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AAVLK03hOTC
- Development stage
- Unknown
- Lead developer
- Bloomsbury Genetic Therapies
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAVLK03hOTC is an investigational adeno-associated virus (AAV) gene therapy designed for the treatment of Ornithine Transcarbamylase Deficiency (OTCD). It utilizes the AAV-LK03 capsid, a chimeric vector engineered for high-efficiency transduction of human hepatocytes. The therapy delivers a functional, codon-optimized version of the human ornithine transcarbamylase (OTC) gene to the liver. By restoring endogenous OTC enzyme activity, the treatment aims to facilitate the normal processing of nitrogen via the urea cycle, thereby preventing the toxic accumulation of ammonia (hyperammonemia). This approach is intended to reduce life-threatening metabolic decompensations and associated neurological damage, potentially serving as a definitive treatment or a 'bridge-to-transplant' for pediatric patients.
- Other names
- AAV-LK03-hOTCAAV-LK-03-hOTCAAV-LK 03-hOTC
02
Targets
OTC (Ornithine carbamoyltransferase)
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