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Benzinga
Benzinga
Technology
Vandana Singh

BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease

  • BridgeBio Pharma Inc (NASDAQ:BBIO) announced promising pharmacodynamic data from the first two participants dosed in CANaspire Phase 1/2 trial of BBP-812, gene therapy for Canavan disease. 
  • Canavan disease is an ultra-rare inherited disorder that damages the ability of nerve cells (neurons) in the brain to send and receive messages.
  • Data from the first two participants show rapid and robust post-treatment decreases in N-acetylaspartate (NAA) in urine and, significantly, in cerebrospinal fluid (CSF) and brain tissue, to a degree not seen in available natural history data. 
  • Also Read: BridgeBio Pharma Posts Early Data From Neurological Movement Disorder Candidate.
  • Reduction in brain NAA is an early signal suggesting that BBP-812 administered IV has reached its target behind the blood-brain barrier and is expressing functional aspartoacylase enzyme. 
  • There is evidence in the scientific literature that lower NAA levels are associated with milder disease. 
  • BBP-812 has been well-tolerated, and no participants have experienced a treatment-related serious adverse event. 
  • The company said that while the data reported are still early and the final safety and efficacy profile of the investigational gene therapy remains to be fully established.
  • BridgeBio believes these data show the potential of BBP-812.
  • Price Action: BBIO shares are up 6.65% at $8.50 during the market session on the last check Wednesday.
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