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Eileen Korey Director of News Services University Hospitals of Cleveland 216/844-3825

or George Stamatis Director of Public Affairs Case Western Reserve University 216/368-3635

        Scientists and physicians in Cleveland have announced encouraging

results from the first-of-its-kind gene therapy trial involving cystic

fibrosis (CF) patients and a new compacted DNA technology. The Phase I

trial involving 12 patients was launched one year ago by University

Hospitals of Cleveland (UHC), Case Western Reserve University (CWRU)

School of Medicine, Children's Hospital of Denver, and Cystic Fibrosis

Foundation Therapeutics, Inc., the nonprofit drug discovery and

development affiliate of the Cystic Fibrosis Foundation.

        Cleveland-based biotechnology firm Copernicus Therapeutics Inc.

produced the non-viral gene transfer system used in the clinical

trial. Working together with UHC and CWRU researchers, Copernicus

formulated a way to "compact" or tightly bind strands of DNA so that

it is tiny enough to pass through a cell membrane and into the

nucleus. The ultimate goal is for the DNA to produce a protein needed

by people with CF to correct the basic defect in CF cells.

        "The primary goal of this Phase I study was to determine if this

gene therapy method is safe and tolerable as administered in this

trial. All participants in this study completed the trial without

significant side effects and the treatment itself was well-tolerated,"

stated Michael W. Konstan, M.D., Associate Professor of Pediatrics at

CWRU and Director of the LeRoy Matthews Cystic Fibrosis Center,

Rainbow Babies & Children's Hospital of UHC. "The secondary goals of

the trial were to evaluate if the CF gene was successfully transferred

to airway cells and if it functioned normally, results which would

suggest that this therapy may be of benefit to people with CF. Our

data were very encouraging with indications that this gene transfer

may have occurred."

        "This gene therapy research has exciting potential as a new

approach to addressing the genetic root cause of CF," said Robert J.

Beall, Ph.D., president and chief executive officer of the Cystic

Fibrosis Foundation. "We are pleased that this novel method of gene

delivery has cleared the first hurdles of clinical research by proving

both safety and tolerability. We eagerly anticipate results of future

clinical studies utilizing this novel approach." CFFT helped fund this

initial trial, along with Copernicus and the National Institutes of

Health through a Core Center Grant to the CF Center at UHC/CWRU and

through the General Clinical Research Center.

        Copernicus Therapeutics recently received more than one million

dollars in state funding through the Technology Action Fund of the

Ohio Department of Development to continue development of compacted

DNA. The company is currently working on an aerosol version that would

enable people with CF to have the healthy gene delivered directly into

their lungs. During the recently completed study, patients received

the compacted DNA in a saline solution dripped into the nasal

passages. Future clinical trials will study the safety and efficacy of

the aerosol approach.

        "Gene transfer technology is expected to revolutionize treatment

of genetic disease by using DNA as a novel therapeutic," said Jeff

Wagener, M.D., Professor of Pediatrics at the Children's Hospital of

Denver, which enrolled three patients in the study. "Striking at the

root cause of CF should ultimately provide an even more effective

treatment for CF than those available today which are aimed at

managing the side effects of the disease."

        The underlying cause of CF, which affects approximately 30,000

Americans, is a defective gene that upsets a delicate salt/water

balance in the lungs. At the crux of the process is a protein,

produced by the CF gene, which controls the flow of salt and water in

and out of cells. In CF patients, this protein does not operate

normally in the cells that line the airways. In turn, the airways

accumulate thick and sticky mucus. Bacteria proliferate in the mucus

and cause chronic infections that permanently damage lungs.

        Under the direction of Dr. Konstan, the research teams for this

recently completed Phase I study delivered the healthy gene into

twelve adult CF patients in a saline solution dripped slowly into

their nasal passages. Investigators monitored salt transport in the

nose, called the "nasal potential difference," as a barometer of the

procedure's success. "CF patients have a markedly abnormal nasal

potential difference," Dr. Konstan said. Through biopsies of nasal

tissue, researchers determined whether the healthy gene was "taken up"

by the cells and produced enough protein to affect the transport of

salt and water in and out of the cells. They found that two-thirds of

patients treated had a meaningful increase in the transport of

chloride ion in the nose.

        The researchers and physicians involved in this study will present

their findings at the American Society of Gene Therapy meeting in

Washington, D.C. in early June, and at the Cystic Fibrosis Foundation

Williamsburg meeting at the end of May.



        University Hospitals Health System (UHHS) is the region's premier

healthcare delivery system, serving patients at more than 150

locations throughout northern Ohio. The System's 947-bed, tertiary

medical center, University Hospitals of Cleveland (UHC), is the

primary affiliate of Case Western Reserve University (CWRU). Together,

they form the largest center for biomedical research in the State of

Ohio. The System provides the major clinical base for translational

researchers at the Case Research Institute, a partnership between UHC

and CWRU School of Medicine, as well as a broad and well-characterized

patient population for clinical trials involving the most advanced

treatments. Included in UHC are Rainbow Babies & Children's Hospital,

among the nation's best children's hospitals; Ireland Cancer Center,

northern Ohio's only National Cancer Institute-designated

Comprehensive Cancer Center (the nation's highest designation); and

MacDonald Women's Hospital, Ohio's only hospital for women.

        Founded in 1843, the Case Western Reserve University School of

Medicine is the largest medical research institution in Ohio and the

14th largest among the nation's medical schools for research funding

from the National Institutes of Health. Seven Nobel Laureates have

been affiliated with the school. The School of Medicine is recognized

throughout the international medical community for outstanding

achievements in research, teaching and service. Annually, the School

of Medicine trains more than 600 M.D. and M.D./Ph.D. students.

        Founded in 1908, The Children's Hospital of Denver is a private,

not-for-profit pediatric health care network. It is consistently

ranked one of the best children's hospitals in America by U.S. News &

World Report and other publications. With more than 1,116 pediatric

specialists and 2,000 full-time employees, Children's is home to a

number of nationally and internationally recognized medical programs.

Children's provides care at its main campus and through a network that

includes four community-based urgent care sites, seven specialty-care

centers and more than 400 outreach clinics in three states each year.

        The Cystic Fibrosis Foundation was created in 1955 to assure the

development of the means to cure and control CF and to improve the

quality of life for people with the disease. CFFT is the nonprofit

drug development affiliate of the CF Foundation that operates drug

discovery, development and evaluation efforts. Total support of CFFT

is provided by the CF Foundation.



CONTACT:

Cystic Fibrosis Foundation

Allison Tobin, 301/841-2665

or

Copernicus Therapeutics, Inc.

Robert C. Moen, M.D., 216/231-0227

or

The Children's Hospital of Denver

Rachel Robinson, 303/861-6388

SOURCE: Copernicus Therapeutics, Inc. & University Hospitals of

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04/29/2003 10:03 EASTERN