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Gene therapy holds the promise of curing disease and improving the quality of life for millions of Americans who suffer from cystic fibrosis, cancer and other illnesses. Since the first clinical trial was initiated in 1990, the area of gene therapy has expanded greatly with an increasing number of sponsors and academic researchers conducting human trials. Thousands of patients have now received experimental gene therapies. Target indications for gene therapies include genetic and metabolic diseases, cancer, acquired diseases such as AIDS, and cardiovascular disease. The field of gene therapy continues to focus its efforts on patients with severe and life-threatening diseases who usually have few treatment options or who have failed all available therapies.
Even after a decade of research and clinical development, many of the gene therapy clinical trials that are active today are in an early stage of development. These early phase studies (Phase I/II) are specifically designed to evaluate the safety of the vector under investigation. These trials continue to be in early development not because of problems establishing a safety profile, but because researchers are exploring their options with indications, routes of administration, dosing regimes, patient populations, combination therapies and novel vectors.
Gene therapy is subject to greater oversight than virtually all other technology. The FDA, through its statutory role as the regulator of drug development, and the NIH/RAC, as the forum for public discussion, have served to protect patients while ensuring that important research moves ahead.
BIO believes that both the FDA and the NIH/RAC play important roles in the oversight process. BIO recommends that any system of oversight for gene therapy be memorialized in an agreement between the key federal agencies and industry that will provide the agencies with safety data while ensuring that patient confidentiality and trade secret data is protected. BIO looks forward to working with the NIH/RAC and the FDA to develop a system that protects patients without hurting the integrity of the drug development process.
Germ Line Gene Therapy Moratorium
For more than a decade, the academic and industrial research communities have observed a voluntary moratorium on gene therapy procedures that would affect the germ line cells — the egg and sperm — that pass on genetic composition to future generations.
- More Information
- BIO comments (145 KB HTML) on FDA proposed rule regarding disclosure of certain information in gene therapy and xenotransplantation experiments (April 18, 2001)
- Testimony of Michael J. Werner, Esq., on behalf of the Biotechnology Industry Organization (BIO), regarding the Proposal That Calls For A Moratorium on Some Human Gene Therapy Protocols before the NIH Recombinant DNA Advisory Committee. (March 10, 2000)
- Testimony of H. Stewart Parker, Targeted Genetics Corporation, on behalf of the Biotechnology Industry Organization (BIO), on oversight of gene therapy before the U.S. Senate Health, Education, Labor and Pensions Subcommittee on Public Health. (February 2, 2000)
- Position of the Biotechnology Industry Organization (BIO), regarding oversight of gene therapy (December 7, 1999)
- Comments (65 KB HTML): Recombinant DNA Research, Proposed Actions Under the NIH Guidelines
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