HRMY vs NTLA Comparison

Harmony Biosciences Holdings Inc is a commercial-stage pharmaceutical company focused on developing and commercializing therapies for patients living with rare neurological diseases who have unmet medical needs. The company's product WAKIX (pitolisant) is a molecule with a novel mechanism of action specifically designed to increase histamine signaling in the brain by binding to H3 receptors and is used for the treatment of cataplexy in adult patients with narcolepsy. The other drug candidates in its pipeline are Pitolisant, HBS-102 (MCHR1 antagonist), BP1.15205 (orexin-2 receptor agonist), ZYN002 (cannabidiol gel), EPX-100 (clemizole hydrochloride), and EPX-200 (lorcaserin), among others.

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.