• understanding-sma-backgrounder
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    Disease Awareness / Fact Sheet
    Spinal Muscular Atrophy

    Understanding spinal muscular atrophy, a rare, neuromuscular genetic disease.

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    Science & Innovation / Fact Sheet
    The Novartis AAV Platform

    Modified viruses – including adeno-associated viruses – are an efficient way to transport therapeutic genetic material to affected cells.

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    The Novartis Gene Therapies Manufacturing Process

    Manufacturing of gene therapy is complex. Novartis has developed a reproducible manufacturing process.

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    Corporate Responsibility / Fact Sheet
    About Novartis Gene Therapies

    Novartis Gene Therapies is the world leader in gene therapy, and is reimagining medicine to transform the lives of patients.

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    Science & Innovation / Fact Sheet
    About Gene Therapy

    Gene therapy – an innovative approach to treating rare, genetic diseases.

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    RTH258 Brolucizumab Clinical Trials

    Novartis brolucizumab (RTH258) demonstrates superiority versus aflibercept in key secondary endpoint measures of disease activity in nAMD, a leading cause of blindness.

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    Kymriah™(tisagenlecleucel) in children and young adults with B-cell ALL that is refractory or relapsed at least twice

    Kymriah (pronounced: Kim-RYE-ah) is the first FDA-approved CAR-T cell therapy available in the US

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    The JULIET Clinical Trial

    The JULIET clinical trial is a global, multi-center Phase II registration trial investigating CTL019 (tisagenlecleucel) for use in diffuse large B-cell lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma (NHL). The Novartis-sponsored JULIET trial was conducted in collaboration with the University of Pennsylvania to evaluate the safety and efficacy of CTL019 in adult patients with relapsed or refractory (r/r) DLBCL. Relapsed/refractory DLBCL is an aggressive (fast-growing), complex and difficult-to-treat disease, and patients with DLBCL often have worse prognosis than other forms of NHL.

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    How does ACZ885 work?

    ACZ885 is a human monoclonal antibody, which is a protein that is designed to bind to only one substance in the body. ACZ885 is designed to bind to human interleukin-1β (IL-1β).

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    Erenumab (AMG 334) Key Clinical Trials By The Numbers

    Erenumab has been studied in several large, global, randomized, double-blind, placebo-controlled studies to assess its safety and efficancy in migraine prevention.

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    About erenumab AMG 334

    Erenumab is designed to specifically block the CGRP receptor, which plays a critical role in migraine activation. It is being co-developed by Novartis and Amgen.

  • 2017 CML Background Fact Sheet
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    Chronic myeloid leukemia Background Fact Sheet

    Chronic myeloid leukemia, or CML, is a cancer of the blood and bone marrow in which the body overproduces white blood cells.