High Complexity AAV3 Capsid Library for Targeted Human Gene Therapy

Technology #15606

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Researchers
Sergei Zolotukhin
Damien Marsic
Managed By
April Kilburn
Assistant Director 352-392-8929
Patent Protection
PCT Patent Application WO2017/070476

Allows Efficient Development of Specialized Vectors for Targeted Gene Delivery

This adeno-associated virus (AAV) vector library combines tens of millions of actual capsid variants in an AAV3 backbone in order to allow the isolation of specialized variants with increased transduction efficiency and high specificity for particular organs or cell populations, as screening experiments based on this library showed. In 2012, worldwide cancer accounted for 8.2 million deaths and $124.6 billion dollars spent on cancer treatments. Gene therapy can be used in the treatment of infections, degenerative disorders, and cancer. An appropriate AAV vector library could make gene therapy attempts more successful, but available AAV libraries suffer from sequence bias or limited vector diversity, limiting their effectiveness. Researchers at the University of Florida have developed a highly complex AAV3 capsid library that can help improve the efficiency of gene therapy treatments. This AAV vector library results in more successful gene therapy by limiting off-target delivery and reducing immune response. In addition, it reduces production costs and increases patient safety, as less vector will be required to achieve therapeutic effect.

Application

Specialized AAV3 capsid variants that can target the human liver and other specific cell types to increase treatment efficiency and reduce production costs

Advantages

  • Makes library variations compatible with capsid structure, producing a high complexity of actual capsid variants
  • Targets gene delivery, allowing the generation of specialized vectors and decreasing the cost of treatments
  • Reduces off-target delivery, dosage and immune response, increasing the efficiency and safety of gene therapy

Technology

This AAV capsid library is based on an AAV3 backbone and includes rationally designed amino acid substitutions at 47 positions in 6 variable regions, with the actual capsid complexity ranging from tens to hundreds of millions.