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Shire Submits IND Application for Hemophilia A Gene Therapy Candidate

July 10, 2017
Shire Submits IND Application for Hemophilia A Gene Therapy Candidate

Shire has announced the submission of an investigational new drug (IND) application to the US Food and Drug Administration (FDA) for SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A.

SHP654, also knowns as BAX 888, was developed using Shire’s proprietary bioengineered adeno-associated virus serotype 8 (rAAV8). rAAV8 acts as a delivery vehicle, or vector, to carry the genetic codes that prompt FVIII production. This approach, which is being tested in mice with hemophilia A, delivers the corrected FVIII gene specifically into liver cells where the protein is normally generated. If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.

According to a Shire press release, the IND filing for SHP654 was based on the results of pre-clinical and phase 1 studies demonstrating the potential utility of this candidate. Details of these trial results will be presented at 26th International Society on Thrombosis and Haemostasis Meeting, July 10, 2017, in Berlin, Germany.

“Shire is leveraging decades of scientific leadership in hemophilia to advance research in gene therapy for this community," said Paul Monahan, MD, Senior Medical Director, Gene Therapy, Shire. “Drawing from our rich heritage, Shire is well equipped to sustainably support the development of gene therapies that aim to advance current standards of care and minimize the burden of this disease. Our goal with gene therapy for hemophilia is to uphold the highest standards for safety and efficacy.”

Source:, July 6, 2017