AAV Technology: Delivery Vehicle of Choice for Gene Therapy

Adeno-associated viruses (AAVs) are attractive delivery vectors in the field of gene therapy. A team from the National Institutes of Health (NIH) developed AAV5-based vectors for delivering gene therapy products into parts of human bodies. Gene therapy is on the brink of becoming a common medical practice; however, developing safe and effective gene therapy products has been challenging. One major issue has been finding a delivery vector to target the diseased tissues in the body without devastating side effects. An AAV is a small virus that infects humans but is not known to cause disease and causes only a very mild immune response. These features make AAV an attractive delivery vector for gene therapy. In fact, the AAV-based vectors developed at the NIH present a popular choice for gene therapy. 

The AAV5 technology has been out-licensed extensively by the NIH Office of Technology Transfer (OTT). One example was a recent exclusive and nonexclusive combination license for the commercial development of AAV5-based gene therapy products. Under this agreement, Amsterdam Molecular Therapeutics (AMT) will have exclusive rights to develop treatments for a restricted number of disease indications, but nonexclusive rights to develop treatments for other diseases. As such, NIH OTT not only allows sufficient incentive for the expedited therapeutic development of AAV5 technology by one licensee, but also ensures the continued availability of AAV technology widely to future commercial partners. In support of the NIH’s public health mission, NIH OTT also agreed to reduce royalties when AMT collaborates with academic institutions on therapies for ultra orphan indications. 

This is just one approach that NIH OTT uses under its license agreements to provide incentives for companies targeting rare and neglected diseases, which broadens the application of NIH technologies to meet public health needs. The technology transfer effort of the NIH OTT has maximized the return on publicly funded research, and commercialization of the scientific discovery of AAV5 technology has advanced the public health mission of the NIH and will provide direct benefit to patients worldwide. 

Awardees

NIH - National Heart Lung and Blood Institute 

• Dr. Robert Kotin 
• Dr. John A. Chiorini