AAV technology and gene therapy
Our global team of research experts, scientists, and collaborators have pioneered gene therapy technology to help change the face of medicine. Our connection to the research community is essential to the discovery of new, innovative genetic technologies and our ability to bring medicines to patients across the globe.
AskBio labs are in Research Triangle Park, North Carolina, Edinburgh, Scotland, Heidelberg, Germany and San Sebastian, Spain. We also have partnerships with many leading research institutions. Our lab facilities, GMP manufacturing sites, and scientific capabilities cover a breadth of AAV technology processes, genetic engineering, and therapeutic development.
Science inspired by collaborative possibilities
The discoveries that our researchers and scientists have brought to life are helping to define how genetic medicine will be commercialized in the future. We bring decades of genetic expertise grounded in commercially validated technologies, some of which are used by the world’s largest biopharmaceutical companies.
Discover possibilities
Our collaborative spirit has driven gene therapy advancements and has culminated in a rich patent portfolio that we hope to translate into clinical success with our research partners.
We continually evolve our science to give every new discovery the chance it deserves to help make a difference in the lives of patients, and advance one of the most promising fields in medicine today – gene therapy.
Genetic technology
- Pioneering AAV technology including self-complementary vectors that are the basis for currently approved FDA-approved gene therapeutics
- Pro10™ cell-line that increases yields 2x-10x greater than most other transfection methods
- An expansive capsid library that is comprised of a wide range of novel chimeric constructs that can be customized for a variety of therapeutics
- Gene control technology for better precision of gene expression including tissue targeting, potency, and optimized cassette development
- Inducible promoter technology research to advance on/off control and to provide flexibility for gene regulation
- Bioanalytic technology and methods to create accurate genome sequences for advanced gene targeting
- neDNA™*, a synthetic benchtop alternative to plasmid DNA that reduces time to manufacture and potentially improves safety of the product due to the exclusion of bacterial components.
- GMP manufacturing facilities that use Pro10™ allow for clinical development and iteration and optimization of clinical candidates.
*Technology for making neDNA™ is licensed from Touchlight IP Ltd