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 |