Our Science

CARAWAY’S INTEGRATED DRUG DISCOVERY PLATFORM

Clearance of toxic cellular components, whether they are damaged organelles, aggregated proteins, or accumulated lipids, is a fundamental function required for cell health. Mutations that impair cellular clearance pathways are associated with multiple neurodegenerative and rare diseases, including neurodegenerative disorders characterized by cognitive dysfunction. Caraway’s targeted approach combines genetic data and unique biological understanding to discover small molecules that activate cells’ clean-up and recycling processes thereby accelerating clearance of toxic materials and defective cellular components

Leveraging patient data

Patient genomic data highlight the critical importance of lysosomal clearance pathways in maintaining healthy neurons.

High-throughput screening High Thoughput Screening and Medicinal Chemistry

One of the challenges facing drug discovery is accurately modeling human diseases in vitro. At Caraway, we overcome this difficulty by utilizing patient-derived induced pluripotent stem cells (iPSCs). These cells can be stimulated to form neurons in the laboratory so that we can recreate aspects of disease and observe how drugs work on them. The cell models allow us to quickly test potential drug molecules for their ability to enhance clearance of toxic accumulations in the cells of interest.

Electrophysiology Electrophysiology

Electrophysiology remains the gold-standard for direct assessment of ion channel function. Caraway employs both manual and automated patch clamp to generate rich datasets capturing channel function and modulation by compounds.  The team performs recordings in a variety of configurations including whole-cell, inside and outside-out patches, as well as recordings directly from the endolysosome.

Lysosomal function High Content Imaging

Probing the impact of compounds on organelle function often requires detailed visualization of the cell. Using our high content imaging system our scientists have implemented a variety of assays to study lysosomal function in living cells

ipsc core iPSC Core

One of the challenges facing drug discovery is accurately modelling human diseases in vitro. At Caraway, we have overcome this difficulty by utilizing patient-derived induced pluripotent stem cells (iPSCs) that are grown on site. These cells can be stimulated to form neurons in the laboratory so that we can recreate key characteristics of disease and assess drug function in a disease-like state.

Development Expertise

The Caraway team has significant expertise in advancing small molecules through clinical trials for neurological and rare diseases.