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.
Surprising no one, Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Chemistry “for the development of a method for genome editing,” that is, the CRISPR/Cas9 system.
Surprising no one, Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Chemistry “for the development of a method for genome editing,” that is, the CRISPR/Cas9 system.
A whole-genome clustered regularly interspaced short palindromic repeats (CRISPR) screening study is the first to identify the gene regulating the inflammatory response and immune cell death during sepsis, Australian researchers reported in the October 5, 2020, edition of Nature Immunology.
Scribe Therapeutics Inc., another CRISPR-based genome editing firm out of the Jennifer Doudna stable, launched with $20 million in series A funding and a deal with Biogen Inc. in amyotrophic lateral sclerosis (ALS), which brings in another $15 million up front and up to $400 million in development and commercial milestones.
Researchers at the University of California at San Diego have used RNA-targeted CRISPR to reverse symptoms in an animal model of myotonic dystrophy type 1 (DM1). They reported their findings in the Sept. 14, 2020, issue of Nature Biomedical Engineering.
