Here is the full PR.
Friday March 24, 7:30 am Eastern Time
Company Press Release
SOURCE: Cell Genesys, Inc.
Cell Genesys Reports Successful Preclinical Studies of
Lentiviral Gene Therapy For Hemophilia A
FOSTER CITY, Calif., March 24 /PRNewswire/ -- Cell Genesys (Nasdaq: CEGE - news)
today announced that Inder Verma, Ph.D., and colleagues at The Salk Institute have reported the successful use of lentiviral
vectors in preclinical studies of hemophilia A, an inherited bleeding disorder. In addition, Flavia Borellini, Ph.D. and colleagues
at Cell Genesys reported the successful development of the scaleable manufacturing technology for lentiviral vectors required
for clinical studies in hemophilia and other serious diseases. These presentations were made in Washington D.C. at the Third
Workshop on Gene Therapies for Hemophilia sponsored by the National Hemophilia Foundation.
Dr. Verma reported that Cell Genesys' proprietary lentiviral gene delivery system efficiently delivered the full-length factor VIII
gene into target cells in a biologically active form. In addition, biologically active factor VIII was detected following
administration to small laboratory animals. Importantly, since other gene delivery systems currently being evaluated by Cell
Genesys in hemophilia A gene therapy (such as the AAV or adeno-associated viral system) are unable to accommodate the
full-length factor VIII gene, lentiviral vectors may be particularly advantageous in gene therapy for this more common form of
hemophilia. Successful delivery of the factor VIII gene is expected to result in production of the factor VIII protein,
representing a potential new approach to the treatment for hemophilia A patients who are deficient in factor VIII, a blood
clotting factor.
Dr. Borellini reported that Cell Genesys has developed technology for scaleable manufacturing of the lentiviral vector system,
which is critical for advancing clinical trials and for potential product commercialization. Using the scaleable manufacturing
process, the company has produced lentiviral vectors that maintain the quality, safety and gene delivery efficiency that had been
previously demonstrated using earlier production systems. This accomplishment represents an important step towards the
initiation of human clinical trials.
``These data further enhance our competitive advantages in the hemophilia A area, as we have overcome the hurdle of
delivering the complete factor VIII gene and have demonstrated the ability to manufacture the lentiviral gene delivery system at
increasing scale,' stated Joseph J. Vallner, Ph.D., executive vice president and chief operating officer at Cell Genesys. ``As a
result of our progress in both hemophilia A and hemophilia B gene therapy preclinical studies, we expect to initiate human
clinical trials in 2001.'
In Cell Genesys' preclinical hemophilia program, a 90 percent reduction in bleeding episodes in a canine model of hemophilia B
was achieved with a single administration of factor IX gene therapy. Additionally, 20 months after the single injection, the
treated animals continued to produce the factor IX protein which was deficient in these animals prior to treatment. These
studies utilized Cell Genesys' proprietary adeno-associated viral (AAV) vector system, which like the lentiviral vector, inserts
the therapeutic gene into the DNA of the patient's cells, thereby allowing a potentially permanent genetic correction. A single
administration of gene therapy could therefore potentially eliminate the need for repetitive treatment regimens.
Successful gene therapy depends to a great extent on the vector systems or gene delivery vehicles used to transfer genes into
cells. Cell Genesys has four different viral vector systems and is using this technology ``toolbox' to capture multiple product
opportunities. The company's proprietary vector technologies include AAV, lentiviral, adenoviral and retroviral vectors
engineered to provide safe, efficient, long-term gene expression. Lentiviral vectors have demonstrated efficient and long-lasting
gene transfer into a variety of human cells including both dividing and non-dividing cells such as nerve, liver, muscle and bone
marrow stem cells potentially providing the opportunity to treat multiple genetic deficiency diseases such as hemophilia, cancer
and Parkinson's disease.
Cell Genesys currently has one of the largest patent portfolios in the gene therapy field including more than 220 issued or
granted patents and over 335 pending patent applications. The portfolio includes issued or granted patents for multiple gene
delivery systems, specific therapeutic genes and gene therapy applications and multiple genetically modified cell types used in
gene therapy independent of the gene delivery system or therapeutic gene. For example, the portfolio currently contains over 90
filings alone pertaining to the two types of gene delivery systems-lentiviral and AAV-with potential applicability to the treatment
of hemophilia and other genetic deficiency diseases.
Cell Genesys is focused on the development and commercialization of gene therapies to treat cancer and other major,
life-threatening diseases. The company is conducting two multicenter Phase II human clinical trials for its GVAX© cancer
vaccine in prostate cancer and plans to initiate a multicenter Phase I/II trial of GVAX© vaccine in lung cancer. Preclinical
stage programs include gene therapy for hemophilia, cancer, cardiovascular disorders and Parkinson's disease. Cell Genesys'
assets outside gene therapy include its approximately 12 percent ownership of Abgenix, Inc. (Nasdaq: ABGX - news) and the
company's licensing program in gene activation technology. For additional information, please visit the company's web site at
www.cellgenesys.com. |
