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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.
The Ripple Therapeutics Epidel® technology is founded on a discovery that drugs can be engineered into controlled release materials without the use of polymers or excipients. The proprietary prodrugs are new chemical entities (NCEs) that have unique properties that allow them to be processed into standalone drug delivery implants (e.g. intravitreal implants, micro/nanoparticles, etc.) or as coatings on medical devices. Ripple Therapeutics implants and coatings are made up entirely from the prodrug without a polymeric carrier to control the implant or drug release properties. The implants and coatings undergo surface erosion to give zero order drug release profiles and are highly engineerable (e.g. different forms, shapes, and sizes) to tailor drug dose and duration for the specific indication of interest. The flexibility in molecular design results in drug doses that are within the therapeutic window, delivering the right amount of drug for the right amount of time. This has the potential to improve patient safety and clinical outcomes.
Galectin Therapeutics (NASDAQ: GALT) is applying its leadership in galectin science and drug development to create new therapies for fibrotic disease and cancer. Drug candidates based on the Company`s unique carbohydrate technology target galectin proteins which are key mediators of biologic and pathologic function.
All of Ankas programs share the same mission: to eliminate the impact of behavioral health problems for all people.
Phathom Pharmaceuticals, launched in 2019 by Frazier Healthcare Partners and Takeda Pharmaceuticals, is a biopharmaceutical company focused on the development and commercialization of novel treatments for gastrointestinal (GI) diseases and disorders. The company is advancing the investigational agent vonoprazan, a novel potassium-competitive acid blocker (P-CAB). Vonoprazan has the potential to address multiple unmet needs among patients with gastroesophageal reflux disease (GERD) and other acid-related disorders.