Expansion Therapeutics Inc., a company developing new drugs for severe RNA-mediated diseases based on the work of its scientific founder, Scripps Research chemistry professor Matthew Disney, has raised $80 million in a series B financing to identify and advance oral small-molecule candidates for the potential treatment of myotonic dystrophy type 1, amyotrophic lateral sclerosis and tauopathies.
"RNA was long thought to be an 'undruggable' target for small molecules, because most cellular RNAs have extensive secondary structure, but only limited tertiary structure," Matthew Disney told BioWorld Science.
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.
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.
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
Avidity Biosciences Inc. CEO Sarah Boyce told BioWorld it's "a little too soon" to say when the company's lead program in myotonic dystrophy type I might reach the clinic, but the firm's $100 million series C financing will help with that effort and the portfolio in rare muscle diseases, as the antibody-oligonucleotide conjugate (AOC) platform undergoes further development.
