Several diseases of the Central Nervous System (CNS), including neurodegenerative diseases (e.g., Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis) and neurodevelopmental disorders (Angelman syndrome, and autism spectrum disorder), in addition to aging-related cognitive impairments and traumatic brain injury (TBI) present a common problem: an overaccumulation of proteins in the brain.  

Stimuli that lead to brain plasticity evoke in the brain, and particularly in neurons, an increase in protein synthesis. This increase, which is significant in neurons and their processes, is necessary for healthy brain functions (e.g. learning and memory) and is linked to the activation of protein degradation via autophagy.

Autophagy is a cellular degradation and recycling process that breaks down proteins through a lysosome-dependent mechanism and, like the stimulus-evoked de novo protein synthesis, is critical for healthy neuronal functions.

Autophagy and lysosomal degradation are impaired in several CNS diseases and in the aged brain; these impairments lead to accumulation of toxic proteins in the brain. 

Ritrova’s new therapies target a membrane receptor that is highly enriched in neurons and controls protein synthesis, autophagy, and the trafficking of proteins to lysosomes for degradation. 

By restoring protein metabolism homeostasis Ritrova is well positioned to reverse the progression of diseases characterized by protein accumulation.