Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
OTXL-101
- Development stage
- Unknown
- Lead developer
- University College London
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
OTXL-101 (formerly BGT-OTCD or AAVLK03hOTC) is an investigational adeno-associated virus (AAV)-based gene therapy designed for the treatment of ornithine transcarbamylase deficiency (OTCD), a rare, life-threatening pediatric metabolic urea cycle disorder. The therapy utilizes the engineered AAV-LK03 capsid, which exhibits high tropism for human hepatocytes, particularly periportal hepatocytes where the urea cycle primarily occurs. OTXL-101 delivers a functional copy of the human ornithine transcarbamylase (OTC) gene to liver cells, restoring OTC enzyme activity and enabling the proper conversion of toxic ammonia into urea. Originally developed by researchers at University College London (UCL) and the Children's Medical Research Institute (CMRI), the program was previously licensed to Bloomsbury Genetic Therapies, which initiated the Phase 1/2 HORACE clinical trial (NCT05092685) before becoming insolvent. In October 2026, the Orphan Therapeutics Accelerator (OTXL) in-licensed the asset to resume its clinical development.
- Other names
- ssAAV-LK03.hAAT.hcoOTCssAAV-LK-03.hAAT.hcoOTCssAAV-LK 03.hAAT.hcoOTC
02
Targets
OTC (Ornithine carbamoyltransferase)
Beyond the preview
Go deeper on OTXL-101.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on OTXL-101.