UK-based start-up AlveoGene announced that its AVG-002 nebulized gene therapy for neonatal surfactant protein B (SP-B) deficiency has received a rare pediatric disease designation from the FDA. The company says that it will advance AVG-002 into clinical development and could submit a marketing application as soon as 2028.
According to Alveogene, studies in SP-B gene knock-out mice have demonstrated extended survival after a single dose of AVG-002. The company also reports that AVG-002 has restored lung function and normal structure in studies using lung tissues with induced disease.
AlveoGene Executive Chair David Hipkiss commented, “This rare pediatric disease designation (RPDD) for AVG-002 marks a significant step forward in our mission to develop transformative treatments for children with fatal genetic lung diseases. Receiving this RPDD not only underscores the urgent need for innovative therapies in this area but also highlights the potential of our approach in SP-B deficiency in newborn infants, which is designed to target the affected lung tissues directly, restore vital function and provide a viable therapeutic solution where presently there is none.”
Hipkiss added that the company thinks that its AVG-003 inhaled gene therapy for ABCA-3 deficiency could also qualify for the rare pediatric disease designation. He also noted “excellent preclinical progress” for AVG-001 for the treatment of alpha-1 antitrypsin deficiency.
Read the AlveoGene press release.
