BridgeBio’s BBP-812 Gains the US FDA’s Regenerative Medicine Advanced Therapy Designation for Canavan Disease
Shots:
- The US FDA has granted RMAT designation to BBP-812 (AAV9 gene therapy) for Canavan disease, based on the 12mos. data from first 8 patients enrolled in P-I/II (CANaspire) study
- The P-I/II (CANaspire) trial assesses the safety, tolerability & PD of BBP-812 (IV) among pediatric patients with Canavan disease. 1EP is change in urine & CNS NAA levels from baseline along with motor function & development evaluation
- Results, to data, showed functional improvements in key parameters like head control, sitting upright, reaching for & grasping objects & visual tracking along with reduced NAA levels in both urine & CNS linked to mild disease. It was well-tolerated & had a safety profile aligning with other AAV9 gene therapies
Ref: BridgeBio | Image: BridgeBio
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
