Cambridge, MA, July 25, 2025 – Lysoway Therapeutics, a biopharmaceutical company developing small molecule modulators of lysosomal ion channels, today announced that it has received a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF). Support comes from MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, which focuses on candidates with strong potential to slow or halt disease progression or alleviate burdensome symptoms for those living with Parkinson’s disease. Lysoway Therapeutics funding of $2.93 million will support the preclinical and translational development of Lysoway’s novel, highly brain-penetrant small molecule TRPML1 agonist.
The study aims to investigate whether activating TRPML1 by a novel, small molecule modulator, will enhance the lysosomal membrane calcium ion channel to restore lysosomal function and help with clearance of alpha-synuclein, the protein that is linked to the disease.
“We are honored to receive this generous grant from The Michael J. Fox Foundation,” said Valerie Cullen, PhD, Principal Investigator and SVP of Research and Translation at Lysoway. “TRPLML1 is a high value target due to its pivotal role in sensing and responding to cellular stress. By activating this ion channel, we can engage multiple beneficial pathways that restore autophagy/lysosomal homeostasis and bolster cellular resilience. Our lead development candidate is both orally bioavailable and highly brain-penetrant, offering strong potential to modify disease progression in Parkinson’s Disease.”
Yongchang Qiu, PhD, Founder and Chief Executive Officer of Lysoway Therapeutics, added “This funding underscores growing confidence in TRPML1 as a compelling target for Parkinson’s disease. It will allow us to accelerate development of our lead TRPML1 agonist and to establish key biomarkers for target engagement, with the goal of initiating first-in-human clinical trials early next year.”
About Lysoway Therapeutics
Lysoway Therapeutics is a leader in lysosomal ion channel disease biology. We have developed unique technological approaches to screen and develop potent modulators of lysosomal ion channels, including TRPML1 and TMEM175. These ion channels serve as vital transducers of cellular signals and play crucial roles in maintaining cellular homeostasis, particularly within the autophagy/lysosomal pathway, which is frequently disrupted in a number of pathological conditions. Lysoway’s small molecule modulators hold immense potential for restoring autophagy/lysosomal function, offering potential treatment for neurodegenerative diseases associated with lysosomal deficiency and other rare diseases characterized by toxic accumulation of cellular wastes.
Contact Info
info@lysoway.com
www.lysoway.com