Drug intelligence / Profile preview

AAV-nov-miR-39

Development stage: Preclinical
Lead developer: UMass Chan Medical School
Modality: Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics

Overview

AAV-nov-miR-39 is a recombinant adeno-associated virus (rAAV)-based gene therapy being developed by the University of Massachusetts Chan Medical School for the treatment of osteoporosis. The therapy utilizes a bone-targeted rAAV9 vector, engineered with bone-targeting peptides (DSS6) grafted onto the capsid, to deliver nov-miR-39, a novel microRNA that acts as a key regulator of the Schnurri-3 (SHN3) pathway in osteoblasts. Overexpression of nov-miR-39 in osteoblasts enhances WNT signaling, osteogenic differentiation, and bone-forming activity, thereby reversing bone loss in postmenopausal and age-related osteoporosis without causing adverse effects in non-skeletal tissues.

Other names: rAAV-nov-miR-39rAAV-nov-miR39rAAV-nov-miR 39rAAV9-nov-miR-39rAAV-9-nov-miR-39rAAV 9-nov-miR-39

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