Patients & Families

Why Gene Therapy?

Gene therapy is a treatment that involves the introduction, removal, or change in genetic material in a person’s cells.

REGENXBIO’s gene therapies are being investigated in clinical trials and are designed to deliver working genes to cells using adeno-associated virus (AAV) vectors as delivery “vehicles.” AAV vectors are modified viruses that cannot increase their numbers or reproduce themselves and are not known to cause disease in humans.

Our investigational gene therapies all use viral vectors from our proprietary gene delivery platform, which we call the NAV® Technology Platform. Thousands of patients have received AAV gene therapies derived from our NAV Technology Platform.