US-based biotech Vertex Pharmaceuticals, in partnership with Crispr Therapeutics, has started recruiting patients for the Phase I/II clinical trial with a treatment developed by the same Crispr Therapeutics–CTX001–for the treatment of beta-thalassemia. The FDA trial will be conducted in Regensburg Hospital (Germany) and will enroll 12 patients. This is the first clinical trial that assesses a treatment using Crispr gene technology.
CTX001 is an ex-vivo therapy: cells are drawn from each individual patient’s blood, reprogrammed and reintroduced into the organism. Blood cells treated through Crispr technology will have a DNA correction enabling them to produce only healthy cells.
Crispr THX has anticipated biotech Editas Medicine, which is almost ready to test its own Crispr therapy on patients with a rare form of blindness.