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Mirum Pharmaceuticals (NASDAQ: MIRM) is dedicated to creating life-changing therapies for patients with liver diseases. We are advancing our lead product candidate for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) — conditions with a profound and devastating impact on quality of life for young patients and their families. Untreated, these conditions can lead to liver failure and inflict life-altering symptoms including stunted growth and severe, non-stop itching. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues. Working closely with patients and caregiver communities, we intimately understand the challenges of living with cholestatic liver diseases. Based on a wealth of data from clinical trials spanning multiple years and involving more than one hundred patients, we believe our approach can address the underlying cause of symptoms and liver damage. Our goal is to give families their lives back.
EQRx operates as a biotechnology company, focused on re-engineering the process from drug discovery to patient delivery with the goal of offering a market-based solution for the rising cost of medicines.
A discovery through early clinical phase pharmaceutical company, Ratio Therapeutics employs a suite of innovative technologies to develop best-in-class targeted radiotherapeutics for the treatment of cancer. Ratio Therapeutics` Trillium™ targeting scaffold combines pharmacokinetic modulation with best-in-class chelation technology to create proprietary small molecule targeting agents to attack a broad array of cancer targets. Our compounds accommodate imaging and therapeutic radioisotopes, ie "theranostics". The tunable nature of our platform combined with small-scale imaging trials in patients results in accelerated, de-risked compound selection.
La Jolla Pharmaceutical Company is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The Company has several product candidates in development. LJPC-501 is La Jolla`s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension. LJPC-401 is La Jolla`s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. LJPC-30S is our next-generation gentamicin derivative program that is focused on therapeutics for the potential treatment of serious bacterial infections as well as rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.