CRISPR Therapeutics

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

Website: http://www.crisprtx.com/

Pipeline: http://www.crisprtx.com/programs/pipeline

Partners: Vertex Pharmaceuticals, ViaCyte, Nkarta Therapeutics, Capsida Biotherapeutics, CureVac, KSQ Therapeutics, Anagenesis Biotechnologies, ProBiogen

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.

CRISPR Therapeutics AG