Apertura Gene Therapy collaborates with NIH on Niemann-Pick disease treatment

Apertura Gene Therapy has partnered with two NIH institutes to advance preclinical gene therapy for Niemann-Pick disease Type C1. This multi-year Cooperative Research and Development Agreement focuses on using Apertura's proprietary capsid for intravenous delivery. The collaboration highlights a commitment to gene-based approaches for this severe pediatric lysosomal disorder.