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Leadiant Biosciences Ltd. is focused on the discovery of molecules and therapies to help improve the lives of people around the world who suffer from specific diseases.

The study of rare diseases is an integral part of Leadiant Biosciences’ research activity and heritage in rare diseases dating back to 1984, we have been the fifth company overall and the first non-American company to receive an Orphan Drug Designation in the United States. Since then, we have obtained several others.

Our most recent achievements as Leadiant Biosciences Group have been the registration of two important products – Chenodeoxycholic Acid Leadiant (chenodeoxycholic acid) for the treatment of Cerebrotendineous Xanthomatosis (CTX) with the European Medicines Agency (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 the Food and Drug Administration (FDA) in 2018. In 2020, Revcovi was the object of a successful transaction with Chiesi which acquired the full rights of the drug.

We continue to honour our commitment to patients by advancing a diverse pipeline with the objective of developing therapeutic candidates and/or to improve existing treatments. Our innovative research programs and development 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 have not yet been established for the proposed therapeutic areas.

Active Ingredient (Compound number) Indication/Disease Area Stage of Development
Chenodeoxycholic Acid (LB101) Cerebrotendinous Xanthomatosis (CTX)/Lipid Storage Disorder Phase III (US)
Cysteamine hydrochoride New Formulation (LB201) Cystinosis (corneal cysteine deposit)/Disorders of Amino Acid Transport Registration (US)
Cysteamine hydrochoride New Delivery System (LB202) Cystinosis (corneal cysteine deposit)/Disorders of Amino Acid Transport Pre-clinical
N-acetyl-D- mannosamine (LB301) GNE Myopathy/Myopathy Phase IIb (pivotal; US)