Spark Therapeutics Announces Launch of aSharedVision, a New Online Resource to Unite and Inform Those Affected by Inherited Retinal Diseases

PHILADELPHIA, Feb. 29, 2016 -- Spark Therapeutics (NASDAQ:ONCE) today announced the launch of aSharedVision (www.aSharedVision.com), a new online resource designed to help patients, caregivers and health care professionals better understand inherited retinal diseases (IRDs) and the importance of genetic testing.

Launched to coincide with the annual observance of international Rare Disease Day, aSharedVision provides the community with an accessible overview of the family of genetic diseases collectively known as IRDs. More than 220 genes have been linked to these diseases, which are believed to affect hundreds of thousands of people in the United States.

“This is a moment of great change and optimism in the field of inherited retinal diseases, where cutting-edge science is showing that, for the first time, it may be possible to reverse, stop or slow vision loss in patients who were progressing toward blindness,” said Rogério Vivaldi, M.D., chief commercial officer at Spark. “In response, the medical system is shifting from clinical diagnoses to more precise and potentially actionable genetic-based diagnoses. As the leader in the development of gene therapies for IRDs, we have a responsibility to help lead this transition, and to provide patients, caregivers and health care professionals with information they need to make the most informed decisions.”

“The launch of aSharedVision is an important step forward for families affected by IRDs,” said Amber Bobnar, founder of the WonderBaby.org website. “By helping to unite and inform our diverse community, aSharedVision will help us find the resources we need to advocate for our families and loved ones.”

Developed in close consultation with members of the IRD community, aSharedVision features information about IRDs, a simple overview of the science behind the emerging field of gene therapy and guidance to help families work with health care professionals to seek a genetic diagnosis. In addition, consistent with the international Rare Disease Day 2016 theme of “Patient Voice,” the site contains profiles of individuals and families living with IRDs, and will continuously feature new content developed by community members.

About Spark Therapeutics
Spark is a gene therapy leader seeking to transform the lives of patients with debilitating genetic diseases by developing one-time, life-altering treatments. Spark’s initial focus is on treating rare diseases where no, or only palliative, therapies exist. Spark’s most advanced product candidate, SPK-RPE65 (voretigene neparvovec), which has received both breakthrough therapy and orphan product designation, recently reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark’s validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Spark 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 www.sparktx.com.

Contacts
Investor Relations
Spark Therapeutics, Inc.
Stephen W. Webster
Chief Financial Officer
(855) SPARKTX (1-855-772-7589)

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

Patients and Family Members
[email protected]