Job title: Vice President, Discovery Biology, Rare Disease Research Unit
John Murphy is currently the head of Discovery Biology for Rare Disease Research at Pfizer. In this role he is responsible for all Rare Disease early discovery efforts, which includes projects on rare genetic diseases in the areas of haematology, neuromuscular, metabolic and cardiology. Dr. Murphy is a molecular biologist by training, having done a PhD in Biochemistry and Molecular Biophysics at Columbia University. Following his PhD work, Dr. Murphy was a Damon Runyon Fellow at UCSF where he studied oncogenic transformation. His first position was in gene therapy at Chiron Corporation where he worked on retroviral, AAV and non-viral gene delivery technologies. Dr. Murphy subsequently moved to Bayer where he started working on Factor VIII and ultimately was responsible for the hematology portfolio from concept to candidate selection.
Live Presenter Q&A 4:30 pm
day: Day One
Gene Therapy for Hemophilia: Learnings from Phase 1/2 studies 3:30 pm
• AAV mediated gene delivery of FIX and FVIII to treat HemB and HemB results in sustained (up to 4 year) therapeutic correction of bleeding with a good safety profile • Assays to monitor factor activity following treatment give variable results and the choice of relevant assay is a topic of interest • While all…Read more
day: Day One