Recent Updates
Recently added Catalysts

AAV9 BBP-812

Phase 1

Canavan Disease | Monoclonal antibody | Other |BridgeBio Pharma, Inc.|Last Updated: Apr 17, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment26
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04998396A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)PHASE1 RECRUITING 26 — — Sep 8, 2021Oct 8, 2032Apr 17, 20264 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Number of Participants with Adverse Events (AEs)
Baseline up to Week 52
Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels
Baseline, Month 12
Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)
Baseline, Month 12
Secondary Endpoints
Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88
Baseline, Week 52
Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4
Baseline, Week 52
Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4
Baseline, Week 52
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)EXPERIMENTALParticipants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)EXPERIMENTALParticipants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
Enrollment Expansion Phase: BBP-812EXPERIMENTALParticipants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.
Interventions
NameTypeDescription
AAV9 BBP-812BIOLOGICALSterile solution for injection for 1-time use via volumetric infusion pump
Unlock Study Design Details
Eligibility Criteria
Age RangeN/A — 30 Months
SexALL
Healthy VolunteersNo
Study Sites4

Key Inclusion Criteria: * Maximum age for inclusion is 30 months. * Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan ...

Countries:United States
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04998396primaryCompletionDate: changed
LOWMay 24, 2026NCT04998396studyFirstPostDate: changed