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.
A team at the Broad Institute of Harvard and MIT has developed a genome editing method that could, in principle, correct 90% of the roughly 75,000 currently known genomic changes that are associated with genetic diseases.
A team at the Broad Institute of Harvard and MIT has developed a genome editing method that could, in principle, correct 90% of the roughly 75,000 currently known genomic changes that are associated with genetic diseases.
The CRISPR genome editing technique has been used to activate a close relative of the gene that is mutated in muscular dystrophy type 1A, preventing development of the disease in mouse-model neonates and reversing symptoms in mice with established pathology.