What We’re Working On

Updated August 2019

ProgramMode of ActionDevelopment PhaseStatusKey Investigator / Affiliation
ASO w LaForaGlycogen synthesis inhibitorAnimal studyIn progress safety, dose completeIonis Pharmaceuticals
Guaiacol/TGM5Glycogen synthesis inhibitor/ membrane stabilizerEarly Clinical-ExUSCRO identified Open Label Phase 1/2IsrAPBDRf (patient sponsored)
Compound A
Hadassah
Aggregate (PB) reductionAnimal studyOngoing pre-IND studiesDr. Or Kakhlon
Hadassah-Hebrew Univ
AAV2/9 UTSW
w/CBh promotor
GBE StabilizerAnimal studyInitial study completedDr. Berge Minassian
UTSW
ASO for Intronic MutationGBE StabilizerAnimal studyStudy prepDr. Orhan Akman
Columbia Univ
Biomarker MRI PilotProof of ConceptAnimal studyIn life startedDr. Or Kakhlon
Hadassah-Hebrew Univ
QOL Patient SurveyNAPatient SurveyStudy complete, Publication in progressDr. Heather Lau
NYU