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AAV-siCnnm1
- Development stage
- Preclinical
- Lead developer
- CIC bioGUNE
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
AAV-siCnnm1 is an experimental adeno-associated virus (AAV) gene therapy vector designed to deliver a small interfering RNA (siRNA) to silence the expression of cyclin M1 (CNNM1), a metal transporter involved in magnesium homeostasis. Developed by researchers at CIC bioGUNE, AAV-siCnnm1 is being investigated for the treatment of type 2 diabetes and insulin resistance. In preclinical models of nutritional distress and type 2 diabetes, hepatic silencing of CNNM1 using AAV-siCnnm1 has been shown to improve systemic insulin sensitivity, decrease liver triglyceride accumulation, and increase serum ketone bodies, primarily through a fibroblast growth factor 21 (FGF21)-mediated pathway.
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