MIRM vs CRSP Comparison

Mirum Pharmaceuticals Inc is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel therapies for debilitating rare and orphan diseases. Its product, Livmarli, a novel, orally administered, minimally-absorbed ileal bile acid transporter IBAT) inhibitor (IBATi), is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.

Crispr 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. The company is focused on using this technology to treat genetically defined diseases. Crispr's first approved drug is Casgevy, which was developed in collaboration with Vertex Pharmaceuticals and targets sickle-cell disease and transfusion-dependent beta-thalassemia, which have high unmet medical needs. The company is advancing a variety of gene editing programs in immuno-oncology, cardiovascular, and a stem cell-derived therapy to treat Type 1 diabetes.