PHAR vs NTLA Comparison

Pharming Group is a biopharmaceutical company focused in transforming the lives of patients with rare, debilitating, and life-threatening diseases. The company is actively developing and commercializing a diverse portfolio of innovative treatments, including both biologics and small molecule therapies. Its commercial products are RUCONEST, approved for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema, or HAE; and Joenja (leniolisib), a small-molecule kinase inhibitor, developed for the treatment of activated phosphoinositide 3-kinase delta syndrome.

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.