CRISPR Therapeutics is developing medicine for serious diseases using the CRISPR-Cas9 platform that co-founder Emmanuelle Charpentier and Jennifer Doudna co-developed and published togehter in Science in 2012 . Charpentier and Doudna were awarded the 2015 Breakthrough Prize in Life Sciences. Charpentier is one of the co-founders and is an advisor to CRISPR Therapeutics which was initially called Inception Genomics AG.
CRISPR Therapeutics’ lead product candidate, CTX001, targets sickle cell disease and beta-thalassemia. Cells are harvested from a patient, treated ex vivo with the CRISPR-Cas9-based therapeutic, and returned to the patient. A clinical trial for CTX001 began in December 2017. Other therapeutic developments include CTX101 and other CAR-T therapies to target cancer. CAR-T therapies are chimeric antigen receptor T cell therapies which are immune cells engineered to find and destroy cancer cells. Hemoglobinopathies and regenerative medicine are other areas they are working in.
CRISPR Therapeutics began separate collaborations with Vertex Pharmaceuticals and Bayer in 2015. The joint venture with Bayer has been named Casebia Therapeutics and focuses of blood disorders, blindness and congenital heart disease.
Co-Founder, Scientific Advisory Board
Co-Founder and President
Co-Founder, former CFO
Documentaries, videos and podcasts
- Cluster: BiotechnologyA cluster of topics related to biotechnology.
- CRISPR/Cas ToolsClustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated (Cas) proteins perform adaptive immune functions in prokaryotic organisms defending against foreign nucleic acids such as viruses. CRISPR/Cas tools have been adapted for use in genome editing and other DNA and RNA targeting applications.
- CRISPRClustered regularly interspaced short palindromic repeats (CRISPR) is a prokaryotic adaptive immune response that provides immunity against foreign nucleic acids, such as viral DNA and bacterial plasmids, through the use of crRNAs (CRISPR RNAs) and associated Cas genes.
- CRISPR-Cas9CRISPR-Cas9 is a genome editing system. CRISPR systems provides immunity to bacteria and archaea from viruses and has been adapted for use as a genome editing tool capable of knocking out genes and rewriting genetic sequences in animal, plant and fungi. CRISPR-Cas9 is being adapted to other applications outside genome editing.
- Caribou BiosciencesCaribou Biosciences is a Berkeley California based private company founded in 2011 by Jennifer Doudna and Rachel Haurwitz based on CRISPR technology.
- Intellia TherapeuticsIntellia Therapeutics is a biotechnology company founded in 2014, based in Cambridge, MA that develops biopharmaceuticals using CRISPR-Cas9 gene editing technology.