Clinical Trial Authorizations

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

27 October 2025   As we enter a new era of drug discovery, where IND authorization can now be granted based on human data only, marking a clean departure from mandatory animal testing, Qureator's human organ-on-chip platform has laid the groundwork for the world’s first IND approval of its kind .   The IND grant concerns SillaJen's BAL0891-tislelizumab combination therapy to treat solid tumors. Qureator's patient-derived vascularized tumor-immune microenvironment (TME) model

12 November 2025   Inventia Life Science's 3D bioprinted patient-derived cancer models has opened the door for Hunter Medical Research Institute to advance its drug repurposing program into Australian New Zealand clinical trials .   The drug in question, efavirenz, is known as an antiretroviral NNRTI (non‑nucleoside reverse transcriptase inhibitor) used for treating HIV. Its repurposing has the potential to improve the survival of patients with high-grade serous ovarian cance