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AAV2-hAADC
- Development stage
- Unknown
- Lead developer
- Krzysztof Bankiewicz
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intracerebral
01
Overview
AAV2-hAADC is an investigational gene therapy developed by Dr. Krzysztof Bankiewicz for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. It utilizes an adeno-associated virus type 2 (AAV2) vector to deliver a functional copy of the human AADC gene (hAADC) directly into the midbrain. The therapy is administered via MR-guided convection-enhanced delivery (CED) into the substantia nigra pars compacta and the ventral tegmental area. By restoring AADC enzyme activity in these regions, the therapy aims to enable the production of dopamine and serotonin, addressing the underlying cause of the disorder. Clinical results from Phase 1 trials (NCT02852213) have shown the therapy to be safe and effective in improving motor function and reducing symptoms like oculogyric crises in pediatric patients.
- Other names
- hAADC gene therapyAAV2-hAADC-BankiewiczAAV-2-hAADC-BankiewiczAAV 2-hAADC-Bankiewicz
02
Targets
DDC (Aromatic L-amino acid decarboxylase)
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