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Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Crispr/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Crispr-associated protein 9 (Cas9), which is a revolutionary technology for precisely altering specific sequences of genomic DNA. Intellia is focused on using this technology to treat genetically defined diseases. It's evaluating multiple gene editing approaches using in vivo and ex vivo therapies to address diseases with high unmet medical needs, including ATTR amyloidosis, hereditary angioedema, sickle cell disease, and immuno-oncology. Intellia has formed collaborations with several companies to advance its pipeline, including narrow-moat Regeneron and wide-moat Novartis.
Enliven Therapeutics Inc is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics. Its product candidates include ELVN-001 is designed to specifically target the BCR-ABL fusion gene product, the oncogenic driver for patients with chronic myeloid leukemia (CML), ELVN-002 is designed to inhibit wild-type HER2 and key mutations of HER2.