# Orphan Therapeutics Accelerator licenses shelved clinical gene therapy

_Published Friday, November 6, 2026 at 7:16 PM EST · Science · Latest · Tier 2 — Notable_

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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.

## Sources

- [GEN - Genetic Engineering and Biotechnology News](https://www.genengnews.com/topics/translational-medicine/orphan-therapeutics-accelerator-in-licenses-its-first-gene-therapy-eyes-pipeline-expansion/)

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Canonical: https://techandbusiness.org/newswire/neyURit83-6G4R0j0wg72l
Published: 2026-11-07T00:16:09.274Z
Story chronology: 2026-11-05T18:29:40.000Z
Retrieved: 2026-11-08T06:09:18.702Z
Publisher: Tech & Business (techandbusiness.org)