CAMBRIDGE, Mass. — Lysoway Therapeutics said it has received an additional research grant from The Michael J. Fox Foundation for Parkinson’s Research to support development of its TMEM175 agonist program for Parkinson’s disease.
The Cambridge, Mass.-based biopharmaceutical company is developing small-molecule modulators of lysosomal ion channels. The new funding, totaling about $3.4 million, will support preclinical and translational work on Lysoway’s brain-penetrant small-molecule TMEM175 agonists.
The grant was awarded through the foundation’s Parkinson’s Disease Therapeutics Pipeline Program, which supports therapeutic candidates that may modify disease progression and improve the lives of people with Parkinson’s disease.
“The Therapeutics Pipeline Program is designed to support the evaluation of promising therapeutic approaches grounded in Parkinson’s biology,” said Jessica Tome Garcia, lead scientific program manager of translational research at The Michael J. Fox Foundation for Parkinson’s Research. “We are interested in better understanding targets like TMEM175 and their role in lysosomal function, and how these pathways may inform future therapeutic strategies.”
Lysoway said the funded studies are designed to evaluate whether activating TMEM175 can affect lysosomal function and protein and lipid homeostasis, as well as how those effects may influence cellular responses tied to aging and cellular stress.
The company said mechanistic and efficacy studies will be conducted across multiple disease-relevant models to support future regulatory submissions and potential clinical development.
“We are honored to receive this generous support from The Michael J. Fox Foundation,” said Valerie Cullen, Ph.D., principal investigator and senior vice president of research and translation at Lysoway Therapeutics. “TMEM175 is genetically linked to Parkinson’s disease risk and plays a critical role in maintaining lysosomal pH, autophagic capacity, and cellular resilience. Our lead development candidate is both orally bioavailable and highly brain-penetrant, addressing key challenges historically associated with targeting lysosomal ion channels for neurodegenerative diseases.”
Yongchang Qiu, Ph.D., co-principal investigator, founder and CEO of Lysoway Therapeutics, said the funding supports broader interest in TMEM175 as a potential Parkinson’s disease target.
“This funding further reinforces the growing recognition of TMEM175 as a promising therapeutic target for Parkinson’s disease,” Qiu said. “The advancement of a second development candidate targeting a lysosomal ion channel further highlights the capabilities of our structure-based discovery platform and our ability to translate complex lysosomal biology into small molecules approaches for therapeutic investigation. With MJFF’s support, we plan to advance the evaluation of our lead TMEM175 agonist, establish translational target engagement biomarkers, and initiate IND-enabling studies.”
