Vertex develops small-molecule and genetic medicines for serious diseases and is best known for its cystic fibrosis drugs. It co-developed Casgevy, the…
CRISPR Therapeutics is a gene-editing company that co-developed Casgevy with Vertex. Its clinical pipeline includes in vivo editing programs for cardiovascular targets…
Retro Biosciences aims to extend healthy human lifespan through programs in autophagy, cell replacement and tissue reprogramming. Its lead oral drug, RTR242,…
Genome + CellsIn December 2023 the FDA approved Casgevy, the first CRISPR-based therapy, for sickle cell disease. Here is how gene editing, gene therapy and RNA medicines differ, and what that approval does and doesn't prove.