C4 Therapeutics (C4T) has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT8634 to treat soft tissue sarcoma. Soft tissue sarcoma is a rare type of cancer that begins in the tissues that connect, support, and surround other body structures, including muscle, fat, blood vessels, nerves, tendons, and the linings of the joints. Patients living with synovial sarcoma currently have limited options for treatment, and patients with metastatic synovial sarcoma tend to do poorly after first-line chemotherapy treatment. CFT8634 may potentially address this dire unmet medical need. CFT8634 is an orally bioavailable, BiDAC™ degrader discovered by C4T’s TORPEDO® platform that targets BRD9 to treat cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1 deleted cancers. Since inhibitors of the bromodomain of BRD9 are not effective in treating these cancers, BRD9 has been considered an “undruggable” target. However, unlike BRD9 inhibition, BRD9 degradation is effective in preclinical models of synovial sarcoma. C4 Therapeutics is a clinical-stage biopharmaceutical company with a mission to deliver on the promise of targeted protein degradation science to create a new generation of medicines that transforms how disease is treated.
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