Lysoway Therapeutics, Inc.
Lysoway Therapeutics is a leader in lysosomal ion channel disease biology, developing potent modulators of lysosomal ion channels such as TRPML1 and TMEM175. The company's mission is to restore autophagy/lysosomal function to treat neurodegenerative diseases and rare diseases characterized by toxic cellular waste accumulation. With technological approaches to screen and develop small molecule modulators, Lysoway aims to create therapies that address lysosomal deficiencies and improve cellular homeostasis.
Industries
Nr. of Employees
small (1-50)
Products
Preclinical TRPML1 agonist candidate (lead development program)
A brain-penetrant, orally bioavailable small-molecule agonist of the TRPML1 lysosomal calcium channel in preclinical/translational development for Parkinson’s disease.
Screening platform for lysosomal ion channel modulators
Technologies and assay approaches used to identify and prioritize potent modulators of lysosomal ion channels (e.g., TRPML1, TMEM175).
Preclinical TRPML1 agonist candidate (lead development program)
A brain-penetrant, orally bioavailable small-molecule agonist of the TRPML1 lysosomal calcium channel in preclinical/translational development for Parkinson’s disease.
Screening platform for lysosomal ion channel modulators
Technologies and assay approaches used to identify and prioritize potent modulators of lysosomal ion channels (e.g., TRPML1, TMEM175).
Services
Discovery and lead optimization for lysosomal ion channel modulators
End-to-end small-molecule discovery services focused on identifying and optimizing modulators of lysosomal ion channels for restoration of lysosomal/autophagy function.
Preclinical translational development and biomarker establishment
Preclinical efficacy testing, translational pharmacology and development of target-engagement and pharmacodynamic biomarkers to support clinical entry.
CMC and regulatory support for small-molecule programs
CMC planning, API/drug-product manufacture coordination and regulatory filing support to enable clinical development of small molecules.
Discovery and lead optimization for lysosomal ion channel modulators
End-to-end small-molecule discovery services focused on identifying and optimizing modulators of lysosomal ion channels for restoration of lysosomal/autophagy function.
Preclinical translational development and biomarker establishment
Preclinical efficacy testing, translational pharmacology and development of target-engagement and pharmacodynamic biomarkers to support clinical entry.
CMC and regulatory support for small-molecule programs
CMC planning, API/drug-product manufacture coordination and regulatory filing support to enable clinical development of small molecules.
Expertise Areas
- Lysosomal ion channel biology
- Small-molecule drug discovery and optimization
- Neurodegenerative disease therapeutics (Parkinson’s disease)
- Translational research and biomarker development
Key Technologies
- Small-molecule ion channel modulation
- High-throughput screening assays
- Medicinal chemistry (SAR-driven design)
- CNS brain-penetrant optimization