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A growing portfolio of novel medicines addressing targets and pathways critical in neurodegenerative and other CNS disorders

What is Neurodegeneration?

SciNeuro is dedicated to developing medicines for neurological diseases. Our initial focus is neurodegenerative diseases in which structure and function of neurons are progressively damaged and lost. As a result, patients lose their natural ability to move, speak, remember, or think. Neurodegeneration can present in various forms, such as Parkinson’s disease (PD) and Alzheimer’s disease (AD), two common neurodegenerative diseases affecting patients worldwide, as well as rare conditions like amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA).

In recent years, the rapid progress of human genetic studies and the advent of genomic technologies have revealed molecular culprits (proteins) of neurodegeneration as promising therapeutic targets.

More research now provides scientific details on what roles these proteins play in the death of neurons. These mechanisms include proteopathy, inflammation, and lysosomal dysfunction. We have developed specific strategies to target each of these mechanisms:

Proteopathy arises from misfolded and toxic proteins such as β-amyloid or α-synuclein. We are developing very potent therapeutic molecules that bind and remove these toxic proteins. As a result, the therapy will alleviate proteopathy and slow down or halt neuron death.

Inflammation is a process where immune cells react to intrinsic cellular changes or damages caused by foreign pathogens. In neurodegenerative diseases, maladaptive inflammation aggravates neuron damage and loss.  Our approach is to curb pro-inflammatory cues (also proteins) with potent and selective therapeutic molecules.

Lysosomal Dysfunction is a common causality in both neurodegenerative diseases such as PD and a host of rare diseases known as lysosomal storage diseases (LSDs). Lysosome is a key cellular organelle that functions as a waste management system by removing intracellular and extracellular debris. One of our strategies is to normalize enzymes central to lysosome function.