Partners & Licensees Pipeline

Partnering the NAV Technology Platform

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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
CLN1 Disease							RESEARCH
CLN3 Disease							RESEARCH
Parkinson’s with GBA							PRECLINICAL
CDKL5 Deficiency							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
PKU							PRECLINICAL
Wilson Disease							PRECLINICAL
Cardiac / Skeletal Muscle XLMTM							PHASE I/II
CPVT							PHASE I/II
Danon Disease							PHASE I/II
Pompe Disease							RESEARCH
Retina Achromatopsia							PRECLINICAL
Choroideremia							PRECLINICAL