PHILADELPHIA, June 18, 2015 (GLOBE NEWSWIRE) -- Spark Therapeutics (Nasdaq:ONCE) today announced that it, along with collaboration partner, Pfizer Inc., will present the results of preclinical testing that supports the safety and efficacy of the lead compound in their SPK-FIX hemophilia B program at the International Society on Thrombosis and Haemostasis (ISTH) during the ISTH 2015 Congress taking place in Toronto, Ontario, Canada from June 20th through June 25th. ISTH is a global, not-for-profit organization advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders and their Congress is the largest global event in the field.

The lead compound being jointly developed by Pfizer and Spark under their SPK-FIX program incorporates a novel, bio-engineered adeno-associated virus (AAV) capsid containing a codon-optimized, high-activity human Factor IX variant (FIX-Padua). Pfizer and Spark will present preclinical safety and efficacy data on the product candidate including its:

  • Safety profile
  • Neutralizing antibody profile in human serum
  • FIX antigen and activity levels
  • Biodistribution.

The poster will be presented on June 22nd by Dr. Xavier Anguela, Spark's Discovery Research Leader.

About Spark Therapeutics

Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating orphan diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, is in a fully enrolled pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark is leveraging the experience and technology utilized in the development of SPK-RPE65 to address a broad spectrum of blinding conditions, starting with the development of SPK-CHM for the potential treatment of choroideremia, currently in a Phase 1/2 clinical trial. Spark also is establishing a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative diseases, including through a global collaboration with Pfizer Inc. for the development and commercialization of its SPK-FIX program for the treatment of hemophilia B. Spark's integrated gene therapy platform builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more, please visit

Cautionary Note on Forward-looking Statements

This press release includes "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. For example, the data seen in our preclinical studies may not be indicative of the results we may see in our planned Phase 1/2 clinical trial or any subsequent clinical trial. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. We do not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Relations

Spark Therapeutics, Inc.
Stephen W. Webster
Chief Financial Officer
(888) 772-7560


Media Contact

Jessica Rowlands
(202) 729-4089