Spark Therapeutics to Participate in Multiple Conferences in October

PHILADELPHIA, Oct. 03, 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 the following October conferences.

European Society of Gene & Cell Therapy (ESGCT) Congress, at the Berlin Congress Center in Berlin

• Federico Mingozzi, Ph.D., chief scientific officer, will present on “SPK-9001 – an Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B” on Thursday, Oct. 19, at 2:10 p.m. CET

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

• Chardan Gene Therapy Conference on Tuesday, Oct. 10, at 11:40 a.m. EDT, at the Westin Grand Central in New York
• Jefferies Gene Technology Investor Summit on Thursday, Oct. 12, at 9:00 a.m. EDT, at the Westin Grand Central in New York

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 SPK-8011. SPK-9001 is in a Phase 1/2 clinical trial for hemophilia B and is being developed in collaboration with Pfizer. It has received both breakthrough therapy and orphan product designations from the Food and Drug Administration (FDA), and access to the PRIority MEdicines (PRIME) Program from the European Medicines Agency (EMA). Our most advanced investigational candidate, with proposed trade name LUXTURNA™ (voretigene neparvovec), is currently under Priority Review with FDA for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated IRD and has been designated as a drug for a rare pediatric disease. The Marketing Authorization Application for LUXTURNA has been validated by EMA for the treatment of patients with vision loss due to Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) IRDs 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, visit www.sparktx.com, and follow us on Twitter and LinkedIn.