Written by BIS Research Analysts Chanda ManuKoushik and Sanya Behl

Chinese have always been at the forefront to go ahead and unleash the complete potential of the genome editing technologies. Recently, in November 2018, a Chinese scientist, He Jiankui’s claim has perplexed the entire scientific community. With his claim to be the first one to edit viable human embryos genetically, He Jiankui has conveyed his reluctance toward following ethical code and conduct. He aims to eliminate the risk of HIV infection in the babies, using gene editing technologies. Despite the aim being noble, the methodology involves several potential risks.

Although, several scientists supported his work for the development of therapeutics, they criticized the unethical methodology incorporated for the experiment. Currently, gene editing technologies are being used for the development of therapeutics. However, the use of gene editing technologies, including CRISPR technology, on viable human embryos, is unethical as it would be practically impossible to track the consequences, which would be phenotypic in later stages of life. In regard with He Jiankui’s case, China’s National Health Commission launched an investigation to evaluate the repercussions of unethical use of CRISPR technology.

Few instances, in which CRISPR technology is being predominantly used in harmony with the current ethical code and conduct are as follows:

• The U.S. company, Vertex Pharmaceuticals, Inc. in collaboration with Swiss-based CRISPR Therapeutics AG launched clinical trials in 2018 to treat Beta-Thalassemia, using CRISPR technology.
• In 2016, a team of  Chinese researchers from Sichuan University has become the first in the world to use the revolutionary CRISPR-Cas9 technology in humans. They edited the immune cells of a lung cancer patient, to kill the cancerous cell by the patient’s immune cells without attacking healthy cells.

The massive potential of the technology has engrossed investments from several conglomerate companies and research institutes in various countries, including Germany, South Korea, China, Singapore, and Australia. These investments are aimed for the development of therapeutics which are intended to cure various diseases, such as beta-thalassemia, sickle cell anemia, Duchenne muscular dystrophy, cystic fibrosis, and hemophilia. On the other side of the coin, contrary to the ethical code and conduct, multiple trials on humans and human embryos have raised several ethical concerns with regards to ‘designer babies’. The reasons due to which ‘designer babies’ are considered as unethical includes an off-targeting effect of the technology, the risk of development of deformed babies, and side effects associated with radiation and chemical exposures for the expecting mother.

Acknowledging the scope for the use of the CRISPR technology for unethical aims, several developed countries have enforced stringent regulatory framework in with regard to the use of CRISPR technology. Few developed countries, including Canada, France, and South Korea, have rolled-out reforms against the use of CRISPR technology in editing human germline. For instance, in Canada, human germline is forbidden under the 2004 Assisted Human Reproduction Act, and punishable by penalties of 500,000 Canadian dollars and the prospect of up to 10 years of imprisonment. On the other side, countries namely, Germany, the U.S., Japan, and the U.K. have formulated reforms to limit the use of CRISPR technology only on nonviable embryos and plants. The European Union and the U.S. possess an established legal and regulatory framework for protecting the development of genetically modified organisms and safeguarding animals, humans, and the environment.

Thus, the investigational reviews of He Jiankui’s case might force the government bodies to refrain pharmaceutical players from using CRISPR technology for the development of therapeutics. Further, government bodies might formulate reforms limiting the use of CRISPR technology only by government organizations. Similar kind of actions by government bodies were witnessed in the case of Terminator technology. The Terminator technology was used by leading agribiotech companies to develop sterile seeds. Acknowledging the unfavorable consequences of developed sterile seeds, the government bodies were forced by several civil society groups across the globe, to impose stringent regulations against the use of Terminator technology by agribiotech companies.

Currently, companies such as CRISPR Therapeutics AG, Editas Medicine Inc., and Intellia Therapeutics Inc. are developing CRISPR-based therapeutics. Owing to the growing concerns against the incorporation of CRISPR technology for unethical methodologies, industry experts anticipate that the clinical trials pertaining to the current CRISPR-based therapeutics would be extended. Further, owing to the increasingly stringent norms regarding the use of CRISPR technology would significantly elevate the clinical trial requirements for the approval of CRISPR-based therapeutics. However, the conclusions of He Jiankui’s case are yet to be observed, while its impact on the industry is still uncertain.

References:

1. https://edition.cnn.com/2018/11/26/health/china-crispr-gene-editing-twin-babies-first-intl/index.html
2. https://edition.cnn.com/2016/11/15/health/china-human-gene-testing-crispr-cas9/index.html
3. https://www.vox.com/science-and-health/2018/11/30/18119589/crispr-technology-he-jiankui
4. https://thebulletin.org/2018/06/crispr-goes-global-a-snapshot-of-rules-policies-and-attitudes/
5. https://www.biosafety-info.net/file_dir/3153248858098ca2f3.doc