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

Friedreich’s Ataxia
PRECLINICAL

Rett Syndrome
PRECLINICAL

ALS SOD1
PRECLINICAL

FTD-GRN
PRECLINICAL

Synucleinopathies (GBA + α-Syn RNAi)
PRECLINICAL

CDKL5 Deficiency
RESEARCH

Undisclosed
RESEARCH

Temporal Lobe Epilepsy (TLE)
RESEARCH
Hematologic/Liver

Hemophilia A
PHASE I/II

Hemophilia A
PHASE I/II

OTC Deficiency
PHASE I/II

GSDIa
PHASE I/II

Wilson Disease
PRECLINICAL

Undisclosed
RESEARCH
Cardiac / Skeletal Muscle

XLMTM
PHASE III

Danon Disease
PHASE I/II

Pompe Disease
PHASE I/II

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.

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