Crossing Barriers in Neurological Disease R&D

Sanofi is developing new medicines built on patient insights and outstanding science to help the millions of people living with neurological disorders. Using revolutionary technologies, Sanofi scientists are developing targeted, potentially disease-modifying therapies for people living with conditions such as multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS). The goal is to design best-in-class medicines that slow or halt neurodegeneration, control neuroinflammation, and protect or even repair the nervous system.

Multiple sclerosis

More than two million people worldwide are affected by MS, a neurological disease with unpredictable symptoms that include vision problems, challenges with cognition, and difficulty with physical activity. Caused by an immune attack on healthy tissues in the brain and spinal cord, the disease can worsen over time in some patients. MS can cause lesions in the brain, some of which can shrink over time while other, "smoldering" lesions can remain unchanged or expand.

How Multiple Sclerosis Affects the Brain

Sanofi R&D teams aim to address MS progression by investigating multiple sources of MS damage in the brain. For example, Sanofi is studying a small molecule that inhibits Bruton's tyrosine kinase (BTK), an enzyme found in immune cells involved in MS. The aim is to create a therapeutic that could potentially act on both peripheral immune cells (e.g., B-cells) and immune cells in the brain (e.g., microglia) that are involved in inflammation and degeneration of the central nervous system. The clinical significance of this BTK inhibitor's activity is unknown and is currently under investigation at Sanofi.

The MS Treatment Landscape
Timothy J. Turner, Global Program Head for Neurology at Sanofi

Amyotrophic Lateral Sclerosis (ALS)

Sanofi R&D teams are studying ALS, a progressive, neurodegenerative disease in which motor neurons in the brain and spinal cord are damaged. ALS is associated with mutations in several genes. Between 5% and 10% of patients have inherited the disease, but most have no family history. Sanofi scientists are studying immune cells called astrocytes, which carry out many important functions in the brain, to understand why and how they interact with motor neurons in ALS. By combining new genomic medicine technologies with long-standing expertise in rare disease, immunology, and lipid biology, they are advancing toward potential new therapies that could tackle the underlying cause of the disease.

Other neurological disorders

Sanofi has a strong commitment to patients with neurologic disorders for which few, if any, treatment options are available. These may include Huntington's disease, an inherited, progressive brain disorder caused by mutations in the HTT gene; chronic inflammatory demyelinating polyneuropathy (CIDP), a chronic disease in which immune cells attack the myelin sheath around nerve fibers in the peripheral nervous system; Parkinson’s disease; and myasthenia gravis, among others.

Technology toolbox

Sanofi R&D teams are developing innovative new medicines that are built on patient insights and expertise in disease biology, and enabled by pioneering technologies such as gene therapies and new techniques that could "shuttle" therapeutic proteins across the blood–brain barrier. Across neurological diseases, they are resetting expectations for targeted therapies and pushing the frontiers of science.

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