Band of gene editing businesses agree to stay away from heritable changes

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Vchal)
(Image: Getty/Vchal)

Related tags: CRISPR, Gene editing, Alliance for Regenerative Medicine, WHO, bluebird bio

The Alliance for Regenerative Medicine released a ‘Statement of Principles’ on gene editing, as controversy over the technology’s potential rumbles on.

The fallout from world’s first gene edited babies continues to spread, as further details about how He Jiankui came to use CRISPR technology at the germline level​ emerge.

Not long after the scientific scandal became public knowledge, the WHO established a multi-disciplinary committee​ to advise on the ethical challenges of applying gene editing technology in humans.

Now those companies that are involved in developing gene editing technology, through the Alliance for Regenerative Medicine (ARM), have put forward their own ‘Statement of Principles’​ on how to develop and safeguard from misuse this treatment modality.

The recurrent message in the published document is that the companies that make up the ‘Gene editing task force’ are not interested in editing germline cells, or those potentially leading to heritable changes, but only somatic cells.

Somatic cells are the cells of a living organism that are not considered the reproductive cells. Therefore, any changes made to them are not passed on to future generations. Somatic cells make up internal organs, skin, bones, blood, and connective tissue, whereas germline cells are sperm, eggs, and fertilized embryos.

The reason He Jiankui’s research has proved so controversial is that CRISPR technology was used to make germline edits to the CCR5 gene, with the aim of protecting the twins involved from the HIV virus that their father had contracted.

As a result of the germline changes, the twin’s edited genome could be passed onto any children they themselves choose to have. In addition, there are fears that the CRISPR may have resulted in ‘off-target’ effects, whereby further unintended genetic modifications arise.

The group of companies, which includes bluebird bio, CRISPR Therapeutics, Editas Medicine, and Sangamo Therapeutics, amongst others, distances itself from the germline approach by making a common commitment to “solely focus[ing] on somatic cell approaches to therapeutic treatments and cures for disease.”

Unless and until ethical and potential safety questions with respect to germline gene editing are adequately addressed, we do not support or condone germline gene editing in human clinical trials or for human implantation,” ​the commitment continues.

The full document contains five principles, which are highlighted, in brief, below:

  1. We endorse investigation of therapeutic applications of somatic cell gene editing
  2. We support the use of gene editing standards to facilitate the development of safe and efficacious gene editing therapies
  3. We call for the continued evolution of national and regional regulatory frameworks governing the development of somatic cell gene editing techniques
  4. We assert that germline gene editing is currently inappropriate in human clinical settings
  5. Common commitment to not support or condone germline gene editing until ethical and potential safety questions have been addressed

Janet Lambert, CEO of ARM, commented, “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in R&D practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles.”

Related topics: Cell & Gene Therapies, Bio Developments

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