Gene Therapy

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PRISM MarketView Spotlights Opus Genetics in New Q&A with CEO George Magrath
Newsfilter· 2025-04-15 14:36
NEW YORK, April 15, 2025 (GLOBE NEWSWIRE) -- PRISM MarketView today published a Q&A feature with Dr. George Magrath, Chief Executive Officer of Opus Genetics, Inc. ("Opus Genetics" or the "Company") (NASDAQ:IRD), a clinical-stage gene therapy company developing treatments for inherited retinal diseases (IRDs) and broader ophthalmic conditions. Dr. George Magrath, Chief Executive Officer of Opus Genetics, Inc. In the interview, Dr. Magrath outlines Opus Genetics' recent milestones, including: Completion of ...
Solid Biosciences: A Strong Buy As An Emerging Biotech In The Gene Therapy Space
Seeking Alpha· 2025-04-08 20:50
Group 1 - Solid Biosciences Inc. (NASDAQ: SLDB) is potentially repositioning itself in a sector that has recently faced significant scrutiny [1] - The company's lead candidate is still in early-stage trials but has demonstrated encouraging results [1]
BioLineRx(BLRX) - 2024 Q4 - Earnings Call Transcript
2025-03-31 14:52
BioLineRx Ltd. (NASDAQ:BLRX) Q4 2024 Earnings Conference Call March 31, 2025 8:30 AM ET Company Participants Irina Koffler - IR, LifeSci Advisors Phil Serlin - CEO Mali Zeevi - CFO Ella Sorani - Chief Development Officer Conference Call Participants Joe Pantginis - HC Wainwright John Vandermosten - Zacks Operator Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Fourth Quarter and Full Year 2024 Financial Results Conference Call. All participants are present in listen-only mode. Foll ...
Opus Genetics Announces Financial Results for Full Year 2024
Newsfilter· 2025-03-31 12:20
Provides update on the Company's transformation and promising portfolio of innovative gene therapy treatments for inherited retinal diseases Strong cash position with $21.5 million financing to supplement $30.3 million year-end balance New capital supports delivery on key milestones for two lead gene therapy candidates OPGx-LCA5 and OPGx- BEST1 RESEARCH TRIANGLE PARK, N.C., March 31, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. ("Opus" or the "Company") (NASDAQ:IRD), a clinical-stage ophthalmic biopharmaceu ...
Abeona Therapeutics(ABEO) - 2024 Q4 - Earnings Call Transcript
2025-03-20 11:30
Abeona Therapeutics (ABEO) Q4 2024 Earnings Call March 20, 2025 07:30 AM ET Company Participants Greg Gin - Vice President, Investor Relations and Corporate CommunicationsVishwas Seshadri - Chief Executive OfficerMadhav Vasanthavada - Senior VP, Chief Commercial Officer & Head of Business DevelopmentBrian Kevany - Senior VP, CTO & CSOJoseph Vazzano - Chief Financial OfficerRaghuram Selvaraju - Managing Director, Healthcare Equity Research Conference Call Participants Kristen Kluska - Equity Research Analyst ...
Solid Biosciences to Present at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Newsfilter· 2025-03-12 12:00
Core Insights - Solid Biosciences Inc. is set to present data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a gene therapy for Duchenne muscular dystrophy, at the 2025 MDA Clinical & Scientific Conference [1] - Duchenne muscular dystrophy is a severe genetic disease affecting approximately 5,000 to 15,000 cases in the U.S., primarily in boys [2] - SGT-003 utilizes a differentiated microdystrophin construct and a next-generation capsid designed to enhance muscle transduction while minimizing liver targeting, indicating its potential as a leading gene therapy for Duchenne [3] - The INSPIRE DUCHENNE trial is a first-in-human, open-label study aimed at evaluating the safety and efficacy of SGT-003 in pediatric patients with confirmed Duchenne diagnosis [4] - Solid Biosciences focuses on developing gene therapies for rare neuromuscular and cardiac diseases, with a mission to improve the lives of patients affected by these conditions [5]
Solid Biosciences to Present at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Globenewswire· 2025-03-12 12:00
CHARLESTOWN, Mass., March 12, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March ...
Voyager Therapeutics(VYGR) - 2024 Q4 - Earnings Call Transcript
2025-03-11 23:35
Voyager Therapeutics, Inc. (NASDAQ:VYGR) Q4 2024 Earnings Conference Call March 11, 2025 4:30 PM ET Company Participants Trista Morrison - Chief Corporate Affairs Officer and Chief of Staff to the CEO Alfred Sandrock - President and Chief Executive Officer Toby Ferguson - Chief Medical Officer Todd Carter - Chief Scientific Officer Nathan Jorgensen - Chief Financial Officer Conference Call Participants Jack Allen - Robert W. Baird Phil Nadeau - TD Cowen Pete Stavropoulos - Cantor Fitzgerald Lili Nsongo - Le ...
Medera Completes Patient Dosing in Phase 1/2a MUSIC-HFrEF Trial of SRD-001 Gene Therapy for Heart Failure with Reduced Ejection Fraction
Newsfilter· 2025-03-11 13:09
Company Overview - Medera Inc. is a clinical-stage biopharmaceutical company focused on developing next-generation therapeutics for difficult-to-treat and currently incurable diseases [8] - The company operates through two business units: Novoheart and Sardocor, which are involved in preclinical and clinical development respectively [8][10] Clinical Trial Update - Medera has completed patient dosing in its MUSIC-HFrEF Phase 1/2a clinical trial for the gene therapy candidate SRD-001, targeting heart failure with reduced ejection fraction (HFrEF) [1][4] - The trial is an open-label, uncontrolled study with two cohorts: Cohort A (low-dose, 6 patients) and Cohort B (high-dose, 3 patients) [3] - Preliminary data presented at the 7th Annual Meeting of the American Society of Gene and Cell Therapy indicated an acceptable safety profile and clinically meaningful improvements in heart function metrics at six and twelve months post-infusion [3][4] Disease Context - HFrEF is a prevalent form of heart disease, accounting for half of the estimated 64.3 million heart failure cases globally, and is currently considered an incurable disease [2][7] - The condition is characterized by progressive and irreversible impairment of cardiac function, leading to debilitating symptoms and high mortality rates, highlighting the urgent need for disease-modifying therapies like SRD-001 [7] Future Prospects - The completion of enrollment in the Phase 1/2a trial is seen as a crucial milestone for the development of cardiac gene therapy for patients with advanced heart failure [4] - Medera's CEO expressed optimism regarding the results, indicating that optimized dosages are making a difference in patient outcomes [5] Additional Information - SRD-001 is an investigational gene therapy using an adeno-associated virus vector to express the SERCA2a transgene in patients with HFrEF [6] - Sardocor, a unit of Medera, is focused on the clinical development of novel therapies and has received FDA IND clearances for multiple ongoing clinical trials targeting various forms of heart failure and related conditions [11]
Imunon(IMNN) - 2024 Q4 - Earnings Call Transcript
2025-02-27 19:32
Imunon, Inc. (NASDAQ:IMNN) Q4 2024 Results Conference Call February 26, 2025 11:00 AM ET Company Participants Peter Vozzo - ICR Healthcare, IR Stacy Lindborg - President & Chief Executive Officer Douglas Faller - Chief Medical Officer David Gaiero - Interim Chief Financial Officer Michael Tardugno - Executive Chairman Khursheed Anwer - Chief Scientific Officer Conference Call Participants David Bautz - Zacks Small-Cap Research James Molloy - Alliance Global Partners Jason Kolbert - D. Boral Capital Kemp Dol ...