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Vertex Pharmaceuticals

Main focus: Many different therapeutic areas including cystic fibrosis, pain and sickle cell disease

Company stage: Clinical

Diseases: Sickle cell disease, beta-thalassemia, cystic fibrosis, duchenne muscular dystrophy

Genome editing technology: CRISPR-Cas9

Funding stage: Public (NASDAQ:VRTX)

Location: Boston, MA, USA



Partners: CRISPR Therapeutics, Mammoth Biosciences, Obsidian Therapeutics, Arbor Biotechnologies

Vertex Pharmaceuticals is a big pharma company that is developing medicines within many different disease areas. Its involvement in gene-editing and CRISPR-Cas9 comes mainly through a partnership with CRISPR Therapeutics. Together, they develop the CTX001 programme for the treatment of sickle cell disease and beta-thalassemia. So far, promising preliminary clinical results have been released and the programme is regarded as the most developed clinical-stage CRISPR programme. Vertex also holds licenses from CRISPR Therapeutics to develop therapies for other genetic diseases such as cystic fibrosis, duchenne muscular dystrophy and myotonic dystrophy type 1.


HashtagVertex Pharmaceuticals, Inc.

Company: Vertex Pharmaceuticals
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