Group Pipeline Leadiant Biosciences, S.p.A is a Rome-based global holding company, with subsidiaries in the US (Leadiant Biosciences, Inc.) and Europe (Leadiant Biosciences, Ltd, Leadiant Biosciences SASU and Leadiant GmbH). For region or country-specific product information, use the dropdown below. Choose a Region or Country We continue to honor our commitment to patients by advancing a diverse pipeline of new drugs and therapeutic candidates. Leadiant Biosciences is a global, research-based pharmaceutical company focused on the discovery of molecules and therapies to help improve the lives of people who suffer from specific high unmet medical need diseases. The study of rare diseases is an integral part of our research activity and heritage in rare diseases dating back to 1984, we have been among the first four Companies in the world to receive an Orphan Drug Designation in the United States. Since then, we have obtained several others. Our most recent achievements have been the registration of two important products as Chenodeoxycholic Acid (CDCA) for the treatment of Cerebrotendineous Xanthomatosis (CTX) with EMA in 2017 and Revcovi (elapegademase-lvlr, a recombinant pegylated adenosine deaminase) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) with FDA in 2018. We continue to honor our commitment to patients by advancing a diverse pipeline of new drugs and therapeutic candidates. Our innovative research programs and development of novel product candidates are led by our scientists in partnership with a wide range of academic and biotechnology organizations. Projects under investigation/development The below chart reflects investigational drugs in various stages of clinical development for potential introduction into various markets. The safety and efficacy of these products have not yet been established for the proposed therapeutic areas. Product Disease Area/Indication Stage of Development Chenodeoxycholic Acid (CDCA)(LB101) Cerebrotendinous Xanthomatosis (CTX)/Metabolic Phase III (US) Cysteamine hydrochoride (LB201) Cystinosis (corneal cysteine deposit)/Lysosomal storage disease New formulation Cysteamine hydrochoride (LB202) Cystinosis (corneal cysteine deposit)/Lysosomal storage disease New delivery system N-acetyl-D- mannosamine (LB301) GNE Myopathy/Myopathy Phase III N-acetyl-D- mannosamine (LB701) Nephrology Phase I Roneparstat (LB401) Hematological Malignancies/Oncology Phase II STP-206 Necrotizing Enterocolitis/Neonatology Phase I Amphotericin B Lipid Complex (LB501) Mycotic keratitis/Infectious Disease Pre-Clinical Elapegademase (LB601) Scleroderma/Immunology-Autoimmune Diseases Pre-Clinical Updated June 2019 Leadiant Biosciences S.p.A. (Corporate) via Sudafrica 20 - 00144 Roma Italia ©2021 Leadiant Biosciences, Ltd. / Roma, ITL GL-NP-2000002 Date of Production: Feb, 2020 About Leadiant Biosciences Group Pipeline Group Marketed Products Contact Us Privacy Policy Sitemap Leadiant Biosciences Ltd. (International) Leadiant Biosciences, Inc. (U.S. and Canada)