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 : Ligand (LGND) Breakout! -- Ignore unavailable to you. Want to Upgrade?

To: Henry Niman who wrote (21877)	6/5/1998 7:34:00 AM
From: Skeeter Bug	Read Replies (1) \| Respond to of 32384

henry, lgnd and rumors leave a bad taste in my mouth ;-) may buy back in today...

To: Henry Niman who wrote (21877)	6/5/1998 10:34:00 AM
From: bob zagorin	Respond to of 32384

off topic but on topic (patents and RNA). this is exciting to me - repairing defecting RNA! Ribozyme Pharmaceuticals, Inc. Signs Worldwide Exclusive Licensing Agreement with Duke University for Novel RNA Repair Technology to Treat Sickle Cell Anemia - Research Published in Current Issue of Science; RNA Repair Technology May Have Broad Applicability Across Many Diseases - BOULDER, Colo., June 5 /PRNewswire/ -- Ribozyme Pharmaceuticals Inc. (RPI) (Nasdaq: RZYM) today announced that it has received a worldwide exclusive license from Duke University for the use of a ribozyme-based approach to treat sickle-cell anemia, an inherited blood disease in which the hemoglobin (oxygen-carrying pigment) of the red blood cells functions abnormally. As published in this week's issue of the journal, Science, Volume 280, Dr. Bruce Sullenger, Assistant Professor of Experimental Surgery at Duke University Medical Center, and his research team describe the use of a ribozyme, a catalytic RNA molecule, to repair a genetic defect in the beta-globin gene that is responsible for causing the disease. Financial terms of the agreement were not disclosed. Dr. Thomas Cech, who shared a Nobel Prize for the discovery of ribozymes, described the work as "impressive progress towards a new kind of therapy for sickle cell anemia. Instead of treating the symptoms, Sullenger and co- workers have devised a way to attack the cause of the disease. Because most genetic diseases (including muscular dystrophy and cystic fibrosis) involve a defective gene product, the RNA repair technology may be very broadly applicable." In the paper titled "Ribozyme-mediated repair of Sickle beta-globin mRNAs in erythrocyte precursors," Dr. Sullenger and his colleagues describe the use of a ribozyme to convert the defective form of beta-globin RNA in peripheral blood cells from sickle cell patients into one that will produce a normal and effective form of hemoglobin. Experiments are currently underway to demonstrate the ability of these ribozymes to repair mutant beta-globin RNA in an appropriate animal model. "This latest discovery increases the potential use of ribozymes to include not only the cleavage of RNA sequences and the inhibition of gene expression, but also the splicing of RNA sequences as well," said Ralph E. Christoffersen, Ph.D., president and chief executive officer of RPI. "This adaptation allows for the possibility of repair of defective RNA molecules, and we look forward to many therapeutic applications in this new area. Our exclusive licensing agreement with Duke, coupled with the extensive patent coverage already present at RPI, further validates our ribozyme technology platform, and we are very pleased to be working with Dr. Sullenger and his research team, who have pioneered this novel use of ribozymes for RNA repair." Approximately 80,000 people in the United States suffer from sickle cell anemia. Characterized by chronic anemia and episodes of pain, the disease can cause breakdown of red blood cells and inhibit red blood cell production. Such incidences can lead to blockage of blood vessels and may damage the kidneys, lungs, bone, liver and central nervous system. Current treatments, such as blood transfusions and pain-killing drugs, address only the symptoms of the disease. To date, there is no available cure for sickle cell anemia. "We are very encouraged by the results achieved in this study and are excited about our exclusive licensing deal with RPI," said Dr. Sullenger. "Sickle cell anemia is the most common heritable blood disease, yet no curative treatment exists. This new approach offers hope that RNA repair may become a useful therapy for treating genetic disorders." Ribozymes are a catalytic form of RNA found in nature that have the ability either to selectively inhibit gene expression or splice RNA together. Separate from the work described in the Sullenger paper, RPI has created proprietary chemically modified ribozymes that are not degraded rapidly in patients' serum. These unique compounds are therefore able to act as powerful therapeutic agents. In addition, these molecules are used as tools for understanding gene function, as well as for discovering new therapeutic targets and validating which genes are appropriate therapeutic targets. RPI has a strong patent position on the basic ribozyme technology, including the dominant patent on the RNA-repair technology described in the Science paper, which has already issued in the United States with broad claims (U.S. Patent No. 5,667,969). With this agreement, RPI has now licensed from Duke University multiple applications of the RNA repair technology for specific diseases, such as sickle cell anemia.