Research & Pipeline

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A mission defined by visionary science

At Biogen, we work toward new discoveries that can make a significant impact in patients’ lives and where there are few or no treatment options.

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Imaging technology is enabling us to gain better insights from our clinical trials.

Core areas of focus

Our research and development efforts are primarily focused on three areas in which we are recognized leaders:

Neurology

The primary mission of neurology discovery at Biogen is to identify novel drug candidates for the treatment of devastating neurological and neurodegenerative diseases.

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Hematology

In hematology, we are focused on exploring novel therapies for the treatment of hemophilia and further expanding our research into related areas of nonmalignant hematology, such as sickle cell disease and beta-thalassemia.

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Immunology

Immunology at Biogen is focused on exploring the immunobiology underlying autoimmune, inflammatory and fibrotic disease.

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Dedicated to new discoveries

Our pipeline is more than a list of medicines in development. It reflects the work we do here every day to break new ground with science that makes a difference in the lives of patients.

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  • Daclizumab High-Yield Process

    Relapsing-remitting multiple sclerosis (MS)

    Developed in collaboration with AbbVie Biotherapeutics.

    This disease causes the body’s immune system to attack myelin, a protective sheath covering nerve fibers. Damage to the myelin “short circuits” communication between the brain, spinal cord and other areas of the body, severely impairing such neurological functions as mobility, vision and thinking.

    How this therapy could help:

    Patients who have relapsing-remitting MS experience attacks, or relapses, of neurologic impairment, followed by periods of partial or complete recovery. Daclizumab HYP is an antibody that binds to CD25, a receptor highly expressed in immune cells abnormally activated in MS. Binding Daclizumab HYP and CD25 may normalize immune cell activity, possibly preventing relapses and slowing the progress of the disease.

  • ISIS-SMNrx

    Spinal muscular atrophy (SMA)

    Developed in collaboration with Isis Pharmaceuticals.

    People with this rare, inherited genetic disease lack a protein called SMN that helps with muscle control. Children born with SMA have difficulty eating, often miss developmental milestones and experience muscle weakness and wasting in the legs, arms and torso. SMA can lead to death by respiratory failure at a young age.

    How this therapy could help:
    ISIS-SMNrx is thought to promote the production of functional SMN protein, potentially improving the motor function and life span of patients with SMA. 


    View enrolling Biogen-sponsored trials for SMA

  • Natalizumab

    Secondary progressive multiple sclerosis (SPMS)

    Currently approved as TYSABRI® for relapsing-remitting multiple sclerosis and Crohn’s disease. Please visit TYSABRI.com for prescribing and safety information.

    MS causes the body’s immune system to attack myelin, a protective sheath covering nerve fibers. Damage to the myelin “short circuits” communication between the brain, spinal cord and other areas of the body, severely impairing such neurological functions as mobility, vision and thinking.

    How this therapy could help:

    After a period of experiencing relapses (increased impairment) and remission (improved ability), many MS patients will eventually enter secondary-progressive MS (SPMS): steady progression of neurological damage. Natalizumab may help by potentially reducing the disability progression.

  • Obinutuzumab (GA101)

    Diffuse large B-cell lymphoma (DLBCL)

    Currently approved as GAZYVA® for chronic lymphocytic leukemia (CLL). Please visit GAZYVA.com for prescribing and safety information.

    DLBCL is a type of non-Hodgkin’s lymphoma (NHL), a cancer of the immune system. NHL involves white blood cells (known as lymphocytes). Under normal conditions, these cells help defend the body from disease. In people with NHL, too many abnormal white blood cells build up in the blood, bone marrow, spleen and/or lymph nodes. Most NHL occurs in white blood cells called B cells. Intermediate and high-grade, or fast-growing, tumors are called aggressive NHL. DLBCL is the most common type of aggressive NHL.

    How this therapy could help:

    Obinutuzumab is a type of antibody therapy that targets and attaches to the CD20 protein found on the surface of NHL cells. Once attached to the CD20 protein, Obinutuzumab works in two different ways:

    • By helping the immune system destroy cancer cells
    • By destroying cancer cells on its own


    We collaborate on obinutuzumab in the U.S. with Genentech, Inc., a wholly-owned member of the Roche Group. The Roche Group and its sub-licensees maintain sole responsibility for the development, manufacturing and commercialization of obinutuzumab.

  • Obinutuzumab (GA101)

    Indolent non-Hodgkin’s lymphoma, front-line and refractory

    Currently approved as GAZYVA® for chronic lymphocytic leukemia (CLL). Please visit GAZYVA.com for prescribing and safety information.

    Non-Hodgkin’s lymphoma, or NHL, is a type of cancer of the immune system. NHL involves white blood cells (known as lymphocytes). Under normal conditions, these cells help defend the body from disease. In people with NHL, too many abnormal white blood cells build up in the blood, bone marrow, spleen and/or lymph nodes. Most NHL occurs in white blood cells called B cells. Low-grade, or slow-growing, tumors are called indolent NHL. Many patients live with indolent NHL for many years.

    How this therapy could help:

    Obinutuzumab is a type of antibody therapy that targets and attaches to the CD20 protein found on the surface of NHL cells. Once attached to the CD20 protein, obinutuzumab works in two different ways:

    • By helping the immune system destroy cancer cells;
    • By destroying cancer cells on its own.


    We collaborate on obinutuzumab in the U.S. with Genentech, Inc., a wholly-owned member of the Roche Group. The Roche Group and its sub-licensees maintain sole responsibility for the development, manufacturing and commercialization of obinutuzumab.

  • Anti-LINGO-1 (BIIB033)

    Multiple sclerosis (MS)

    This disease causes the body’s immune system to attack myelin, a protective sheath covering nerve fibers. Damage to myelin “short circuits” communication between the brain, spinal cord and other areas of the body, severely impairing such neurological functions as mobility, vision and thinking. Over time, the nerves themselves can become damaged permanently.

    How this therapy could help:

    LINGO is a neurologic protein that is involved in the development of myelin. In patients with MS, LINGO may inhibit myelin growth when it binds with its normal receptor. Data suggest that the antibody anti-LINGO-1 could block this process, potentially allowing for the re-myelination and restoration of nerve communication in MS patients

  • Anti-TWEAK

    Lupus nephritis

    This kidney disorder is caused by systemic lupus erythematosus (SLE or lupus). SLE is a chronic inflammatory disease. It occurs when the body’s own immune system mistakenly attacks healthy tissue in skin, joints, kidneys, the brain and other organs. It is a difficult disease to diagnose because it resembles several other conditions.

    How this therapy could help:

    Scientists have linked TWEAK, an immune cell protein, to increased disease activity in lupus nephritis. When TWEAK binds to its normal receptor, inflammation can be the result. TWEAK binding to the anti-TWEAK antibody could block this process and potentially diminish inflammation.

  • BG00011 (STX-100)

    Idiopathic pulmonary fibrosis (IPF)

    Pulmonary fibrosis is a disease in which lung tissue becomes thickened and stiff due to scarring. The formation of scar tissue is called fibrosis. As the disease progresses, it becomes harder for the lungs to work properly and the body cannot get the oxygen it needs. This debilitating disease is almost always fatal.

    How this therapy could help:

    The TGF-beta brain cell pathway is involved in several fibrotic diseases. The BG00011 (STX-100) antibody may selectively block the TGF-beta pathway, and may slow the development of fibrosis in IPF patients.

  • BAN2401 (Humanized anti-amyloid beta mab)

    Alzheimer’s disease

    Developed in collaboration with Eisai Co., Ltd.

    The most common cause of dementia, Alzheimer’s disease is a brain disorder that leads to impairment of intellectual functions and social skills, resulting ultimately in significant disturbance of daily living activities. Dementia is caused by the destruction of cells in certain parts of the brain primarily involved in memory and mental function.

    How this therapy could help:

    The memory loss and functionality decline of Alzheimer’s disease have been linked to amyloid plaques, abnormal protein deposits that build up in the brain. BAN2401 is an antibody that binds to amyloid, which could reduce its presence in the brain and potentially slow the progress of the disease.

  • CNV1014802

    Trigeminal neuralgia (TGN)

    Acquired in acquisition of Convergence Pharmaceuticals

    Trigeminal neuralgia is an extremely painful condition usually involving one side of the face. Trigeminal neuralgia is characterized by sudden, brief, stabbing, recurrent episodes of pain which frequently occurs spontaneously but is also commonly evoked by trivial stimuli including washing, shaving, smoking, talking and/or brushing the teeth (trigger factors). The pain follows one or more branches of the trigeminal nerve, which provides nerve sensation from the mouth, face and the front of the scalp. The intensity of the pain and its unpredictability results in profound effects on the quality of life of patients.

    How this therapy could help:

    CNV1014802 is a novel state dependent small molecule sodium channel blocker that preferentially inhibits the Nav 1.7 ion channel, a therapeutic target implicated by genetics in human pain conditions. CNV1014802 is thought to penetrate the central nervous system and block Nav channels in a novel manner, and has the potential to provide a new and effective option for the treatment of trigeminal neuralgia.

  • E2609 (BACE1 inhibitor)

    Alzheimer's disease

    Developed in collaboration with Eisai Co., Ltd.

    The most common cause of dementia, Alzheimer’s disease is a brain disorder that leads to impairment of intellectual functions and social skills, resulting ultimately in significant disturbance of daily living activities. Dementia is caused by the destruction of cells in certain parts of the brain primarily involved in memory and mental function.

    How this therapy could help:

    The memory loss and functionality decline of Alzheimer’s disease have been linked to amyloid plaques, abnormal protein deposits that build up in the brain. E2609 is a small-molecule inhibitor of beta-secretase, a protein that cleaves enzyme 1 (BACE1). By inhibiting BACE1, E2609 blocks amyloid production, potentially slowing the progress of the disease.

  • Natalizumab

    Acute ischemic stroke

    Currently approved as TYSABRI® for relapsing-remitting multiple sclerosis and Crohn’s disease. Please visit TYSABRI.com for prescribing and safety information.

    It is estimated that nearly two million people in the United States and the European Union will have a stroke each year. Stroke is a leading cause of mortality and serious long-term disability. There is a substantial unmet medical need for new therapies that can improve outcomes in acute stroke. 

    How this therapy could help:

    Natalizumab is thought to reduce the inflammatory cells in the brain by blocking the lymphocytic infiltration, known to occur in the brain following stroke, which may reduce the extent of injury.

  • Aducanumab (BIIB037)

    Alzheimer’s disease

    The most common cause of dementia, Alzheimer’s disease is a brain disorder that leads to impairment of intellectual functions and social skills, resulting ultimately in significant disturbance of daily living activities. Dementia is caused by the destruction of cells in certain parts of the brain primarily involved in memory and mental function.

    How this therapy could help:
    The memory loss and functional decline of Alzheimer’s disease have been linked to amyloid plaques, abnormal protein deposits that build up in  the brain. BIIB037 is an antibody that binds to and may reduce amyloid plaques from the brain, potentially slowing the progress of the disease.


    More information about our aducanumab clinical trials

  • Anti-alpha Synuclein mAb (BIIB054)

    Parkinson's disease

    Parkinson's disease is a disorder of the central nervous system. People who have this disease experience tremors, slow movement, muscle stiffness, and impaired balance.  As these symptoms become progressively worse, patients have difficulty walking, talking, or completing other simple tasks.

    How this therapy could help:
    BIIB054 targets abnormal alpha Synuclein protein that is associated with the destruction of nerve cells. This progressive degeneration is the cause of Parkinson's disease.

  • Anti-BDCA2

    Systemic lupus erythematosus (SLE)

    This chronic inflammatory disease occurs when the body’s own immune system mistakenly attacks healthy tissue in skin, joints, kidneys, the brain and other organs. It is a difficult disease to diagnose because it resembles several other conditions.

    How this therapy could help:

    BDCA2 is a protein present in specific cells within the immune system. An antibody against BDCA2 can potentially interrupt production of interferons, inflammatory molecules that are increased in patients with SLE and contribute to disease activity. 

  • BIIB061 (Oral re-myelination)

    Multiple sclerosis (MS)

    This disease causes the body’s immune system to attack myelin, a protective sheath covering nerve fibers. Damage to myelin “short circuits” communication between the brain, spinal cord and other areas of the body, severely impairing such neurological functions as mobility, vision and thinking. Over time, the nerves themselves can become damaged permanently.

    How this therapy could help:

    Data suggest that BIIB061 treatment may lead to re-myelination, or the replacement of myelin on damaged nerve fibers.

  • Dapirolizumab Pegol (Anti-CD40L)

    Systemic lupus erythematosus (SLE)

    Developed in collaboration with UCB, Inc.

    This chronic inflammatory disease occurs when the body’s own immune system mistakenly attacks healthy tissue in skin, joints, kidneys, the brain and other organs. It is a difficult disease to diagnose because it resembles several other conditions.

    How this therapy could help:

    CD40L is a protein in B and T cells, which helps regulate the immune system. Dapirolizumab pegol (Anti-CD40L) is an antibody that blocks CD40L, potentially lessening disease activity in SLE patients.

  • ISIS-DMPKRX

    Myotonic dystrophy, type 1

    Developed in collaboration with Isis Pharmaceuticals

    People with this rare inherited genetic disease have a mutation in the DMPK gene. The mutation can affect multiple organ systems. People with the disease can suffer from weakness and slowed relaxation of contracted muscles (myotonia), as well as heart or breathing problems.

    How this therapy could help:

    ISIS-DMPKRX is anticipated to dampen the expression of the DMPK gene, slowing the disease progression and improving quality of life for patients with myotonic dystrophy, type 1.

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Gaining insight through clinical trials

Biogen conducts clinical trials to assess new treatments and to investigate potential new uses and benefits for existing therapies.

Powered by the world's leading scientists

Pavan
Auluck
Associate Director, Postdoc Mentor Translational Sciences, Neurology
Linda
Burkly
Distinguished Investigator, Postdoc Mentor Immunology, Tissue Injury and Fibrosis
Ekta Seth
Chhabra
Senior Scientist, Postdoc Mentor Hematology
Janaky
Coomaraswamy
Scientist II, Postdoc Mentor Neurology
Mark
Cosentino
Director, Postdoc Mentor Precision Medicine
Olivier
Danos
Senior Vice President Cell & Gene Therapy
Abhishek
Datta
Scientist II Biologics Drug Discovery
Jeremy
Duffield
Senior Research Fellow, Postdoc Mentor Inflammation and Fibrosis
Stefan
Hamann
Senior Scientist Translational Sciences, Immunology
Edward
Lin
Scientist I Medicinal Chemistry
John
Lydeard
Scientist I, Postdoc Mentor Epigenetics
Alexander
McCampbell
Principal Scientist, Postdoc Mentor Neurology
Benjamin
Smith
Scientist I Biologics Drug Discovery
Meena
Subramanyam
Vice President Translational Sciences
Qin
Wang
Principal Scientist, Postdoc Mentor Translational Sciences
Guangqing
Xiao
Principal Scientist, Postdoc Mentor Translational Sciences
Zhili
Xin
Scientist I Medicinal Chemistry
Mingxuan
Zhang
Scientist II Hematology

Excellence in research & development

Maintaining our scientific leadership requires that we engage the best minds in our efforts as we strive to make breakthroughs in discovery and translational sciences.

Our scientists are at the forefront of their fields and, in partnership with leading academic institutions and companies, work to identify and validate novel targets that will catalyze the discovery of important new approaches to treating disease.

Opportunities for postdoctoral fellows

Our goal is to create an environment where scientists can flourish. Our postdoctoral program is one of the many ways we help support promising young scientists.

In addition to being able to do basic science and understand cellular molecular process, we offer the opportunity to rapidly convert ideas into something tangible.

Jeremy Duffield

Senior Research Fellow

Working together toward innovation and discoveries

Biogen is continuously working to form new alliances, partnerships and collaborations to accelerate the discovery of new targets and help deliver new therapies.