Core Insights - Beam Therapeutics Inc. announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 for treating sickle cell disease (SCD), demonstrating strong clinical outcomes and a consistent safety profile [2][3][4] Efficacy and Safety Data - All patients treated with BEAM-101 achieved hemoglobin F (HbF) induction levels exceeding 60% and a reduction in sickle hemoglobin (HbS) to below 40%, with no vaso-occlusive crises reported post-engraftment [3][5][11] - Patients required a median of one mobilization cycle for treatment, with rapid neutrophil and platelet engraftment observed, taking a median of 16.5 days and 19.5 days respectively [11] - Key markers of hemolysis normalized or improved in all patients, indicating significant enhancement in oxygen delivery to tissues [11] Clinical Trial Progress - Enrollment in both adult and adolescent cohorts of the BEACON trial is complete, with 26 patients dosed as of June 13, 2025, and an expectation to dose 30 patients by mid-2025 [7][8] - The follow-up period for treated patients ranged from 0.2 to 15.1 months, with durable responses observed in HbF and HbS levels [4][11] Manufacturing and Process - BEAM-101 is manufactured using an advanced, largely automated process that has shown high yields and viability, facilitating successful manufacturing for all patients in the trial [3][11] - The safety profile of BEAM-101 aligns with that of busulfan conditioning and autologous hematopoietic stem cell transplantation, with common treatment-emergent adverse events being consistent with busulfan [11] Company Vision and Future Plans - Beam Therapeutics aims to deliver life-long cures through its precision genetic medicines platform, focusing on base editing technology [12] - The company is committed to advancing its BEAM-101 program and plans to share additional data from the trial by the end of 2025 [4][7]

Solid Biosciences (SLDB) 2025 Conference June 04, 2025 02:00 PM ET Speaker0 Everyone. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the SolidBio team. We've got Bo Kumbow and Gabe Brooks joining us today. And we're going to do fireside chat format. So, for those who are new to the story, maybe give a brief intro to Solid, your key programs and platform. Speaker1 Yeah, thank you, Mari. I appreciate the invitation. Solid Bioscienc ...

Solid Biosciences Investor Contact: CHARLESTOWN, Mass., May 28, 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, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, at 2:00 pm ET. A live webcast of the fireside chat will b ...

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 ...

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 ...

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 ...

1. Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy Session Title: Late-Breaking Abstracts II Date/Time: May 17, 9:15 – 9:30am CT Location/Room: Room 391-392 Presenter: Kevin Flanigan, MD, Advisor Consultant & Researcher, Nationwide Children's Hospital and investigator in the INSPIRE DUCHENNE clinical trial 2. Title: Insight into the Mechanism of Action of AAV-SLB101, a Novel Muscle-Tropic Capsid for Neuromuscular an ...