AlveoGene’s AVG-0020 Secures the US FDA’s Rare Pediatric Disease Designation (RPDD) for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency
Shots:
- The US FDA has granted Rare Pediatric Disease Designation (RPDD) to AVG-002 for the treatment of lethal neonatal Surfactant Protein B (SP-B) deficiency
- Preclinical studies show significantly prolonged survival in SP-B gene knockout models with single dose of AVG-002 & restores normal lung function, offering a potential treatment. AlveoGene plans its clinical development, targeting marketing authorization by 2028
- AVG-002, developed by utilizing AlveoGene’s InGenuiTy platform, transfers a functional SP-B gene to the neonatal lung's alveolar region through a pseudotyped lentiviral vector for efficient respiratory instillation
Ref: AlveoGene | Image: AlveoGene
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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.
