Isis Pharmaceuticals (Nasdaq: ISIS) says that it has earned a $10 million milestone payment from fellow USA-based biotech company Biogen Idec (Nasdaq: BIIB) related to the selection and advancement of ISIS-DMPKRx to treat myotonic dystrophy type I (DM1).
"We are very pleased with the successes we are having in our alliance with Biogen Idec. The progress we have made is evidenced in our spinal muscular atrophy and myotonic dystrophy programs. In less than two years, we discovered and advanced ISIS-DMPKRx into development, and we plan to begin human clinical studies next year," said B Lynne Parshall, chief operating officer at Isis.
"Myotonic dystrophy represents an ideal opportunity for antisense as the disease-causing gene produces a toxic RNA that is not easily targeted with traditional therapeutic approaches," said C Frank Bennett, senior vice president of research at Isis. "In our preclinical studies, we and our collaborators, Drs Charles Thornton and Thurman Wheeler at the University of Rochester, have been able to target the toxic RNA with antisense, remove the toxic RNA and restore normal cell function. We look forward to working with Biogen Idec to move this program into human clinical trials."
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