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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.