What We’re Working On

Updated April 2021

Program	Mode of Action	Development Phase	Status	Key Investigator & Affiliation
DRUG DEVELOPMENT
ASO w LaFora	Glycogen synthesis inhibitor	Plan to Start Ph1/2 for Lafora 2021	For Lafora only in progress safety, dose complete	Ionis Pharmaceutical
ASO for Intronic Mutation Columbia	Glycogen synthesis inhibitor/stabilizer	Animal POC	POC completed Publication in progress	Ionis supplying ASO Dr Orhan Akman Columbia Univ
Guaiacol/TGM5	Glycogen synthesis inhibitor/ membrane stabilizer	preparing for Clinical-Israel	reformulating Guaiacol for sustained release	GHF LLC Dr Or Kakhlon Hadassah Medical Center
TGM5	GBE1 enzyme stabilization(membrane), mild synthesis inhibitor	POC Clinical	Need POC Study Design & review w FDA	GHF LLC Dr Or Kakhlon Hadassah Medical Center
Compound A Hadassah & GHF	Aggregate (PB) reduction	small Compassionate Use study in Isrl	awaitng IRB approval	GHF LLC Dr Or Kakhlon Dr A Lassos Hadassah Medical Center
CompoundsD&E GHF	Glycogen synthesis inhibitor or PB reduction	ADMET studies completed	awainting funding	Dr Or Kakhlon Hadassah Medical Center
AAV2/9 w/CBh promotor UTSW & Taysha	GBE Stabilizer	IND enabling	need to address 2nd dose immune response	Berge Minassian UTSW Taysha Gene Therapies
AAV-9 GT Duke	GBE Stabilizer	Animal POC	Dose Range study using AAV-mGBE	Duke
GSD molecule Maze Therap	Glycogen synthesis inhibitor	IND enabling	start FIH ’22 lead indication Pompe	Maze Therapeutics
BIOMARKER
CSF Biomarker UTSW	Biomarker	Clinical POC	study design	Souad Messahel Berge Minassian UTSW Joseph Carroll Wisconsin
EARLY RESEARCH
AAV9 express of amylase	Aggregate (PB) reduction	Early Research	in progress	Dr Felix Nitschke UTSW
OTHER PHARM DEV ENABLING PROJECTS
QOL Patient Survey NYU	Observational Study	Analysis Complete	Preparing Publication	Dr Heather Lau,NYU