Gaithersburg, MD (PRWEB) August 18, 2003
Intronn Inc. announced today that it corrected the genetic defect responsible for hemophilia A in animals using a new approach to genetic repair. The study, published in the August issue of Nature Medicine, shows for the first time, phenotypic correction of a genetic defect by RNA repair in knockout hemophilic mice.
IntronnÂs technology, known as spliceosome mediated RNA trans-splicing or SMaRT(TM), produced sufficient quantities of normal Factor VIII to correct the hemophilia A phenotype in hemophilic mice. The work was done in collaboration with scientists at the Mount Sinai School of Medicine in New York.
ÂSMaRT(TM) is an entirely new approach to gene medicine,Â said Dr. Gerard J. McGarrity, IntronnÂs President and Chief Executive Officer. ÂUsing SMaRT(TM) we replace only the mutated portion of the gene, not the entire gene, to achieve phenotypic correction of the disease. Another advantage of SMaRT(TM) is that it utilizes the target cellÂs natural systems of RNA splicing and regulation of gene expression. We look forward to submitting an application for clinical trials in the second half of 2004.Â
Intronn recently announced that it received an SBIR Phase I grant of $605,791 from the National Heart Lung and Blood Institute of the National Institutes of Health to support further studies in hemophilia A under the direction of IntronnÂs Dr. Gary Mansfield and in collaboration with Mount Sinai.
SMaRT(TM) re-programs gene expression at the level of pre-messenger RNA through the use of IntronnÂs pre-trans-splicing molecules or PTMs. PTMs contain a binding domain, a splicing domain and a coding domain. The application depends on the nature of the sequences in the coding domain. The coding sequences can be a single exon, a series of exons or entire genes.
For more information contact:
Gerard J. McGarrity, Ph.D.
910 Clopper Road
South Building, Suite 210
Gaithersburg, MD 20878
Christopher E. Walsh, M.D., Ph.D.
Mount Sinai School of Medicine
One Gustave Levy Place
New York, NY 10029