TORONTO, Ontario & CAMBRIDGE, Mass.—ProMIS Neurosciences Inc., a biotechnology company focused on the discovery and development of precision treatments for neurodegenerative diseases, recently announced the identification of a novel therapeutic epitope target on misfolded forms of TDP43, implicated in the development and progression of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The company filed a provisional patent application for this target with the United States Patent Office on May 30, 2017.

 

Commenting on the announcement, Dr. Neil Cashman, chief scientific officer of ProMIS, stated: “Using our proprietary discovery engine, scientists at UBC and ProMIS identified both the sequence and conformational shape of this novel epitope target on misfolded TDP43 that plays a key role in ALS and frontotemporal dementia.”

 

TDP43 is present in every cell, and plays a critical role in the response of cells to oxidative stress. However, in ALS, FTD and other neurodegenerative diseases, TDP43 can lose its normal function, forming intracellular aggregates of misfolded TDP43 that disrupt cellular energy generation and normal protein degradation.

 

“Our goal is to selectively target misfolded TDP43 without disrupting the critical role that normally folded TDP43 plays in cell biology,” stated Dr. Elliot Goldstein, ProMIS president and CEO. “We plan to create and validate monoclonal antibodies against misfolded TDP43 to select optimal therapeutic candidates for advancement into the clinic.”