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Main focus: Gene editing for the treatment of genetic diseases and cancer
Company stage: Clinical
Diseases: Sickle cell disease, beta-thalassemia, multiple myeloma, haematological malignancies, solid tumours, Type 1 diabetes mellitus, Glycogen storage disease type 1a (GSD 1a), duchenne muscular dystrophy, myotonic dystrophy type 1, cystic fibrosis
Genome editing tool: CRISPR-Cas9
Funding stage: Public (NASDAQ:CRSP)
Location: Zug, Switzerland and Cambridge, MA, USA
CRISPR Therapeutics is a clinical-stage genome-editing company primarily working with haemoglobinopathies, immunooncology, regenerative medicine and other rare diseases. The company has a clinical-stage programme, CTX001 in sickle cell disease and beta-thalassemia, with promising preliminary results so far. Furthermore, the company is investigating three distinct CRISPR-Cas9-modified CAR-T cell therapies. The company is co-founded by Dr. Emanuelle Charpentier and holds multiple important patents within the gene-editing space.