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AAV1-CMV-hIns
- Development stage
- Preclinical
- Lead developer
- Children's Hospital of Philadelphia
- Modality
- Gene Therapies
- Administration
- Intramuscular
01
Overview
AAV1-CMV-hIns is an investigational adeno-associated virus serotype 1 (AAV1) gene therapy vector designed to express human insulin (hIns) under the control of a cytomegalovirus (CMV) promoter. Developed by researchers at the Universitat Autònoma de Barcelona (UAB) and the Children's Hospital of Philadelphia (CHOP), this vector is designed for intramuscular injection to transduce skeletal muscle cells, effectively turning them into a biofactory for the continuous production and secretion of human insulin. In preclinical studies, AAV1-CMV-hIns has been evaluated both as a monotherapy and in combination with a glucokinase-expressing vector (AAV1-CMV-Gck) to achieve synergistic glucose-sensing and insulin-secreting activity in skeletal muscle, offering a potential long-term treatment for type 1 diabetes.
- Other names
- AAV1-hInsAAV-1-hInsAAV 1-hInsAAV1-ohINSAAV-1-ohINSAAV 1-ohINS
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