The first Investigational New Drug (IND) application for base-editing technology has been cleared by the Food and Drug Administration. BEAM-101, developed by Beam Therapeutics, is an ex vivo base-editing product candidate, meaning that it uses a modified form of CRISPR capable of making single base changes without double-stranded DNA cleavage.
The treatment is described by the company as “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 to potentially alleviate the effects of mutations causing sickle cell disease or beta-thalassemia”.
Furthermore, Beam has initiated IND-enabling studies for a second candidate treatment called BEAM-102, aiming to directly correct the causative mutation in sickle cell disease by recreating a naturally-occurring normal human hemoglobin variant. This is further evidence that when we come to advanced therapies for SCD, the level of innovation and competition is high.
The list of developers is long and getting longer. Bluebird Bio, Aruvant Sciences, CRISPR Therapeutics & Vertex Pharmaceuticals, Editas Medicine, Intellia Therapeutics & Novartis, Sangamo Therapeutics & Sanofi), California Institute for Regenerative Medicine & UCLA, Boston Children’s Hospital, Graphite Bio & Stanford University, University of California (San Francisco, Berkeley & Los Angeles), and now Beam Therapeutics.
Don’t miss Nature Biotechnology’s article on current clinical trials for SCD to learn more.