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Copernicus Therapeutics, Inc. announced today that it received a milestone payment from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), a non-profit affiliate of the Cystic Fibrosis Foundation. This milestone payment is part of a research, development, and commercialization agreement between CFFT and Copernicus to develop gene therapy to treat cystic fibrosis.
Copernicus earned the milestone payment by demonstrating a significant improvement in the level of CFTR gene activity in an animal model. This increased improvement in the level and duration of CFTR gene activity represents an important step in the path to developing a clinically relevant therapy.
Copernicus' unique, non-viral nanoparticle formulation is intended to deliver a normal copy of the CF gene to the affected lung cells of CF patients, and may provide a therapy that treats the root cause of CF regardless of the specific mutations resulting in CF. Copernicus' first clinical trial, supported by CFFT, demonstrated the desired safety profile and encouraging biological changes were observed.
Cystic fibrosis is a life threatening genetic disease that affects approximately 30,000 children and adults in the United States. People with CF develop serious lung infections and digestive complications. About 10 million Americans are unknowing carriers of a defective CF gene.
"We have had a productive relationship for a number of years with CFFT and we share the common goal of providing a safe and effective therapeutic for people with CF," said Robert C. Moen, M.D., Ph.D., president and CEO of Copernicus. "We look forward to working with CFFT in meeting the critical need for an effective gene-based therapy for the lung manifestations of CF."
"We are encouraged by the promise of Copernicus' technology," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "Gene-based therapies offer hope for potentially lifesaving treatments that can be applied to all patients with CF, regardless of the specific type of gene mutation."
About Copernicus Therapeutics, Inc.
Copernicus Therapeutics, Inc., a privately held biotechnology company, is dedicated to delivering the promise of nucleic acid therapeutics. The Copernicus multi-component delivery platform can be used to develop nucleic acid therapies for numerous human diseases. The same technology that is being tested for its ability to deliver the CF gene to the lung of CF patients can be applied to treating serious disorders of the brain, such as Parkinson’s disease, as well as for treating a variety of blinding disorders. Additionally, this lung delivery technology may be utilized for RNAi-based therapeutics to treat lung infections caused by influenza A, bird flu, and other respiratory viruses. Additional information about Copernicus is available at http://www.cgsys.com .
The Cystic Fibrosis Foundation is the leading organization devoted to curing and controlling cystic fibrosis. Headquartered in Bethesda, Md., the Foundation funds CF research, has 80 chapter and branch offices throughout the country, and supports and accredits a nationwide network of 115 CF care centers, which provide vital treatments and other CF resources to patients and families. For more information, visit www.cff.org.
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Copernicus Therapeutics, Inc.
Robert C. Moen, M.D., Ph.D.
President & CEO
Mark J. Cooper, M.D.
Sr. V.P. of Science and Medical Affairs
Cystic Fibrosis Foundation
Director of Media Relations
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