Spark Therapeutics Announces Multiple Presentations on Updated Hemophilia A and Hemophilia B Phase 1/2 Clinical Trials at 59th American Society of Hematology Annual Meeting and Exposition

PHILADELPHIA, Dec. 05, 2017 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that Lindsey A. George, M.D., attending physician in the Division of Hematology at Children’s Hospital of Philadelphia, will present data on the company’s hemophilia A and B investigational gene therapies at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition at the Georgia World Congress Center in Atlanta.

Dr. George will be presenting twice on Monday, Dec. 11, during the “Gene Therapy and Transfer: Gene Therapy for Hemophilia and Improving Lentiviral Vectors” session, to be held in auditorium C101, on level 1 in building C at the Georgia World Congress Center:

• “SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B – 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence” at 7 a.m. ET
• “SPK-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia A” at 7:45 a.m. ET

Additionally, Daniel J. Hui, Ph.D., cell-based assay lead, Spark Therapeutics, will present a poster, “SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response,” on Saturday, Dec. 9, at 5:30 p.m. ET during the “Gene Therapy and Transfer: Poster I” session, to be held in auditorium A2, on level 1 in building A at the Georgia World Congress Center.

Investor Teleconference
Spark Therapeutics will host a teleconference at 12:30 p.m. ET, on Monday, Dec. 11, 2017, to discuss these data and address inquiries from analysts and investors. To access a live audio webcast, please visit the “Investors” section at www.sparktx.com, or dial (855) 851-4526 (domestic) or (720) 634-2901 (international), and enter the passcode 8486925. Materials related to the call will be available at the same website prior to the call.

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 8486925, or by visiting the “Investors” section at www.sparktx.com.

About Spark Therapeutics
At Spark Therapeutics, a fully integrated company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia and neurodegenerative diseases. We have successfully applied our technology directed to the retina and liver, and currently have four programs in clinical trials or under regulatory review, including the first potential gene therapy for a genetic disease in the United States and product candidates that have shown promising early results in patients with hemophilia. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.

Investor Contact:                                                                                             
Ryan Asay                                                                                                           
[email protected]                                                                                 
(215) 239-6424                                                                                                               

Media Contact:
Monique da Silva
[email protected]
(215) 282-7470