Partners & Licensees Pipeline

Partnering the NAV Technology Platform

PARTNERS & LICENSEES PIPELINE

SORT BY

PROGRAM AREA RESEARCH PRECLINICAL PHASE I/II PHASE III APPROVED LICENSEE PHASE
Central Nervous System

SMA

APPROVED

MPS IIIA
PHASE III

SMA Type II / III
PHASE I/II

Parkinson's with GBA mutation & Neuronopathic Gaucher
PHASE I/II

MPS IIIA
PHASE I/II

MPS IIIA
PHASE I/II

MPS IIIB
PHASE I/II

CLN1
PHASE I/II

Friedreich’s Ataxia
PRECLINICAL

Rett Syndrome
PRECLINICAL

ALS SOD1
PRECLINICAL

CLN3
PRECLINICAL

FTD-GRN
PRECLINICAL

Synucleinopathies (GBA + α-Syn RNAi)
PRECLINICAL

CDKL5 Deficiency
RESEARCH

Undisclosed
RESEARCH
Hematologic/Liver

Hemophilia A
PHASE I/II

Hemophilia A
PHASE I/II

OTC Deficiency
PHASE I/II

GSDIa
PHASE I/II

Crigler-Najjar
PHASE I/II

Wilson Disease
PRECLINICAL

PKU
RESEARCH
Cardiac / Skeletal Muscle

XLMTM
PHASE III

CPVT
PHASE I/II

Danon Disease
PHASE I/II

Pompe Disease
PRECLINICAL

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

9600 Blackwell Road, Suite 210, Rockville, MD 20850
240.552.8181

| | | | |

Follow Us
REGENXBIO_Logo-RGB

©2018 REGENXBIO Inc. “NAV®” is a registered trademark of REGENXBIO Inc. All rights reserved.

|

Recent Comments

    Archives

    Buy Themo

    Sign Up

    Receive updates to stay connected on REGENXBIO programs and our commitment to patients.

    Sign Up