While pharmaceutical companies sift through vast libraries of
chemical compounds to come up with the magic bullets that may one day help subdue
diseases, biotechnology companies pursue alternative strategies known collectively as gene
therapy. Gene therapy attacks diseases by replacing defective or missing genes--long
strands of DNA which code for production of a particular protein--with normal copies of
the genes to restore normal cell function.
As gene therapy has moved from laboratory into clinical trials, the technology has been
refined to the basic areas of gene identification, gene expression and gene delivery. With
volumes of DNA sequence information being revealed through the Human Genome Project--in
which Berkeley Lab plays a major role--new genes are identified on a daily basis,
providing more fuel for the promise of gene therapy. Challenges remain in the areas of
developing appropriate vehicles for the expression and delivery of these genes into
The state of this technology and its prospects for the future was the subject of a
conference hosted by Berkeley Lab on May 13.
One of Life Sciences Division's largest industrial partners, Gencell--the gene therapy
subsidiary of pharmaceutical giant Rhône-Poulenc Rorer (RPR)--gathered more than 30
international business journalists on the Hill--from Le Monde to The Times of London--to
hear the gene therapy gospel from biotech industry experts.
The event was kicked off by the self-professed "biogoddess" Cynthia
Robbins-Roth. Founder of BioVenture Publishing Inc. and BioVenture Consultants,
Robbins-Roth has been a biotech industry mainstay since 1981. The tall order of the day
for her was to explain to business reporters the basics of biology, from DNA to gene
therapy, all in an hour and with simultaneous translation into French.
Constance McKee, president of Xavos Corporation, a start-up company developing drug
delivery technology for improved treatment of viral diseases and conditions which affect
the peripheral nervous system, outlined the industry players and later commented on the
significance of gene therapy.
"It is not just the technologies in gene therapy that are causing a
revolution," she said. "Gene therapy will affect the entire chain of events in
the health care delivery system as we know it. One of the key challenges to the industry
will be to bring gene therapy technologies into conformity with the current health care
delivery system: training doctors and nurses in medical and nursing schools, doctors
offices, specialized treatment centers, and hospitals, to administer new therapies."
Over lunch, Elizabeth Silverman, a senior research analyst focusing on genomics and new
technologies related to drug discovery, provided a prospectus on the latest market
Berkeley Lab's own Eddy Rubin, head of the Life Sciences Division's Department of
Genome Sciences, provided the view from the interface of academia and industry. Rubin
described his lab's studies entailing the use of genetically engineered mice to decipher
and add biological value to the data flowing from DOE's human genome sequencing program.
Linkages, both with academic institutions and start-up companies, are critical to the
rapid delivery of gene therapies to the market, said RPR Senior Vice President of Research
Thierry Soursac. In 1994 Rhône-Poulenc Rorer launched RPR Gencell as an integrated
division dedicated to the discovery, development, production and marketing of gene therapy
products. David Nance, president & CEO of Texas-based Introgen Therapeutics, Inc., a
leader in the field of anti-cancer gene therapy products, echoed the importance of
establishing linkages from the perspective of an entrepreneur. Through its own discovery
and partnerships with various biotechnology companies and research institutes worldwide,
including Berkeley Lab, Gencell has sought to accelerate the development of novel
gene-based therapies in the areas of oncology, cardiovascular diseases and disorders of
the central nervous system.
Soursac said that Gencell's long-term investment in the future of gene therapy is
paying off in the near term, with two projects currently in clinical trials and five in
preclinical, three of which are expected to enter the clinical phase by 1999.
The industry executives as well as the journalists concluded their day at the Lab with
tours of the user facilities, including NERSC and the ALS.