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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.

All disease areas
  • All disease areas
  • Alzheimer's Disease
  • Fibrosis
  • Inflammatory Bowel Disease (IBD)
  • Lupus
  • Lymphoma
  • Multiple Sclerosis (MS)
  • Myotonic Dystrophy
  • Ophthalmology
  • Parkinson's Disease
  • Sjögren's Syndrome
  • Spinal Muscular Atrophy (SMA)
  • Stroke
  • Trigeminal Neuralgia
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  • Filing
  • 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.

  • Aducanumab (BIIB037)

    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. 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

  • Nusinersen (IONIS-SMNRx)

    Spinal muscular atrophy (SMA)

    Developed in collaboration with Ionis 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:
    Nusinersen (IONIS-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 trials for SMA

  • 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

    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.

  • Ocrelizumab

    Primary progressive and relapsing multiple sclerosis (MS)

    The 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 be damaged permanently.

    How this therapy could help:
    Ocrelizumab is a humanized monoclonal antibody designed to selectively target CD20-positive B cells. CD20-positive B cells are a specific type of immune cell thought to be a key contributor to myelin and nerve cell damage. Based on preclinical studies, ocrelizumab binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, and therefore important functions of the immune system may be preserved.

    The Roche Group and its sub-licensees maintain sole responsibility for the development, manufacturing, and commercialization of ocrelizumab.

  • Amiselimod (MT-1303)

    Inflammatory bowel disease (IBD)

    Acquired exclusive rights in a worldwide licensing agreement, excluding Asia, from Mitsubishi Tanabe Pharma Corporation (MTPC)

    IBD describes a group of inflammatory conditions of the digestive tract. The two most common inflammatory bowel diseases are ulcerative colitis and Crohn’s disease. Ulcerative colitis is characterized by inflammation in the large intestine. Crohn’s disease can feature areas of swelling anywhere along the digestive tract. Both conditions are autoimmune disorders caused by an abnormal response of the body’s immune system.

    How this therapy could help
    Amiselimod (MT-1303) is an oral compound that targets the sphingosine 1-phosphate (S1P) receptor. In the immune system, SIP receptors regulate the trafficking of lymphocytes in the body. Lymphocytes are the immune cells that in an abnormal response, attack the gastrointestinal tract as seen in IBD. Inhibition of S1P receptors is associated with lymphocyte suppression and the lowering of immune cells circulating in the body.

    MT-1303 is currently in a Phase 2 study for Crohn’s disease and is being assessed for ulcerative colitis, multiple sclerosis and other autoimmune indications.

  • 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.

  • 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.

  • 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.

  • 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:
    Opicinumab is a neurologic protein that is involved in the development of myelin. In patients with MS, opicinumab 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.

  • Raxatrigine (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:
    Raxatrigine (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. Raxatraigine 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.

  • 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.

  • Anti-alpha-synuclein mAb (BIIB054)

    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.

  • 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.

  • BIIB063

    Sjögren’s Syndrome

    Sjögren’s Syndrome is a chronic autoimmune disease where the immune system attacks moisture-producing glands. Although many patients experience dry eyes, dry mouth, fatigue and joint pain, Sjögren’s also causes dysfunction in other organs such as the kidneys, gastrointestinal system, blood vessels, lungs, liver, pancreas, and the central nervous system. Patients may also experience extreme fatigue and joint pain and have a higher risk of developing lymphoma.

    How this therapy can help:
    CD40/CD40L is a premier immunological pathway associated with autoimmune diseases. BIIB063 targets the CD40L protein and blocks the pathway that initiates the immune cell hyperactivity seen in Sjögren’s Syndrome.

  • 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.

  • IONIS-DMPK-2.5Rx

     

     

    Myotonic dystrophy, type 1

    Developed in collaboration with Ionis 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:
    IONIS-DMPK-2.5Rx 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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