Rare Daily Staff

The cell and gene therapy advocacy group Alliance for Regenerative Medicine issued a Statement of Principles that calls editing of germline cells for use in human clinical studies “inappropriate,” but endorsed investigation of therapeutic applications of somatic cell gene editing.

“Gene editing technologies have not matured to the point where human trials of edited germline cells are appropriate,” the statement said. “Many important safety, ethical, legal, and societal issues involved with this type of gene editing remain unresolved.”

Gene editing technology enables users to insert, replace, remove, or modify DNA at particular locations in the human genome. It has the potential to provide a durable or curative effect for patients suffering from a wide variety of serious or potentially fatal genetic disorders. Unlike germline cells, somatic cells do not pass on genetic material to the next generation.  

The statement provides an industry view on a bioethical framework for the use of gene editing in therapeutic applications. The statement, developed by ARM’s Gene Editing Task Force and signed by 13 therapeutic developers using gene editing technologies, specifies a set of principles for the ethical use of gene editing and genetic modification.

The group said regulated, clinical validation of somatic cell-based gene editing technologies for non-inherited genetic modification is, and should remain, the primary objective of the therapeutic development community and, in contrast to germline gene editing, offers the most acceptable near-term path to potentially transformative therapeutic benefits for patients.

It also supports the use of gene editing standards to facilitate the development of safe and efficacious gene editing therapies. There is ongoing work by the NIST Genome Editing Consortium, US Pharmacopeia, International Organization for Standardization (ISO), and other standards development organizations to formulate gene editing standards.

While the statement called for the “continued evolution” of regulatory frameworks governing the development of somatic cell gene editing techniques, it said “arbitrary and ancillary oversight bodies or processes may carry the risk of delaying research and development efforts, which in turn would adversely impact afflicted patient populations.”

“Gene editing is a rapidly developing technology that represents one of the most exciting developments in medicine. These techniques will be integral to the next generation of advanced therapeutics and we welcome their potential to provide important, and potentially life-saving, treatments for patients,” said Janet Lambert, CEO of ARM. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles.”

Photo: Janet Lambert, CEO of ARM

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