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Main focus: Gene editing for the treatment of genetic diseases and cancer
Company stage: Clinical
Diseases: Non-hodgkins lymphoma (NHL), multiple myeloma, B-cell acute lymphoblastic leukaemia (ALL), chronic lymphocytic leukaemia (CLL), small lymphocytic lymphoma (SLL), Duchenne muscular dystrophy, primary hyperoxaluria type 1, chronic hepatitis B infection.
Genome editing tool: megaNuclease
Funding stage: Public (NASDAQ:DTIL)
Location: Durham, North Carolina, US
Partners: Eli Lilly, SpringWorks Therapeutics, iECURE
Precision Biosciences utilises its proprietary ARCUS genome-editing plaform to develop novel CAR-T and gene-editing therapies. The platform is based on the homing endonuclease I-CreI that allows for single, highly specific DNA edits. The company has advanced multiple CAR-T programmes to clinical trials, mainly for the treatment of haematological cancers. Furthermore, the company has a broad in vivo gene correction pipeline under development for the treatment of genetic diseases.