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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.
Liquidia Technologies is a biopharmaceutical company transforming the development of new therapies by precisely engineering drug particles.
RDMD`s mission is to empower patients and communities to accelerate the development of treatments for rare diseases of all kinds.
Argonaut Manufacturing Services provides contract manufacturing for pharmaceutical, diagnostics and life science organizations.
Topaz Pharmaceuticals LLC is a Horsham, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.