A Mission Rooted in Neuroscience

For billions of people worldwide who live with a neurological disease,¹ changes in muscle strength and function, movement and cognition can turn once-simple moments into daily challenges. Driven by the profound impact these diseases can have on patients, care partners and the clinicians who care for them, we are working to better understand these diseases and are committed to researching them:

• For adults living with generalized myasthenia gravis (gMG), their day-to-day lives are often unpredictable, ranging from intermittent symptoms such as fluctuating muscle weakness to fatigue, as well as severe exacerbations, including myasthenic crisis, a potentially life-threatening condition that can impair breathing.²
• Amyotrophic Lateral Sclerosis (ALS) begins as stiffness and weakness gradually leading to progressive loss of muscle control, taking away a person’s ability to live independently.³
• For people living with Parkinson’s disease, tremors and impaired movement can make once-routine activities, like buttoning a shirt or holding a cup of coffee, increasingly difficult.⁴

Regeneron’s Deeply Rooted Commitment to Neuroscience

The pursuit of finding answers to neurological diseases has been part of our story since the beginning.

Regeneron was co-founded by neurologist, Leonard S. Schleifer, MD, PhD, and scientist, George D. Yancopoulos, MD, PhD, with the original goal of “REGENErating neuRONs.” While working as a practicing neurologist and professor, Len became frustrated with the lack of effective treatments for patients with serious neurodegenerative diseases like Parkinson’s and Alzheimer’s. He wondered if new biotechnologies could be harnessed to potentially make an impact for these people, their families and many others. Meanwhile, George was inspired to pursue medicine and research fueled by his personal experience seeing his grandmother, affectionately known as Yaya, battle dementia.

These deeply rooted motivations drove our early research efforts, with our first investigational drug – a neurotrophic factor – entering clinical development in 1992. Decades later, that same drive continues to shape Regeneron’s vision: to deepen our understanding of these hard-to-treat neurological diseases through insights from human genetics and disease biology.

Len (left) and George (right) in the early days of Regeneron. The name Regeneron comes from “REGENErating neuRONS” with “gene” embedded in the middle, a reflection of the science that inspired our founding.

What is siRNA and How Does it Work?

What Other Technologies Are Being Explored in Neuroscience?

While siRNA is one approach being explored, neurological conditions are often complex and can involve multiple biological pathways. That is why we are also investigating a range of other innovative approaches that may help address different aspects of disease biology.

• CRISPR is a technology that allows scientists to make precise changes to DNA. We are exploring how it can be used to study and potentially influence genes associated with neurological disease.
• Gene therapies are designed to deliver genetic material to cells. Depending on the approach, they may be used to replace, modify or introduce genetic instructions.
• Antibodies are proteins that can be engineered to recognize and bind to specific targets in the body, such as those associated with disease processes. They may also allow for more precise targeting.

At Regeneron, our work is about more than scientific innovation. We are guided by the possibility that advances in research may one day translate into meaningful progress for people living with neurological diseases. That purpose remains at the core of our commitment to helping advance research for people living with neurological conditions.