Spark Therapeutics Announces Collaboration with Leading Gene Therapy Center

Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company seeking to transform the lives of patients with debilitating genetic diseases by developing investigational, potentially one-time, life-altering treatments, announced today a multi-year research agreement with Guangping Gao, Ph.D., the Penelope Booth Rockwell Chair in Biomedical Research, director of the Horae Gene Therapy Center, and professor of microbiology and physiological systems at the University of Massachusetts Medical School. Dr. Gao will collaborate broadly with Spark Therapeutics to identify adeno-associated virus (AAV) vectors from a proprietary library of AAV capsids and evaluate their efficacy, with the goal of enhancing the efficiency of gene delivery to cells in the retina, liver and central nervous system.

“We’re excited to have selected Spark Therapeutics as a collaborator to develop next-generation AAV vectors that deliver genetic information to certain cell types more efficiently and to cell types that are otherwise hard to target,” said Dr. Gao, an internationally renowned AAV and gene therapy researcher.
Under the agreement, Spark Therapeutics has the option to an exclusive, world-wide license for the intellectual property developed under the research agreement.
“We are delighted to be working with Professor Gao, a long-time leader in gene therapy and capsid development,” said Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics. “Strategically, these types of relationships and agreements help us build on our research in gene therapy and continue to advance the utility of our proven and proprietary gene therapy platform.”
About Spark Therapeutics
Spark Therapeutics, a fully integrated gene therapy company, is seeking to transform the lives of patients with debilitating genetic diseases by developing investigational, potentially one-time, life-altering treatments. Spark Therapeutics’ validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal diseases, liver-mediated diseases such as hemophilia, and neurodegenerative diseases. Spark Therapeutics’ validated platform successfully has delivered proof-of-concept data with investigational gene therapies in the retina and liver. Spark Therapeutics has reported top-line results from a pivotal Phase 3 clinical trial for its most advanced product candidate, voretigene neparvovec (formerly referred to as SPK-RPE65), a potential treatment of a rare genetic blinding condition. Voretigene neparvovec has received both breakthrough therapy and orphan product designations. Spark Therapeutics’ hemophilia franchise has two lead assets: SPK-9001 in a Phase 1/2 trial for hemophilia B being developed under a collaboration with Pfizer and SPK-8011, a preclinical candidate for hemophilia A to which Spark Therapeutics retains global commercialization rights. To learn more, please visit www.sparktx.com.
About the University of Massachusetts Medical School
The University of Massachusetts Medical School, one of five campuses of the University system, is comprised of the School of Medicine, the Graduate School of Biomedical Sciences, the Graduate School of Nursing, a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. Its mission is to advance the health of the people of the Commonwealth through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education. The Medical School attracts more than $266 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2006, UMMS’s Craig C. Mello, Ph.D., Howard Hughes Medical Institute Investigator and the Blais University Chair in Molecular Medicine, was awarded the Nobel Prize in Physiology or Medicine, along with colleague Andrew Z. Fire, Ph.D., of Stanford University, for their discoveries related to RNA interference (RNAi).

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