Our Mission

At Vanguard Clinical Rare Disease Foundation, our mission is to accelerate the development and delivery of innovative therapies for ultra-rare pediatric patients, by reimagining the drug development process itself—making it faster, more efficient, and infinitely scalable. We believe the process is our product: a revolutionary approach where the methods used to create therapies are as transformative as the treatments themselves. We challenge conventional timelines, break through systemic barriers, and push the boundaries of what’s possible. By embracing creative ideas, innovative science, and bold regulatory strategies, our vision is to deliver treatments at the speed of need. By thinking beyond traditional models and forging new pathways, we turn the impossible into reality. For families waiting on hope, we make hope happen—faster, smarter, and without limits.

Donate

Inquiries

Email: info@vcrdf.org

Press

Vanguard Clinical Rare Disease Foundation Launches Development of VCRDF-CLN3, a Precision ASO Therapy for CLN3 Batten Disease
June 30, 2025