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Biotech / Medical : Gene therapy -- Ignore unavailable to you. Want to Upgrade?

To: nigel bates who wrote (244)	5/5/2001 3:02:38 AM
From: nigel bates	Respond to of 319

WESTPORT, CT (Reuters Health) Apr 27 - Using a viral vector gene delivery technique, researchers have successfully restored sight in a canine model of Leber congenital amaurosis (LCA), a disease that causes near-total blindness in human infants. In a study reported in the May issue of Nature Genetics, Dr. Jean Bennett, from the University of Pennsylvania in Philadelphia, and colleagues assessed the ability of gene therapy to restore vision in dogs with a naturally occurring mutation in the retinal pigment epithelium (RPE) 65 gene. Similar mutations have been seen in infants with LCA. The researchers used a recombinant adeno-associated virus (AAV) vector to deliver the wild-type RPE65 gene to dogs. After intraocular injection of AAV-RPE65, various visual function tests confirmed that vision was, in fact, restored. "A logical question leading from our results is whether subretinal injection of AAV-RPE65 would also correct the functional defects found in humans with LCA due to RPE65 mutations," the authors state. "Currently, there is no treatment for LCA and related early-onset retinal degenerative diseases." Dr. Bennett's team believes that "if long-term preservation of structure despite loss of function is a feature of RPE-associated retinopathy, delivery of RPE65 to defective RPE cells could conceivably restore some functional vision to humans as well." ...

To: nigel bates who wrote (244)	5/9/2001 12:23:21 AM
From: scaram(o)uche	Respond to of 319

Wow. Sounds like hype, but Scollay is a well-respected scientist and the backgrounds of the scientific founders is intriguing.

To: nigel bates who wrote (244)	8/29/2001 1:58:08 AM
From: tuck	Read Replies (1) \| Respond to of 319

Another contestant on non-viral gene therapy, also private: >>CLEVELAND--(BW HealthWire)--Aug. 28, 2001--Copernicus Therapeutics, Inc. announced that the United States Patent Office has issued Automated Nucleic Acid Compaction Device, US Patent Number 6,281,005. Mark J. Cooper, M.D., Senior Vice President of Science and Medical Affairs said, ``This patent provides very broad coverage for scale-up manufacture of compacted nucleic acids for gene therapy. Our non-viral formulation consists of single molecules of nucleic acid that are condensed to their minimal possible size using polycationic carriers. These compacted nucleic acid complexes have a diameter of approximately 18-25 nm as visualized in electron micrographs, and are especially effective in transfecting post-mitotic, differentiated cells. The automated compaction device permits a several log production scale up in manufacturing these complexes, and yields a very homogeneous population of condensed nanoparticles. We are currently preparing compacted DNA for a planned phase I trial in patients with cystic fibrosis in early 2002.'' ``Copernicus has established a very broad and enabling position in the field of gene therapy,'' said Robert C. Moen, M.D., Ph.D., President and CEO of Copernicus. ``Our intellectual property positions us uniquely in this field. Given the dangers inherent with viral vectors, our technology enables us to access the favorable aspects of viral vectors while providing the safety and pharmaceutical qualities inherent in non-viral gene delivery systems. This patent covers our scale-up and manufacture of compacted DNA, and effectively extends our patent protection for production of gene therapy clinical trial reagents. We expect to broadly partner this gene transfer platform with other pharmaceutical and biotechnology companies wishing to exploit the advantages of our proprietary technology.'' Copernicus Therapeutics, Inc. is advancing novel targeting and delivery systems with broad applications in human therapeutics and vaccines. Copernicus' technologies include a multi-component delivery platform that can be applied to nucleic acids to develop therapies for a variety of human diseases and a targeting platform enabling the efficient uptake of drugs by specific cells and tissues. The Company's targeting and delivery platforms are complementary and can be combined to enhance the efficacy and safety of existing drugs or to create novel therapeutics. << snip Cheers, Tuck