$900 million to help develop CRISPR gene editing therapy Vertex renews cooperation

Today, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the two companies have renewed their cooperation agreement for the joint development of the treatment CTX001 under investigation.

CRISPR-Cas9 gene editing technology uses artificially designed guide RNA (guide RNA) to identify the target genome sequence, guide Cas9 protease to effectively cut DNA double strands, and modify genes through repair or recombination after DNA double strand breaks.

CTX001 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of TDT and SCD by the US FDA, as well as fast track and orphan drug designation.

According to the revised terms of the agreement, Vertex will lead the global development, production and promotion of CTX001 with the support of CRISPR Therapeutics to maximize the potential of CTX001 to change the lives of tens of thousands of patients.

Reference materials:

[1] Vertex Pharmaceuticals and CRISPR Therapeutics Amend Collaboration for Development, Manufacturing and Commercialization of CTX001™ in Sickle Cell Disease and Beta Thalassemia.

Note: This article aims to introduce the progress of medical and health research, not to recommend treatment options.