What We’re Working On

Updated April 2021

ProgramMode of ActionDevelopment PhaseStatusKey Investigator & Affiliation
DRUG DEVELOPMENT
ASO w LaForaGlycogen synthesis inhibitorPlan to Start Ph1/2 for Lafora 2021For Lafora only in progress safety, dose completeIonis Pharmaceutical
ASO for Intronic Mutation ColumbiaGlycogen synthesis inhibitor/stabilizerAnimal POCPOC completed Publication in progress Ionis supplying ASO
Dr Orhan Akman
Columbia Univ
Guaiacol/TGM5Glycogen synthesis inhibitor/ membrane stabilizerpreparing for Clinical-Israelreformulating Guaiacol for sustained release GHF LLC
Dr Or Kakhlon
Hadassah Medical Center
TGM5GBE1 enzyme stabilization(membrane), mild synthesis inhibitorPOC ClinicalNeed 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 GHFGlycogen synthesis inhibitor or PB reductionADMET studies completedawainting fundingDr Or Kakhlon
Hadassah Medical Center
AAV2/9
w/CBh promotor
UTSW & Taysha		GBE StabilizerIND enablingneed to address 2nd dose immune responseBerge Minassian UTSW
Taysha Gene Therapies
AAV-9  GT
Duke		GBE StabilizerAnimal POCDose Range study using AAV-mGBEDuke
GSD molecule
Maze Therap		Glycogen synthesis inhibitorIND enablingstart FIH ’22 lead indication PompeMaze Therapeutics
BIOMARKER
CSF Biomarker UTSWBiomarkerClinical POCstudy designSouad Messahel Berge Minassian
UTSW
Joseph Carroll
Wisconsin
EARLY RESEARCH
AAV9 express of amylaseAggregate (PB) reductionEarly Researchin progressDr Felix Nitschke  UTSW
OTHER PHARM DEV ENABLING PROJECTS
QOL Patient Survey
NYU		Observational StudyAnalysis CompletePreparing PublicationDr Heather Lau,NYU