The scientific agenda that gene therapy pioneer W. French Andersonand his colleagues at the National Institutes of Health (NIH) set backin the early 1980s has fostered the formation of at least two dozengene therapy and genomics companies as well as the intense interestof virtually every major pharmaceutical house in the world.

Yet, even when Anderson first realized that his long-standing dreamof curing or even preventing human diseases by fixing the defects atthe genetic level was actually going to become a reality, he neverthought much about the process or the technology as the basis of abusiness. "I've always expected that gene therapy would ultimatelybe used in millions of patients. In retrospect, I should have alsorecognized that we couldn't possibly have offered gene therapytreatments except through companies," Anderson told BioWorld.

And now the venture capital community, which provided thefinancial resources to fuel the growth of just such entrepreneurialfirms, is awarding Anderson its National Biotechnology VentureAward for his pioneering research and clinical contributions to theadvancement of gene therapy. (For an update on Anderson's views ofthe future of gene therapy, see BioWorld Today, June 16, p. 1.)

Anderson, who is currently the director of the gene therapylaboratories and a professor of biochemistry and pediatrics at theUniversity of Southern California School of Medicine in LosAngeles, will be honored on Nov. 7, 1995 at the annual NationalConference on Biotechnology Ventures, which is sponsored byOxford Bioscience Partners and Ernst & Young of San Francisco.

The conference organizers also sponsor the annual award. Andersonjoins a distinguished roster of previous awardees: Leroy Hood (1994;automation); James Watson (1993; lifetime achievement); MarvinCaruthers (1992, oligonucleotide synthesis); Kary Mullis (1991;polymerase chain reaction technology); Cesar Milstein (1990;monoclonal antibodies); and Stanley Cohen and Herb Boyer (1989;gene splicing).

Anderson is primarily recognized for initiating the first clinicalapplication of gene therapy in humans. His research efforts have alsomade it possible to use retroviruses as a means to deliver geneticinformation to targeted cells. In 1986, he co-founded GeneticTherapy Inc. based on the strength of a patent _ co-invented withNIH scientists Michael Blaese and Steven Rosenberg _ covering exvivo human gene therapy. That recently issued patent is the first NIHpatent that has been licensed to a commercial entity, GeneticTherapy, of Gaithersburg, Md.

"Patenting is absolutely critical [to the success of gene therapy],"Anderson said. "Patent protection provides an incentive to investors;it's starting to bring money back into biotech and gene therapy. It'sgoing to take a lot of resources to make gene therapy work _ andthat requires money." n

-- Jennifer Van Brunt Editor, BioWorld Financial Watch

(c) 1997 American Health Consultants. All rights reserved.