PARTNERS & LICENSEES PIPELINE PARTNERING THE NAV TECHNOLOGY PLATFORM PARTNERS & LICENSEES PIPELINE SORT BY All Program Areas Central Nervous System Hematologic/Liver Cardiac / Skeletal Muscle All Licensees Novartis Ultragenyx Lilly Esteve Lysogene/Sarepta Ultragenyx/Bayer Takeda Rocket Pharma Astellas Pfizer Corlieve Therapeutics PROGRAM AREA RESEARCH PRECLINICAL PHASE I/II PHASE III APPROVED LICENSEE Central Nervous System SMA MPS IIIA SMA Type II / III Parkinson's with GBA mutation & Neuronopathic Gaucher MPS IIIA Friedreich’s Ataxia Rett Syndrome ALS SOD1 FTD-GRN Synucleinopathies (GBA + α-Syn RNAi) CDKL5 Deficiency Undisclosed Temporal Lobe Epilepsy (TLE) Hematologic/Liver Hemophilia A Hemophilia A OTC Deficiency GSDIa Wilson Disease Undisclosed Cardiac / Skeletal Muscle XLMTM Danon Disease Pompe Disease Zolgensma® is approved in the U.S. for the treatment of pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene. SIGN UP Receive updates to stay connected on REGENXBIO programs and our commitment to patients. Subscribe CONTACT US 9804 Medical Center Drive, Rockville, MD 20850 240.552.8181 General Info | Medical Info | Patient Advocacy | Partners | Investors | Media FOLLOW US ©2021 REGENXBIO Inc. “NAV®” is a registered trademark of REGENXBIO Inc. All rights reserved. Privacy Policy | Terms and Conditions