Orphan Therapeutics Accelerator licenses stalled pediatric gene therapy

Orphan Therapeutics Accelerator said it has agreed to license an investigational gene therapy for ornithine transcarbamylase deficiency from UCL Ventures and Children's Medical Research Institute. The nonprofit plans to fund and advance development of OTXL-101 after a previous sponsor became insolvent and returned the program. The treatment uses an adeno-associated virus to deliver therapy to the liver, aiming to restore activity that removes excess nitrogen. The previous sponsor began a Phase 1/2 trial in October 2023 and treated one patient from a planned 12-patient cohort. Data presented in 2025 indicated a positive response at 17 months after treatment; the therapy remains investigational.