BridgeBio Pharma Shares Preliminary Data From Gene Therapy Candidate In Ultra Rare Disorder

BridgeBio Pharma Inc BBIO presented pharmacodynamic, tolerability, and preliminary functional efficacy data from the first three participants dosed in the CANaspire Phase 1/2 trial of BBP-812.
BBP-812 is an investigational intravenous gene therapy for Canavan disease, an ultra-rare neurodevelopmental disorder.
The disease is caused by an inherited mutation of the ASPA gene that codes for aspartoacylase, a protein that breaks down a compound called N-acetylaspartate (NAA).
At Month 3 post-treatment, Participant 3 showed an 89% reduction of NAA in cerebrospinal fluid (CSF) and a 45% reduction in urine NAA.
In addition, Participant 2 showed a continued decline in urine NAA at Month 6, with an 85% decrease compared to pre-treatment.
In June, BridgeBio Pharma reported, at Month 6 post-treatment, Participant 1 showed a 77% reduction of NAA in CSF, a 15% reduction in NAA in brain white matter by MRS imaging, and a 45% reduction in urine NAA.
At Month 3 post-treatment, Participant 2 showed an 89% reduction of NAA in CSF, a 53% reduction in NAA in brain white matter by MRS imaging, and an 81% reduction in urine NAA.
BBP-812 has been well-tolerated by all 3 participants, with no reported treatment-related serious adverse events.
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