Silicon Investor (SI) -- The First Internet Community

STOCKTALK

We've detected that you're using an ad content blocking browser plug-in or feature. Ads provide a critical source of revenue to the continued operation of Silicon Investor. We ask that you disable ad blocking while on Silicon Investor in the best interests of our community. If you are not using an ad blocker but are still receiving this message, make sure your browser's tracking protection is set to the 'standard' level.

Biotech / Medical : Cell Genesys (CEGE) -- Ignore unavailable to you. Want to Upgrade?

To: Madharry who wrote (1133)	6/19/2001 9:30:24 AM
From: tuck	Read Replies (2) \| Respond to of 1298

This might help, but there is already competition here (from GNT, for example): >>FOSTER CITY, Calif., June 19 /PRNewswire/ -- Cell Genesys, Inc. (Nasdaq: CEGE - news) today announced that in preclinical studies of hemophilia gene therapy, the delivery of the factor IX gene, a blood clotting gene, into the liver demonstrated clear treatment advantages compared to intramuscular injection. Animals that received the factor IX gene in the liver using an AAV (adeno-associated viral) gene delivery system exhibited stable production of the factor IX protein throughout the duration of the study whereas those that received intramuscular injections had no detectable levels of the protein primarily due to the formation of anti-factor IX antibodies. Based on these and other findings, the company has determined that it will not pursue intramuscular delivery in human hemophilia gene therapy studies. These data were published in a June issue of the journal, Blood, the official journal of the American Society of Hematology, by Dr. James G. McArthur and colleagues at Cell Genesys. ``These newly published data further support our long-standing strategy to emphasize liver-directed gene delivery in our hemophilia gene therapy program,'' stated Joseph J. Vallner, PhD., executive vice president and chief operating officer at Cell Genesys, Inc. ``This study and others will provide the preclinical data needed to advance this program to human clinical trials which are targeted to begin within the next year.'' In the studies described in Blood, different methods of administration of AAV-based gene therapy were evaluated in genetically deficient mice which serve as a model of human hemophilia. The mice received a single injection of the human factor IX gene either by delivery into the liver or by intramuscular injection and were then monitored for the presence of the factor IX protein in their blood. Gene delivery to the liver, where the factor IX clotting factor is naturally produced, resulted in easily measurable levels of factor IX for the duration of the study (270 days) and no detectable antibodies or other inhibitors of factor IX. In contrast, intramuscular injection produced a strong antibody response to factor IX and as a result, no measurable levels of factor IX in the blood of the test animals. Based on these findings liver directed gene delivery was concluded to be the preferred route of administration for human gene therapy of hemophilia. Cell Genesys has previously reported successful preclinical studies of hemophilia gene therapy in canine models, where normal levels of factor IX were restored following a single injection into the liver. In a canine model of hemophilia B, a significant reduction in bleeding episodes was achieved with a single administration of factor IX gene therapy. Additionally, production of the factor IX protein has now been observed for more than three years following a single injection. These studies also utilized Cell Genesys' AAV gene delivery system which permanently inserts the therapeutic gene into the DNA of the cells, thereby allowing potential long term therapeutic benefit when applied to the treatment of human diseases. For example, a single administration of gene therapy could potentially reduce the need for repetitive treatment regimens in hemophilia patients and decrease the serious and disabling complications arising from spontaneous bleeding episodes. Cell Genesys is pursuing gene therapies for hemophilia A and hemophilia B -- genetic deficiencies in factor VIII and factor IX clotting factor genes, respectively. Cell Genesys has four different gene delivery 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 and efficient therapeutic gene expression. In Cell Genesys' preclinical program for hemophilia B, an AAV vector system is employed since the factor IX gene is small enough to fit in this vector. For hemophilia A, the company has the option of employing an AAV vector system if a truncated form of the factor VIII gene can be successfully applied or a lentiviral vector system if the full length gene is required for optimal production of the deficient clotting factor protein. Successful delivery of the clotting factor genes is expected to result in production of the clotting factor proteins, representing a potential new approach to the treatment for hemophilia patients.<< snip Cheers, Tuck