Powering a New Field of Discovery

REGENXBIO is seeking to improve lives through the curative potential of gene therapy. Our NAV® Technology Platform has ushered in a new era of gene therapy innovation.

Therapeutic Programs

Retinal

Diseases

Metabolic

Diseases

Neurodegenerative

Diseases

Patients & Families

We are deeply committed to discovering and developing innovative gene therapies to improve treatment options for people with severe and debilitating diseases with significant unmet needs.

Gene Therapy    |    Wet AMD     |     HoFH     |     MPS I     |     MPS II

Partners & Licensees

Partnerships and licensing are a key part of REGENXBIO’s commitment to enabling the ongoing development of gene therapy treatments using our NAV Technology Platform.

What We Do

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes.

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