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Chemistry: Generation 2.5

Danvatirsen, formerly known as IONIS-STAT3-2.5Rx and AZD9150, is an investigational antisense medicine designed to reduce the production of signal transducer and activator of transcription 3, or STAT3, for the treatment of patients with cancer. STAT3 is a protein involved in the translation of key factors critical for tumor cell growth and survival. STAT3 is over-active in a variety of cancers, including brain, lung, breast, bone, liver and multiple myeloma. Overactivity in STAT3 is believed to prevent cell death and promote tumor cell growth.

About STAT3 and Cancer

STAT3, or signal transducer and activator of transcription 3, is an important mediator of signaling in the JAK2/STAT3 pathway. Mutations in STAT3 and other regulatory genes could result in constitutively active STAT3. Numerous studies have demonstrated constitutive STAT3 activation promotes tumor cell growth and survival. Indeed, activated STAT3 are present in a wide variety of human tumors, including hematological malignancies (leukemia, lymphomas, and multiple myeloma) as well as diverse solid tumors (head and neck, breast, lung, gastric, hepatocellular, colorectal and prostate cancer).