Findings show improved lysosomal function, reduced inflammation, and neuroprotection in Alzheimer’s and Parkinson’s disease models
Cambridge, MA, April 8, 2025 – Lysoway Therapeutics, a biotechnology company pioneering small-molecule agonists that restore lysosomal ion channel function to treat neurodegenerative diseases, today announces the presentation of new preclinical data for its lead brain-penetrant TRPML1 agonist at the AD/PD™ 2025 International Conference on Alzheimer’s & Parkinson’s Diseases, held April 1–5, 2025, in Vienna, Austria and online.
The poster, titled “A Novel, Brain-Penetrant TRPML1 Agonist Ameliorates Pathology, Inflammation, and Neurodegeneration in Mouse Models of Parkinson’s Disease and Alzheimer’s Disease,” is presented on April 2–3 (Poster #140). The data highlight the therapeutic potential of targeting lysosomal ion channels as an upstream strategy to restore autophagy-lysosomal pathway function — a core pathological driver across multiple neurodegenerative diseases.
Key findings presented include:
- Evidence of robust brain penetration and direct TRPML1 activation
- Restoration of lysosomal function, including improved cargo processing and reduced lysosomal stress
- Significant reductions in neuroinflammatory markers and proteinopathy-related pathology
- Demonstrated neuroprotection in both Parkinson’s and Alzheimer’s disease mouse models
“These results provide strong support for TRPML1 activation as a new therapeutic modality for neurodegenerative diseases,” said Yongchang Qiu, Ph.D., Founder & CEO of Lysoway Therapeutics. “By targeting lysosomal ion channels upstream of many pathological processes, we aim to develop treatments that fundamentally modify disease progression rather than address downstream symptoms.”
Lysoway continues to advance its TRPML1 agonist toward Phase I clinical readiness, alongside its TMEM175 agonist program for Parkinson’s disease and platform expansion across additional lysosomal ion channel targets.
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
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