At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.
Founded in March 2013 as a result of the technology and know-how accumulated over two decades at Children’s Hospital of Philadelphia (CHOP), our investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed our scientists to tailor investigational therapies to patients suffering from very specific genetic diseases. This approach holds great promise in developing effective treatments to a host of inherited diseases. Our initial focus is on treating orphan diseases.
Headquartered in dynamic Philadelphia, we are a diverse, experienced team united in our goal to break down barriers for people and families affected by genetic diseases. As our name suggests, our investigational, one-time therapies are designed to spark healthy biology, and deliver potentially life-altering transformation for people and families affected by genetic disease. Spark is a member of the Roche Group.
Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now.
A world where no life is limited by genetic disease.
Dr. Federico Mingozzi is the chief scientific officer at Spark Therapeutics, bringing two decades of experience in gene therapy, immunology, as well as biochemistry and molecular biology in both industry and academic settings.
Federico began his scientific career studying the genetic bases of bleeding disorders. At the Children’s Hospital Philadelphia (CHOP) he conducted pioneering studies on liver gene transfer with adeno-associated virus (AAV) vectors and immunology. Federico was involved in several first-in-human clinical studies of gene therapy based on the AAV vector platform while serving as the director of translational research the Center for Cellular and Molecular Therapeutics, at CHOP. He also led studies aimed at the characterization of human immune responses to AAV vectors and the development of strategies to modulate vector immunogenicity.
He then joined the French National Institute of Health and Medical Research (INSERM) as Research Director and Genethon, a leading French nonprofit R&D organization focused on gene therapy for rare diseases, as Team Leader. There, he spearheaded the development of in vivo gene therapies for inherited diseases. His work continued to focus on the characterization of human immune responses to AAV vectors and on the development of strategies to overcome immune responses in gene transfer.
Federico currently serves as faculty at the Pierre and Marie Curie University in Paris, France, and Universitat Autonoma de Barcelona, Spain. He received his bachelor’s degree in biology and his Ph.D. in biochemistry and molecular biology from the University of Ferrara in Italy, and his M.B.A. from Drexel University. Throughout his distinguished career, Federico has received several awards and has contributed to more than 90 publications, including seminal findings in the field of AAV gene therapy.
Jeff Marrazzo founded and has led the growth of Spark Therapeutics from a research center within the Children’s Hospital of Philadelphia to a fully integrated, commercial gene therapy company that is challenging the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginable – until now.
Under Jeff’s leadership, Spark received regulatory approval for the first gene therapy for a genetic disease approved in both the U.S. and EU, launched the first gene therapy for a genetic disease in the United States, and established human proof-of-concept of Spark’s gene therapy platform in both the retina and liver. In less than seven years, Jeff has built an organization of more than 450 colleagues while securing over $1 billion in capital and successfully orchestrating a merger with Roche. Spark has been named to the top 10 of Science Magazine’s 2019 Top Employer list, listed on the Philadelphia Business Journal’s Best Places to Work list three consecutive years and was awarded the Prix Galien for Best Biotechnology Product in 2019.
Prior to Spark, he helped build the first genetic testing benefit management up to and through the acquisition of a majority of the company’s shares by CVS Caremark. Previously, Jeff served as an advisor to former Pennsylvania Governor Edward G. Rendell, where he led reforms in the financing and delivery of healthcare.
Jeff currently serves as on the boards of the Life Science Cares Philadelphia and The Rendell Center for Civics and Civic Engagement. Jeff received a B.A. in economics and B.S.E. in systems science and engineering from the University of Pennsylvania. He also holds a dual M.B.A. / M.P.A. from The Wharton School of the University of Pennsylvania and Harvard University, a program which he founded.
Jeff can be found on Twitter @JeffMarrazzo.
Dr. Kathy High, an accomplished hematologist with a longstanding interest in gene therapy for genetic disease, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders.
Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to adeno-associated viral (AAV) vectors in a variety of target tissues. Kathy’s work has evolved to encompass clinical translation of potential gene therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia (CHOP), Kathy assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application.
As president at Spark Therapeutics, she has led the development and received regulatory approval of the first gene therapy for a genetic disease approved in both the U.S. and EU. Spark has shown human proof-of-concept of its leading gene therapy platform in both the retina and the liver, and has received breakthrough therapy designations for three different therapeutics across two different tissue targets, the eye and the liver. In addition to her operational role, Kathy serves as a member of the Board of Directors of Spark.
Kathy was a long-time member of the faculty at the University of Pennsylvania and of the medical staff at CHOP, where she was also an Investigator of the Howard Hughes Medical Institute. She served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy (ASGCT). She received her A.B. in chemistry from Harvard University, an M.D. from the University of North Carolina School of Medicine, a business certification from the University of North Carolina Business School Management Institute for Hospital Administrators and an M.A. from the University of Pennsylvania.
Ron Philip leads all aspects of product strategy at Spark, including new product and commercial development activities for Spark’s early and late-stage pipeline assets. He is also responsible for all commercial operations including sales, marketing, diagnostics, market access and patient services functions for inline products. At Spark, he has successfully led the development and implementation of commercial strategy, novel distribution and reimbursement models for the first gene therapy approved for a genetic disease in the U.S.
Ron has more than 20 years of experience in the pharmaceutical industry with a proven track record of improving operating performance in multiple therapeutic categories and global markets. Prior to joining Spark Therapeutics, he served in several senior leadership roles at Pfizer including Regional President – Africa Middle East, Head of Global Business Unit Strategy, Head of Business Development Search & Evaluation and Commercial Development within Pfizer’s Innovative Products Business.
He previously worked at Wyeth Pharmaceuticals in U.S. commercial operations, with Deloitte Consulting in their strategy and operations practice and at Merck and Co. in the manufacturing and marketing divisions.
Ron is an alumnus of Drexel University.
Lisa Dalton, who has more than 20 years of human resources experience across the biopharmaceutical and financial services industries, is responsible for four core functions — HR, IT, Corporate Facilities and Corporate Communications — focused on how to enable and empower Spark’s most important resource – its talented people. Lisa has been instrumental in helping lead the company’s growth from a few dozen people then to more than 450 people today. She began her tenure at Spark Therapeutics in July 2014 as the Head of Human Resources and joined the company’s corporate management team as Chief Human Resource Officer in 2018.
Lisa was previously vice president, human resources at Shire, where she led various human resources functions to meet the needs of a high-growth organization, including human resources-related merger and acquisition efforts, the total rewards function and the human resources operations function. While on international assignment in Switzerland, Lisa played a critical role in planning and launching Shire’s European hub, where she was a member of the site leadership team, championed the culture and values of the organization and had oversight of the talent strategies for the newly established site.
Lisa also has held roles in human resources with Franklin Templeton Investments, Applied Biosystems and Goldman Sachs.
Lisa serves as a member of the Board of Advisors of Life Science Cares Philadelphia. She holds an M.B.A. from Rutgers University School of Business and a B.A. in psychology from Pennsylvania State University.
Hari Pujar, Ph.D., serves as Chief Technology Officer of Spark Therapeutics, leading the technical operations, process and technology development and quality assurance organizations. He is responsible for growing and scaling the company’s cutting-edge technology capabilities for an expanding development pipeline.
Prior to joining Spark, Hari was most recently vice president, Technical Development & Manufacturing, at Moderna Therapeutics. There he built and led the technology and early manufacturing organizations that delivered on supply for more than a dozen clinical programs in a brand-new technology area. Previously, he held a variety of scientific and cross-functional leadership positions during 18 years at Merck & Co in the company’s commercial, R&D and manufacturing divisions.
Hari has a Ph.D. in Chemical Engineering from the University of Delaware and an MBA from the Wharton School at the University of Pennsylvania.
Joe serves as the Chief Legal Officer of Spark Therapeutics, Inc., where he oversees legal, compliance, government affairs and pricing and reimbursement. Spark is a fully integrated biotechnology company committed to discovering, developing and delivering gene therapies that challenge the inevitability of genetic diseases, including blindness, hemophilia and neurodegenerative diseases. Spark has received regulatory approval for the first gene therapy approved in both the U.S. and EU, launched the first gene therapy for a genetic disease in the United States, and has established human proof-of-concept of Spark’s gene therapy platform in both the retina and liver.
In his five years at Spark, Joe has led multiple public and private financings, raising more than $1 billion in capital since its founding in 2013 and been instrumental in the development of novel distribution and reimbursement models for one-time therapeutics. Joe currently serves on the Boards of Directors of Life Sciences Pennsylvania and the National Pharmaceutical Council.
Kathy Reape is the Chief Medical Officer of Spark Therapeutics and oversees clinical, pharmacovigilance and medical affairs activities. With more than 18 years of pharmaceutical industry experience in clinical drug development, Kathy has overseen numerous clinical trials and had a hands-on role in the approvals of more than two dozen products including small molecules, biologics, biosimilars, and devices spanning a wide range of therapeutic areas.
Prior to joining Spark, Kathy was the president of Ark Medical Consulting LLC where she provided pharmaceutical and medical consulting and the Senior Vice President, Head of Clinical Development, Global Brands R&D for Allergan, plc where she led the Clinical Development function for the entire brand R&D portfolio, including projects in ophthalmology, cardiovascular, gastrointestinal, anti-infectives, respiratory, women’s health, urology, dermatology/aesthetics, and central nervous system (CNS). She also oversaw all development work related to Allergan’s onabotulinumtoxinA (BOTOX®). Kathy has also held leadership positions at Teva Pharmaceuticals, Duramed Research and Wyeth Pharmaceuticals.
She received her M.D. from the University of Pennsylvania and completed her internship and residency at the University of Florida and the University of Medicine and Dentistry of New Jersey.