Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.

New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology’s annual meeting, Dec. 5-8, including: Actinium, Allogene, Allovir, Amgen, Amphivena, Apellis, Arch Oncology, Astrazeneca, Attralus, Autolus, Beigene, Bergenbio, Biocryst, Bioniz, Bluebird, BMS, Cellectis, Constellation, Corvus, Crispr, Fate, Fulcrum, Gamida, GBT, Genentech, Gilead, Glycomimetics, IGM, Immunogen, IMV, Intellia, Janssen, Kite, Kura, Lilly, Macrogenics, Magenta, Orca Bio, Poseida, Regeneron, Rhizen, Rocket, Syndax, Syros, Vertex, Viracta.

Cambridge, Mass.-based Agios Therapeutics Inc.’s encouraging phase III data from a pair of trials with allosteric activator mitapivat in pyruvate kinase deficiency brought more attention to the space, where Rocket Pharmaceuticals Inc. – at a much earlier stage – is trying a gene therapy called RP-L301.