Spark Therapeutics Announces Multiple Presentations on Updated Hemophilia B Phase 1/2 Data at American Society of Hematology Annual Meeting

PHILADELPHIA, Nov. 30, 2016 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that Katherine High, M.D., president and chief scientific officer of Spark Therapeutics, and Lindsey A. George, M.D., hematologist and attending physician at the Children’s Hospital of Philadelphia, will present updated Phase 1/2 clinical trial data for SPK-9001 in hemophilia B at the 58th American Society of Hematology (ASH) Annual Meeting in San Diego.

• Dr. High, senior study author, will present during the ASH press briefing scheduled on Saturday, Dec. 3, 2016, at 8 a.m. PT
   
• Dr. George, lead study investigator, will present during the Plenary Scientific Session on Sunday, Dec. 4, 2016, between 4 and 6 p.m. PT

Spark Therapeutics management will also host a conference call on Monday, Dec. 5, 2016, at 8 a.m. ET to discuss the data presented at the meeting. The conference call can be accessed by dialing (855) 851-4526 (domestic) or (720) 634-2901 (international), and entering passcode 29837698. To access a live audio webcast, please visit the “Investors” section at www.sparktx.com.

A replay of the call will be available for one week following the call and can be accessed by dialing (855) 859-2056 (domestic) or (404) 537-3406 (international), and entering passcode 29837698 or by visiting the “Investors” section at www.sparktx.com.

About Spark Therapeutics
Spark Therapeutics, a fully integrated company, is challenging the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), liver-mediated diseases such as hemophilia, and neurodegenerative diseases. 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 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 in a Phase 1/2 trial for hemophilia B being developed in collaboration with Pfizer (which also has received both breakthrough therapy and orphan product designations) and SPK-8011, a preclinical candidate for hemophilia A to which Spark Therapeutics retains global commercialization rights. To learn more about us and our growing pipeline, visit www.sparktx.com.

Spark Therapeutics Corporate Contacts
Stephen W. Webster, Chief Financial Officer
Daniel Faga, Chief Business Officer
(855) SPARKTX (1-855-772-7589)

Media Contact
Dan Quinn
Ten Bridge Communications
(781) 475-7974
[email protected]