Our R&D Expertise

Research and Development (R&D) Standard of Excellence

Neurological diseases are the leading cause of disability and the second-leading cause of death worldwide.1 We believe that the pathways of neurological diseases are interrelated and so are the potential approaches for treating them.

We leverage our knowledge about pathophysiological processes and disease characteristics to pioneer new potential treatments and further other areas of research and development. There’s more to come as we continue to chase the possibilities and strive to make a difference for patients everywhere.

Biogen R&D is driven by the potential to transform the lives of patients. We work in disease areas with some of the greatest unmet need. We work relentlessly knowing that bold, new possibilities await discovery. As pioneers in neuroscience, we work toward those once-in-a-lifetime moments that could become a historic point in our pursuit of innovative treatments.

 

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Scientific Expertise and Disease Areas

Because neurological diseases devastate so many people worldwide, we believe no other disease area holds as much unmet need or as much promise.

 

Core Growth Areas

Multiple Sclerosis and Neuroimmunology

We have a diverse portfolio of approved drugs to treat MS. We are taking a variety of approaches to further our understanding of this disease and how to treat it.

Alzheimer’s Disease and Dementia

Our diverse portfolio of investigational products includes several assets targeting early-stage Alzheimer’s disease. We hope to capitalize on advances in molecular and cellular biology and connect them with discoveries in human genetics.

Neuromuscular Disorders Including SMA and ALS

Our efforts in neuromuscular disorders are focused on two main areas: spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). We have brought forward the first approved therapy for SMA, and our scientists continue to work toward new potential therapeutics for SMA patients while advancing potential therapies for ALS.

Parkinson’s Disease and Movement Disorders

We currently have a preclinical and clinical portfolio around Parkinson’s disease (PD) and other movement disorders, targeting core disease genetics and pathology.

Ophthalmology

Biogen’s ophthalmology portfolio bolsters our pipeline across complementary modalities while leveraging our extensive expertise in disorders of the central nervous system. Our leading gene therapy programs target two inherited retinal disorders: choroideremia and X-linked retinitis pigmentosa.

Neuropsychiatry

Neuropsychiatry is an area of significant unmet patient need that intersects with other neurological disorders in our core therapeutic areas.

 

Emerging Disease Areas

Immunology

Leveraging our expertise in neuroimmunology, we are excited by the possibility of pioneering new scientific breakthroughs in immunology.

Acute Neurology

We have an innovative portfolio of compounds that target peripheral immune cell infiltration, brain edema, glial cell activation and cell death pathways, which have the potential to transform clinical care paradigms in acute brain injury.

Neuropathic Pain

Our approach to pain therapeutics includes selecting drug targets and pathways in rare genetic conditions, testing new drug candidates in patient populations with severe pain conditions, and exploring the possibility of combining multiple approaches for engaging a drug target.

 

Modalities

With capabilities in multiple drug modalities, including small molecules, protein therapeutics, antisense oligonucleotides and gene therapy, Biogen pursues the most appropriate modality for each target.

Small Molecules

Small molecule therapeutics are often taken orally, as a pill, and can readily distribute into cells and tissues, which differentiates them from other modalities. Medicinal chemists aim to optimize the potency of starting molecules towards the desired biological target along with physical properties to maximize the potential of the medicine to treat disease.

Protein Therapeutics

Therapeutic proteins, also referred to as biologics, are often as much as a thousand times the size of small-molecule drugs. Although this type of drug is much more complicated to make and characterize, using biologics allows scientists to attack serious disease in ways that might not be possible with other technologies.

Antisense Oligonucleotides

Antisense oligonucleotides (ASOs) are synthetic, single-stranded nucleic acids consisting of 8 to 50 nucleotides which bind to RNA and are designed to interfere with target gene expression. ASOs utilize several distinct mechanisms to alter RNA and either restore desired protein expression or reduce and modify toxic proteins.

Gene Therapy

Gene therapies target the root causes of genetic diseases. They expand our potential to treat previously intractable diseases by capitalizing on recent advances in our understanding of human genetics.

 

Partner with Us

We welcome the opportunity to explore working with other innovators. If your company or institution would like to share a potential asset or platform technology for collaboration, contact us today at partnering@biogen.com.