News

Cambridge, MA, November 17, 2025 – Lysoway Therapeutics is pleased to announce that Yongchang Qiu, Founder and CEO, has been invited to speak at the 14th Annual Alzheimer’s & Parkinson’s Drug Development Summit in February 2026, a premier global forum bringing together scientific leaders, industry innovators, and clinicians advancing next-generation therapies for neurodegenerative diseases.

Dr. Qiu will deliver a featured seminar titled:

“Lysosomal Ion Channels as a New Therapeutic Class in Neurodegeneration: Advancing TRPML1 and TMEM175 Small-Molecule Agonists”
Thursday, February 5, 2026 – 1:30 PM

His presentation will highlight Lysoway’s pioneering work in developing first-in-class small-molecule agonists targeting lysosomal ion channels—an emerging and highly promising therapeutic strategy in neurodegeneration. Key topics will include:

• Scientific and genetic rationale for targeting TRPML1 and TMEM175 in Alzheimer’s and Parkinson’s disease
• Development considerations and translational insights gained while advancing Lysoway’s highly brain-penetrant, Phase 1–ready TRPML1 agonist
• Emerging preclinical data supporting our lead TMEM175 agonist as a genetically validated therapeutic for Parkinson’s disease

Lysosomal ion channels play a critical upstream role in regulating autophagy, lysosomal function, protein/lipid clearance, and overall cellular resilience—pathways broadly impaired in age-related neurodegenerative disorders. Lysoway’s differentiated, brain-penetrant small-molecule agonists aim to restore these essential mechanisms and deliver transformative benefits to patients.

“We are honored to share our latest scientific progress at this year’s Alzheimer’s & Parkinson’s Drug Development Summit,” said Yongchang Qiu, Founder & CEO of Lysoway Therapeutics. “The strong convergence of genetic, mechanistic, and translational evidence underscores the potential of TRPML1 and TMEM175 agonists as a new therapeutic class capable of addressing the upstream drivers of neurodegeneration.”

For more information about the conference, visit the 2026 Alzheimer’s & Parkinson’s Drug Development Summit website.
To learn more about Lysoway’s platform and programs, please visit www.lysoway.com.

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

Cambridge, MA, October 16, 2025 – Lysoway Therapeutics’ Senior Vice President and Head of Research and Translational Science, Valerie Cullen, PhD, today presented an invited talk and participated in a panel discussion at the 17th Michael J Fox Foundation’s Annual Parkinson’s Disease Therapeutics Conference (PDTC) in New York City. This prestigious conference is the only scientific meeting worldwide focused exclusively on Parkinson’s disease drug development. Each year, it brings together over 300 professionals to showcase the most exciting and innovative research from MJFF’s research portfolio.

Dr. Cullen delivered a presentation entitled “Development of a Potent, Selective, and Brain-Penetrant TRPML1 Agonist for Parkinson’s Disease Treatment” and also participated in a panel discussion focused on “Exploring Innovative Therapeutic Approaches for Endolysosomal Dysfunction in PD”.

Her presentation highlighted Lysoway’s compelling data on its Development Candidate, LW-1017, which is a highly brain penetrant, orally bioavailable, and potent small-molecule TRPML1 agonist. She presented robust in vivo efficacy data demonstrating a significant reduction of alpha-synuclein aggregation and inflammation, improved neuronal cell survival and augmented motor function in highly relevant PD models; outlined key results from our successfully completed IND-enabling studies; and confirmed plans to initiate a First-in-Human Phase I clinical study of LW-1017 in healthy volunteers beginning in 2026.

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

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

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

info@lysoway.com
www.lysoway.com

Cambridge, MA, May 30, 2024 – Lysoway Therapeutics, a leading discovery-stage biotech company dedicated to developing next-generation treatments for patients with neurodegenerative diseases, today proudly announces the appointment of Valerie Cullen, PhD as Senior Vice President and Head of Research and Translational Science.

Dr. Cullen brings a wealth of cross-functional experience as a research leader, translational medicine expert and company builder and has a proven track record of success in building and translating innovative therapeutic programs.

“We are thrilled to welcome Valerie to the executive leadership team of Lysoway Therapeutics,” said Yongchang Qiu, CEO, President and Co-Founder of the company. “Valerie brings deep expertise in lysosomal biology and neurological disease and has led complex CNS drug development programs at the intersection of research, translation and clinical development. Her leadership will be invaluable to Lysoway as we build our pipeline and navigate key milestones in 2024 and beyond.”

Dr. Cullen brings almost 25 years of operational and leadership experience in research and development, with particular emphasis on drug discovery for neurodegenerative disorders. Most recently, she served as SVP of Research at Expansion Therapeutics, where she built the research group and helped raise over $80M in VC funding. Prior to that, she served as Vice President, Translation and Development at Generian Pharmaceuticals, Inc. At Lysosomal Therapeutics, Inc. Dr. Cullen was Vice President, Program and Alliance Management and acted as program lead for a clinical-stage asset in Parkinson’s Disease. She previously held roles of increasing responsibility in NeuroPhage, Aldeyra Therapeutics and Link Medicine.

Dr. Cullen earned a PhD and BSc (Hons) in pharmacology from the University of Dublin, Ireland and held postdoctoral fellowships at the Institute of Psychiatry, King’s College London and at the Center for Neurologic Diseases, Harvard Medical School. Her work has won various accolades including the Annals of Neurology Clinical Impact Award and she has been granted numerous patents worldwide for innovative therapies for neurological diseases.

“I’m delighted to join Lysoway’s executive team at this exciting juncture, as the organization advances towards delivering new treatment options for patients with neurodegenerative diseases,” said Dr. Cullen. “I look forward to making significant and meaningful contributions towards the company’s development of novel therapeutics with the potential to highly impact patient lives.”

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

Cambridge, MA, April 2, 2024 – Lysoway Therapeutics, a leading discovery-stage biotech company dedicated to developing next-generation treatments for patients with neurodegenerative diseases, today proudly announces the appointment of Chris Adams, PhD to its board of directors. Dr. Adams brings a wealth of experience as a distinguished business leader, entrepreneur and mentor, with a proven track record of guiding biotechnology companies towards maximizing program value and realizing their full potential.

“We are thrilled to welcome Chris to the Lysoway board of directors as we advance our pipeline into clinical studies next year,” said Yongchang Qiu, CEO & President, Co-Founder of Lysoway Therapeutics. “Chris’s extensive industry expertise and strategic guidance will be invaluable to Lysoway as we bolster our team and navigate key milestones in 2024 and beyond.”

Dr. Adams is a strategic biotech executive with over three decades of broad cross-functional expertise spanning all facets of biotech operations. His impressive background includes in-depth experience with start-up operations, fundraising, M&A, business development, strategic transactions, and company building. In 2013, he co-founded Cydan, an orphan drug accelerator focused on improving the lives of patients with rare genetic diseases. Together with the Cydan team, he successfully raised over $200M and co-founded  and served on the Board of three startups: Vtesse, Imara and Tiburio. Prior to Cydan, he served as the Chief Business Officer of FoldRx Pharmaceuticals Inc., where he played a pivotal role in the development of VYNDAQEL® (tafamidis) for TTR amyloidosis, leading to its acquisition by Pfizer in 2010. Previously, he held various business development and marketing roles at  ViaCell Inc., TKT Inc. and Ciba-Geigy AG. Currently Dr. Adams serves on the Board of Directors of Alkeus Pharmaceuticals Inc., Aviceda Therapeutics Inc. and Reveal Pharmaceuticals Inc. He holds a PhD in organic chemistry and a diploma in organic chemistry and biochemistry from the University of Zurich, and an MBA from INSEAD of Fontainebleau, France.

“It is a privilege to join Lysoway’s board as the organization advances towards delivering new treatment options for patients with neurodegenerative diseases, many of whom have been significantly underserved for decades,” said Dr. Adams “I look forward to working alongside this team and advancing a promising pipeline through development and towards the market.”

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

Cambridge, MA and New York, NY, Jan. 5, 2023 – Lysoway Therapeutics, a startup biotech developing small molecule modulators of lysosomal ion channels and leader in lysosomal ion channel disease biology, announced today receiving a research grant from the Silverstein Foundation for Parkinson’s with GBA.

“We appreciate the generous support from the Silverstein Foundation, which underlines the importance of lysosomal ion channels as drug targets for Parkinson’s disease.” said Dr. Yongchang Qiu, chief executive officer of Lysoway Therapeutics. “This grant and our relationship with the Silverstein Foundation will bring us closer to the Parkinson’s disease research community and expedite our overall translational research effort. Lysoway plans to use the grant to investigate the therapeutic potential of its channel modulators in preclinical models of GBA-deficient Parkinson’s disease, particularly focusing on discovery and development of target engagement and pharmacodynamic biomarkers.”

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 are essential transducers of cellular signals and are required to help maintain cellular homeostasis, especially the delicate balance within the autophagy/lysosomal pathway, which is often disrupted in a number of pathological conditions. Lysoway’s small molecule modulators have great potential to re-balance lysosomal function and autophagy to treat neurodegenerative diseases with lysosomal deficiency and rare diseases characterized by toxic accumulation of cellular wastes.

About The Silverstein Foundation for Parkinson’s with GBA

The Silverstein Foundation is a 501(c)(3) non-profit organization focused on investing in cutting-edge therapeutic approaches for the treatment and prevention of Parkinson’s disease in glucocerebrosidase (GBA-PD) mutation carriers. The Foundation collaborates with clinicians, scientists, and biotechnology companies to accelerate research and clinical trials in an effort to rapidly bring new disease-modifying therapeutic options to patients. The Foundation has funded 39 projects across a diverse set of therapeutic approaches since its inception in 2017. The Foundation applies a unique flexible funding model including both new company formation and traditional research grants to deliver in real-time on its mission of rapidly developing novel treatments for GBA-PD patients.

Contact Info

info@lysoway.com
www.lysoway.com

info@silversteinfoundation.org
www.silversteinfoundation.org

Lysoway Therapeutics, a Cambridge, MA based startup developing small molecule modulators of lysosomal ion channels, announced today raising a total of $22m in Series A financing since founding, led by Highlight Capital, 3E Bioventures, and Oceanpine Capital. Lysoway was founded in 2020 by CEO and biotech veteran Yongchang Qiu and Professor Haoxing Xu (University of Michigan, Zhejiang University), who is leading the frontier in lysosomal biology.

“We appreciate Highlight Capital, 3E Bioventures, and Oceanpine Capital’s strong support and confidence in our therapeutic approach and experienced team,” said Yongchang Qiu, chief executive officer of Lysoway Therapeutics.  “Lysosomal deficiency has long been associated with many neurodegenerative diseases and metabolic disorders, both genetically and phenotypically. Lysosomal ion channels play key roles in the regulation of lysosome function and yet these channels have been neglected as therapeutic targets up until now.  Having developed unique technological approaches to screen and develop potent modulators of lysosomal ion channels, we see tremendous potential to apply the breadth of our scientific expertise to advance the field. We are looking forward to accelerating the development of our lysosomal ion channel modulators to bring novel and best-in-class therapies to patients”.

Professor Xu added, “The lysosome, the cell’s recycling center, can mediate the degradation of a variety of biomaterials (proteins, lipids, and membranes) into smaller building-block molecules, which will be subsequently transported out of lysosomes for reutilization or energy.  Activation of lysosomal channels by synthetic modulators, like those being developed at Lysoway, may boost lysosome function to maintain cellular health and promote cellular clearance in lysosome storage disorders.”

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 are essential transducers of cellular signals and are required to help maintain cellular homeostasis, especially the delicate balance within the autophagy/lysosomal pathway, which is often disrupted in a number of pathological conditions. Lysoway’s small molecule modulators have great potential to re-balance autophagy/lysosomal function to treat neurodegenerative diseases with lysosomal deficiency and rare diseases characterized by toxic accumulation of cellular wastes.

About Highlight Capital

Highlight Capital is an investment company with a mission to promote technology innovations based on deep scientific and industrial insights. We focus on enabling high growth sectors such as healthcare, biotech and consumer, and we strive to create long-term value and deliver life wellness.  We wholeheartedly partner with our entrusted entrepreneurs to drive long-term value creation and to deliver sustainable return.

About 3E Bio

3E Bioventures Capital is dedicated to investing in cutting-edge life sciences and biomedical technologies, with a focus on breakthrough first-in-class therapies and disruptive cross-disciplinary innovations in medical devices and diagnostics.  3E Bioventures takes on a science-driven, entrepreneur-friendly investment philosophy by working closely with companies and research institutions to develop drugs or products that have strong unmet medical needs.

About Oceanpine Capital

Oceanpine Capital is an institutional growth equity investment company formed by seasoned entrepreneurs and sophisticated investors with 20 years of in-depth industry operational and business building experiences in both China and the US. With a perspective of achieving successful growth over the long term, Oceanpine partners visionary entrepreneurs to build world-class companies together over a period of years rather than on the short-term impact.

Contact Info

info@lysoway.com
www.lysoway.com