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