Aspa Therapeutics, Inc.
A biotechnology subsidiary focused on developing an AAV-based gene therapy to treat Canavan disease. The company advances preclinical and clinical programs, runs a natural history study, and engages with patient advocacy groups and the wider scientific community to support clinical development.
Industries
N/A
Products
Investigational AAV gene therapy for Canavan disease (clinical program)
A program delivering an investigational AAV-based therapy encoding ASPA intended to correct the underlying genetic defect in Canavan disease; currently administered as a one-time intravenous infusion within a clinical trial framework.
Investigational AAV gene therapy for Canavan disease (clinical program)
A program delivering an investigational AAV-based therapy encoding ASPA intended to correct the underlying genetic defect in Canavan disease; currently administered as a one-time intravenous infusion within a clinical trial framework.
Services
Investigational AAV gene therapy clinical trial for Canavan disease
Clinical program administering a one-time IV AAV-based gene therapy to eligible pediatric patients with ASPA deficiency, including screening, inpatient infusion, scheduled assessments and multi-year follow-up.
CANInform natural history study for Canavan disease
Longitudinal natural history study that collects retrospective medical records and prospective clinical data to characterize disease progression and support clinical development.
Educational webinars and scientific presentations
Public-facing webinars and conference presentations describing the gene therapy approach, clinical program updates, and scientific background for families and clinicians.
Investigational AAV gene therapy clinical trial for Canavan disease
Clinical program administering a one-time IV AAV-based gene therapy to eligible pediatric patients with ASPA deficiency, including screening, inpatient infusion, scheduled assessments and multi-year follow-up.
CANInform natural history study for Canavan disease
Longitudinal natural history study that collects retrospective medical records and prospective clinical data to characterize disease progression and support clinical development.
Educational webinars and scientific presentations
Public-facing webinars and conference presentations describing the gene therapy approach, clinical program updates, and scientific background for families and clinicians.
Expertise Areas
- Gene therapy development (AAV-based)
- Clinical trial design and conduct for rare genetic neurologic diseases
- Natural history study design and data collection
- Preclinical animal model testing
Key Technologies
- AAV-based gene delivery
- AAV9 serotype screening
- Intravenous (IV) vector administration
- Mouse disease models