Targeted Genetics Phase I Cystic Fibrosis Research Featured in Current Issue of The Lancet
06:32 a.m. Jun 08, 1998 Eastern

SEATTLE, June 8 /PRNewswire/ -- Targeted Genetics Corporation announced today that the Company's proprietary gene therapy for cystic fibrosis (CF), tgAAV-CFTR, is the subject of a research paper appearing in the June 6 issue of The Lancet. The paper, titled "Efficient and Persistent Gene Transfer of AAV-CFTR in the CF Maxillary Sinus," discussed previously announced data from a 10-patient randomized, non-blinded, dose-escalation Phase I trial of tgAAV-CFTR.

"The results of this Phase I trial demonstrated safe, dose-dependent and effective gene transfer by means of the AAV-CFTR vector," said principal investigator Phyllis Gardner, M.D., of Stanford University's School of Medicine. "In addition, the trial supported use of the maxillary sinus as a surrogate for cystic fibrosis lung disease." Targeted Genetics and a team of researchers at Stanford are currently completing a follow-up Phase II double-blinded, randomized, placebo-controlled study of tgAAV-CFTR. Enrollment in this trial, designed to test the safety and efficacy of tgAAV-CFTR in preventing sinusitis in patients with CF, has been completed and preliminary results are expected by the end of the third quarter of this year.

"Our Phase I study of tgAAV-CFTR in patients with CF demonstrates the feasibility of gene therapy as a treatment for the disease," said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. "Targeted Genetics remains on track to initiate Phase I trials of an aerosolized formulation of tgAAV-CFTR during the second half of 1998." An aerosolized formulation will provide widespread delivery of tgAAV-CFTR to the airways of the human lung. Targeted Genetics expects to take this product for the treatment of CF through the regulatory process for approval as a commercial product.

Background: tgAVV-CFTR Phase I Study at Stanford

Initiated in late 1995, the Phase I clinical trial was designed to evaluate the safety and feasibility of in vivo gene therapy using tgAAV-CFTR. The studies followed preclinical research conducted in rabbits and primates in which long-term gene expression was achieved with no observed side effects. The Stanford Phase I study, along with studies performed at Johns Hopkins University and the University of Florida, were unique in that they marked the first instance where an adeno-associated virus (AAV) vector had been used for gene delivery to humans, and the first time an AAV vector-based gene therapy had been evaluated to treat CF. Targeted Genetics remains the only company to take an AAV vector into Phase II clinical trials and the only company to take a CF gene therapy into Phase II clinical trials.

A total of 10 patients who had undergone bilateral endoscopic antrostomies were enrolled in the trial. tgAAV-CFTR was delivered to patients' sinuses via a catheter at five escalating doses. Biopsies were taken from patients' sinuses and analyzed. The patients at the three highest dose levels all showed evidence of gene transfer, with those at the highest level averaging one copy of the vector per cell. Long-term persistence of the CFTR gene for at least 70 days was observed, which far exceeded that found historically using other types of vectors. Overall, tgAAV-CFTR was well tolerated and demonstrated no adverse effects or neutralizing immune response, including after repeat delivery.

Targeted Genetics Corporation develops gene and cell therapy products for the treatment of certain acquired and inherited diseases. The Company has three lead product development programs targeting cystic fibrosis, cancer, and infectious disease, as well as an extensive technology platform.

This news release contains forward-looking statements that involve risks and uncertainties, including the timing and results of clinical trials and other product development and commercialization risks. Risks associated with liquidity, capital resources and other risks are detailed in Targeted Genetics' filings with the U.S. Securities and Exchange Commission (SEC). SOURCE Targeted Genetics Corporation

Copyright 1998, PR Newswire

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