Expert Details

Expert in Virology, Anti-infectives, Gene Therapy, Bioterrorism Research

Expert 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

Expert may consult nationally and internationally, and is also local to the following cities: Honolulu, Hawaii

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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 Responsibilities
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: 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: 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: 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: 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

Government Experience

Years Agency Role Description
Years: 2004 to 2005 Agency: Dept of Defense Role: Principal Investigator Description: He served as Principal Investigator for a contract research project investigating small molecule inhibitors of anthrax lethal toxin protease
Years: 2004 to 2005 Agency: NIH Role: Principal Investigator Description: He served as Principal Investigator for a Phase II SBIR grant from NIAID investiagting anthrax lethal toxin ihhibitors

International Experience

Years Country / Region Summary
Years: 1977 to 1981 Country / Region: United Kingdom Summary: Graduate research leading to PhD degree at the University of London
Years: 1997 to 1997 Country / Region: Belgium Summary: Member of External Scientific Advisory Board, CTTGT, Leuven, Belgium
Years: 1985 to 1992 Country / Region: Germany Summary: He was employed by Molecular Therapeutics inc., a wholly owned subsidiary of Bayer AG. Position involved reguar travel to Germany to particpate in scientific planning and strategy meetings and management of R&D collaborations
Years: 1995 to 1999 Country / Region: Switzerland Summary: He was Vice President of Research for Genetic Therapy Inc. a subsidiary of Novartis. In this capacity Expert was a member of the Novartis Research Management Board and participated in oversight and review of pharmaceutical research programs, including an annual review of the entire pharma research portfolio

Career Accomplishments

Associations / Societies
Member of the American Society for Gene Therapy
Member of the National Hemophilia Foundation
Professional Appointments
Member of External Scientific Advisory Board, Center for Transgene Technology and Gene Therapy, University of Leuven, May 1997
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

Additional Experience

Training / Seminars
He was a lecturer in the Gene Therapy Course offfered in the Johns Hopkins University Masters in Biotechnology program from 1994 to 1996. He has given numerous scientific presentations at academic institutions and national and international scientific meetings. He organized and chaired two Corporate Symposia that were part of the scientific program of the American Society of Gene Therapy in 2001 and 2002
Vendor Selection
He has selected CROs for the conduct of GLP toxicology studies that were included in IND submissions to the FDA
Other Relevant Experience
He has written and submitted successful grant applications to the NIH including recent SBIR grants in the areas of bioterrorism preparedness and emerging infectious disease

Fields of Expertise

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