31 January 2026   Mahzi Therapeutics-sponsored Phase 1/2 study  to assess safety and efficacy of MZ-1866 gene therapy for children and adults with Pitt Hopkins syndrome is about to start. The trial was announced  by Mahzi in 2025, highlighting the sponsor's collaboration with the Pitt Hopkins Research Foundation and the Muotri Lab at UC San Diego in developing this long awaited treatment.   Pitt-Hopkins syndrome (PHS) is a rare genetic disorder caused by mutations in the tran

30 June 2025   The Ministry of Health, Labour and Welfare in Japan has granted orphan drug designation to riliprubart for treating chronic inflammatory demyelinating polyneuropathy (CIDP), Sanofi reports . Riliprubart also holds this designation for CIDP in the EU and US.   By engineering its human-relevant CIDP-on-chip as preclinical tool for disease modelling and drug efficacy testing, Hesperos has played an instrumental role in achieving this outcome.   CIDP is an inflamma