BEAM-101 IND cleared by FDA for evaluation as a treatment for Sickle Cell Disease

Beam Therapeutics Inc. (Beam), announced recent business and pipeline updates, as well as third quarter 2021 financial results, on 8 of November. Beam announced that its Investigational New Drug (IND) application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration (FDA). BEAM-101, the company’s lead ex vivo base editing product candidate, is a patient-specific, autologous hematopoietic investigational cell therapy which incorporates base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin (HPFH) to potentially alleviate the effects of mutations causing sickle cell disease or beta-thalassemia. This is the first open IND for base editing technology, a next-generation form of CRISPR capable of making single base changes without creating double strand breaks in the DNA. Beam is preparing to initiate a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which Beam refers to as the BEACON-101 trial. More info: Beam Therapeutics, press release.