MJFF backs Lysoway's TMEM175 Parkinson's therapy with $3.4M grant
What's the deal? The Michael J. Fox Foundation for Parkinson's ResearchDealroom has a profile for this one. Try Dealroom → (MJFF) has awarded Lysoway TherapeuticsDealroom has a profile for this one. Try Dealroom → $3.4 million to develop a therapy targeting the TMEM175 protein — an ion channel critical for maintaining the acidity of lysosomes, the cellular structures that clear waste and recycle damaged molecules.
The grant falls under MJFF's Parkinson's Disease Therapeutics Pipeline Program, which funds potential disease-modifying therapies that can prevent, halt, or delay progression.
Lysoway is building a small-molecule, brain-penetrant agonist designed to restore TMEM175 function, fix lysosomal pH, and help neurons clear toxic clumps of misfolded alpha-synuclein — a hallmark of Parkinson's disease. The funding will help advance the company's lead candidate, establish biomarkers for target engagement, and kick off preclinical studies required before human trials.
Why now? Mutations in the TMEM175 gene have been identified as a genetic risk factor for Parkinson's. When lysosomal acidity is disrupted, the autophagy process that normally clears misfolded proteins breaks down, allowing toxic alpha-synuclein to accumulate and kill dopamine-producing neurons.
"TMEM175 is genetically linked to Parkinson's disease risk and plays a critical role in maintaining lysosomal pH, autophagic capacity, and cellular resilience," said Valerie Cullen, principal investigator and senior vice president of research and translation at Lysoway. She added that the company's lead candidate is "both orally bioavailable and highly brain-penetrant," addressing long-standing challenges in targeting lysosomal ion channels.
This is Lysoway's second MJFF grant. It previously received $2.93 million to advance a therapy targeting TRPML1, a protein that regulates calcium signalling in autophagy — bringing its total MJFF funding to roughly $6.3 million.
What could go wrong? The therapy is still preclinical, meaning it hasn't been tested in humans. Many drug candidates fail to clear IND-enabling studies, and even those that do face long timelines and high attrition rates in clinical trials. Targeting lysosomal ion channels in the brain remains a technically complex endeavour with limited precedent.
The signal: Lysoway's cumulative $6.3 million from MJFF — a non-profit investor that funds disease-modifying research rather than symptom-management drugs — underscores a broader shift in Parkinson's funding toward lysosomal biology and root-cause therapeutics. For an early-growth company with no clinical-stage assets yet, repeated non-dilutive grants from the field's most prominent funder provide both validation and runway to derisk preclinical programmes before seeking venture capital.
Read more: parkinsonsnewstoday.com
