GeneMedicine Inc. announced Friday that it is licensing newtechnologies for delivering gene therapy from the University ofCalifornia, San Francisco (UCSF). The technologies include "lipidand polymer-based approaches to deliver genes and othermacromolecules across membranes for their targeting to cellsand organs," the Houston company said.
In return for an exclusive, worldwide license to thetechnologies, GeneMedicine will provide UCSF with certainlicensing fees, milestone payments and royalties.
GeneMedicine said the UCSF technologies "represent potentiallysignificant advancements in vector delivery that may enable awide variety of gene therapy products to be administereddirectly to patients by conventional routes, without the use ofviral carriers."
Furthermore, GeneMedicine explained, these technologies"enable DNA expression vectors to be formulated, targeted tospecific cells within the body and transported through thecellular membrane to the nucleus, where gene expression cantake place. Once within the nucleus, DNA expression vectorsproduce therapeutic gene products."
GeneMedicine's current vector delivery systems introducesynthetic DNA expression vectors into specific cells in the body.These expression vectors are comprised of circular pieces ofDNA that incorporate a therapeutic gene and flankingsequences that control gene expression, including a cell-specificpromoter and a sequence that increases RNA transcriptstability. GeneMedicine was founded by scientists from theBaylor College of Medicine, who transferred their inventions tothe company.
GeneMedicine's vectors are being developed to treat specificclinical indications. Proteins from genes are to be administeredto the muscle for a local or systemic effect. The company's twolead compounds are insulin-like growth factor-I (IGF-I) to treatmuscle wasting, osteoporosis, peripheral neuropathies andmuscle injury, and Factor IX to treat hemophilia B.GeneMedicine licensed rights to the Factor IX gene sequencefrom British Technology Group USA Inc. in November.
The company also has a non-exclusive licensing agreementwith the University of Texas Southwestern in Dallas for genetherapy rights to the LDL receptor gene sequence. In addition,it has a Cooperative Research and Development Agreement(CRADA) with the U.S. Department of Agriculture to improvelivestock and meat products through development of a strain ofswine with greater muscle mass and lower fat content.
The UCSF vector systems were developed by a research groupled by Frank Szoka, of UCSF. Szoka, a GeneMedicine consultantand member of the company's clinical scientific advisory board,is now assuming a position as a founding scientist ofGeneMedicine. Szoka was also a founding scientist of LiposomeTechnology Inc.
-- Brenda Sandburg News Editor
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