Spark Therapeutics to Participate in Multiple Upcoming Conferences

PHILADELPHIA, Sept. 21, 2016 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company seeking to transform the lives of patients with debilitating genetic diseases by developing potential one-time, life-altering treatments, announced today that members of company management or key investigators will present at the following upcoming conferences:

2016 Leerink Partners Rare Disease Roundtable Series, at the Lotte NY Palace in New York City

• Stephen Webster, chief financial officer, will participate in the Rare Disease and Immuno-Oncology roundtable discussion on Wednesday, Sept. 28, at 3:40 p.m. (EDT)

XII International Conference of the Argentinian Society of Retina and Vitreous, at the Hotel Arakur in Ushuaia, Argentina

• Paolo Falabella, M.D., Medical Affairs Ophthalmic Lead, will talk about “Investigational ocular gene therapy for RPE65-mediated retinal dystrophy” on Monday, Oct. 3, at 12:50 p.m. (EDT)

2016 European Association for Vision and Eye Research (EVER) Congress, at the Acropolis Convention Center in Nice, France

• Bart Leroy, M.D., director of the Ophthalmic Genetics and Retinal Degenerations clinics in the Division of Ophthalmology and Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, will speak about “Current gene therapy trials for inherited retinal disorders” on Wednesday, Oct. 5, at 5:56 p.m. (CET). Dr. Leroy is an investigator in the clinical development program for voretigene neparvovec, Spark Therapeutics’ lead investigational gene therapy for RPE-65 mediated inherited retinal disease.

2016 NORD Rare Diseases & Orphan Products Breakthrough Summit 2016, at the Hyatt Regency Crystal City in Arlington, VA

• Jeffrey D. Marrazzo, chief executive officer, will participate in the "Potential Advances Through Genetic Innovation" panel discussion, on Monday, Oct. 17, at 11 a.m. (EDT)           

MIT Review emTech Conference, at the MIT Media Lab in Boston

• Katherine High, M.D., president and chief scientific officer, will speak about “Gene Therapy - A New Era of Medicine” on Tuesday, Oct. 18, at 11 a.m. (EDT)

2016 European Society of Gene & Cell Therapy (ESGCT), at the Palazzo dei Congressi e degli Affari in Florence, Italy

• Katherine High, M.D., president and chief scientific officer, will participate in the In vivo Gene Therapy session on Thursday, Oct. 20, at 10:30 a.m. (CET)

Additionally, company representatives will attend the following conference for one-on-one meetings:

• Jefferies Gene Editing/Gene Therapy Summit, at the Jefferies Conference Center, in New York City on Tuesday, Oct. 11

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 potential 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-associated diseases, such as hemophilia, and neurodegenerative diseases.  Spark Therapeutics’ validated platform has successfully delivered proof-of-concept data with investigational gene therapies in the eye and liver. Spark Therapeutics’ most advanced product candidate, voretigene neparvovec (formerly referred to as SPK-RPE65), which has received both breakthrough therapy and orphan product designations, reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of a rare blinding condition. Spark Therapeutics’ hemophilia franchise has two lead assets: SPK-9001 in a Phase 1/2 trial for hemophilia B and SPK-8011, a preclinical candidate for hemophilia A.  To learn more, please visit www.sparktx.com.

Spark Therapeutics Corporate Contacts
Stephen W. Webster
Chief Financial Officer

Daniel Faga
Chief Business Officer

(855) SPARKTX (1-855-772-7589)

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