Expert Details

Virology, Anti-infectives, Gene Therapy, Bioterrorism Research

ID: 723081 Hawaii, USA

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He has extensive experience in the development and use of viral vectors for gene transfer and in particular has worked with both adenovirus and AAV vector delivery systems. His work with adenovirus vectors included some of the earliest research focused on the development of cell targeted vectors through genetic manipulation of the adenovirus fiber protein. This work resulted in two issued US patents and several publications in peer-reviewed journals. The concept of retargeting adenoviruses using the fiber modification approach is in widespread use today and is of particular importance in oncology applications where fiber modified adenovirus vectors are being evaluated in clinical trials.



He headed a research team at Bayer that succeeded in identifying Inter-Cellular Adhesion Molecule-1 (ICAM-1) as the receptor for human rhinoviruses, the primary cause of the common cold. This work led to important new insights into the mechanism of rhinovirus infection and the development of a soluble receptor decoy that was one of the first antiviral entry inhibitor drugs to be tested in humans. His recent work in antiviral research has focused on efforts to identify inhibitors of dengue virus entry based on the X-ray crystal structure of the viral envelope protein.

The scale up of manufacturing technology for viral vectors used in gene therapy and vaccine applications continues to present significant technical and regulatory challenges, due to the complexity of these biological products.He has worked with both adenovirus and AAV vectors and oversaw the work of a development team that made several advances in the scale up of AAV vectors for human gene transfer studies. These included the development of a partially automated cell culture system allowing for consistent scalable production of AAV using a plasmid DNA based transfection system and the use of column chromatography based purification methods.

He has over a decade of experience in the development of gene transfer approaches to treat disease, and has published extensively in this field. He joined Genetic Therapy Inc in 1992 as Director of Molecular and Cell Biology and headed research programs in adenovirus vector development, non-viral gene transfer, hemophilia and cardiovascular disease. Following the acquisition of GTI by Novartis in 1995, He was promoted to Vice-President of Research, assuming responsibility for the research programs in respiratory disease and oncology, in addition to the hemophilia and cardiovascular programs. He also established and managed several collaborations within the Novartis research organization as well as with external academic research groups. From 1999 to 2003 he headed R & D at Avigen Inc, a biotechnology company that was focused exclusively on the use of adeno-associated virus (AAV) as a gene delivery vector for the treatment of inherited and acquired diseases. Under his leadership Avigen successfully completed two Phase I clinical trails for the treatment of hemophilia B using AAV vectors and initiated a clinical trial for Parkinson’s disease. He recruited and assembled a process development team that was able to scale up the production and purification process for AAV vectors as well as developing new and improved analytical methods for QC testing of vector product used in human clinical studies



He has over 10 years of experience in the development of gene therapy approaches to the treatment of hemophilia A and B. At Genetic Therapy Inc. his research included some of the first studies to show the potential for gene transfer approaches to correct hemophilia in canine models of this genetic disease. Subsequently, at Avigen Inc., he oversaw a research team that collaborated with investigators at Children’s Hospital of Philadelphia and Stanford University in the first human clinical studies to evaluate the use of AAV vectors for treatment of hemophilia B

He was a member of the External Scientific Advisory Board of the Center for Transgene Technology and Gene Therapy in Leuven, Belgium which conducted a two day site visit and scientific review of the research programsProvided scientific advice and project evaluation to a Contract Research Organization in the area of gene therapy, assisting the CRO in preparing recommendations for a product development plan to a client organization. This assignment included participation in client meetings and preparation of reports.Currently advising a client in the area of biodefense research, providing strategic and technical input to the formulation of the business plan and R & D direction

Education

Year Degree Subject Institution
Year: 1981 Degree: PhD Subject: Biochemistry Institution: University of London
Year: 1977 Degree: BS Subject: Molecular Biology Institution: University of Edinburgh

Work History

Years Employer Title Department
Years: 2003 to 2005 Employer: Hawaii Biotech Inc Title: Vice President Department: Discovery Research
Responsibilities:
He was responsible for leading research programs in small molecule drug discovery, focusing on infectious disease and bioterrorism targets, and was PI on several NIH grants and Dept. of Defense contracts
Years Employer Title Department
Years: 1999 to 2003 Employer: Avigen Inc Title: Vice President, Research and Development Department:
Responsibilities:
He was responsible for preclinical research and development, leading a group of approximately 50 scientists dedicated to the development of adeno-associated virus (AAV) based human therapeutic products for inherited and aquired disease including hemophilia and Parkinson's disease
Years Employer Title Department
Years: 1997 to 1999 Employer: Novartis Title: Vice President, Research Department: Genetic Therapy Inc
Responsibilities:
He was responsible for scientific leadership and management of 80 researchers developing gene delivery based therapies, and was a member of the Novartis Research Management Board.
Years Employer Title Department
Years: 1992 to 1997 Employer: Genetic Therapy Inc Title: Laboratory Director Department:
Responsibilities:
He established and led a research team comprising up to 40 scientists conducting research in gene therapy for inherited and aquired diseases including hemophilia, cardiovascular disease and organ transplantation rejection. He led vector development research in targeted gene delivery that resulted in issued US patents for this technology
Years Employer Title Department
Years: 1985 to 1992 Employer: Bayer Title: Principal Staff Scientist Department: Molecular Therapeutics Inc
Responsibilities:
He planned, initiated and built an anti-viral research discovery program which discovered the major human rhinovirus receptor (ICAM-1). He headed group which designed, produced and tested soluble receptor molecules based on ICAM-1, developed mammalian production cell lines and advanced a candidate molecule through preclinical testing in primates

Career Accomplishments

Associations / Societies
Member of the American Society for Gene Therapy
Member of the National Hemophilia Foundation
Awards / Recognition
He was a recipient of a Science and Engineering Research Council postdoctoral fellowship award, and received the Miles Corporate Science Award in 1990 for research leading to the discovery of the human rhinovirus receptor
Publications and Patents Summary
He has over 40 publications in peer reviewed journals and 17 issued US patents

Fields of Expertise

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