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Note: The below is an edited press release from Dimension Therapeutics, Inc. The original release can be read here.


Dimension Therapeutics, Inc. announced on January 7 the initiation of a phase 1/2 study of DTX101, the company鈥檚 lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression of blood clotting Factor IX (FIX) in patients with hemophilia B, a rare genetic bleeding disorder resulting from a deficiency in FIX. The current standard of care, chronic replacement of FIX protein through routine intravenous infusion, often does not achieve sustained, durable FIX expression, potentially putting patients at risk for spontaneous bleeds and long term joint destruction. In 2013, the World Federation of Hemophilia estimated there were more than 28,000 hemophilia B patients worldwide, including 4,000 patients in the United States.

Dimension received orphan drug designation and fast track status from the U.S. Food and Drug Administration for DTX101 in hemophilia B last year.

鈥淭he progression of the Dimension pipeline into the clinic represents an important milestone for the company,鈥 said Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension. 鈥淲e are pleased with the progress of DTX101, and believe it holds substantial potential to address the need for new, improved therapies for hemophilia B patients who are still struggling to manage the ongoing challenges, including joint destruction and recurrent episodes of bleeding.鈥

Dimension鈥檚 phase 1/2 clinical trial of DTX101 is a single arm, open-label, multi-center study, designed to evaluate the safety, dose and early efficacy of DTX101 in adult patients with moderate/severe to severe hemophilia B. Additional information about Dimension鈥檚 Phase 1/2 study of DTX101 may be found at ClinicalTrials.gov, using Identifier NCT: NCT02618915.

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