Neuroscience
Pairing genetic insights and innovative technologies to impact the treatment of neurodegenerative disease, pain and neuropsychiatric disorders
After decades of setbacks in hard-to-treat neurological diseases, the field is now on the cusp of exciting breakthroughs. Powerful insights from human genetics and disease biology paired with emerging technologies are enabling new approaches to potentially improve lives.
Regeneron was founded by a neurologist with the original goal of "REGENErating neuRONs,” and has been focused on neurological conditions since its early days, with our first investigational drug – a neurotrophic factor – entering clinical development in 1992.
Today, we are investigating potential treatments for a variety of neurological diseases and for pain. Efforts to apply genetic medicine technologies like gene therapy and gene editing are underway and take advantage of Regeneron’s innovative delivery technologies that go beyond the liver. Regeneron’s genetic medicine approaches have enabled a new vision for neurological disorders, which are areas of urgent and unmet societal need.
Genetic medicine technologies
With our collaborator Alnylam, we are investigating siRNA oligonucleotide therapeutics for a variety of ocular and central nervous system (CNS) diseases, including ALS, Huntington’s disease, tauopathies, Parkinson’s disease, and other serious neurodegenerative conditions.
We are exploring adeno-associated virus (AAV) gene therapy for the treatment of genetic hearing loss. We are investigating our novel approach for individuals with profound, congenital hearing loss caused by rare variants of a single gene. We are also advancing additional hearing loss programs into the clinic to address more common forms of hearing loss.
With our collaborator Intellia, we are investigating therapeutic approaches for transthyretin amyloid polyneuropathy (ATTR-PN), a rare, hereditary disease that causes a buildup of a misfolded protein called transthyretin, or TTR, in the nerves. This can lead to difficulty walking, tingling and numbness in the hands and feet, limb weakness and pain. Our research also includes potential therapeutics for the cardiovascular form of the disease (ATTR-CM).
Addressing Painful Memories From Within
One of our research scientists explains the challenges of treating neurological conditions like pain