NOVA-AAV9

NOVA-AAV9 is an experimental gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). Developed by researchers at the Indian Institute of Technology Kanpur, the therapy utilizes a rationally engineered adeno-associated virus serotype 9 (AAV9) capsid, designated NeddyAAV9, which has been modified at post-translational modification sites to enhance transduction efficiency in muscle tissues. The vector carries a codon-optimized microdystrophin (µDys) transgene under the control of a ubiquitous CAG promoter. In preclinical studies using the mdx mouse model, systemic administration of NOVA-AAV9 demonstrated significant restoration of dystrophin expression (approximately 60%), improved muscle contraction force, and enhanced cardiac performance for up to 14 months post-treatment, highlighting its potential for long-term therapeutic efficacy in DMD patients.

Other names

NeddyAAV9-CAG-microdystrophinNeddyAAV-9-CAG-microdystrophinNeddyAAV 9-CAG-microdystrophin