Ultragenyx grew revenues more than 25% on a year-over-year basis in 2024 and should see close to 20% sales growth in 2025. The company's Biologic License Application, or BLA, was accepted by the FDA in December for its AAV gene therapy to treat Sanfilippo syndrome type A (MPS IIIA), a rare and lifelong genetic disorder. This would mark the company's sixth approved therapy.