Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)
AskBio is a pioneering gene therapy company dedicated to developing life-saving treatments for genetic diseases. With a focus on AAV gene therapy technology, AskBio aims to improve patient outcomes through innovative research, technology, and collaborations. The company is committed to transforming medicine and changing lives by advancing genetic technology and providing curative therapies for rare and complex diseases.
Industries
Nr. of Employees
large (251-1000)
Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)
20 T.W. Alexander Drive, Suite 110, Research Triangle Park, NC 27709, USA
Patents
Products
Clinical-stage GDNF gene therapy for Parkinson’s disease (AAV-delivered)
A clinical-stage AAV-delivered gene therapy encoding GDNF evaluated in a Phase 1b study to assess safety and preliminary efficacy following targeted intracerebral delivery.
AAV-based FKRP gene therapy candidate for LGMD2I/R9 (clinical trial)
An investigational systemic AAV gene therapy designed to restore FKRP enzyme activity for the treatment of limb-girdle muscular dystrophy type 2I/R9; being evaluated in a randomized, placebo-controlled, dose‑escalation clinical trial.
Preclinical AAV CYP46A1 gene therapy program for Huntington’s disease
Preclinical AAV program to increase CYP46A1 expression in the brain with proof-of-concept reported in animal models and regulatory orphan designation in the EU.
Proprietary producer cell line for suspension AAV manufacturing
A high-yield suspension producer cell line intended for AAV vector production to increase stability and overall therapeutic output in manufacturing processes.
Synthetic promoter design platform
A platform combining bioinformatics and experimental validation to produce cell-selective and inducible regulatory elements for gene therapy payloads and manufacturing optimization.
Oligonucleotide-regulated gene-editing control system
A gene-control approach using oligonucleotide small molecules to regulate in vivo gene-editing components and transgene expression, intended to provide temporal control and reduce off-target activity.
Clinical-stage GDNF gene therapy for Parkinson’s disease (AAV-delivered)
A clinical-stage AAV-delivered gene therapy encoding GDNF evaluated in a Phase 1b study to assess safety and preliminary efficacy following targeted intracerebral delivery.
AAV-based FKRP gene therapy candidate for LGMD2I/R9 (clinical trial)
An investigational systemic AAV gene therapy designed to restore FKRP enzyme activity for the treatment of limb-girdle muscular dystrophy type 2I/R9; being evaluated in a randomized, placebo-controlled, dose‑escalation clinical trial.
Preclinical AAV CYP46A1 gene therapy program for Huntington’s disease
Preclinical AAV program to increase CYP46A1 expression in the brain with proof-of-concept reported in animal models and regulatory orphan designation in the EU.
Proprietary producer cell line for suspension AAV manufacturing
A high-yield suspension producer cell line intended for AAV vector production to increase stability and overall therapeutic output in manufacturing processes.
Synthetic promoter design platform
A platform combining bioinformatics and experimental validation to produce cell-selective and inducible regulatory elements for gene therapy payloads and manufacturing optimization.
Oligonucleotide-regulated gene-editing control system
A gene-control approach using oligonucleotide small molecules to regulate in vivo gene-editing components and transgene expression, intended to provide temporal control and reduce off-target activity.
Services
Manufacture of clinical‑grade AAV vectors under GMP standards, including process development, technology transfer and operation of production facilities.
Collaborative, bioinformatics-driven design and optimization of synthetic promoters for cell-type specificity and regulated transgene expression.
Access to engineered capsid libraries and collaborative support for capsid selection and vector optimization to meet tissue targeting and transduction requirements.
Alternative DNA supply for AAV production using enzymatic DNA workflows intended to reduce reliance on plasmid DNA and animal‑origin materials.
Collaborative development of non-viral nanocapsule delivery approaches for intracellular and nuclear delivery of therapeutic cargoes, intended to improve delivery precision and support potential repeat dosing.
Program-based support for early-phase clinical trial design, operational execution and regulatory interactions, including assistance pursuing regulatory designations.
Manufacture of clinical‑grade AAV vectors under GMP standards, including process development, technology transfer and operation of production facilities.
Collaborative, bioinformatics-driven design and optimization of synthetic promoters for cell-type specificity and regulated transgene expression.
Access to engineered capsid libraries and collaborative support for capsid selection and vector optimization to meet tissue targeting and transduction requirements.
Alternative DNA supply for AAV production using enzymatic DNA workflows intended to reduce reliance on plasmid DNA and animal‑origin materials.
Collaborative development of non-viral nanocapsule delivery approaches for intracellular and nuclear delivery of therapeutic cargoes, intended to improve delivery precision and support potential repeat dosing.
Program-based support for early-phase clinical trial design, operational execution and regulatory interactions, including assistance pursuing regulatory designations.
Expertise Areas
- AAV vector and capsid engineering
- Synthetic promoter design and regulatory elements
- GMP AAV manufacturing and process transfer
- Proprietary suspension producer cell line development
Key Technologies
- AAV vector engineering
- Capsid engineering and selection libraries
- Self-complementary AAV vectors
- Proprietary suspension producer cell lines
News & Updates
AskBio announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The trial is evaluating safety and preliminary efficacy, with ongoing enrollment.
AskBio announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The trial is evaluating safety and preliminary efficacy, with ongoing enrollment.