Cure GM1 Associated Research

Projects in which Cure GM1 participated in funding – $512K in total funding provided

2017 Dorphan Small Molecule Research and Mouse Study

Cure GM1 most’s recent grant was for a drug repurposing project and mouse study to study a small molecule drug in collaboration with a small biotech company called Dorphan located in Switzerland.

2017 UC Davis Lentiviral Gene Therapy Research

Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using stem cells.  The research is being conducted at University of California Davis.

2016 Cyclodextrin GM1 Pilot Study

GM1 Cyclodextrin Pilot Study Initiated

2015-2017 Auburn/UMass Research AAV9 IV Gene Therapy

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.

About IntraBio

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.

Mallory Factor

Chairman of IntraBio Inc.