GAITHERSBURG, Md., June 6 /PRNewswire/ -- Intronn Inc announced today that it significantly increased the levels of high density lipoprotein (HDL) or "good" cholesterol in animal studies. HDL levels were raised 30-35% in mice using the company's proprietary RNA re-programming technology. The results were presented at the Annual Meeting of the American Society of Gene Therapy.
Using Intronn's spliceosome-mediated RNA trans-splicing (SMaRTTM) technology, the RNA sequences for human ApoAI, a major component of HDL, were inserted into the RNA elements for mouse albumin, thereby enabling the cell's albumin machinery to make increased amounts of ApoAI. ApoAI then made additional HDL. Albumin was selected because it constitutes more than half of the proteins in plasma and is the most abundant gene product in liver. The prevalence of the albumin-producing machinery presents an ideal target to make therapeutic proteins in the liver.
"This is an innovative approach utilizing Intronn's SMaRTTM technology," said Gerard J. McGarrity, Ph.D., Intronn's President and Chief Executive Officer. "It is well known that increasing blood levels of HDL will reduce the risk of heart disease. While these animal results represent an early stage of study and significant challenges remain, we are cautiously optimistic that HDL levels can be raised still further. We are certainly encouraged and want to speed the development of this approach to test its effectiveness in humans."
Medical estimates state that increasing HDL levels one milligram can reduce the risk of heart disease by 2-3%. The animals in Intronn's study had increases of 20-25 milligrams.
The study was a collaboration between Intronn and the National Institutes of Health in Bethesda, MD. The NIH collaborators were Alan Remaley, M.D., of the NIH Clinical Center and Bryan Brewer, M.D., recently moved from the NIH's National Heart, Lung, and Blood Institute to the Medstar Research Institute at the Washington Hospital Center.
Intronn is a leading RNA biology firm. Its patented, broad-based SMaRT(TM) technology re-writes the genetic message at the level of pre- messenger RNA. RNA reprogramming is achieved through trans-splicing, a naturally occurring process. SMaRT(TM) can trans-splice virtually any desired sequence into a pre-mRNA target. Applications are very broad and include RNA therapeutics, real time molecular imaging, determination of alternate splice sites, and molecular evolution. Intronn focuses on RNA therapeutics.
CONTACT: Gerard J. McGarrity, Ph.D., President and CEO of Intronn Inc.,+1-240-683-2452, or firstname.lastname@example.org