- Vertex is partnering with CRISPR Therapeutics in a four-year pact to develop therapies for cystic fibrosis (CF) and sickle cell anemia. Vertex is investing $105 million upfront, with a potential investment over the four years of $2.6 billion, including milestone payments.
- Vertex changed the treatment landscape for CF in December 2014 when it introduced Kalydeco (ivacaftor), and in 2015, Orkambi (lumacaftor). Kalyeco addresses the following gene deficiencies: G551D, G1244E, G1349D, G178R, G551S, G970R, S1251N, S1255P, S549N, and S549R.
- CRISPR Therapeutics was founded based on technology developed by an award-winning scientists, Emanmanuelle Charpentier.
Vertex and CRISPR are both part of the Cambridge, MA-area biotech community, with long-time ties between the CEOs. As the company with the leading CF therapy, Kalydeco has a certain cache in this arena, despite the fact that a lot of the attention has been focused on the $259,000 cost of its therapy. Despite the cost, Kalydeco's ability to target mutations and go after them during R&D has been vetted and tested. Now the goal is to use that methodology, along with CRISPR's gene-editing technique to go after more CF targets, as well as sickle cell anemia targets.
All told, CF affects 30,000 people in the U.S. and 70,000 people globally, while sickle cell anemia affects 30,000 people in the U.S.—including 1,000 babies born each year with the disease. The medical needs are acute, and in this case it seems that the scientific prowess of the team is prepared to advance the standard of care in both cases.