Beam Therapeutics Inc.

Beam Therapeutics Reports Initial Clinical Data from Phase 1/2 Trial of BEAM-301, Demonstrating Proof of Concept for Genetic Correction in Glycogen Storage Disease Type Ia (GSDIa) at ESGCT 33rd Annual Congress

Beam Therapeutics reported initial Phase 1/2 data for BEAM-301 in glycogen storage disease type Ia, showing proof of concept for genetic correction of the R83C mutation via a single infusion. In the 40 mg cohort, two of three patients achieved 15 hours of controlled fasting, improved glucose control, and no longer required daily starch supplementation; median time to fasting hypoglycemia rose from 2.5 to 15.0 hours at Day 36. Safety was consistent with LNP-based therapies, with no dose-limiting toxicities or Grade 4/5 events, though four of six patients had serious treatment-related TEAEs including three Grade 3 infusion-related reactions; premedication and infusion regimens were modified. Beam is advancing to a 60 mg dose cohort and plans to engage the FDA on a potential path to approval.

•5d ago

Beam Therapeutics to Present Initial Data from Phase 1/2 Trial of BEAM-301 in Glycogen Storage Disease Type Ia (GSDIa) at the ESGCT 33rd Annual Congress

Beam Therapeutics announced initial Phase 1/2 data for BEAM-301 in glycogen storage disease type Ia, achieving clinical proof of concept for base-editing correction of the R83C mutation. Both evaluable 40 mg patients achieved the protocol-defined maximum of ≥15 hours of controlled fasting, consistent with restored endogenous glucose production, and starch intake fell 71-92% in that cohort. Safety was consistent with LNP-based therapies, though three Grade 3 infusion-related reactions occurred; the steroid premedication regimen has since been optimized and no dose-limiting toxicities or deaths were reported. Updated data will be presented at ESGCT on October 28, with an investor webcast on October 27 at 8:00 a.m. ET.

•19d ago

Beam Therapeutics Presents Updated Clinical Data from the Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the European Respiratory Society (ERS) Congress 2026

Beam Therapeutics presented updated Phase 1/2 data for BEAM-302, its lead liver-targeted base-editing program for alpha-1 antitrypsin deficiency, in a late-breaking oral session at the ERS Congress 2026. A single 60 mg dose lifted steady-state total AAT to a mean of 14.4 µM in Part A versus 5.0 µM at baseline, above the 11 µM protective threshold, alongside an 84% reduction in circulating mutant Z-AAT and 93% corrected M-AAT at steady state, with durability out to 18 months of follow-up. Safety remained consistent with LNP-based therapies, with mild-to-moderate infusion-related reactions in 41% of patients. Based on FDA feedback, Beam is pursuing accelerated approval on a 12-month AAT biomarker endpoint at 60 mg and has dosed the first patient in a global pivotal cohort expected to enroll roughly 50 additional patients.

•1mo ago

Beam Therapeutics Appoints Eric Foster as Chief Commercial Officer

Beam Therapeutics appointed Eric Foster as chief commercial officer to lead the company's commercial organization and strategy. Foster brings over 25 years of experience, including roles at Amgen, Horizon Therapeutics, and Ardelyx, where he managed franchises generating $2.75 billion in net sales. The hire positions Beam for the potential commercial launch of risto-cel in 2027.

•2mo ago

Beam Therapeutics Reports Second Quarter 2026 Financial Results and Announces First Patient Dosed in Global Pivotal Cohort of BEAM-302 Trial in Alpha-1 Antitrypsin Deficiency (AATD)

Beam Therapeutics announced the first patient was dosed in the global pivotal cohort of its Phase 1/2 BEAM-302 trial for Alpha-1 Antitrypsin Deficiency (AATD), marking a significant milestone for its lead genetic disease program. The company completed dosing in all adult and adolescent patients for the Phase 1/2 BEACON trial of risto-cel in Sickle Cell Disease, with a U.S. Biologics License Application (BLA) submission expected as early as year-end 2026, and received FDA clearance for the IND application of BEAM-304 in Phenylketonuria (PKU). Financially, Beam ended Q2 2026 with $1.2 billion in cash and expects its current resources, plus a $200 million draw from its Sixth Street facility, to support operations into mid-2029, covering the anticipated launch of risto-cel and the pivotal development of BEAM-302.

•2mo ago

Beam Therapeutics to Present Updated Data from Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) in Late-Breaking Oral Presentation at the European Respiratory Society (ERS) Congress 2026

Beam Therapeutics announced it will present updated Phase 1/2 clinical data for BEAM-302, its lead genetic disease program for Alpha-1 Antitrypsin Deficiency, at the ERS Congress in September. The company describes the upcoming dataset as robust, reinforcing the therapy's potential to address both lung and liver disease by correcting the underlying PiZ mutation. An investor webcast and conference call are scheduled for September 8, 2026, to review the updates.

•3mo ago

Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration

Beam Therapeutics received FDA clearance of its IND application for BEAM-304, an in vivo base editing therapy designed to treat phenylketonuria (PKU). The upcoming Phase 1/2 trial will initially evaluate safety and Phe reduction in patients with the R408W mutation, utilizing a platform-based approach to address multiple mutations within a single program. Preclinical data showed normalized plasma Phe levels in mouse models at clinically relevant doses, with updated data to be presented at the FASEB conference in July.

•4mo ago

Beam Therapeutics Presents Recently Reported Topline Clinical Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the American Thoracic Society (ATS) 2026 International Conference

Beam Therapeutics presented topline clinical data for BEAM-302 at the American Thoracic Society 2026 International Conference, highlighting durable restoration of normal AAT function in patients with Alpha-1 Antitrypsin Deficiency. The data, covering 29 patients treated in the Phase 1/2 trial, demonstrated that a single dose can correct the PiZ mutation at the DNA level, addressing both lung and liver manifestations of the disease. Beam intends to pursue an accelerated approval pathway with the FDA and plans to initiate a pivotal cohort of approximately 50 additional patients in the second half of 2026 to support a future BLA submission.

•5mo ago

Beam Therapeutics Supports AlphaDetect to Accelerate Detection of Alpha-1

Beam Therapeutics has announced its support for AlphaDetect, a nonprofit organization dedicated to accelerating the detection of Alpha-1 Antitrypsin Deficiency (Alpha-1) in patients with liver and/or lung disease. The sponsorship will help expand proven strategies for routine targeted detection of Alpha-1, consistent with clinical practice guidelines, and aims to identify more individuals earlier. Alpha-1 remains significantly underdiagnosed, with over 90% of affected individuals estimated to be unidentified. Detection can inform timely decisions and provide access to comprehensive information and support.

•5mo ago

Beam Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Updates

Beam Therapeutics reported Q1 2026 revenue of $31.7 million, up significantly from the prior year, while narrowing its net loss to $0.91 per share. The company ended the quarter with $1.2 billion in cash, which is expected to fund operations into mid-2029. Pipeline progress included the publication of Risto-cel data in the New England Journal of Medicine and a reaffirmed target to submit a BLA for the sickle cell disease treatment by year-end 2026. The company also selected 60 mg as the optimal biological dose for BEAM-302 in alpha-1 antitrypsin deficiency and plans to initiate a pivotal cohort in the second half of the year. Additional milestones anticipated in 2026 include an IND filing for BEAM-304 in PKU and initial clinical data for BEAM-301 in GSDIa.

•5mo ago