Core Insights - The FDA has granted Breakthrough Therapy designation to AMT-130 for Huntington's disease, highlighting the urgent need for effective treatments and the promising interim data indicating potential disease progression slowing [1][2] - The designation is supported by clinical data from ongoing Phase I/II trials, with interim data showing dose-dependent slowing of disease progression based on the cUHDRS after 24 months [2] Regulatory Updates - Additional regulatory updates and guidance on the Biologics License Application (BLA) submission for AMT-130 are expected in the second quarter of 2025 [1] - The Breakthrough Therapy designation allows for expedited development and review processes, including intensive guidance on drug development and senior management involvement from the FDA [3] Huntington's Disease Overview - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies to slow its progression [4] - The disease is characterized by motor symptoms, behavioral abnormalities, and cognitive decline due to a genetic mutation in the huntingtin gene [4] Company Background - uniQure is focused on advancing gene therapies, including AMT-130 for Huntington's disease, and has previously achieved significant milestones with its gene therapy for hemophilia B [5] - The company is developing a pipeline of gene therapies for various severe diseases, including ALS and Fabry disease [5]

Core Viewpoint - Precision BioSciences' shares surged 23.8% following the FDA's Fast Track designation for PBGENE-HBV, a gene editing program aimed at curing chronic hepatitis B by targeting cccDNA and integrated HBV DNA [1][2][3] Company Overview - PBGENE-HBV is the first gene editing program with a unique mechanism of action to enter clinical stages as a potential cure for chronic hepatitis B [3] - The company has received FDA clearance to initiate the phase I ELIMINATE-B study for PBGENE-HBV in the U.S., with simultaneous studies in other regions to enhance patient recruitment [4] - Currently, the company is enrolling patients in the low-dose cohort of the ELIMINATE-B study, with plans to escalate to higher doses in subsequent cohorts [5][7] Market Context - Chronic hepatitis B affects approximately 300 million people globally, with around 2 million in the U.S. facing significant health risks, including cirrhosis and liver cancer [8] - Existing antiviral treatments do not eliminate the virus, necessitating lifelong therapy without achieving a functional cure, highlighting the unmet medical need that PBGENE-HBV aims to address [9] Pipeline and Future Prospects - In addition to PBGENE-HBV, Precision BioSciences has several other gene therapy programs in preclinical evaluation, including a partnered candidate, ECUR-506, for treating neonatal onset ornithine transcarbamylase deficiency [10]

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 ...

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) 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 ...

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, 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 ...

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 ...

Imunon (IMNN) Q4 2024 Earnings Call February 27, 2025 11:00 AM ET Company Participants Peter Vozzo - Managing DirectorStacy Lindborg - President, CEO & DirectorDouglas Faller - Chief Medical OfficerDavid Gaiero - Chief Financial OfficerJoey Brusca - Equity Research AssociateKhursheed Anwer - Executive VP & Chief Scientific OfficerJason Kolbert - Managing Director Conference Call Participants David Bautz - Senior AnalystJames Molloy - Managing Director, Senior Biotechnology & Specialty Pharmaceuticals Equity ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q4 2024 Earnings Conference Call February 26, 2025 4:30 PM ET Company Participants Mary Jenkins - Associate Director, Investor Relations and Corporate Communications Doug Ingram - President and CEO Ian Estepan - Executive Vice President and CFO Dallan Murray - Executive Vice President and CCO Dr. Louise Rodino-Klapac - Executive Vice President, Chief Scientific Officer and Head, Research & Development Conference Call Participants Tazeen Ahmad - Bank of America Securi ...