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

biotechnology
medical-device
therapeutics

Nr. of Employees

small (1-50)

Lysoway Therapeutics, Inc.


Patents

Piperazine derivatives as TRPML modulators

US-10851084-B2

View Details

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).


Services

End-to-end small-molecule discovery services focused on identifying and optimizing modulators of lysosomal ion channels for restoration of lysosomal/autophagy function.

Preclinical efficacy testing, translational pharmacology and development of target-engagement and pharmacodynamic biomarkers to support clinical entry.

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
  • Show More (4)

Key Technologies

  • Small-molecule ion channel modulation
  • High-throughput screening assays
  • Medicinal chemistry (SAR-driven design)
  • CNS brain-penetrant optimization
  • Show More (3)

News & Updates

Lysoway Therapeutics received a $2.93 million research grant from The Michael J. Fox Foundation for Parkinson’s Research to support the development of a highly brain-penetrant small molecule TRPML1 agonist. The study aims to investigate whether activating TRPML1 can restore lysosomal function and help clear alpha-synuclein, a protein linked to Parkinson’s disease.


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