Vertex and CRISPR Therapeutics Partner on Gene Therapy for Blood Disorders

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The companies will co-develop and co-commercialize the lead candidate generated from their earlier collaboration to treat genetic blood disorders.

On Dec. 12, 2017, Vertex Pharmaceuticals announced that it will co-develop and co-commercialize an investigational gene-editing treatment, known as CTX001, for blood disorders with CRISPR Therapeutics, a gene-editing company with a proprietary CRISPR/Cas9 gene-editing platform. The move is part of the companies' previously announced collaboration in October 2015 aimed at discovering and developing new gene-editing treatments that use the CRISPR/Cas9 technology.

The lead candidate is the first gene-based treatment that Vertex exclusively licensed from CRISPR Therapeutics as part of their collaboration. For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide. The companies filed a clinical trial application earlier in December 2017 to initiate a Phase I/II trial in β-thalassemia, a genetic blood disorder, in 2018 in Europe. The companies also plan to file an investigational new drug application in the US in 2018 to initiate a Phase I/II trial in sickle cell disease.

"Over the past two years, we've made significant progress with CRISPR Therapeutics on the discovery and preclinical development of multiple CRISPR/Cas9-based treatments, and we're pleased to select CTX001 as the first of these treatments to move into clinical development as part of our collaboration," said David Altshuler, MD, PhD, Vertex's executive vice president, global research and chief scientific officer, in a company press release. "The addition of CTX001 to our clinical development pipeline provides us with a near-term opportunity to generate the first proof-of-concept clinical data for a CRISPR/Cas9-based medicine in two genetic diseases that are highly aligned with our research strategy."


"The study of CTX001 in β-thalassemia will be the first company-sponsored clinical trial of a CRISPR-based therapy and is a major step forward for both the treatment of certain inherited blood diseases and for our collaboration with Vertex," said Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics, in the press release.

Source: Vertex Pharmaceuticals