Vertex, CRISPR Therapeutics To Co-develop And Co-commercialize CTX001

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) and Switzerland-based CRISPR Therapeutics AG (CRSP) said that the companies will co-develop and co-commercialize CTX001, an investigational gene editing treatment.

The move is part of the companies' previously announced collaboration aimed at the discovery and development of new gene editing treatments that use the CRISPR/Cas9 technology.

CTX001 represents the first gene-based treatment that Vertex has exclusively licensed from CRISPR Therapeutics as part of the collaboration. For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

According to the companies, a Clinical Trial Application was submitted earlier this month for CTX001 to support the initiation of a Phase 1/2 trial in ß-thalassemia in 2018 in Europe.

In addition, the companies plan to submit an Investigational New Drug or IND Application for submission in 2018 to support the initiation of a Phase 1/2 trial in sickle cell disease in the U.S.

The companies noted that preclinical data presented for CTX001 at the American Society for Hematology on December 10, 2017 showed clinically relevant increases in fetal hemoglobin and a high editing rate that support the advancement of CTX001 into the planned trials in ß-thalassemia and sickle cell disease in 2018.