Spark Therapeutics Further Expands Gene Therapy Expertise with Appointment of Federico Mingozzi, Ph.D., as Chief Scientific Officer

PHILADELPHIA, Sept. 25, 2017 -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Federico Mingozzi, Ph.D., as chief scientific officer. He will report to Katherine A. High, M.D., who will continue as the company’s president and head of R&D. Dr. High will also continue to serve on the board of directors.

“We are excited to have Federico join and expand our exceptional scientific team at Spark Therapeutics. He brings a wealth of experience in research and early-stage clinical investigation of gene therapies for rare inherited diseases to Spark Therapeutics,” said Dr. High. “His deep understanding of cutting-edge vector design and human immune responses to adeno-associated viral (AAV) vectors further bolsters our ability to deliver on our growing pipeline of clinical programs and platform capabilities.”

Dr. Mingozzi joins Spark Therapeutics from the French National Institute of Health and Medical Research (INSERM) and Genethon, a leading French nonprofit R&D organization focused on rare diseases, where he led the development of liver-targeted gene therapies for inherited diseases such as Crigler-Najjar syndrome, and worked on the characterization of immunity and tolerance in gene transfer. He serves as faculty at the Pierre and Marie Curie University in Paris and Universitat Autonoma de Barcelona, Spain.

Dr. Mingozzi has had a distinguished career in gene therapy research, including earlier as director of translational research, Center for Cellular and Molecular Therapeutics, at Children’s Hospital of Philadelphia, where he was involved in the development of gene therapies based on the AAV vector platform.

“I am thrilled to work with Spark Therapeutics’ team to help advance gene therapy research programs directed at targets in the retina, liver and central nervous system,” said Dr. Mingozzi. “Spark Therapeutics is an established leader in the gene therapy industry. I believe it is one of the places where true innovation is breaking down barriers, and the company has the potential to change the lives of people with rare genetic diseases.”

Throughout his career, Dr. Mingozzi has contributed to more than 90 publications, including seminal findings in the field of AAV gene therapy. He has a Ph.D. in biochemistry and molecular biology from the University of Ferrara, Italy and an MBA from Drexel University in Philadelphia.

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.

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