"TPP1 deficiency exacts a terrible toll on young children and their families, and effective therapies that slow or stop the progression of disease are urgently needed," said Dr. Davidson. "We are highly encouraged by these results, including the clinical impact seen in this study and the successful use of a novel route of administration of gene therapy to induce the production of an enzyme like TPP1 throughout the central nervous system.""Dr. Davidson's team has made important strides in demonstrating the potential of one-time administration of gene therapy to alter the course of this fatal neurodegenerative disease," said Katherine A. High, MD, co-founder, president and chief scientific officer of Spark. "As we advance this program toward the clinic, we are building on the success of our SPK-RPE65 program, including applying the same vector and manufacturing technology to potentially overcome the formidable challenge of delivering enzyme replacement therapies across the blood-brain barrier to the CNS." 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, 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. Cautionary Note on Forward-looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company's lead SPK-TPP1 program as well as its most advanced product candidate, SPK-RPE65. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the data from the early pre-clinical studies of SPK-TPP1 will not be replicated in IND-enabling studies or, that the results of additional preclinical studies will not be sufficient to support advancing into human clinical trials and that the company may not be able to successfully develop and/or commercialize any products from our SPK-TPP1 program or otherwise. 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, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark undertakes no duty to update this information unless required by law.
ContactsInvestor RelationsSpark Therapeutics Inc.Stephen W. WebsterChief Financial Officer(855) SPARKTX (1-855-772-7589)MediaTen Bridge Communications Inc.Dan Quinn(781) firstname.lastname@example.orgFinancial MediaTeneo StrategyAndy Maas(212) email@example.com