CRISPR Therapeutics
The FDA approved three new gene therapy products in 2024 and expanded indications for two others, all for rare diseases.
Gene-Editing Company Exsilio Therapeutics Launches With $82M Series A Funding
The firm emerged from stealth mode to develop redosable genomic medicines by inserting entire genes using mRNA templates and lipid nanoparticles.
If Novartis and others show that lowering Lp(a) improves cardiovascular outcomes, it may spur a new class of drugs that change treatment and testing paradigms.
NICE Does Not Recommend Sickle Cell CRISPR Drug Casgevy in Draft Guidance, Calls for More Data
The agency said it wants to see additional data on effectiveness and duration of response and said Vertex should submit an updated managed access proposal.
CRISPR Therapeutics Prices $280M Registered Direct Offering
The firm will use these funds to advance current and new therapeutic candidates, invest in drug development, and bolster its manufacturing infrastructure.
Nov 1, 2023