Recombinant Adeno-Associated Virus (AAV) Vector Capsid for Efficient Gene Therapy

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The Novel Technology:

 
The technology relates to recombinant adeno-associated virus (AAV) vector serotype, wherein the capsid protein of AAV serotypes is mutated at single or multiple sites. Further, this technology relates to an improved transduction efficiency of mutant AAV serotypes (AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9 and AAAV10).
 
Applications:
  • To use novel AAV vectors in clinical trials
  • To use novel vectors to improve gene delivery for many human diseases in pre-clinical or clinical trials for gene therapy (eg: AAV3-liver target diseases, AAV8-hemophilia)
Advantages:
  • Increased transduction efficiency from the novel AAV mutant vectors translates into enhanced therapeutic benefit in patients undergoing AAV-mediated gene-therapy
  • Broader applicability in the gene therapy field for various diseases
  • Lower cost of gene therapy due to administration of low doses of vectors
  • Promotion of safety of the AAV vectors by limited dose-dependent immune-toxicity seen with conventional WT-AAV vectors
Business Model:
 
The technology is at early-stage with proof-of-concept in-vivo studies in mice showing increase in transduction efficiency of mutant AAV serotypes. Looking for industrial partners or licensee(s) for taking the technology further.
 
Principal Investigator(s): Dr. Jayandharan, CMC
 
Invention ID: CMP-022
 
IP Status: Patent Pending
 
Jurisdictions: India (IN)
 
For more details, please write to:
techtransfer [at] ccamp.res.in