ReGenX

Pipeline

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• Pipeline

• Metabolic Disorders
  • Familial Hypercholesteremia
  • Hurler Syndrome (MPS I)
  • Maroteaux-Lamy Syndrome (MPS VI)
• Hematologic Disorders
  • Hemophilia B
  • Hemophilia A
• Ocular Diseases
  • X-Linked Retinitis Pigmentosa
  • Age‐Related Macular Degeneration
  • Leber Congenital Amaurosis (LCA)
• Neurodegenerative Disorders
  • Batten Disease
  • Sanfilippo Syndrome (MPS IIIA)
  • Metachromatic Leukodystrophy
  • Alzheimer’s Disease

• REGENX Programs
• Licensed Programs
• Investigator Sponsored Programs

The therapeutic pipeline was designed to take advantage of the central properties of NAV™ Technology:

• Stable, long-term gene expression
• Highly efficient rates of gene delivery
• Efficient delivery of therapeutic genes to tissues whose cells are not actively dividing, such as the liver, retina, and brain
• Gene expression regulation that provides control of gene expression and the potential for greater safety and efficacy in certain disease populations.

Types of therapeutics in the current pipeline:

• Gene replacement/correction for inherited disorders
• One-time delivery of biologics for acquired diseases, with stable therapeutic protein expression

To learn more about the use of NAV Therapeutics research, development, and therapeutic deployment, please see the chart below. Click on the disease/disorder category to learn more.