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
Nr. of Employees
small (1-50)
Aspa Therapeutics, Inc.
421 Kipling St., Palo Alto, CA 94301
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
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.
Longitudinal natural history study that collects retrospective medical records and prospective clinical data to characterize disease progression and support clinical development.
Public-facing webinars and conference presentations describing the gene therapy approach, clinical program updates, and scientific background for families and clinicians.
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.
Longitudinal natural history study that collects retrospective medical records and prospective clinical data to characterize disease progression and support clinical development.
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
News & Updates
Webinar featuring the scientific founder and an expert panel providing background on gene therapy and the clinical development program.
Scientific publication describing metabolic pathway relevance and preclinical findings related to N-acetylaspartate and Canavan disease.
Preclinical study reporting immune findings and systemic pathology in an AAV gene therapy mouse model.
Preclinical report demonstrating efficacy of late postnatal IV AAV administration in mouse models.
Announcement that the first clinical trial participant received the investigational AAV-based gene therapy and was discharged from the hospital research unit as planned.
Webinar featuring the scientific founder and an expert panel providing background on gene therapy and the clinical development program.
Scientific publication describing metabolic pathway relevance and preclinical findings related to N-acetylaspartate and Canavan disease.
Preclinical study reporting immune findings and systemic pathology in an AAV gene therapy mouse model.
Preclinical report demonstrating efficacy of late postnatal IV AAV administration in mouse models.
Announcement that the first clinical trial participant received the investigational AAV-based gene therapy and was discharged from the hospital research unit as planned.