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.

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