Thiogenesis Therapeutics, Inc.

University of California

Patrice P Rioux

Vincent P Stanton, Jr

Ranjan Dohil

Piotr Barski

Dariusz Witt

The invention features compositions, methods, and kits containing (i) one or more cysteamine precursor compounds convertible to cysteamine in vivo, and (ii) optionally agents to enhance that conversion, formulated to produce a spectrum of pharmacokinetic profiles of cysteamine that can be tailored to individual patients and diseases. The invention also features varying modes of administration of the therapeutic substances in the treatment of cystinosis and other cysteamine sensitive disorders. In particular, formulations combining active ingredient(s) with pharmaceutical excipients that permit sustained cysteamine plasma concentrations are featured.

Compositions for controlled release of cysteamine and systemic treatment of cysteamine sensitive disorders

The invention features methods for the treatment of cystinosis and other cysteamine sensitive disorders in a subject including administration of a disulfide convertible to cysteamine in vivo. The methods can include the separate administration of a reducing agent to the subject to increase the bioavailablity and extend the plasma pharmacokinetic profile of the cysteamine produced following administration of the disulfide. The methods permit sustained cysteamine plasma concentrations in a subject.

Methods for the treatment of cysteamine sensitive disorders

The disclosure relates, in general, to treatment of fatty liver disorders comprising administering compositions comprising cysteamine or cystamine compositions. The disclosure provides administration of enterically coated cysteamine compositions to treat fatty liver disorders, such as non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH).

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San Diego, CA, United States of America

Patrice P Rioux