GM1 Research Overview June 2015

Although GM1 is a rare disease, thankfully, promising research is ongoing.  Scientists are working on developing a cure.

At this time, the 2 most active categories of GM1 research are small molecules and gene therapy. This post enumerates some of the startups and research groups working on GM1.

Be aware that this post is time-sensitive.  Worse, in biotech, there is a term called the “valley of death.”  This particular term refers to research that may have been promising, but ultimately does not progress to clinical trials primarily due to lack of funding.  At the time this post was written, all the entities listed are still active.

Many, many researchers have contributed to a body of work over a period of decades worldwide to better understand GM1 Gangliosidosis.  We gratefully acknowledge all the researchers work, but not every researcher is listed below.

This is a recent report published in February 2015 that highlights challenges with respect to GM1 gene therapy and also provides some recent background information on various approaches: Gene therapy for GM1 gangliosidosis: challenges of translational medicine

Gene Therapy Research

Lysogene, a fast growing biopharmaceutical company developing IND-supporting pre-clinical studies in GM1-gangliosidosis using adeno-associated virus (AAV) gene therapy technology.  Lysogene is collaborating with UMass Medical School and Auburn University and aiming for its first clinical trial in 2017.

Small Molecule Research

At this time, all the small molecule treatments are pre-clinical.

Minoryx Therapeutics, located in Barcelona, developing a pharmacological chaperone through its See-Tx platform

Division of Functional Genomics, Research Center for Bioscience & Technology, Faculty of Medicine, Tottori University, Yonago, Japan

The Carbohydrate Bioorganic Chemistry Research Group at University of Seville

Dorphan, a company dedicated to the development of drugs for rare and orphan diseases founded by Sanfilippo Foundation Switzerland

Enzyme Replacement Therapy Research

BioStrategies LC, Lectin-assisted transnasal delivery of corrective enzyme for GM1 gangliosidosis

If you are aware of other treatments being developed or startups that may be relevant, please contact us.

GM1 Research Overview June 2015
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