Beam Therapeutics Inc. Stock Price, News & Analysis

Beam Therapeutics (Nasdaq: BEAM) reported updated BEACON Phase 1/2 data for ristoglogene autogetemcel (risto-cel) in 31 adults and adolescents with severe sickle cell disease (data cut-off Aug 6, 2025; follow-up 0.3–20.4 months).

Key results: mean HbF >60%, mean HbS <40%, durable editing (peripheral editing 67.4% at Month 6; 72.8% at Month 12), median 1 stem cell collection cycle (median 3 total collection days), median neutrophil engraftment 17.5 days and platelet engraftment 19 days, and no investigator-reported severe VOCs post-engraftment. One patient died 4 months post-infusion; investigator attributed death likely to busulfan conditioning.

Beam Therapeutics (Nasdaq: BEAM) announced that company management will participate in a fireside chat at the 2025 Jefferies London Healthcare Conference on Wednesday, November 19, 2025 at 3:30 p.m. GMT in London.

The event will be available via a live webcast in the company's investor section at www.beamtx.com and the webcast will be archived for 60 days following the presentation.

Beam Therapeutics (Nasdaq: BEAM) will present updated safety and efficacy data from the BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease at the 67th American Society of Hematology Annual Meeting, December 6-9, 2025 in Orlando.

Two presentations: a poster on Dec 6, 2025, 5:30–7:30 p.m. ET (Abstract 2532) reporting robust fetal hemoglobin induction and improvements in anemia and hemolysis; and an oral presentation on Dec 8, 2025, 5:30–5:45 p.m. ET (Abstract 1049) on CD34+ mobilization, collection, and safety comparing fixed-dose versus weight-based plerixafor dosing. Presenters: Ashish Gupta, M.D., MPH, and Haydar Frangoul, M.D.

Beam Therapeutics (Nasdaq: BEAM), a biotechnology company focused on precision genetic medicines through base editing, has announced its participation in three major investor conferences in September 2025.

The company's management will engage in fireside chats at Citi's 2025 Biopharma Back to School Conference (September 3), the 2025 Wells Fargo Healthcare Conference (September 4), and the H.C. Wainwright 27th Annual Global Investment Conference (September 8). All presentations will be available via webcast on the company's investor relations website for 60 days following each event.

Beam Therapeutics (NASDAQ:BEAM) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, its investigational cell therapy for sickle cell disease. The designation follows the previously granted orphan drug status and provides opportunities for accelerated development and enhanced FDA collaboration.

The company's BEACON Phase 1/2 trial has now treated 30 patients, with updated data from 17 patients showing promising results including increased fetal hemoglobin, reduced sickle hemoglobin, and improved markers of hemolysis and oxygen delivery. The treatment demonstrated rapid neutrophil and platelet engraftment, with no VOCs reported post-engraftment. Additional trial data is expected by the end of 2025.

Beam Therapeutics (NASDAQ:BEAM) reported significant progress in its Q2 2025 clinical programs and financial results. The company's lead candidate BEAM-302 for alpha-1 antitrypsin deficiency (AATD) has shown promising results with 17 patients dosed across four cohorts, demonstrating durable correction of disease-causing mutations and restored AAT physiology. All doses tested were well-tolerated with no serious adverse events.

In their hematology franchise, Beam completed dosing of 30 patients in the BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease, including the first adolescent patient. The company ended Q2 2025 with a strong financial position of $1.2 billion in cash and equivalents, extending their runway into 2028. Q2 financial results showed an R&D expense of $101.8 million and a net loss of $102.3 million ($1.00 per share).

Beam Therapeutics (NASDAQ: BEAM) reported promising new data from its BEACON Phase 1/2 clinical trial of BEAM-101, a base-editing therapy for severe sickle cell disease. The updated results from 17 patients showed consistent efficacy with all patients achieving hemoglobin F levels over 60% and hemoglobin S reduction below 40%. Patients required only one median mobilization cycle and demonstrated rapid cell engraftment. The therapy showed durable responses up to 15 months with normalized hemolysis markers and no vaso-occlusive crises post-engraftment. The safety profile aligned with standard busulfan conditioning and stem cell transplantation. Enrollment is complete in both adult and adolescent cohorts, with 26 patients dosed and a target of 30 patients by mid-2025. Additional data is expected by year-end 2025.

Beam Therapeutics (NASDAQ: BEAM) has received FDA orphan drug designation for BEAM-101, its investigational cell therapy for sickle cell disease (SCD). The designation provides benefits including tax credits, user fee exemptions, and potential 7-year market exclusivity post-approval. Clinical data from seven patients in the BEACON Phase 1/2 trial showed promising results, with robust increases in fetal hemoglobin, reduced sickle hemoglobin, and improved markers of hemolysis. The therapy demonstrated consistent manufacturing success at Beam's North Carolina facility. The company plans to treat 30 patients in the ongoing BEACON trial by mid-2025. SCD affects approximately 100,000 people in the U.S., causing severe complications including anemia, pain, stroke, and early death.

The FDA has granted orphan drug designation to Beam Therapeutics' (NASDAQ: BEAM) BEAM-302 for treating Alpha-1 Antitrypsin Deficiency (AATD). BEAM-302 is a liver-targeting treatment designed to correct the disease-causing mutation in AATD patients. This follows the recent RMAT designation received in May 2025.

Initial Phase 1/2 trial results showed positive safety and efficacy data, with single doses leading to durable, dose-dependent mutation correction. The 60 mg dose cohort achieved AAT protein levels above therapeutic threshold. The company has begun dosing the fourth cohort at 75 mg and plans to start Part B trials with AATD patients with mild to moderate liver disease in H2 2025.

The orphan drug designation provides benefits including tax credits for clinical trials, user fee exemptions, and potential seven-year market exclusivity post-approval.