Guest guest Posted May 1, 2003 Report Share Posted May 1, 2003 Subject: Development of Novel Treatment For Cystic Fibrosis;Phase IIb Clinical Trial to Begin in Mid-2003 PR Newswire April 24, 2003, Thursday HEADLINE: Targeted Genetics and Cystic Fibrosis Foundation Therapeutics Announce Collaboration to Advance Clinical Development of Novel Treatment For Cystic Fibrosis;Phase IIb Clinical Trial to Begin in Mid-2003 DATELINE: SEATTLE and BETHESDA, Md., April 24 Targeted Genetics Corporation (Nasdaq: TGEN) and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation, today announced a collaboration agreement to advance the development of Targeted Genetics' product candidate, tgAAVCF, to treat patients with cystic fibrosis (CF). The focus of this collaboration is to conduct a Phase IIb clinical trial to evaluate the efficacy of tgAAVCF in patients with mild to moderate CF. CFFT will award approximately $1.7 million in funding to the clinical trial sites in this study. " We are pleased to continue the development of our CF program in partnership with CFFT. CFFT's unique approach for developing new therapies specifically for CF is a significant factor in our ability to move tgAAVCF forward, " said H. , president and CEO of Targeted Genetics Corporation. " CFFT's network of clinical care centers, the Therapeutics Development Network, played a critical role in executing successful clinical trials for Targeted Genetics in our most recent study. Now that we have established a strong safety profile with tgAAVCF, we look forward to combining the knowledge and expertise of both organizations to further evaluate the efficacy of our product candidate to treat the underlying cause of CF. " Targeted Genetics has recently completed a Phase II clinical trial testing the safety of tgAAVCF in patients with mild to moderate CF. Preliminary results suggest a strong safety profile associated with the product candidate, and positive trends were observed in activity endpoints such as lung function and levels of inflammatory cytokines, both considered important measurements of disease. Based on the preliminary results of this study, Targeted Genetics and CFFT will initiate a larger, confirmatory Phase IIb clinical trial with the primary activity endponge in lung function after 30 days. This trial is scheduled to begin patient recruitment in mid-2003. " The CF Foundation places great emphasis on the importance of research to develop novel approaches to treating this life-threatening disease. Currently approved therapies treoms of CF; we believe that tgAAVCF may hold significant potential for treating the underlying cause of this disease, " said J. Beall, Ph.D., president and CEO of the CF Foundation and CFFT. " If successful, this therapy could have profound implications for individuals with CF and their families. We appreciate the efforts of Targeted Genetics in this arena and look forward to collaborating with them going forward. " About CF CF is caused by a defective CFTR gene. Normally, the protein encoded by this gene is found in the airways, pancreas, salivary glands and sweat glands, where it helps to regulate chloride in the cells. When this protein malfunctions, it leads to the production and build-up of thick, sticky mucus in the lungs and digestive system. The abnormal mucus leads to chronic bacterial infections in the lungs and the invasion of white blood cells, causing inflammation and scarring. The lungs deteriorate over time leading to a loss of respiratory function. Common symptoms include very salty-tasting skin, persistent coughing, wheezing or pneumonia, excessive appetite but poor weight gain, and bulky stools. It is estimated that 70,000 people worldwide have CF, including approximately 30,000 people in the United States. The median age of survival for patients with CF is currently 33.4 years. Quote Link to comment Share on other sites More sharing options...
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