Mateusz Brzeziński

The FDA approved Casgevy, the first CRISPR-based therapy for sickle cell disease, in December 2023 after similar approval in the UK. In 2020, Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize for their CRISPR work. Dozens of clinical trials are underway worldwide. The 2023 Human Genome Editing Summit reaffirmed the ban on heritable genome editing.

In December 2023, the FDA approved Casgevy as the first CRISPR-based gene therapy for sickle cell disease and beta-thalassemia, with 29 of 31 patients free of pain crises after one year. Other recent approvals include Luxturna for congenital blindness, Zolgensma for SMA in infants, Hemgenix and Roctavian for hemophilia, and Vyjuvek for epidermolysis bullosa. FDA counted about 10 approved gene therapies in early 2024.