Cure GM1 funded pre-clinical studies and IND-enabling studies for AAV9 intravenous GM1 gene therapy being conducted by investigators at UMass Medical School and Auburn University.
Additional GM1 Research and Pre-Clinical Programs
Funded by sources other than Cure GM1 Foundation
Syner-G Clinical Study
There is clinical study being conducted at University of Minnesota where the ketogenic diet is combined with a medicine called miglustat/Zavesca. Unfortunately, this regimen is not considered a cure. The regimen may only slow the course of GM1 for certain patients. It is thought to possibly be effective for certain genetic mutations or only Juvenile patients or those with residual enzyme activity. Please refer to the description on the clinical trials web site.
BioStrategies, LLC Enzyme Replacement Therapy
IntraBio Inc is pleased to announce significant progress in the treatment of rare neurodegenerative diseases with IB1000, a well-tolerated, modified amino acid that is administered orally. Most notably, IB1000 is showing promising effects in pre-clinical and Phase 1 observational clinical studies in patients with late-onset Tay-Sachs (TS) disease. These findings are also supported by results from in vitro cellular studies and studies in a Sandhoff animal model. In addition, patients with Niemann-Pick Type C (NPC) disease, and various subtypes of Cerebellar Ataxias (CA) have been treated with IB1000 and observed symptomatic benefit. A multi-national, randomized, controlled Phase 2/3 clinical trial of IB1000 in 108 CA patients was recently completed, with the final results expected in 2018.
IntraBio is now planning multi-national randomized, controlled pivotal clinical trials to evaluate the safety and efficacy of IB1000 as a therapeutic intervention in patients with TS, NPC and specific CA diseases. IntraBio hopes to commence enrollment in one or more of these pivotal trials in the EU in early 2018 to be followed by recruitment in North America. In these trials, the safety and efficacy of IB1000 will be determined for each indication using validated clinical and quality of life assessment scales. IntraBio will provide full details of clinical site locations and the clinical trial design including inclusion criteria as soon as they are available.
IntraBio is a global leader developing novel therapies and treatments for “orphan” and neurodegenerative diseases, including lysosomal storage disorders, which have high unmet medical needs. IntraBio has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for two drug compounds, including IB1000 for the treatment of Niemann-Pick Types A, B & C diseases.
Chairman of IntraBio Inc.