Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101 Beam to Host Investor Webcast on Friday, June 13, 2025, at 4:00 p.m. ET CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present new data from the BEACON Phase 1/2 clinical trial of BE ...

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Advancements Lay the Groundwork for Future Genetic Medicines for Rare and Prevalent Forms of Heart DiseaseSOUTH SAN FRANCISCO, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it will present five abstracts at the upcoming American Society of Gene and Cell Therapy (ASGCT 2025) 28th Ann ...

Core Insights - The company EG 427 is at the forefront of developing non-replicating HSV-1 (nrHSV-1) vector technology for neurological applications, with data to be presented at the ASGCT meeting [1][4] - The innovative dual-payload nrHSV-1 vector allows for the delivery of multiple transgenes with distinct expression dynamics, providing a significant advantage over existing vector technologies [3][6] - The company has initiated a phase 1/2 clinical study of its lead candidate, EG110A, targeting neurogenic bladder-related incontinence, marking a significant milestone in its development pipeline [4][7] Vector Technology - The nrHSV-1 vector technology enables the expression of two different transgenes controlled by independent regulatory elements, allowing for varied expression durations [2][6] - In vivo studies demonstrated that the vector can achieve high transgene expression in specific neuronal populations, with stable expression levels observed for at least six weeks post-administration [2][6] - The HERMES platform utilized by the company allows for targeted biodistribution of therapeutic proteins within the brain, addressing a range of neurodegenerative diseases [8] Clinical Development - The first patient has been treated in a clinical study of EG110A, which aims to address multiple severe bladder diseases, potentially improving patient care and reducing healthcare costs [4][7] - The ongoing research supports the company's vision of developing genetic medicines that are safe, targeted, and cost-effective for chronic neurological diseases [4][6] Future Prospects - The advancements in nrHSV-1 vector technology are expected to facilitate pipeline growth by addressing challenges in genetic medicine for prevalent pathologies [6][8] - The ability to deliver gene editing proteins and corrected genes through a single vector could revolutionize treatment approaches for autosomal dominant diseases [3][6]

—Interim readout from Phase 1/2a REWRITE clinical trial of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD) on track for the second half of 2025 —Streamlining operations and focusing on delivering potential value generating program milestones, with workforce reduction of approximately 20% and cost reductions expected to extend cash runway into 2027 —Second development candidate expected to be announced in 2025 —Ended first quarter 2025 with $139.0 million in cash, cash equivalents and marketable secur ...

PITTSBURGH, May 07, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) today announced that the Company will participate in the BofA Securities 2025 Health Care Conference on May 14, 2025, in Las Vegas. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 11:20 am PT and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 11:20 am PT on Wednesday, May 14, 2025 and will be po ...

Core Insights - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on genetic diseases, participating in the Bank of America Merrill Lynch Global Healthcare Conference 2025 [1][3] - The company aims to discover, create, test, and deliver transformative medicines for patients with genetic diseases, with a pipeline that includes early science to advanced clinical trials [3] Company Overview - Founded in 2015, BridgeBio is committed to applying advances in genetic medicine to expedite patient care [3] - The company has a team of experienced professionals in drug discovery and development [3] Event Participation - Management will engage in a fireside chat at the conference on May 14, 2025, at 2:20 pm PT [1] - A live webcast of the presentation will be available on the company's website, with a replay accessible for 30 days post-event [2]

First Patient Dosed in the Phase 1/2 Study of BEAM-301 in Glycogen Storage Disease Type Ia, Beam’s Second Clinical Stage In Vivo Editing Program Updated Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Accepted for Presentation at the European Hematology Association 2025 Congress in June Following Positive Initial Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency, Company Initiated Dosing of Fourth Cohort in Part A of Phase 1/2 Trial and Received Clearance of U.S. IND; Updated Data Expected to be Pre ...

enGene Holdings (ENGN) 2025 Conference May 05, 2025 02:00 PM ET Speaker0 For our next presentation, it's from NGIN, and we have the pleasure of having Ron Cooper, the CEO, here to present for us. So take it away, Ron. Thank you. Speaker1 Well, good afternoon, everybody. It's a real pleasure to be here. You know, first of all, thanks to the conference organizers. It's great to be back in back in Canada. NGEN is a company form formed in Canada. Our headquarters is in Montreal. Our labs are there, and we have ...

CHARLESTOWN, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, will participate in a fireside chat at The Citizens Life Sciences Conference on Wednesday, May 7, at 12:30 pm ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by cli ...