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.
Scribe Therapeutics Inc. raised $100 million in a series B round to continue its engineering-intensive approach to developing CRISPR-based therapies that employ custom-designed CasX enzymes.
Berkeley, Calif.-based Caribou Biosciences Inc. has raised $115 million in an oversubscribed series C financing co-led by Farallon Capital Management, PFM Health Sciences and Ridgeback Capital Investments. Proceeds from the round will be used to advance its CRISPR technology platform and pipeline of off-the-shelf genome-edited CAR T and CAR-NK cell cancer therapies, including CB-010, its lead CAR T program, now in a phase I trial for patients with relapsed/refractory B-cell non-Hodgkin lymphoma.
Excision Biotherapeutics Inc.’s CEO is specific when he talks about his company’s therapies and what they may achieve: a functional cure. “When you treat someone and they become cancer free, you can’t use the world ‘cured’ because the cancer may come back decades later,” Daniel Dornbusch told BioWorld. “But you can talk about a functional cure, meaning the cancer didn’t come back for a very long time. It’s functionally cured for maybe 10, 20 or 30 years."
Transient gene editing of the herpes simplex virus type 1 using mRNA-based CRISPR was shown for the first time to be effective against herpetic stromal keratitis in mice and blocked HSV-1 replication in human corneas, Chinese researchers reported in the January 11, 2021, online edition of NatureBiotechnology.
It might be difficult to view the past year through anything other than a COVID-shaped hole. But 2020 brought some remarkable and impactful news for the biopharma sector that had little to do with the novel coronavirus. In this end-of-year recap, BioWorld takes a look at some of achievements and trends affecting the industry that were completely unrelated to – or, in some cases, in spite of – the COVID-19 pandemic.
Metagenomi Inc. has raised a $65 million series A financing to expand its gene editing abilities, advance its research and validate its pipeline in preclinical studies. The company’s CRISPR-based systems use algorithms for screening thousands of genomes from microorganisms to advance therapies for use in oncology, genetic diseases and possibly much more.
Perkinelmer Inc. is looking to bolster its life sciences offerings with the purchase of cell engineering company Horizon Discovery Group plc for $383 million. The all-cash acquisition will add gene-editing and gene-modulation tools to Perkinelmer’s existing portfolio of discovery and applied genomics solutions. Headquartered in Cambridge, U.K., Horizon provides CRISPR and RNA interference (RNAi) reagents, cell models, cell engineering and based editing products to aid in drug discovery and development.
Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.
Crispr Therapeutics AG's first stab at developing an allogeneic CAR T-cell therapy, CTX-110, looks promising, but the efficacy data were overshadowed by a death in the study.
