Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc. is a biotechnology company that develops and commercializes therapies for rare and ultra-rare genetic diseases. Its product development pipeline, as described in its SEC filings, requires the “completion of extensive preclinical laboratory tests and preclinical animal studies” for its investigational gene therapies and biologics. This reliance on animal models is a standard, mandatory component of the regulatory approval process for novel pharmaceuticals, particularly for complex biologics like its adeno-associated virus (AAV) gene therapy candidates.

The company’s FDA submissions for product candidates, such as the UX111 gene therapy Biologics License Application, include data from nonclinical animal studies. These studies are conducted to establish safety and efficacy profiles required by regulators before human trials can commence. As a clinical-stage biopharmaceutical company, Ultragenyx’s core business model is inherently tied to animal testing as part of its research, development, and regulatory compliance activities.