Lysoway Therapeutics, a Cambridge, Massachusetts-based biopharmaceutical company focused on lysosomal ion channel modulators, has received approximately USD 3.4 million from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance its TMEM175 agonist program toward clinical development for Parkinson's disease.
The award, made through MJFF's Parkinson's Disease Therapeutics Pipeline Program, will fund preclinical and translational studies of Lysoway's lead small-molecule TMEM175 agonists, which the company describes as orally bioavailable and highly brain-penetrant. TMEM175 encodes a lysosomal potassium channel genetically linked to Parkinson's disease risk, and its activation is hypothesized to restore lysosomal pH, autophagic capacity, and cellular homeostasis under conditions of aging and cellular stress.
Studies will be conducted across multiple disease-relevant models, with the stated goals of establishing translational target engagement biomarkers and initiating investigational new drug (IND)-enabling studies.
"TMEM175 is genetically linked to Parkinson's disease risk and plays a critical role in maintaining lysosomal pH, autophagic capacity, and cellular resilience," said Dr. Valerie Cullen, principal investigator and SVP of Research and Translation at Lysoway, in the company's announcement. "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."