Precision BioSciences' proprietary gene-editing system ARCUS - using the naturally occurring homing endonuclease I-CreI - has been licensed to the American pharmaceutical company Lilly. The license includes a gene target for Duchenne Muscular Dystrophy and two other undisclosed targets as well as another three optional targets. Precision will receive $100 million cash upfront and an equity investment by Lilly of $35 million as well as potential milestone payments and royalties.
American geneticist George Church has launched 64x Bio, a new startup company. The startup will discover and design enhanced mammalian cell lines to increase the efficiency of viral vector production and reduce the cost of gene therapy manufacturing. The startup uses the VectorSelect platform that leverages leading-edge DNA library synthesis, next-generation sequencing, and a proprietary genetic barcoding method.
Another digital one-pot CRISPR/Cas assay for rapid and sensitive detection of SARS‐CoV-2 has been developed by researchers at the Johns Hopkins University, USA. The so-called deCOViD assay is realized through tuning and discretizing a one‐step, fluorescence‐based, CRISPR/Cas12a‐assisted reverse transcription recombinase polymerase amplification assay into sub‐nanoliter reaction wells within commercially available microfluidic digital chips.
Researchers from the University of Minnesota Medical School gives an update on emerging liver targeted gene therapies in a review in Drug Discovery Today: Technologies. Advances in CRISPR and similar technologies like base editing and prime editing are discussed as well as other forms of gene therapy.