Beam Therapeutics Reports Promising Results for BEAM-103 in Sickle Cell Disease

Beam Therapeutics has announced promising results from its BEAM-103 treatment, showing rapid and sustained reactivation of fetal hemoglobin (HbF) in sickle cell disease (SCD) patients post-transplant. The treatment was well tolerated, with no need for transfusions, antibiotics, or additional supportive care. In non-human primates (NHPs), BEAM-103 treatment resulted in only minor declines in neutrophil counts and platelet levels, contrasting with the more severe effects seen with traditional busulfan conditioning. The CD117 base-editing showed normal receptor function both in vitro and in vivo, with no changes to CD117 signaling, structure, or expression observed. Post-transplantation, normal hematopoietic reconstitution was seen in NHP studies.

Beam Therapeutics will host a live and webcast investor event at 8:00 p.m. PT on December 8, 2024, in San Diego to review key presentations from the ASH meeting. The event will be accessible via the "Events & Presentations" section of Beam's website, and an archived webcast will be available approximately two hours after the event.

Beam Therapeutics, listed on NASDAQ as BEAM, is a biotechnology company focused on precision genetic medicines, utilizing base editing technology to enable precise and efficient single base changes at targeted genomic sequences without causing double-stranded DNA breaks. This technology supports a range of therapeutic strategies aimed at providing life-long cures for serious diseases.