What We’re Working On

Updated August 2019

Program Mode of Action Development Phase Status Key Investigator / Affiliation
ASO w LaFora Glycogen synthesis inhibitor Animal study In progress safety, dose complete Ionis Pharmaceuticals
Guaiacol/TGM5 Glycogen synthesis inhibitor/ membrane stabilizer Early Clinical-ExUS CRO identified Open Label Phase 1/2 IsrAPBDRf (patient sponsored)
Compound A
Hadassah
Aggregate (PB) reduction Animal study Ongoing pre-IND studies Dr. Or Kakhlon
Hadassah-Hebrew Univ
AAV2/9 UTSW
w/CBh promotor
GBE Stabilizer Animal study Initial study completed Dr. Berge Minassian
UTSW
ASO for Intronic Mutation GBE Stabilizer Animal study Study prep Dr. Orhan Akman
Columbia Univ
Biomarker MRI Pilot Proof of Concept Animal study In life started Dr. Or Kakhlon
Hadassah-Hebrew Univ
QOL Patient Survey NA Patient Survey Study complete, Publication in progress Dr. Heather Lau
NYU