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, announced today that Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, will present a corporate update at Chardan's 9th Annual Genetic Medicines Conference on Tuesday, October ...

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]

Core Insights - Uniqure NV (NASDAQ:QURE) experienced a significant stock price increase of up to 275% following positive clinical trial results for its drug candidate AMT-130, aimed at treating Huntington's disease [1][2] - The drug candidate demonstrated a 75% reduction in the progression of Huntington's disease over a 36-month period, with no adverse effects reported since 2022 [2][3] - The FDA has granted AMT-130 Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations, indicating its potential significance in treatment [3] Company Performance - On the day of the announcement, shares peaked at $51.21 before closing at $47.50, reflecting a 247.73% increase [1] - The Chief Medical Officer of Uniqure expressed excitement about the trial results and their implications for patients and families affected by Huntington's disease [3][4] Industry Impact - The findings from the clinical trial are seen as a potential game-changer for the treatment landscape of Huntington's disease and support the use of one-time, precision-delivered gene therapies for neurological disorders [4]

Core Insights - CRISPR Therapeutics (CRSP) and Sirius Therapeutics have initiated a phase II study for SRSD107, a long-acting siRNA therapy targeting thromboembolic disorders [1][7] - The study focuses on the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA) [2] - CRISPR Therapeutics has seen a 57.7% increase in share price year-to-date, significantly outperforming the industry average of 3.5% [2] Collaboration and Pipeline Expansion - In May 2025, CRISPR Therapeutics entered a collaboration with Sirius Therapeutics to develop and commercialize siRNA therapies, sharing costs and profits equally [4][5] - The collaboration allows CRISPR to exclusively license up to two siRNA programs and retain rights for clinical development and commercialization [5] - This partnership diversifies CRISPR's pipeline into RNA therapeutics, expanding beyond its traditional gene therapies [8] Potential of SRSD107 - If successful, SRSD107 could become a best-in-class therapy for patients at risk of life-threatening thromboembolic events due to co-morbid conditions [6] Casgevy Sales and Future Prospects - CRISPR's gene therapy, Casgevy, approved for sickle cell disease and transfusion-dependent beta-thalassemia, is gaining sales momentum, with $30.4 million recorded in Q2 2025 [9][10] - The company is advancing multiple next-generation gene-edited cell therapy programs, including CAR T candidates for various cancers and autoimmune diseases [10]

Company Overview - Ultragenyx is a rare disease company focused on transformative treatments where none have existed in the past [2] - The company is 15 years old and is on a pathway to full year GAAP profitability in 2027 [2] - Ultragenyx has 4 current commercial programs that have significant and growing revenue [2] Upcoming Developments - The company has 3 near-term launches planned, including two gene therapies (401 program and 111 program) and an OI program (143) [3] - These upcoming programs are expected to transform the company [3] - The Angelman program is also a point of interest, receiving numerous inquiries [3]

Company Overview - 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] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Lead gene therapy candidates include OPGx-LCA5, currently in a 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] Corporate Update - George Magrath, MD, CEO, and Ash Jayagopal, Ph.D., MBA, Chief Scientific and Development Officer, will present a corporate update at the H.C. Wainwright 27th Annual Global Investment Conference from September 8-10, 2025 [1] - The presentation will be available on demand starting September 8, 2025, at 7:00 a.m. ET [1] Investor Relations - Recent investor event replays are accessible on the Opus Genetics website under the Investors section [2]

Core Points - A securities class action lawsuit has been filed against Rocket Pharmaceuticals, Inc. for alleged material misstatements and omissions affecting investors [1][3] - The lawsuit pertains to the period from September 17, 2024, to May 26, 2025, during which investors may seek compensation [2] - The lawsuit claims that Rocket Pharmaceuticals failed to disclose significant safety-related updates regarding its RP-A501 clinical trial, leading to a fatal adverse event and subsequent FDA clinical hold [3] Company Overview - Rocket Pharmaceuticals is headquartered in Cranbury, New Jersey, and specializes in gene therapies for rare and life-threatening diseases [2] - Following the public confirmation of the trial hold and patient death on May 28, 2025, Rocket Pharmaceuticals' stock price plummeted from $6.27 to $2.33 [3]

Core Viewpoint - A securities class action lawsuit has been filed against Rocket Pharmaceuticals, alleging material misstatements and omissions that led to investor losses [1][3]. Group 1: Lawsuit Details - The lawsuit is on behalf of investors who purchased Rocket Pharmaceuticals stock between September 17, 2024, and May 26, 2025 [2]. - The lawsuit claims that Rocket Pharmaceuticals failed to disclose significant safety-related updates regarding its RP-A501 clinical trial, including the use of an immunomodulatory agent, prior to a fatal adverse event and subsequent FDA-imposed clinical hold [3]. - Following the public confirmation of the trial hold and patient death on May 28, 2025, Rocket Pharmaceuticals' shares plummeted from $6.27 to $2.33 [3]. Group 2: Company Overview - Rocket Pharmaceuticals is headquartered in Cranbury, New Jersey, and specializes in gene therapies for rare and life-threatening diseases [2].

Core Insights - Eli Lilly (LLY) and Merck (MRK) are significant players in the U.S. pharmaceutical industry, focusing on oncology, immunology, diabetes, and cardiovascular therapies [1][2] - Lilly has a strong position in cardiometabolic health with GLP-1 drugs, while Merck excels in oncology with its PD-L1 inhibitor, Keytruda [1][2] Company Overview: Eli Lilly - Lilly's diabetes drug Mounjaro and weight loss medicine Zepbound have become key revenue drivers, accounting for approximately 50% of total revenues [3][10] - Despite slower-than-expected sales in the second half of 2024, Mounjaro and Zepbound's sales rebounded in Q1 2025 due to international market launches and improved production [4][5] - Lilly anticipates continued growth from Mounjaro and Zepbound, alongside new drug approvals and expanded uses of existing drugs [5][6] - The company is advancing its pipeline in obesity, diabetes, and cancer, with several mid- and late-stage data readouts expected [7] - Lilly is diversifying its portfolio through acquisitions, including Verve Therapeutics for heart disease gene therapies and oncology and pain management candidates [8] - However, Lilly faces challenges such as declining product prices in the U.S. and competition from Novo Nordisk's GLP-1 drugs [9][10] Company Overview: Merck - Merck's portfolio includes over six blockbuster drugs, with Keytruda being the primary revenue driver, particularly in early-stage non-small cell lung cancer [12] - The company has made significant regulatory and pipeline advancements, with its phase III pipeline nearly tripling since 2021 [13] - New products like Capvaxive and Winrevair are showing strong launches, and Merck is pursuing acquisitions to enhance its pipeline [14][15] - Despite these strengths, Merck is experiencing declining sales for Gardasil in China and challenges in its diabetes franchise [16] - Merck's heavy reliance on Keytruda raises concerns about its ability to diversify its product lineup ahead of the drug's patent expiration in 2028 [17] Financial Performance and Estimates - Lilly's 2025 sales and EPS estimates suggest a year-over-year increase of 33.0% and 68.4%, respectively, with a forecasted revenue range of $58.0 billion to $61.0 billion [19][32] - In contrast, Merck's 2025 sales and EPS estimates imply a modest year-over-year increase of 1.02% and 15.7%, with declining EPS estimates over the past 60 days [19][22] - Year-to-date, Lilly's stock has risen 2.7%, while Merck's stock has declined by 15.6% [23] Valuation and Dividend Yield - From a valuation perspective, Lilly's shares trade at a forward P/E ratio of 29.54, significantly higher than Merck's 8.87 [25] - Lilly's dividend yield is 0.76%, while Merck's is higher at 3.93% [29]