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14 December 2017Americas

Vertex and CRISPR Therapeutics to develop gene-editing treatment

US-based Vertex Pharmaceuticals and CRISPR Therapeutics have revealed plans to jointly develop and commercialise an investigational gene-editing treatment.

The announcement stems from a research partnership between the two companies focused on discovering and developing new treatments for genetic causes of human diseases through the use of CRISPR/Cas9 technology.

In October 2015, LSIPR reported on the collaboration, a four-year project initially designed to focus on mutations and genes which are known to contribute and cause cystic fibrosis and sickle cell disease.

The treatment, CTX001, is the first gene-based treatment that Vertex exclusively licensed from CRISPR Therapeutics as part of the collaboration.

As part of the agreement, which was announced on Tuesday, December 12, CRISPR Therapeutics and Vertex will share the research and development costs and profits worldwide.

A clinical trial application was submitted earlier this month in support of a trial in β-thalassemia, a blood disorder, to take place in 2018 in Europe. And an Investigational New Drug Application is planned for submission in 2018 to support the initiation of a phase 1/2 trial in sickle cell disease in the US.

David Altshuler, Vertex’s executive vice president, global research and chief scientific officer, said that the parties have made significant progress over the past two years.

He added: “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.”

Samarth Kulkarni, CEO of CRISPR Therapeutics, added that 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”.

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