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cbin1 gene therapy
- Development stage
- Preclinical
- Lead developer
- TikkunLev Therapeutics
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
cBIN1 gene therapy is an adeno-associated virus (AAV) vector-based treatment, specifically utilizing the AAV9 serotype, designed to deliver the human cardiac bridging integrator 1 (cBIN1) gene to cardiomyocytes. cBIN1 is a T-tubule-associated scaffold protein essential for the structural organization of the calcium-handling machinery within the heart, including L-type calcium channels and ryanodine receptors. In conditions such as heart failure and pulmonary arterial hypertension (PAH), cBIN1 expression is often significantly reduced, leading to T-tubule disruption and impaired excitation-contraction coupling. By restoring cBIN1 levels, the therapy aims to stabilize T-tubule microdomains, improve cardiac contractility, and prevent the progression of right ventricular (RV) remodeling and failure. It is currently being investigated in preclinical models for right heart failure associated with PAH.
- Other names
- AAV9-cBIN1AAV-9-cBIN1AAV 9-cBIN1AAV9-hcBIN1AAV-9-hcBIN1AAV 9-hcBIN1exogenous cBIN1 gene therapy
02
Targets
BIN1 (Bridging integrator 1)
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