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We are a biopharmaceutical company specializing in the development of novel therapeutics to treat rare metabolic diseases. Our lead product candidate is INZ-701, an enzyme replacement therapy (ERT) in the early stages of clinical development for the potential treatment of patients with a variety of calcification disorders linked primarily to mutations in the ENPP1 and ABCC6 genes, or ENPP1 Deficiency and ABCC6 Deficiency respectively. We have demonstrated proof of concept with INZ-701 in both GACI and ARHR2 two phenotypes of ENPP1 Deficiency.
Noramco, Inc. is a Wilmington, DE-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
GENFIT is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative therapeutic and diagnostic solutions in metabolic and liver related diseases where there are considerable unmet medical needs, corresponding to a lack of approved treatments. GENFIT is a leader in the field of nuclear receptor-based drug discovery with a rich history and strong scientific heritage spanning almost two decades. Its most advanced drug candidate, elafibranor, is currently evaluated in pivotal Phase 3 clinical trial (“RESOLVE-IT”) as a potential treatment for nonalcoholic steatohepatitis, or NASH. NASH is considered by regulatory authorities as a medical emergency because of its potentially severe consequences, although often asymptomatic until late stages, and because its prevalence is on the rise. Elafibranor has also obtained positive preliminary results in a Phase 2 clinical trial in primary biliary cholangitis (PBC), a severe chronic liver disease. As part of GENFIT’s comprehensive approach to clinical management of NASH patients, the Company is also developing a new, non-invasive and easy-to-access blood-based in vitro diagnostic, or IVD, test to identify patients with NASH who may be appropriate candidates for drug therapy. With facilities in Lille and Paris, France, and Cambridge, MA, USA, the Company has approximately 170 employees. GENFIT is a public company listed on the Nasdaq Global Select Market and in compartment B of Euronext’s regulated market in Paris.
CIMA Labs Inc. develops and manufactures orally disintegrating tablet and enhanced-absorption oral drug delivery systems. The Company's lead, orally disintegrating tablet technologies, OraSolv and DuraSolv, allow an active drug ingredient, which is
Arrowhead Research Corporation is a biopharmaceutical company developing targeted RNAi therapeutics. The company is leveraging its proprietary Dynamic Polyconjugate delivery platform to develop targeted drugs based on the RNA interference mechanism that efficiently silences disease-causing genes. Arrowhead`s pipeline includes ARC-520 for chronic hepatitis B virus, ARC-AAT for liver disease associated with Alpha-1 antitrypsin deficiency, and partner-based programs in obesity and oncology.