Katherine High, M.D. | President And Head, Research And Development
Spark Therapeutics

Katherine High, M.D., President And Head, Research And Development, Spark Therapeutics

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 AAV vectors in a variety of target tissues. Kathy’s work has evolved to encompass clinical translation of potential 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 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.

Appearances:



Conference Day 1: Thursday 16th May 2019 @ 09:00

Fireside chat: A new era in medicine- introducing gene therapy

Conference Day 1: Thursday 16th May 2019 @ 09:30

(ex vivo) Gene edited therapeutics and (in vivo) gene therapy: how can gene technology be used now and, in the future, to treat and cure diseases in the clinic

  • How does gene editing apply to the clinic?
  • How is it currently being applied?
  • How can we improve delivery of AAVs into the cell?
  • Improving the volume of viral particles: what stages do we need to cover to get to a commercial scale?
  • Moving into larger diseases from rare diseases, what challenges need to be overcome?
  • Risk/benefit acceptance of the general population and the medical system.
  • What is the real risk of CRISPR causing cancer as published by Novartis and KI?
  • How do we want to use it in the future and what challenges need to be overcome to do so? (immunogenicity, off target effects and efficacy, being vigilant with safety profiles, potency and manufacturing, moving towards direct, in vivo [bluebird bio live delivery usingnanoparticles], viral and non-viral)
  • How do you move towards that goal in a way that engages stakeholders? Applications
last published: 25/Mar/19 17:15 GMT

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