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PHILADELPHIA, July 08, 2016 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE) announced today that it will participate in the following conferences in July:
About Hemophilia B
Hemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot as a result of a deficiency in one of several blood clotting factors, and occurs almost exclusively in males. People with hemophilia face specific risks as they are not able to form blood clots efficiently and are at risk for excessive and recurrent bleeding from modest injuries, which have the potential to be life threatening. People with severe hemophilia often bleed spontaneously into their muscles or joints. The incidence of hemophilia B is one in 25,000 male births. People with hemophilia B have a deficiency in clotting factor IX, a specific protein in the blood. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. Current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor IX to control and prevent bleeding episodes. There exists a significant need for novel therapeutics to treat people living with hemophilia.
About the SPK-FIX Program
Spark's proprietary technology platform for selecting, designing, manufacturing and formulating highly optimized gene therapies was applied to developing compounds in the SPK-FIX program. The SPK-FIX program leverages a long track record of hemophilia gene therapy research and clinical development conducted by Spark and its founding scientific team over nearly three decades. SPK-9001 is a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized high-activity human factor IX variant enabling endogenous production of factor IX. SPK-9001 is being developed under a collaboration with Pfizer. Spark and Pfizer entered into a collaboration in 2014 for the SPK-FIX program, including SPK-9001, under which Spark is responsible for conducting all Phase 1/2 studies for any product candidates, while Pfizer will assume responsibility for pivotal studies, any regulatory activities and potential global commercialization of any products that may result from the collaboration.
About Spark Therapeutics
Spark Therapeutics, a fully integrated gene therapy company, is seeking to transform the lives of patients with debilitating genetic diseases by developing one-time, life-altering treatments. Spark’s validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal diseases, liver-associated diseases, such as hemophilia, and neurodegenerative diseases. Spark’s validated platform has successfully delivered gene therapies with proof-of-concept data in the eye and liver. Spark’s most advanced product candidate, voretigene neparvovec (formerly referred to as SPK-RPE65), which has received both breakthrough therapy and orphan product designation, reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark’s hemophilia franchise has two lead assets: SPK-9001 in a Phase 1/2 trial for hemophilia B and SPK-8011, a preclinical candidate for hemophilia A. To learn more, please visit www.sparktx.com.
ContactsCorporate Contacts:Stephen W. WebsterChief Financial OfficerSpark Therapeutics, Inc.Daniel FagaChief Business OfficerSpark Therapeutics, Inc.(855) SPARKTX (1-855-772-7589)Media Contact:Ten Bridge CommunicationsDan Quinn(781) firstname.lastname@example.org
Source:Spark Therapeutics, Inc.