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BridgeBio?Pharma: Early CANaspire Trial Results Show Potential Of BBP-812

BridgeBio?Pharma, Inc. (BBIO) reported promising pharmacodynamic data from the first two participants dosed in CANaspire, a phase 1/2 clinical trial of BBP-812, an investigational gene therapy for the treatment of Canavan disease. The company said the initial pharmacodynamic results for two participants show unprecedented decreases in N-acetylaspartate in the brain and urine, suggesting the therapy is producing functional ASPA enzyme.

From a safety standpoint, IV infusions of BBP-812 have been well-tolerated. No participants have experienced a treatment-related serious adverse event.

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