Spark Therapeutics to Present Updated Hemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress

PHILADELPHIA, June 23, 2017 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that Lindsey George, M.D., attending physician in the Division of Hematology at Children's Hospital of Philadelphia, and a clinical investigator in the Phase 1/2 clinical trial of SPK-9001 for hemophilia B, will present updated interim data at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress in Berlin.

Additionally, Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics, will provide an overview of gene therapy research in hemophilia, including updated interim data on SPK-9001, during Tuesday’s “State-of-the-Art” session. Sylvia von Mackensen, Ph.D., senior scientist at the Institute of Medical Psychology, University Medical Centre Hamburg-Eppendof, Germany, will present a poster on quality-of-life data of participants in the SPK-9001 clinical trial.

Specific dates and times for the presentations are:

• “SPK-9001: Adeno-associated Virus Mediated Gene Transfer for Haemophilia B Achieved Durable Endogenous Prophylaxis at Levels of Activity Sufficient to Achieve Significant Mean Reduction in Annual Bleeding and Infusions Rates in Preliminary Data from an Ongoing Phase 1/2a Trial,” Lindsey George, M.D., on Monday, July 10, from 5:45 to 6 p.m. CET
• “Gene Therapy Replacement,” Katherine A. High, M.D., on Tuesday, July 11, from 8 to 8:25 a.m. CET
• “Preliminary Results of SPK-9001 Gene Transfer Demonstrate Statistical Improvements on the Health-related Quality-of-Life in Adults with Haemophilia B Speaker,” Sylvia von Mackensen, Ph.D., poster session on Tuesday, July 11, from 12 to 1:15 p.m. CET

About Spark Therapeutics
Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Our validated platform successfully has delivered proof-of-concept data with investigational gene therapies in the retina and liver. Our most advanced investigational candidate, voretigene neparvovec, in development for the treatment of biallelic RPE65-mediated IRD, has received orphan designations in the U.S. and European Union, and breakthrough therapy designation in the U.S. The pipeline also includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two hemophilia development programs: SPK-9001 (which also has received both breakthrough therapy and orphan product designations by the FDA, and access to the PRIority MEdicines (PRIME) Program by the EMA) in a Phase 1/2 trial for hemophilia B being developed in collaboration with Pfizer, and SPK-8011, in a Phase 1/2 trial for hemophilia A to which Spark Therapeutics retains global commercialization rights. To learn more about us and our growing pipeline, visit www.sparktx.com.

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