Our team includes world leaders in the research, development and manufacture of gene therapies, as well as seasoned commercial, entrepreneurial and executive management.
Our team’s experience in the clinical study of gene therapy - from designing and manufacturing vectors to conducting clinical trials - is unparalleled. Clinical-grade vectors prepared by our team have been used to safely treat more than 150 human subjects in 12 clinical trials in the U.S. and EU, across five routes of administration.
Senior members of our scientific team are responsible for numerous development milestones in the gene therapy field, including the first clinical trials of AAV in skeletal muscle tissue, the liver and the sub-retinal space, and the first gene therapy trial for a non-lethal disorder including pediatric patients. To this group, we have added a team of industry executives with experience in commercialization, manufacturing, finance, business development, human resources and legal affairs of novel biotechnology companies and products.
Jeffrey D. Marrazzo is the co-founder and chief executive officer of Spark Therapeutics, Inc. (NASDAQ: ONCE) and serves on the board of directors. Jeff has led the creation and growth of Spark from a research center within The Children's Hospital of Philadelphia to a publicly-traded, integrated gene therapy company developing one-time, life-altering treatments to transform the lives of patients and re-imagine the treatment of debilitating diseases. Under Jeff's leadership, Spark has raised more than $265 million in capital, established a global collaboration with Pfizer for the development and commercialization of a gene therapy for hemophilia B, recruited some of the world's pioneers in gene therapy together with a team of biotechnology industry executives, and advanced the company's lead product candidate for the treatment of rare, blinding conditions to a fully-enrolled Phase 3 clinical trial. Jeff is an experienced entrepreneur dedicated to curing disease through precision medicine, having helped build and sell the first genetic testing benefit management and pharmacogenomics medicines company to CVS Caremark. Previously, Jeff served as an advisor to former Pennsylvania Governor Edward G. Rendell. Jeff received his B.A. in economics, magna cum laude, and B.S.E. in systems science and engineering, magna cum laude, from the University of Pennsylvania, and holds a dual M.B.A. / M.P.A. from The Wharton School and Harvard University, a program which he founded.
Dr. Katherine High, a world renowned hematologist, 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 virus (AAV) vectors in a variety of target tissues. Kathy’s work has evolved to encompass clinical translation of genetic therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia, 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. Kathy 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, business certification from the UNC Business School Management Institute for Hospital Administrators and a master’s degree from the University of Pennsylvania.
Dr. Rogério Vivaldi is the former CEO and president of Minerva Neurosciences, Inc., a public, clinical-stage biopharmaceutical company focused on the development and commercialization of a portfolio of product candidates to treat patients suffering from neuropsychiatric diseases. He previously held a variety of senior roles at Genzyme, ultimately leading the company’s rare disease business and earning a reputation as a passionate advocate for patients. Dr. Vivaldi led the successful approval and reimbursement of more than 15 orphan products across more than 20 countries throughout the world. Bringing his medical and professional expertise to Genzyme, Dr. Vivaldi served as senior vice president of Rare Diseases having responsibility for Genzyme’s rare diseases business and the renal and endocrinology group. He also served as senior vice president and general manager of the company’s Latin America group. Dr. Vivaldi began his career as a physician, spending 18 years in private practice specializing in diabetes and endocrinology, and was the first physician in Brazil to treat Gaucher disease using enzyme replacement therapy. He received his medical degree from Rio de Janeiro University (UNIRIO) – School of Medicine, and completed his residency in endocrinology at Rio de Janeiro State University (UERJ) as well as his fellowship at Mount Sinai Hospital Center in New York in the department of genetics, where he focused on Gaucher disease. He received his M.B.A. degree from COPPEAD, Rio de Janeiro Federal University.
Stephen Webster has over 25 years of experience serving as a financial professional in the life sciences industry. Prior to Spark, Stephen served as senior vice president, finance and chief financial officer at Optimer Pharmaceuticals, Inc., a commercial-stage company in the antibiotic field. Before that, he served in the same capacity at Adolor Corporation, a commercial-stage company in the gastro-intestinal space. Stephen played an integral role in the sale of both Optimer and Adolor to Cubist Pharmaceuticals, Inc. Prior to taking his first operating role, for 15 years Stephen was an investment banker to life sciences companies, raising over $3 billion in financings and advising clients on over $3 billion in aggregate mergers and acquisitions value. Stephen received his A.B. in economics, cum laude, from Dartmouth College and his M.B.A. in finance from the Wharton School at the University of Pennsylvania.
Dan Faga is the chief business officer of Spark Therapeutics. Dan brings 15 years of biopharma industry experience to the company. Prior to joining Spark, Dan was a Managing Director at Centerview Partners, where he had served since 2009 as a founding member of Centerview's healthcare advisory practice. Primarily focused in the life sciences sector, Dan has deep expertise in corporate strategy, M&A and partnering, including advising on more than $50 billion in biopharma transactions. Prior to Centerview, Dan worked at Merrill Lynch in its healthcare investment banking group and as a management consultant in the Life Sciences Practice at PRTM. Dan earned a B.S. in Engineering from Cornell University and a M.B.A. in Health Care Management from the Wharton School of the University of Pennsylvania.
Joseph La Barge is a seasoned executive and legal advisor with over 15 years of experience counseling life science companies and healthcare institutions. Joe was the vice president, general counsel and chief compliance officer at Tengion, Inc., a clinical-stage, regenerative medicine company, where he oversaw legal affairs, compliance and quality assurance. While at Tengion, Joe helped lead the company’s initial public offering in 2010. Joe also played an integral role in developing Tengion’s intellectual property strategies for their novel, first-in-class, cell-based therapies. Joe was previously of counsel at Ballard Spahr LLP in Philadelphia where he advised biotechnology companies in private and public financings, mergers and acquisitions and collaboration and licensing transactions. He also served as the deputy general counsel to the Kennedy Health System in New Jersey. He received his J.D. from Temple University and a B.A. from Bucknell University.
Dr. Fraser Wright has been engaged for more than 20 years in the development of viral vector-based new biologic products in both industry and academic settings. Fraser previously served as the director of the Clinical Vector Core Laboratory at The Children’s Hospital of Philadelphia, a world-class manufacturing facility that has designed, manufactured, characterized and certified recombinant adeno-associated viruses (AAV) and lentiviruses (rLenti) for human gene therapy trials. He also was a professor of pathology and laboratory medicine at the University of Pennsylvania. Fraser has led efforts in basic research, process and analytical methods development and GMP manufacturing and quality systems supporting translational research. He has overseen investigational product chemistry, manufacturing and controls for gene therapy vectors administered to more than 150 human subjects in 12 clinical studies. With basic training in biochemistry, immunology and virology, his primary research interests focus on the development and clinical translation of gene therapy vectors with the goal of achieving effective new treatments for serious human diseases. He previously served as director of development and clinical manufacturing at Avigen, Inc. He received his B.S. and Ph.D. in biochemistry from the University of Toronto.
Carol Greve-Philips has more than 20 years of biotech industry experience focused on strategic planning, direct sales and business development. Prior to joining Spark, Carol served as Pronota’s chief business officer and, before that, helped build Genzyme Corporation’s Personalized Genetic Health business. In her role as vice president of corporate development at Genzyme she led collaborations with industry partners and created a new strategic focus for the PGH business unit focusing on six neurologic disease targets. Carol has also held roles in direct sales, sales management and marketing for Amersham Corporation, Chemsyn Science Laboratories, Watson Laboratories and Toxikon Corporation. She is a member of the BioPharm America Planning Committee and the Licensing Executive Society. Carol has a B.Sc. in zoology and chemistry from the University of Massachusetts at Amherst.
Dr. Daniel Takefman has more than 15 years of regulatory experience across a range of categories including gene therapy, chemistry, manufacturing and control (CMC) and biologics. Previously, Dan was chief, gene therapy branch, of the division of cellular and gene therapies within the center for biologics evaluation and research at the U.S. Food and Drug Administration (FDA). He has authored numerous regulatory documents, including: Draft Guidance for Industry: Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products; Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines and Related Recombinant Viral and Microbial Products; and Guidance for Industry: Potency Measures for Cell and Gene Therapy Products. Dan began his career at FDA in 1999 as a Postdoctoral Fellow and became a Staff Fellow shortly thereafter. He served as a microbiologist at FDA from 2001 to 2006. He had oversight of the CMC review process of approximately 425 active gene therapy Investigational New Drug (IND) applications, approximately 150 active therapeutic vaccine INDs and three Biologics License Applications. Dr. Takefman holds a Ph.D. in microbiology from Rush University and a B.S. in microbiology from the University of Iowa.
Diane Blumenthal has 30 years of experience in the use of recombinant DNA technology for the expression and manufacture of biotechnology products. Throughout her career, Diane has played a key role in the development and commercialization of five pharmaceutical products, including Erbitux® and Cyramaz®. She most recently served as vice president of technical services and manufacturing sciences for Eli Lilly and Company, following the acquisition of ImClone Systems. While in this role, Diane was responsible for leading a multi-disciplinary team of scientists and engineers tasked with providing technical support to manufacturing, development, quality control, quality assurance and regulatory teams. Diane has extensive experience in the transfer and scale-up of processes from the laboratory bench to commercial scale manufacturing with expertise in both microbial and mammalian cell culture expression systems and the corresponding downstream purification processes. Prior to her time at Lilly, Diane served as a scientific and manufacturing consultant to multiple start-up biotechnology companies and held scientific leadership positions at Zymquest, Inc., Scios, Inc. and the Eastman Kodak Company. She received her M.S.E. in chemical engineering from Lehigh University and a B.S.E. in bioengineering at the University of Pennsylvania.
Romu Corbau has more than 15 years of experience leading drug discovery efforts across industry and academic settings. At Pfizer, he led various discovery efforts from preclinical research to proof-of-concept, and was instrumental in efforts to initiate the company’s first gene therapy program for HCV. He subsequently served as director of biology at the Center for Innovation and Stimulation of Drug Discovery at the Catholic University of Leuven in Belgium, evaluating investigational therapeutics discovered in academic settings across Europe for further development. Most recently, he was associate director at The Children’s Hospital of Philadelphia Center for Cellular and Molecular Therapeutics.
Lisa Dalton has more than 15 years of strategic and operational human resources experience across the biopharmaceutical and financial services industries. Lisa was previously vice president, human resources at Shire, where she most recently led HR merger and acquisition activity and implementation. While at Shire, Lisa played many other critical roles within HR, including building and leading the Compensation function, implementing an HR systems platform, and establishing the HR Operations function. Lisa has also held roles in compensation and HR business partnering with Franklin Templeton and Applied Biosystems. Lisa holds an M.B.A. from Rutgers University School of Business and a B.A. in psychology from Penn State University.
Dr. Paul Gil has more than three decades of experience in regulatory affairs and quality across a range of categories, including biologics, plasma-derived therapeutics, recombinants and oncology products. Previously, Paul was vice president, global regulatory affairs CMC at ViroPharma (acquired by Shire), a biopharmaceutical company developing treatments for rare diseases. While at ViroPharma, he served as the U.S. agent, overseeing all global regulatory filings, licenses and regulatory compliance activities, and played a major role in the company’s orphan disease product Cinryze™. Prior to ViroPharma, Paul spent 13 years at Bayer in positions of increasing responsibility and served as associate director global regulatory affairs CMC. Paul has directed hundreds of successful biologics submissions globally, including BLAs and CBE-30s to the FDA. He holds a doctorate degree in management and organizational leadership from the University of Phoenix, a MBA from Mt. St. Mary’s University, a master’s degree in medical biology from Long Island University and a bachelor’s degree in biology from the Franklin and Marshall College.
Jennifer Wellman has more than 15 years of R&D, clinical and regulatory experience specifically related to adeno-associated virus (AAV)-mediated gene therapy. Prior to joining Spark, she spent eight years at the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia where she directed regulatory interactions for the Center’s gene therapy studies. She also has performed clinical study site management for these international trials. Jen is well versed not only in federal, local and institutional regulatory issues and clinical trial methodology, but also has hands-on knowledge of product manufacturing and the research studies used to support clinical testing from her previous role as associate scientist at Avigen, Inc. She received her B.S. in microbiology and immunology from Queen’s University and her M.S. from the University of New Haven.
Dr. Guang Qu has served as Technical Director for the Clinical Vector Core Laboratory at The Children’s Hospital of Philadelphia, a world-class facility for clinical vector manufacturing. In his role there, he has led process development, striving to optimize product purity and yield. Guang has more than 15 years working experience in the gene therapy field, including six years in the biotechnology industry directing process development at Avigen, Inc. He developed several breakthrough techniques in adeno-associated virus (AAV) vector production and purification, including scalable purification methods for eight serotypes of AAV vectors and a patented column-based purification process capable of removing most of the empty capsids that are naturally formed during the AAV production process. In addition, he participated in the designing of two cGMP facilities for manufacturing gene therapy vectors and is intimately familiar with cGMP regulation and operation. Guang is a member of the American Society for Gene Therapy and served on the AAV National Reference Standard Working Group from 2003 to 2005. He received his B.S. from Liaoning General University and his M.S. from Academia Sinica, both in China, and his Ph.D. from The Ohio State University.
Linda Hearne has more than 20 years of finance and accounting experience that spans multinational corporations and startup biotechnology companies. Her areas of expertise include mergers and acquisitions, valuation modeling, fundraising and cash management. Linda was previously vice president, finance and principal financial officer at Tengion, Inc. and began her career as a public accountant at Ernst & Young. She holds a B.S. in accountancy from Miami University of Ohio and an M.B.A. from Duke University Fuqua School of Business.
Dr. Linda Couto has 20 years of experience with AAV-mediated delivery of therapeutic genes, including roles as senior research director at Benitec LLC and senior director of preclinical research at Avigen, Inc., where she led the company’s hemophilia B gene therapy project. Most recently, she was associate director of the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia where she worked to advance the hemophilia B program now within Spark, designing more potent coagulation FVIII and FIX AAV-based expression vectors and evaluating novel AAV vectors for gene transfer efficiency. In addition, she developed a treatment for hepatitis C virus infection using AAV-mediated delivery of a combination of therapeutic microRNAs. Linda holds a B.S. in biology from Northeastern University, a Ph.D. in molecular toxicology from Massachusetts Institute of Technology and was a postdoctoral fellow in the Department of Pathology at Stanford University School of Medicine.
Dr. Bernd Hauck previously served as the assistant director of production in the Clinical Vector Core Laboratory at The Children’s Hospital of Philadelphia. During his tenure, he established the standard operating procedures for the AAV vector manufacturing process and contributed significantly to the rapid development and improvement of AAV production in the facility. Under Bernd’s careful day-to-day supervision, the clinical vector core has successfully produced 38 GMP comparable vector lots and nine full GMP lots, four of which have been administered to patients, and have a demonstrable safety record. A trained molecular biologist and geneticist, he has studied regulation of conserved genes that are involved in eye development. Bernd received his Ph.D. from the Universität Hohenheim in Stuttgart, Germany.