Lysoway Therapeutics Secures Additional MJFF Funding to Advance TMEM175 Agonist Program for Parkinson’s Disease
Lysoway Therapeutics, a biopharmaceutical company focused on developing small-molecule therapies that target lysosomal ion channels, has announced that it has received an additional research grant from the renowned Parkinson’s disease advocacy and research organization, The Michael J. Fox Foundation for Parkinson’s Research. The new funding award, valued at approximately $3.4 million, will support the continued development of the company’s TMEM175 agonist program, a novel therapeutic approach aimed at addressing underlying disease mechanisms associated with Parkinson’s disease.
The grant was awarded through MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, an initiative dedicated to accelerating the development of promising therapies that have the potential to slow, halt, or modify disease progression. Through this program, the foundation seeks to advance scientific discoveries from the laboratory toward clinical evaluation, helping to bridge critical gaps in drug development for Parkinson’s disease.
The newly awarded funding will enable Lysoway to further advance its portfolio of highly brain-penetrant TMEM175 agonists through a series of preclinical and translational studies. The company believes that targeting TMEM175, a lysosomal ion channel genetically linked to Parkinson’s disease risk, could offer a new pathway for treating neurodegenerative disorders characterized by impaired cellular waste management and protein accumulation.
Expanding Research into a Promising Parkinson’s Disease Target
Parkinson’s disease remains one of the most common neurodegenerative disorders worldwide, affecting millions of people and creating a significant burden for patients, caregivers, and healthcare systems. While existing treatments primarily focus on managing symptoms, there remains a substantial need for therapies that address the underlying biological processes driving disease progression.
TMEM175 has emerged as an increasingly important target in Parkinson’s disease research. Genetic studies have identified variants in the TMEM175 gene as being associated with an elevated risk of developing Parkinson’s disease. Scientists believe the protein plays a critical role in maintaining proper lysosomal function, a cellular process essential for recycling damaged proteins, lipids, and other cellular components.
Lysosomes act as the cell’s waste disposal and recycling centers. When lysosomal function becomes impaired, toxic proteins and cellular debris can accumulate, contributing to neuronal dysfunction and degeneration. This process is widely believed to play a key role in the progression of Parkinson’s disease and several other neurodegenerative disorders.
Through activation of TMEM175, Lysoway aims to restore or enhance lysosomal activity, thereby improving cellular resilience and reducing the harmful effects associated with protein aggregation and cellular stress.
Support from The Michael J. Fox Foundation
According to representatives from MJFF, the organization’s Therapeutics Pipeline Program is specifically designed to support innovative scientific approaches that are grounded in a strong understanding of Parkinson’s disease biology.
Jessica Tome Garcia, Lead Scientific Program Manager for Translational Research at The Michael J. Fox Foundation, emphasized the importance of exploring biological targets such as TMEM175 and understanding how they influence disease-relevant cellular pathways.
She noted that the program seeks to evaluate promising therapeutic strategies that may ultimately contribute to future treatments for Parkinson’s disease. By supporting investigations into lysosomal biology and related pathways, the foundation hopes to deepen scientific understanding while fostering the development of potential disease-modifying therapies.
The additional grant reflects growing scientific interest in lysosomal dysfunction as a driver of neurodegeneration and highlights the increasing recognition of TMEM175 as a potentially valuable therapeutic target.
Advancing Preclinical and Translational Development
The funding will support a comprehensive research program aimed at evaluating the effects of pharmacologically activating TMEM175 across a variety of disease-relevant experimental models.
Researchers plan to investigate whether stimulation of the ion channel can improve lysosomal performance, regulate protein and lipid homeostasis, and enhance cellular responses to aging and stress-related conditions. These studies are expected to provide important mechanistic insights into how TMEM175 influences cellular health and whether its activation could produce meaningful therapeutic benefits.
In addition to mechanistic investigations, the research program will include efficacy studies designed to evaluate the therapeutic potential of Lysoway’s lead compounds. Data generated through these efforts are expected to support future regulatory submissions and contribute to the advancement of the company’s drug candidates toward human clinical trials.
The preclinical work will also help establish a stronger translational framework, ensuring that findings from laboratory studies can be effectively applied to future clinical development programs.
Building on Advances in Lysosomal Biology
Lysoway’s scientific strategy centers on targeting lysosomal ion channels, an area that has historically presented significant challenges for drug discovery. Developing small molecules capable of effectively modulating these channels while achieving adequate brain penetration has been a longstanding obstacle for researchers.
The company believes it has overcome many of these challenges through its proprietary structure-based drug discovery platform. Using advanced molecular design techniques, Lysoway has developed compounds that are both orally bioavailable and capable of efficiently reaching the brain, characteristics that are particularly important for therapies intended to treat central nervous system disorders.
Dr. Valerie Cullen, Principal Investigator and Senior Vice President of Research and Translation at Lysoway Therapeutics, expressed appreciation for the continued support from MJFF and highlighted the biological significance of TMEM175.
She noted that the ion channel plays an essential role in maintaining lysosomal pH balance, supporting autophagic processes, and preserving cellular resilience. Because disruptions in these processes are closely associated with Parkinson’s disease pathology, enhancing TMEM175 activity could represent a promising strategy for addressing disease progression rather than simply treating symptoms.
Dr. Cullen further emphasized that the company’s lead development candidate combines strong brain penetration with oral administration, characteristics that may improve patient convenience and therapeutic effectiveness if the program successfully reaches clinical development.
Strengthening the Therapeutic Pipeline
Lysoway’s leadership views the new grant as further validation of the company’s scientific approach and its broader efforts to translate discoveries in lysosomal biology into therapeutic opportunities.
Dr. Yongchang Qiu, Founder, Chief Executive Officer, and Co-Principal Investigator at Lysoway Therapeutics, stated that the funding underscores the growing recognition of TMEM175 as a compelling therapeutic target for Parkinson’s disease.
He explained that the advancement of an additional development candidate targeting a lysosomal ion channel demonstrates the capabilities of the company’s discovery platform and its ability to generate novel small-molecule therapeutics addressing complex biological mechanisms.
According to Dr. Qiu, the company intends to use the grant funding to further evaluate its lead TMEM175 agonist, develop translational biomarkers capable of measuring target engagement, and begin the critical studies required to support an Investigational New Drug (IND) application.
These IND-enabling studies represent a key milestone in the drug development process, helping establish the safety profile and pharmacological characteristics necessary before a therapy can be tested in human subjects.
The award marks another significant step forward for Lysoway Therapeutics as it works to advance innovative treatments for Parkinson’s disease. By focusing on TMEM175 and lysosomal biology, the company is pursuing a therapeutic strategy that targets fundamental cellular processes implicated in neurodegeneration.
With approximately $3.4 million in additional support from The Michael J. Fox Foundation, Lysoway is positioned to expand its preclinical research efforts, generate critical translational data, and move its lead candidate closer to clinical evaluation. The collaboration also highlights the broader commitment within the Parkinson’s research community to explore new biological pathways that may ultimately lead to disease-modifying therapies.
As scientific understanding of lysosomal dysfunction continues to grow, therapies designed to restore cellular recycling mechanisms and improve neuronal health may become increasingly important components of future treatment strategies. Through this latest funding award, Lysoway and MJFF aim to accelerate progress toward that goal, advancing research that could one day improve outcomes for individuals living with Parkinson’s disease worldwide.
Lysoway Therapeutics is a biotechnology company pioneering therapeutics targeting lysosomal ion channels for age-related neurodegenerative diseases. Leveraging structure-based drug design, high-resolution cryo-EM, and proprietary insights into lysosomal biology, Lysoway is developing highly brain-penetrant small-molecule modulators of TRPML1 and TMEM175 designed to restore autophagy–lysosomal function and cellular homeostasis.
