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GENETIC THERAPY, INC. PLANS TO FUND USC GENE THERAPY RESEARCH WITH $30 MIL.

Executive Summary

GENETIC THERAPY, INC. PLANS TO FUND USC GENE THERAPY RESEARCH WITH $30 MIL. earmarked for the new lab of W. French Anderson, MD, who will retire from the National Heart, Lung and Blood Institute as the chief of the Molecular Hematology Branch on Sept. 19 to take a position at the University of Southern California ("The Pink Sheet" June 8 and June 1, In Brief). In return for providing Anderson's lab with $30 mil. through a multi-year research grant, Gaithersburg, Md.-based Genetic Therapy, Inc. would get exclusive licensing rights to any patent that may result from the research conducted by Anderson, one of the pioneers of genetic therapy. GTI now has five Cooperative Research and Development Agreements with the National Institutes of Health and has supplied vectors for NIH's gene therapy clinical trials. Anderson is the lead researcher for all five CRADAs. Anderson expects to maintain close and active contact with NIH during his first year at USC. The continuing commitment to NIH includes plans to stay in almost daily contact with gene therapy collaborators Michael Blaese, MD, and Steven Rosenberg, MD, on ongoing gene therapy protocols and to visit the NIH campus in Bethesda, Md. every month. Anderson's lab at NIH currently includes about 30 researchers. At USC, he plans to build a program of virtually identical size. Rather than duplicating or competing with NIH's ongoing gene therapy research, Anderson wants to engage in the next major step: making gene therapy a low-tech, inexpensive procedure, instead of a high-tech, expensive procedure. One of the objectives to effect that change is to develop an injectable, targetable vector. The goal is to create a gene therapy that is pre-engineered for clinical use. In effect, physicians would then "be able to insert the gene of choice directly into the tissue that's wanted, into the appropriate place in the chromosome that's wanted, and have it regulated in the way that's wanted," Anderson explains. To reach this point, Anderson believes it will be necessary to examine "the various approaches that various viruses now use, and take the best features from each virus and build them into a sort of artificial virus-like structure which will transfer a gene the way that we want to transfer it." He added that he is particularly interested in researching the development of a vector that would enter liver cells carrying the low-density cholesterol (LDL) receptor gene and produce higher LDL receptor levels. Such a vector would theoretically be useful in reducing the incidence of coronary disease and strokes. In the near term, the first major report of NIH's adenosine deaminase (ADA) deficiency gene therapy trial is scheduled to be published in the New England Journal of Medicine in November or December. The trial was designed to evaluate the possible therapeutic efficacy of administering autologous lymphocytes transduced with a normal human ADA gene. Research has shown the half life of mature T cells to be in the range of three to five months, giving rise to a second ADA protocol, which will evaluate the efficacy of gene-corrected peripheral blood stem cells from a CD34-enriched population together with the gene-corrected mature T cells. That protocol, which has been approved by NIH's Recombinant DNA Advisory Committee but not yet by FDA, could begin by the end of the year. There are only about 15 ADA patients worldwide. The first NIH AIDS gene therapy protocol is scheduled to be considered at the December NIH rDNA Advisory Committee meeting, Anderson said. All told, there are 28 approved gene therapy protocols in the U.S. and abroad. The first commercial AIDS gene therapy protocol, for Viagene's HIV immunotherapeutic, was recommended for approval by FDA's Vaccines & Related Biological Products Advisory Committee on June 16. Viagene expects to begin the trials by year-end ("The Pink Sheet" June 22, p. 8).
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