Partners & Licensees Pipeline

Partnering the NAV Technology Platform

PARTNERS & LICENSEES PIPELINE

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

SMA Type I

PHASE III

SMA Type II / III

PHASE I/II

MPS IIIA

PHASE I/II

MPS IIIA

PHASE III

MPS IIIA

RESEARCH

MPS IIIB

PHASE I/II

Rett Syndrome

RESEARCH

ALS SOD1

RESEARCH

ALS SOD1

RESEARCH

CLN1 Disease

RESEARCH

CLN3 Disease

RESEARCH

Parkinson’s with GBA

PRECLINICAL

CDKL5 Deficiency

PRECLINICAL

Undisclosed

PRECLINICAL
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

Citrullinemia Type I

PRECLINICAL

PKU

PRECLINICAL

Wilson Disease

PRECLINICAL
Cardiac / Skeletal Muscle

XLMTM

PHASE I/II

CPVT

PHASE I/II

Danon Disease

PHASE I/II

Pompe Disease

RESEARCH

Friedreich’s Ataxia

PRECLINICAL
Retina

Achromatopsia

PRECLINICAL

Choroideremia

PRECLINICAL
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