MJFF grant supports therapies for PRKN-related disease

$4.5 million award to Nine Square Therapeutics targets genetic form of Parkinson's

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded a $4.5 million grant to Nine Square Therapeutics to advance the development of potential treatments for a genetic form of Parkinson’s disease.

“The Michael J. Fox Foundation grant will go toward accelerating the identification of drug candidates that may one day deliver meaningful benefits to people with [Parkinson’s disease],” Tina Schwabe, PhD, vice president and head of biology at Nine Square Therapeutics, said in a press release.

Jessica Tome Garcia, PhD, associate director of research programs at MJFF, added: “Research in neurodegenerative disease is experiencing rapid innovation, and MJFF is pleased to support researchers at emerging companies like Nine Square that have a differentiated approach to drug and target discovery and are committed to ushering in a brighter future for patients around the globe.”

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This grant specifically will help to advance treatments for Parkinson’s associated with mutations in the PRKN gene. PRKN mutations are the most common cause of early-onset Parkinson’s, with patients often developing the disease before age 35.

This gene provides instructions for making parkin, an enzyme that plays a protective role in neurodegenerative disease by tagging defective mitochondria (the cells’ powerhouses) for degradation. By doing so, it prevents the accumulation of damaged structures within cells, maintaining cellular health.

As such, PRKN gene mutations disrupt the normal regulation of mitochondrial quality control, allowing for the toxic buildup of dysfunctional mitochondria inside cells. Neurodegeneration in Parkinson’s is thought to be associated with impaired functioning of mitochondria and problems in mitochondrial quality control.

Nine Square is developing small molecules that can boost the activity of the parkin enzyme, helping to clear away damaged mitochondria and preventing nerve cell death.

Seeking to improve mitochondrial function

“In preclinical testing, our Parkin activators have demonstrated the ability to promote mitochondrial turnover and improve mitochondrial function in cellular models of Parkinson’s disease,” Schwabe said.

The MJFF grant will be used to help further develop these small molecules, with the aim of identifying the best candidate to move into clinical testing as an experimental treatment.

“We look forward to using these funds to advance the nomination of our clinical candidate,” Schwabe said.

Just a few months ago, MJFF made another large investment in a different treatment strategy for PRKN-associated Parkinson’s. It awarded a grant to NysnoBio to help move NB001, its investigational gene therapy for this genetic form of Parkinson’s, into clinical testing.