We supported Dorphan SA, a small start-up active in the development of treatment of lysosomal storage diseases, in the preclinical development of their lead compound DO-1, an iminosugar derivative by working on the process development and scale-up of this highly complex molecule on up to 100 g’s of the API.
CTI Project “Intermediate scale manufacturing and in vivo therapeutic assessment of a promising drug candidate for the treatment of GM1-gangliosidosis” with Dorphan SA.
Patent: 4-Epi-Isofagomine Enantiomer Derivatives for the Treatment of Lysosomal Storage Diseases - Stephane Demotz, Olivier Martin, Estelle Boivineau, Sophie Front, Julie Charollais-Thoenig, Roger Marti, Jean-Pascal Bourgeois, Sofia Almeida, WO2016174131 (A1).
Publication: 5a-C-Substituted 4-Epi-Isofagomine Derivatives as Pharmacological Chaperones for the Treatment of Lysosomal Diseases Linked to b-Galactosidase Mutations: Improved Synthesis and Biological Investigations - Sophie Front, Sofia Almeida, Vincent Zoete, Julie Charollais-Thoenig, Estelle Gallienne, Cécile Marmy, Vincent Pilloud, Roger Marti, Tim Wood, Olivier R. Martin, Stéphane Demotz, Bioorg. Med. Chem. 2018, 26, 5462–5469 -