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Drug intelligence / Profile preview
AAV9-hamartin
- Development stage
- Preclinical
- Lead developer
- GondolaBio
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-hamartin (BBP-822) is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy designed to deliver a functional copy of the human TSC1 gene, which encodes the protein hamartin. Developed initially by BridgeBio and subsequently transferred to GondolaBio, this therapy is being evaluated for the treatment of Tuberous Sclerosis Complex type 1 (TSC1). Hamartin, in complex with tuberin, acts as a critical negative regulator of the mechanistic target of rapamycin complex 1 (mTORC1) pathway. Mutations in TSC1 lead to loss of hamartin function, resulting in constitutive mTORC1 hyperactivation, abnormal cell growth, and neurological manifestations such as epilepsy, cognitive impairment, and subventricular zone cell proliferation. AAV9-hamartin aims to restore hamartin expression in the central nervous system, thereby normalizing mTORC1 signaling and ameliorating TSC1-associated pathologies.
- Other names
- AAV-hamartinAAV9-CB6-TSC1AAV-9-CB6-TSC1AAV 9-CB6-TSC1AAV9-CB6-hamartinAAV-9-CB6-hamartinAAV 9-CB6-hamartinAAV9-mediated hamartinAAV-9-mediated hamartinAAV 9-mediated hamartin
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