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Wellstat Therapeutics Corporation has announced in September that the US Food and Drug Administration granted marketing approval for XURIDEN™ (uridine triacetate), a pyrimidine analog for uridine replacement indicated for the treatment of hereditary orotic aciduria (HOA). With the approval of XURIDEN for the treatment of HOA, the FDA also granted Wellstat Therapeutics a Rare Pediatric Disease Priority Review Voucher. HOA is an extremely rare, potentially life-threatening, genetic disorder in which patients cannot synthesize adequate amounts of uridine and consequently can suffer from hematologic abnormalities, failure to thrive, a range of developmental delays, and episodes of crystalluria leading to obstructive uropathy.
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Third Rock Ventures portfolio company, Voyager Therapeutics, a gene therapy player in the field of CNS based out of Cambridge, Mass. have appointed Michael Higgins to the Board of Directors. Mr. Higgins is an experienced biotechnology executive and is currently an entrepreneur-in-residence at Polaris Partners. Previously, he served as chief operating officer and chief financial officer at Ironwood Pharmaceuticals, Inc. from 2003 through 2014. In 2014, Mr. Higgins was named the Boston Business Journal’s CFO of the Year. Mr. Higgins worked at Genzyme Corporation, with which Voyager Therapeutics now has a partnership, from 1997 through 2003 in a variety of leadership roles, including vice president of corporate finance and vice president of business development. While at Genzyme, he was involved with multiple business units, including Cell Therapy, Gene Therapy and Orphan Diseases.