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MYOAAV3A-Pik3c2b miRNA

Development stage: Preclinical
Lead developer: University of Toronto
Modality: Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

MYOAAV3A-Pik3c2b miRNA is a preclinical-stage gene therapy designed to downregulate the expression of phosphoinositide 3-kinase class II beta (PIK3C2B) specifically in skeletal muscle. The therapeutic construct consists of a muscle-tropic adeno-associated virus capsid (MyoAAV 3A) encapsulating a synthetic microRNA (miRNA) targeting the Pik3c2b gene. By knocking down Pik3c2b in skeletal muscle, the therapy aims to improve insulin sensitivity, reduce adiposity, and regulate body weight. It is being investigated for the treatment of insulin resistance, obesity, and related metabolic co-morbidities.

Other names: MYOAAV-miRNAMYOAAV3A-Pik3c2b-targeted miRNAMYOAAV-3A-Pik3c2b-targeted miRNAMYOAAV 3A-Pik3c2b-targeted miRNA

Targets

PIK3C2B (PI3K-C2β)

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