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Canavan Disease

2
Pipeline Programs
3
Companies
4
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Aspa Therapeutics
Aspa TherapeuticsCA - Palo Alto
2 programs
1
AAV9 BBP-812Phase 1/21 trial
Natural History Study of Patients With Canavan Disease (CANinform Study)N/A1 trial
Active Trials
NCT04126005Completed67Est. Aug 2025
NCT04998396Recruiting26Est. Oct 2032
Myrtelle
MyrtelleMA - Wakefield
1 program
1
rAAV-Olig001-ASPAPhase 1/21 trial
Active Trials
NCT04833907Enrolling By Invitation24Est. Aug 2027
Eppendorf
EppendorfGermany - Hamburg
1 program
A Natural History Study of Canavan DiseaseN/A1 trial
Active Trials
NCT02851563Completed21Est. Apr 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Aspa TherapeuticsAAV9 BBP-812
MyrtellerAAV-Olig001-ASPA
Aspa TherapeuticsNatural History Study of Patients With Canavan Disease (CANinform Study)
EppendorfA Natural History Study of Canavan Disease

Clinical Trials (4)

Total enrollment: 138 patients across 4 trials

NCT04998396Aspa TherapeuticsAAV9 BBP-812

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Start: Sep 2021Est. completion: Oct 203226 patients
Phase 1/2Recruiting
NCT04833907MyrtellerAAV-Olig001-ASPA

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Start: Apr 2021Est. completion: Aug 202724 patients
Phase 1/2Enrolling By Invitation
NCT04126005Aspa TherapeuticsNatural History Study of Patients With Canavan Disease (CANinform Study)

Natural History Study of Patients With Canavan Disease (CANinform Study)

Start: Oct 2019Est. completion: Aug 202567 patients
N/ACompleted
NCT02851563EppendorfA Natural History Study of Canavan Disease

A Natural History Study of Canavan Disease

Start: May 2016Est. completion: Apr 201921 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 actively recruiting trials targeting 138 patients
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.