Beam Therapeutics Inc. (BEAM) Reports Updated Data From BEACON Phase 1/2 trial Of Ristoglogene

Beam Therapeutics Inc. (NASDAQ:BEAM) is among the 12 Best Genomics Stocks to Invest In.

TheFly reported on December 2, 2025, that Beam Therapeutics Inc. (NASDAQ:BEAM) has released revised safety and effectiveness information from its BEACON Phase 1/2 clinical trial evaluating ristoglogene autogetemcel (previously BEAM-101). It is an investigational genetically engineered ex vivo base editing cell therapy for sickle cell disease patients suffering from severe vaso-occlusive crises. The information was shown during the 67th Annual Meeting and Exposition of the American Society of Hematology in Orlando.

A total of 31 adults and adolescents were treated with risto-cel and were included in the updated results. The company reported a decline of hemoglobin S to less than 40%, a remission of anemia that lasted up to 20 months, and a mean fetal hemoglobin induction of more than 60%. A median of one cell collection cycle was needed for each patient. After treatment, rapid neutrophil and platelet engraftment were seen. According to the firm, busulfan conditioning, autologous hematopoietic stem cell transplantation, and preexisting sickle cell disease did not affect the safety profile.

Separately, Beam Therapeutics Inc. (NASDAQ:BEAM) was covered by Evercore ISI on November 24, 2025, with a $35 price target and an Outperform rating. BEAM-302 was mentioned by the analyst as a possible treatment for alpha-1 antitrypsin insufficiency. Levels of adjusted alpha-1 antitrypsin production were seen in early data. The analyst noted that new data anticipated in the first quarter of 2026 will be critical.

Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology firm focused on developing a new class of precision genetic therapies based on its own base editing technique to give patients with severe illnesses permanent solutions.

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