Chaperone Therapeutics Partners With ALS Therapy Development Institute for ALS Research

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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The ALS Therapy Development Institute has just signed a research partnership with Chaperone Therapeutics, Inc., a company that specializes in treatments that address protein misfolding and neuronal cell loss, the underlying causes of neurodegenerative disease. The new partners will be working on potential treatments for amyotrophic lateral sclerosis (ALS), in order to facilitate preclinical development of lead product candidates.

“Like many neurodegenerative disease indications, ALS appears to be a protein misfolding disorder,” said Steve Perrin, Ph.D., CEO and CSO of the ALS Therapy Development Institute. “The accumulation of these errant proteins results in cellular stress in motor neurons, loss of innervation of skeletal muscle, and paralysis in ALS patients. We are excited about the partnership with Chaperone. These lead molecules could be first in class treatments for neurodegenerative diseases such as ALS.”

Chaperone Therapeutics’ preclinical studies on small molecules have established a base at the ALS Therapy Development Institute’s facility in Cambridge, Massachusetts. Together the two companies have created cell-based assays capable of predicting treatments’ ability to address protein misfolding, preserve neurons from stress-related death and assess the treatment’s effect on ALS disease progression. The Institute is determined to maximize what Chaperone has to offer in ALS with its novel technology, and will jointly publish findings in a peer-reviewed journal as soon as the results are available.

“Protein misfolding is the cause of many neurodegenerative diseases, and data from a number of disease models demonstrate efficacy through enhancing the cellular protein folding capacity,” said Dennis J. Thiele, Ph.D., CSO at Chaperone Therapeutics. “We are delighted to work closely with the outstanding team at the ALS Therapy Development Institute to test our molecules that increase protein chaperone activity, as a potential disease modifying therapy for ALS.”

The ALS Therapy Development Institute and Chaperone Therapeutics will hold a webinar about the research partnership on August 18 at 2:00 PM ET. To register, you can visit

Researchers at the University of Massachusetts Medical School in the United States and the Babraham Institute in the United Kingdom recently revealed a new fly model to study motor neuron degeneration. The study was published in the journal Current Biology and is entitled “Age-Dependent TDP-43-Mediated Motor Neuron Degeneration Requires GSK3, hat-trick, and xmas-2”. The team reports the development of a new model to study neurodegeneration based on the common fruit fly (Drosophila melanogaster), specifically focusing on the neurons in the fly’s leg.