Company Overview

A targeted approach to activate cellular clearance

Caraway’s targeted approaches leverage genetic data and unique biological understanding to develop small molecules that activate neurons’ clearance and recycling processes – accelerating removal of toxic materials and defective cellular components. New insights in genetics highlight the link between lysosomal processes that normally maintain order inside neurons, and the risk of deterioration that occurs in neurodegenerative diseases. Caraway is a leader in targeting these mechanisms with potentially disease-modifying compounds for patients suffering the devastating effects of neurodegenerative disease including Parkinson’s Disease, Amyotrophic Lateral Sclerosis (ALS) and lysosomal storage diseases.

Urgent and increasing medical need

More than six million Americans currently suffer from neurodegenerative diseases with great unmet need. Available therapies address symptoms but do not stop the loss of neurons that causes the progression of disease. Caraway is developing therapeutics with the potential to alter disease progression and help patients maintain neuronal health and function.

 Why Now

The discovery and development of therapeutics for neurodegenerative disease has lagged behind many other major diseases such as cancer. Improved genetic diagnoses and imaging techniques now allow visualization of brain pathologies, enabling more accurate diagnosis and objective measurements of damage. Recent genetic insights have highlighted, that like cancer, neurodegenerative diseases are not monolithic. Targeting patients affected by the same mutations can increase the probability of therapeutic benefit. In addition, large-scale population studies highlight key disease drivers, such as defects in lysosomal processing. The focus on genetically defined diseases also enables more robust modeling of disease pathology at the cellular level. Integrating such key advances in our approach provides us with increased capabilities to develop meaningful therapeutics and to focus on the patients who are most likely to benefit.

Defining disease using genetics

Neurodegenerative diseases are complex with diverse causes of symptoms even among patients with the same disease diagnosis. Caraway is using genetically-defined populations to identify patients who have a greater chance of benefiting from enhanced cellular clearance.

Leveraging Induced Pluripotent Stem Cells (IPSCs) to model human disease

Because Caraway focuses on patients who have specific gene signatures, we can model their neurons using differentiated IPSCs. These cells allow us to assess the impact of our molecules on human neurons of patients with the disease.

Novel imaging and biomarker techniques

Recent advances in brain imaging allow more accurate diagnosis and objective ways to assess disease progress and therapeutic impact.

Caraway is positioned to make a difference

The Caraway team includes world-class scientists experienced in drug discovery for novel disease targets. Caraway’s programs take advantage of the endogenous cellular cleaning mechanisms. Because accumulations of toxic materials occur in many diseases including neurodegenerative and lysosomal storage diseases, the Caraway approach has broad therapeutic potential. The company is backed by investors with world-class expertise in central nervous system diseases.