BioTech IP Review

Patent Title: Gene therapy for treating propionic acidemia
Patent Number: US 12472268 B2
Filing Date: October 1, 2019 (priority filing)
Patent Issue Date: November 18, 2025 (grant date)
Assignee: Ultragenyx Pharmaceutical Inc.

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What This Patent Covers

This patent protects gene‑therapy vectors and methods for treating propionic acidemia (PA) — a rare, life‑threatening metabolic disorder:

• Recombinant AAV Vectors: The invention describes adeno‑associated virus (AAV) vectors engineered to deliver functional copies of genes PCCA or PCCB, which encode subunits of the enzyme propionyl‑CoA carboxylase. Lack of this enzyme causes PA.
• Therapeutic Use: These vectors are formulated into pharmaceutical compositions for gene therapy, enabling long‑term expression of functional enzyme in patients’ cells to correct the underlying metabolic defect rather than just manage symptoms.
• Methods of Administration: The patent includes methods of administering these gene‑therapy vectors to patients to effectively treat or prevent manifestations of PA, a severe and otherwise hard‑to‑treat pediatric condition.

In essence, the patent claims AAV gene‑delivery technology designed to permanently address the genetic cause of propionic acidemia.

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Why This Patent Is Important

1) First‑in‑Class & High Unmet Need
Propionic acidemia has no broadly effective curative therapies today. A successful gene therapy that corrects the genetic defect could dramatically improve outcomes and become a premium rare‑disease therapy with strong pricing power.

2) Strong Patent Protection Provides Competitive Advantage
The granted U.S. patent gives Ultragenyx broad IP coverage over its gene‑therapy approach for PA. This exclusivity can be leveraged to secure market leadership, support premium reimbursement, and potentially license or cross‑license with partners.

3) Platform Importance in Gene Therapy Portfolio
Ultragenyx’s pipeline includes multiple gene‑therapy programs (e.g., UX111 for Sanfilippo syndrome type A and DTX401 for GSD Ia). A PA gene therapy expands the company’s portfolio of one‑time curative candidates, which investor analysts often value highly in biotech strategic assessments.