Expert Details
Virology, Anti-infectives, Gene Therapy, Bioterrorism Research
ID: 723081
Hawaii, USA
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 |
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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 |
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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 |
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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 |
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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. |
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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 |
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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 |
Government Experience
Years | Agency | Role | Description |
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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 |
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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 |
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Member of the American Society for Gene Therapy Member of the National Hemophilia Foundation |
Professional Appointments |
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Member of External Scientific Advisory Board, Center for Transgene Technology and Gene Therapy, University of Leuven, May 1997 |
Awards / Recognition |
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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 |
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He has over 40 publications in peer reviewed journals and 17 issued US patents |
Additional Experience
Training / Seminars |
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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 |
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He has selected CROs for the conduct of GLP toxicology studies that were included in IND submissions to the FDA |
Other Relevant Experience |
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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
adenovirus, antiviral agent, common cold virus, biological virus production, gene therapy, hemophilia, bioterrorism, gene therapy vector purification, eukaryotic gene, Flavivirus, animal disease model, genetic disease, viral culture, viral assay, delivery system, biotechnology scale-up, retrovirology, pharmaceutical research, molecular medicine, genome, DNA technology, applied molecular biology, human genetics, drug development, genetic sequencing, drug delivery, gene expression regulation, molecular genetics, cell membrane transport, DNA sequencing, gene, DNA, drug, virology, genetic vector, picornavirus, monoclonal antibody, molecular biology, investigational drug, immunology, genetics, genetic manipulation, genetic engineering, gene expression, common cold, cell surface interaction, cell culture, biotechnology