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Main focus: Gene editing for the treatment of genetic diseases
Company stage: Clinical
Diseases: Beta-thalassemia, other undisclosed anaemias, lymphoma, solid tumours, neuromuscular diseases, Hurler syndrome
Genome editing technology: CRISPR-Cas9, LEAPER (RNA Editing)
Funding stage: Private
Location: Beijing, China
EdiGene is a clinical-stage, gene-editing company, mainly focused on the development of novel therapies for haematopoietic diseases and cancer. The company holds licenses on proprietary CRISPR-Cas and RNA-editing platforms. The company's most advanced programme is ET-01 which has entered Phase I clinical development. This therapy is developed for the treatment of beta-thalassemia, and thus, is competing with similar programmes from CRISPR Therapeutics/Vertex Pharmaceuticals and Graphite Bio. The company is also developing next-generation CAR-T cell therapies, and these are still at pre-clinical stage.