Turbocharging gene delivery with AAV-booster technology

Section: 

   KEYWORDS

  •   Adeno-associated virus (AAV)
  •   Interaction
  •   Cell nucleus
 

 

 

 

 

 

 

Description  

Adeno-associated virus (AAV) has become the gene delivery vector of choice in both basic research and clinical development. However, because AAV particles are relatively ineffective at transducing most cell types, high titers are required for efficient gene transfer. This in turn raises production costs substantially.

We have developed technology to dramatically increase AAV infectivity and drive down costs. Our approach relies on chemical modification of the viral coat to increase its interaction with cells. The technology can be applied to any AAV vector post-production, reducing the amount of virus required for gene delivery by more than tenfold.

Scientific Background

Through chemical modification of the viral capsid, we increase binding of the virus to cell surface carbohydrates. This in turn improves transduction through increased internalization and transportation of AAV particles to the cell nucleus.

Results

We have tested the technology with several viral serotypes in vitro (cell culture) and in vivo (injections in mouse). We are currently quantifying efficiency increases across different serotypes. Upon completion, the technology is ready to be marketed.

Advantages and Innovative features

There are enormous challenges in producing AAV in sufficient quantities for efficient gene transfer. This is reflected in the multitude of different manufacturing platforms that are used for AAV production and purification. The advantage of our technology is it can be easily applied to any AAV produced using any of these platforms. This means that AAV suppliers or users are not restricted to a specific serotype or production method, and any AAV they have in stock can be easily modified. The results are a significant increase in gene transfer efficiency, which translates into major savings in production time and cost.

Potential Applications / Target Companies

Potential targets include AAV users/scientists, AAV production core facilities (TIGEM, ICGEB, UNC Vector Core, Penn Vector Core, UZH Viral Vector Facility etc), and AAV production companies (Vector Biolabs, Vigene Biosciences, GeneCopoeia, Creative Biogene etc).

Scientific Director

Prof. Paul Heppenstall