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Gene Therapy Developers Issue Principles for Human Genome Editing

Posted 27 August 2019 | By Michael Mezher 

Gene Therapy Developers Issue Principles for Human Genome Editing

The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology.
While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions.
“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,” said ARM CEO Janet Lambert.
The framework comes less than a year after Chinese scientist He Jiankui announced he had edited the genes of infant twin girls using CRISPR in an attempt to make them more resistant to HIV infection. Jiankui’s announcement prompted swift international backlash, with regulators in China proposing rules to restrict gene editing in humans and experts around the world calling for a moratorium on human germline editing.
The World Health Organization (WHO) also reacted to the announcement by forming an advisory panel on human genome editing, which called for the development of a central registry for all human genome editing studies last March.
A Framework for Gene Editing
Among the five principles included in the framework are an endorsement of therapeutic applications of somatic cell gene editing; support for the development of gene editing standards and “evolving” regulatory frameworks for gene editing techniques; and opposition to human germline gene editing until unresolved scientific, legal and ethical questions are addressed at an international level.
The companies endorsing the principles include Audentes, bluebird bio, BlueRock, Caribou Biosciences, Casebia Therapeutics, CRISPR Technologies, Editas, Homology Medicines, Intellia Therapeutics, LogicBio, Precision Biosciences, Sangamo and Tmunity.
In addition to opposing human germline gene editing, the task force also opposes the implantation of human embryos that carry gene modified cells.
“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,” the task force writes.
The principles offer support for efforts by the National Institute of Standards and Technology (NIST) Genome Editing Consortium, US Pharmacopoeia, International Organization for Standardization (ISO) and other standards bodies that are working to develop gene editing standards that could support the development and assessment of gene therapies.
The task force also argues that existing regulatory frameworks from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) offer appropriate oversight for somatic cell gene therapies, but caution that “arbitrary and ancillary oversight bodies or processes” could delay the development of promising new treatments.


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