Brainvectis
AskBio is a pioneering gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. With a focus on AAV gene therapy, the company advances genetic technology to treat a wide range of diseases, including Parkinson's disease, neuromuscular disorders, and more. AskBio's mission is to transform medicine and change lives through innovative research, technology, and collaboration.
Industries
Nr. of Employees
small (1-50)
Brainvectis
20 T.W. Alexander Drive, Suite 110, Research Triangle Park, NC 27709, USA
Patents
Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
US-12138298-B2
View Details
Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
US-12138298-B2
View DetailsProducts
Capsid and promoter libraries for AAV vectors
Extensive collections of capsid variants and promoter/expression cassettes for selecting tissue tropism and controlling transgene expression.
High-yield suspension cell-line process for AAV production
A suspension cell-line-based manufacturing process optimized to increase AAV output per batch and reduce manufacturing costs.
Enzymatic DNA production (plasmid alternative)
Enzymatically produced DNA intended as an alternative to plasmid DNA to reduce use of animal-origin materials and lower cost in AAV manufacturing workflows.
Regulated gene-editing platform with oligonucleotide control
A gene-editing system that uses viral delivery combined with an oligonucleotide-based on/off switch to modulate gene-editing protein activity and limit off-target effects and immunogenicity.
Capsid and promoter libraries for AAV vectors
Extensive collections of capsid variants and promoter/expression cassettes for selecting tissue tropism and controlling transgene expression.
High-yield suspension cell-line process for AAV production
A suspension cell-line-based manufacturing process optimized to increase AAV output per batch and reduce manufacturing costs.
Enzymatic DNA production (plasmid alternative)
Enzymatically produced DNA intended as an alternative to plasmid DNA to reduce use of animal-origin materials and lower cost in AAV manufacturing workflows.
Regulated gene-editing platform with oligonucleotide control
A gene-editing system that uses viral delivery combined with an oligonucleotide-based on/off switch to modulate gene-editing protein activity and limit off-target effects and immunogenicity.
Services
On-site Good Manufacturing Practice production of AAV vectors, incorporating high-yield suspension cell-line processes and alternative DNA production methods to support clinical and commercial supply.
Partnership-based support for discovery and translational research including capsid and promoter screening, preclinical vector development, and translational study design.
Design and execution of early-phase gene therapy clinical trials, safety monitoring, long-term follow-up and support for publication of clinical results.
On-site Good Manufacturing Practice production of AAV vectors, incorporating high-yield suspension cell-line processes and alternative DNA production methods to support clinical and commercial supply.
Partnership-based support for discovery and translational research including capsid and promoter screening, preclinical vector development, and translational study design.
Design and execution of early-phase gene therapy clinical trials, safety monitoring, long-term follow-up and support for publication of clinical results.
Expertise Areas
- AAV gene therapy development
- Capsid engineering and tropism optimization
- Promoter and expression cassette engineering
- GMP manufacturing and scale-up for viral vectors
Key Technologies
- Adeno-associated virus (AAV) vector engineering
- Capsid libraries and chimeric capsids
- Self-complementary AAV vectors
- Promoter engineering and inducible expression cassettes
News & Updates
AskBio published complete results of its Phase 1b trial of AB-1005, a gene therapy for Parkinson’s disease, showing it was well tolerated and associated with stability and improvement in motor function.
An overview of how AAV vectors work in gene therapy, their safety, and their application in treating genetic diseases.
In February 2025, AB-1005 was granted RMAT designation by the FDA, recognizing its potential in regenerative medicine.
AskBio published complete results of its Phase 1b trial of AB-1005, a gene therapy for Parkinson’s disease, showing it was well tolerated and associated with stability and improvement in motor function.
An overview of how AAV vectors work in gene therapy, their safety, and their application in treating genetic diseases.
In February 2025, AB-1005 was granted RMAT designation by the FDA, recognizing its potential in regenerative medicine.