Spark Therapeutics to Participate in Multiple Conferences in September

PHILADELPHIA, Aug. 30, 2017 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that company management will present at or attend several September conferences.

Company management will present at the following conferences:

• Dan Faga, chief business officer, will participate in a panel discussion at the Global Genes Rare Partnering & Investor Forum on “Alternatives to Standard Newcos: Spin-offs, Build-to-Buys, Joint Ventures and Alliances” on Thursday, Sept. 14, at 11:20 a.m. PDT, at the Hotel Irvine in Irvine, Calif.
• Jeffrey D. Marrazzo, chief executive officer, will participate in a panel discussion at Life Sciences Future on “Hear from the Heart of the Cellacon Valley” on Tuesday, Sept. 19, at 10:15 a.m. EDT, at the Philadelphia Downtown Marriott in Philadelphia
• John Furey, chief operating officer, will participate in a panel discussion at the Pharmaceutical Care Management Association Annual Meeting on “Challenges in Payment and Delivery Systems for Some Novel Therapies” on Tuesday, Sept. 26, at 10:30 a.m. MST, at the Westin Kierland in Scottsdale, Ariz.
• Katherine A. High, M.D., president and chief scientific officer, will present at the Deutsche Ophthalmologische Gesellschaft Genetics Congress on Thursday, Sept. 28, at 4:30 p.m. CET, at the Estrel Congress Center in Berlin

Additionally, company management will present at the following investor conferences:

• Wells Fargo Securities Healthcare Conference on Wednesday, Sept. 6, at 3:35 p.m. EDT, at the Westin Boston Waterfront in Boston
• Citi Biotech Conference in a gene therapy panel discussion on Thursday, Sept. 7, at 11:00 a.m. EDT, at the Mandarin Oriental in Boston
• Morgan Stanley Global Healthcare Conference on Wednesday, Sept. 13, at 10:30 a.m. EDT, at the Grand Hyatt in New York
• Cantor Fitzgerald Global Healthcare Conference on Tuesday, Sept. 26, at 3:25 p.m. EDT, at the InterContinental New York Barclay Hotel in New York
• Leerink Partners Roundtable Series: Rare Disease & Immuno-Oncology on Wednesday, Sept. 27, at 10:30 a.m. EDT, at the Lotte New York Palace in New York

Company management will also participate in the Goldman Sachs Biotech Symposium on Friday, Sept. 8, in London.

About Spark Therapeutics
Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. Spark Therapeutics has ongoing clinical trials investigating gene therapies in hemophilia A and B. SPK-8011 is in an ongoing, dose-escalation Phase 1/2 clinical trial as a potential one-time therapy for hemophilia A. The company retains full global commercialization rights to the SPK-FVIII program for hemophilia A. SPK-9001, which has received both breakthrough therapy and orphan product designations by FDA, and access to the PRIority MEdicines (PRIME) Program by EMA, is in a Phase 1/2 clinical trial for hemophilia B and is being developed in collaboration with Pfizer. Our most advanced investigational candidate, with proposed trade name LUXTURNA™ (voretigene neparvovec), is currently under Priority Review with FDA for the treatment of biallelic RPE65-mediated IRD and has been designated as a drug for a rare pediatric disease. The MAA for LUXTURNA has been submitted to EMA for the treatment of vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. LUXTURNA has received breakthrough therapy and orphan product designations from FDA and orphan product designations from EMA. The pipeline also includes SPK-7001 in an ongoing Phase 1/2 clinical trial for choroideremia. For more information on our pipeline, visit www.sparktx.com.

Investor Contact:
Ryan Asay
[email protected] 
(215) 239-6424

Media Contact:
Monique da Silva
[email protected]	
(215) 282-7470