Innovative therapies

We develop medicines for debilitating medical conditions with the goal of making a meaningful difference in patients’ lives.

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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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  • Alzheimer's Disease
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  • Multiple Sclerosis (MS)
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  • Parkinson's Disease
  • Sjögren's Syndrome
  • Spinal Muscular Atrophy (SMA)
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  • Aducanumab (Aβ mAb)

    Alzheimer’s disease

    Developed in collaboration with Neurimmune.

    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. Aducanumab 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

  • 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.

  • Obinutuzumab

    Front-line Indolent non-Hodgkin’s lymphoma

    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.

  • BAN2401 (Aβ 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.

  • 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.

  • BIIB059 (anti-BDCA2)

    Lupus

    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 lupus and contribute to disease activity. 

  • BIIB074 (Nav1.7 inhibitor)

    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:
    BIIB074 (Nav1.7 inhibitor) 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. BIIB074 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.

  • Dapirolizumab pegol (anti-CD40L)

    Lupus

    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 lupus patients.

  • Intravenous glyburide

    Stroke

    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. The target indication for intravenous glyburide is large hemispheric infarction (LHI), a severe form of ischemic stroke where brain swelling (cerebral edema) often leads to a disproportionately large share of stroke-related morbidity and mortality.

    How this therapy could help:
    In pre-clinical studies, intravenous glyburide has been shown to block SUR1-TRPM4 channels that mediate stroke related brain swelling. Clinical proof-of-concept studies have demonstrated the potential of CIRARA to reduce brain swelling, disability and the risk of death in patients with LHI.

  • Natalizumab (α4-integrin inhibitor)

    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.

  • Opicinumab (anti-LINGO)

    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-1 is a CNS specific-protein that is involved in the development of myelin. In patients with MS, LINGO-1 may inhibit myelin growth when it binds with its 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.

  • XLRS Gene Therapy

    X-linked retinoschisis (XLRS)

    Developed in collaboration with AGTC

    XLRS is a disease that affects young males beginning in their teenage years. It can lead to loss of vision and serious complications such as vitreous hemorrhage or retinal detachment during adulthood.

    How this therapy could help:
    The XLRS gene therapy is a recombinant adeno-associated virus vector (rAAV) delivering a transgene (RS1) that expresses retinoschisin protein. Normal expression of retinoschisin could potentially improve long-term retinal function and preserve structural integrity.

  • BIIB054 (anti-α-synuclein)

    Parkinson's disease

    Acquired from Neurimmune.

    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.

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May 31, 2017

Living with MS: Understanding the Patient Experience

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