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AAV9-cTuberin
- Development stage
- Preclinical
- Lead developer
- BridgeBio Pharma
- Modality
- Gene Therapies
- Administration
- Intracerebral, Intrathecal, Intravenous
01
Overview
AAV9-cTuberin is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy designed for the treatment of tuberous sclerosis complex type 2 (TSC2). Developed by Massachusetts General Hospital and BridgeBio, the therapy delivers a "condensed" form of human tuberin (cTuberin) to overcome the packaging size limitations of standard AAV vectors, which cannot accommodate the full-length 5.4 kb TSC2 coding sequence. The cTuberin construct (approximately 2.3 kb) contains the essential hamartin-binding and GTPase-activating protein (GAP) domains of tuberin but lacks the Akt phosphorylation site Thr1462. By restoring tuberin function, AAV9-cTuberin suppresses the hyperactivation of the mammalian target of rapamycin complex 1 (mTORC1) pathway, thereby reducing cellular proliferation, brain lesions, and seizures associated with TSC2.
- Other names
- AAV9-CBA-cTuberinAAV-9-CBA-cTuberinAAV 9-CBA-cTuberincTuberin
02
Targets
RHEB (GTP-binding protein Rheb)
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