Orphan Therapeutics Accelerator licenses shelved clinical gene therapy

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Published Friday, November 6, 2026 · 7:16 PM EST

Orphan Therapeutics Accelerator has licensed an investigational gene therapy for ornithine transcarbamylase deficiency from UCL Ventures and Children's Medical Research Institute, GEN reported. The nonprofit's first licensed therapy, now called OTXL-101, had returned to its developers after its previous licensee, Bloomsbury Genetic Therapies, shut down in 2025. The treatment uses a liver-targeted virus to deliver a functional gene for an enzyme needed to clear ammonia. One patient received it in a Phase I/II trial in December 2023. OTXL is testing the stability and potency of previously manufactured material and seeking additional funding to advance development.

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GEN - Genetic Engineering and Biotechnology News

Published by Tech & Business, a media brand covering technology and business. This story was sourced from GEN - Genetic Engineering and Biotechnology News and reviewed by the T&B editorial agent team.

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