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AAV9 BBP-812

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment26

FDA Designations

RMATORPHAN_DRUGRARE_PEDIATRIC_DISEASEFAST_TRACK

Clinical trial landscape

AAV9 BBP-812 · 1 trial · 1 indication

Phase 1 1
NCT04998396A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)Canavan Disease
RECRUITING26 Analytics
PHASE1RECRUITING
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Canavan Disease
NCT0499839626
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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
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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
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Eligibility Criteria

Age RangeN/A to 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
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Frequently asked questions about AAV9 BBP-812

What is AAV9 BBP-812 used for in Canavan Disease?

AAV9 BBP-812 is an investigational gene therapy being studied for the treatment of Canavan Disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by the FDA. The therapy is designed to address the underlying cause of the disease.

What does AAV9 BBP-812 target?

AAV9 BBP-812 is a gene therapy that uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional gene to cells. It is being developed to treat Canavan Disease by targeting the genetic defect responsible for the condition.

Who makes AAV9 BBP-812?

AAV9 BBP-812 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company. BridgeBio's stock is traded under the ticker symbol BBIO on the NASDAQ exchange.

What phase is AAV9 BBP-812 in?

AAV9 BBP-812 is currently in Phase 1 clinical trials. It is an investigational therapy and has not yet received FDA approval. The drug has been granted several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, and Fast Track.

What clinical trials is AAV9 BBP-812 in?

AAV9 BBP-812 is being evaluated in a Phase 1 clinical trial with the identifier NCT04998396, known as the CANaspire Clinical Trial. This study is recruiting participants with Canavan Disease in the United States and has an estimated enrollment of 26 participants.

Is AAV9 BBP-812 the same as BBP-812?

Yes, AAV9 BBP-812 is the same as BBP-812. The full name includes the AAV9 vector, which is the delivery system used in this gene therapy. It is being developed by BridgeBio Pharma for the treatment of Canavan Disease.