A CDMO partnership shows how gene therapy manufacturing is scaling for chronic disease
Dr. Dereck Mush, MD, MBA
Ultragenyx Pharmaceutical
Rare-disease biotechnology company behind the first FDA-approved gene therapy for pediatric MPS IIIA.
What they do
Ultragenyx develops therapies for rare and ultrarare diseases. FDA approved Fayuvi, a one-time intravenous AAV9 gene therapy for eligible pediatric patients with MPS IIIA, on September 17, 2026.
The record
- Vertical
- Pharma & Drug Discovery
- Sub-segment
- Rare disease gene therapy
- Product type
- Service
- Stage
- Public
- Headquarters
- Novato, CA, USA
- Region
- North America
- Founded
- 2010
- Total funding
- Undisclosed
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