Spark Therapeutics Announces Acquisition of Genable Technologies

PHILADELPHIA, March 07, 2016 -- Spark Therapeutics (NASDAQ:ONCE) today announced that it has acquired Dublin, Ireland-based Genable Technologies, a private gene therapy innovator with which Spark has collaborated since 2014 in the development of Genable's therapeutic program targeting one of the most prevalent forms of inherited retinal disease (IRD).

With the acquisition, Spark acquires RhoNova™, a potential treatment targeting rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP), an IRD that routinely leads to visual impairment and in the most severe cases to blindness. Using an adeno-associated virus (AAV) vector developed and manufactured at Spark, RhoNova™ is designed to both suppress the expression of a faulty gene and deliver normal copies of the RHO gene to restore normal expression. RhoNova™ has been granted Orphan Drug Designation in both the U.S. and Europe in addition to the Advanced Therapy Medicinal Product designation from the European Medicines Agency. There is currently no approved pharmacologic treatment for RHO-adRP, which affects an estimated 12,000 patients in the United States and the five major European markets (EU5).

“This transaction advances our strategy of leveraging Spark’s best-in-class gene therapy platform through a combination of internal innovation, acquisition and collaboration,” said Jeffrey D. Marrazzo, Spark co-founder and chief executive officer. “Genable’s technology and promising RhoNova™ development program further strengthens our portfolio of treatments for IRDs, which is led by our Phase 3 program for RPE65-mediated blindness which recently reported overwhelmingly positive pivotal stage data.”

“Genable has provided convincing data in animals to support their innovative scientific approach for the treatment of autosomal dominant diseases, where it is critical to both suppress faulty genes and replace them with a functional copy in order to have a meaningful therapeutic effect,” said Katherine A. High, MD, co-founder, president and chief scientific officer of Spark. “We look forward to using our validated gene therapy development platform and expertise to accelerate the clinical development of RHoNova, while examining other potential applications for the Genable suppression/augmentation approach outside of this initial indication.”

The consideration paid to Genable shareholders consisted of $6 million in cash and 265,000 shares of Spark common stock. Additional financial terms were not disclosed.

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.

About Genable Technologies 
Genable Technologies Ltd. is a privately held, venture-capital-backed, Dublin (Ireland)- based gene therapy company. Genable is developing new gene therapies to treat "dominant" genetic diseases. Genable has received significant support and investment form Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland, Foundation Fighting Blindness Clinical Research Institute (U.S.), Trinity College Dublin and Enterprise Ireland. To learn more please visit www.genable.net.

Cautionary Note on Forward-looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. 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 preclinical studies of RhoNova 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 Genable 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.

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]