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DTX601
- Development stage
- Preclinical
- Lead developer
- Ultragenyx Pharmaceutical
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
DTX601 is an investigational gene therapy designed to treat **citrullinemia type I**, a rare genetic disorder affecting the urea cycle, leading to accumulation of ammonia and neurological complications. It is developed as part of a suite of gene therapies addressing inherited metabolic disorders. DTX601 utilizes an adeno-associated virus (AAV) vector, likely based on the AAV8 serotype given the developer’s prior pipeline, to deliver a functional copy of the relevant gene to liver cells, aiming to restore urea cycle function and reduce toxic metabolite buildup[11]. Gene therapies for urea cycle disorders intend to increase enzymatic activity and reduce disease symptoms by achieving durable expression of the corrective gene following a single administration.
- Brand names
- DTX601DTX-601DTX 601
- Other names
- DTX601DTX-601DTX 601
02
Targets
ASS1 (Argininosuccinate synthase 1)
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