Spark Therapeutics to Participate in Multiple Meetings in May

PHILADELPHIA, May 03, 2017 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that members of company management or key investigators will present scientific data at the following May meetings:

Association for Research in Vision and Ophthalmology (ARVO) 2017 Annual Meeting, at the Baltimore Convention Center

• Kathleen Z. Reape, M.D., head of clinical R&D, will present data on the “Natural History of Individuals with Retinal Degeneration Due to Biallelic Mutations in the RPE65 Gene,” on Monday, May 8, from 8:30 to 10:15 p.m. EDT.
• Daniel C. Chung, D.O., M.A., clinical ophthalmic lead, will present data on the “Correlation of multi-luminance mobility testing with visual function tests in a Phase 3 trial of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease,” on Tuesday, May 9, between 11 a.m. - 12:45 p.m. EDT.
• Stephen R. Russell, M.D., clinical director of the Stephen A. Wynn Institute for Vision Research, University of Iowa, will present “Year 2 results for a Phase 3 trial of voretigene neparvovec in biallelic RPE65-mediated inherited retinal disease,” on Wednesday, May 10, from 8:30 to 10:15 a.m. EDT.
• Tomas S. Aleman, M.D., associate professor of ophthalmology at the Perelman School of Medicine, University of Pennsylvania, will present “AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: Preliminary Six Month Safety Results of an Ongoing Phase I/II Gene Therapy Trial” on Wednesday, May 10, from 11:00 a.m. to 12:45 p.m. EDT.

American Society of Gene & Cell Therapy 2017 Annual Meeting, at the Marriott Wardman Park Hotel in Washington, DC

• Katherine A. High, M.D., president and chief scientific officer, will present on “Clinical-Development Pathways in Gene-based Therapeutics,” as part of the Clinical Pathways from Lab to (Almost) Licensure session on Tuesday, May 9, from 2:20 to 4 p.m. EDT
• Xavier M. Anguela, Ph.D., head of discovery, will present “SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B at the Low Vector Dose Achieved Sustained, Continuous Factor IX Activity Levels Adequate for Endogenous Prophylaxis Preventing Bleeding Episodes and Reducing the Risk of Immune Response,” during the Clinical Trials Spotlight Symposium on Saturday, May 13, from 8:15 to 8:35 a.m. EDT.

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. Our validated platform successfully has delivered proof-of-concept data with investigational gene therapies in the retina and liver. Our most advanced investigational candidate, voretigene neparvovec, in development for the treatment of biallelic RPE65-mediated IRD, has received orphan designations in the U.S. and European Union, and breakthrough therapy designation in the U.S. The pipeline also includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two hemophilia development programs: SPK-9001 (which also has received both breakthrough therapy and orphan product designations by the FDA, and access to the PRIority MEdicines (PRIME) Program by the EMA) in a Phase 1/2 trial for hemophilia B being developed in collaboration with Pfizer, and SPK-8011, in a Phase 1/2 trial for hemophilia A to which Spark Therapeutics retains global commercialization rights. To learn more about us and our growing pipeline, visit www.sparktx.com.

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(215) 239-6424			

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