December 6, 2021

Arkuda Therapeutics Awarded Michael J. Fox Foundation Grant to Accelerate Development of Compounds for Parkinson’s Disease

Watertown, MA – December 6, 2021 – Arkuda Therapeutics, a biotechnology company applying new insights at the intersection of lysosomal and microglial biology to develop medicines to change the trajectory of neurodegenerative disease, today announced that it has received a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to study the effects of its compounds on cellular and animal models of Parkinson’s disease (PD), including models of Parkinson’s with GBA1 mutations (PD-GBA).

“It is an honor to have the support of The Michael J. Fox Foundation in our efforts to develop novel therapies for Parkinson’s disease, a devastating neurological condition affecting millions of patients,” said Gerhard Koenig, PhD, Co-Founder, President and CEO of Arkuda Therapeutics. “Growing understanding of the significance of reduced beta-glucocerebrosidase activity in the pathophysiology of PD as well as in other neurodegenerative diseases suggests that this may be a promising avenue for treatment, both for PD patients with GBA mutations and potentially for the broader PD population. Our work will provide critical insights into these questions, and we hope that this research will support the advancement of new medicines into clinical development for this disease.”

MJFF funding will support preclinical studies in multiple cellular and in vivo models to evaluate the ability of Arkuda compounds to increase beta-glucocerebrosidase (GCase) activity as well as their effects on other relevant biomarkers.

Previously released data demonstrated that a novel Arkuda small molecule increases the enzymatic activity of GCase, which plays a key role in normal lysosome function and is essential for the health of cells in the central nervous system. Mutations in the GBA1 gene result in a deficiency in GCase activity and collectively are the most common known genetic risk factors for PD, occurring in an estimated 10% of patients with the disease. Therapeutics that increase GCase activity may offer potential for the treatment of PD, for which no disease-modifying therapies currently exist.


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About Arkuda Therapeutics
Arkuda Therapeutics is a biotechnology company applying insights into lysosomal and microglial biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead program aims to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. Beyond progranulin, Arkuda’s compounds enhance the activity of several other key lysosomal enzymes, including GCase, and the company is further exploring the therapeutic potential of its molecules in Parkinson’s disease patients with GBA1 mutations (PD-GBA) and other neurodegenerative diseases. Arkuda is backed by leading life science investors including Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and Mission BioCapital. To learn more visit


Investor Contact:
Andrew Funderburk
Kendall Investor Relations


Media Contact:
Lissette Steele
Verge Scientific Communications


Lissette Steele
Verge Scientific Communications