HEMOPHILIA A CORRECTED IN HEMOPHILIC ANIMALS

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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.

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.

Intronn’s 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.

“SMaRT(TM) is an entirely new approach to gene medicine,” said Dr. Gerard J. McGarrity, Intronn’s President and Chief Executive Officer. “Using 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 cell’s 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.”

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 Intronn’s Dr. Gary Mansfield and in collaboration with Mount Sinai.

About SMaRT(TM)

SMaRT(TM) re-programs gene expression at the level of pre-messenger RNA through the use of Intronn’s 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.

Intronn Inc.

910 Clopper Road

South Building, Suite 210

Gaithersburg, MD 20878

240-683-2450

240-683-2451 (fax)

Christopher E. Walsh, M.D., Ph.D.

Mount Sinai School of Medicine

Hematology/Oncology

One Gustave Levy Place

Room 24-2C

New York, NY 10029

212-241-3443

212-426-4390 (fax)

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Lucy Salah
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