China's Gene-Editing Baby Scientist Stirs Up Rare Disease Controversy

Jiankui He, the researcher behind China's world-shocking gene-edited babies scandal, now leaps into rare diseases research area but ethics oversight remain murky.

Four years ago, a rouge scientist shocked the world disclosing he had edited the human embryos and led to the birth of twin babies.

Jiankui He, an associate professor at China’s Southern Institute of Technology in Shenzhen, was later sentenced to three years in prison after he was found to have violated ethnics review requirements, and dodged a regulatory oversight process, even as the editing of human embryos is prohibited by law in China. (Also see "Chinese Scientist Behind Gene Edited Babies Gets 3 Year Jail Term " - Pink Sheet, 31 Dec, 2019.)

Last September, He was released and moved to Beijing, where he began to focus on using gene-editing to treat rare disease, starting with Duchenne Muscular Dystrophy. In his Twitter banner, he said his goal is to make the cutting-edge technology affordable to patients with rare medical conditions.

He has since set up a lab in the city and claimed to have support from over 600 families of DMD patients in China. His plan is to raise billions to use the technology to treat genetic deficiencies. However, He has avoided touching up how he would address enhancing ethic oversight for his research.

At a recent BioGovernance Commons Initiative event hosted by Global Science and Epistemic Justice at the University of Kent, He talked about his latest research and “building public confidence and trust in human genome editing.” However, He didn’t address any of multiple questions seeking clarifications about ethnic oversight over his current research.

He announced in a 21 February briefing in Beijing that he had obtained a visa from the Hong Kong government to continue his research, and that he had been in touch with universities, research institutes and groups for collaboration. Hours later, though, the Hong Kong government said the visa has been revoked, citing lapses in the review and approval process.

He’s failure to address the ethical concerns about his latest gene editing project has many worried about the potential ripple effects of what a reckless scientist could do to a novel and promising technology.

In November 2018, He disclosed during the second International Summit on Genome Editing held in Hong Kong that his team had edited the genes of two babies. But he failed to disclose that a third baby was also being edited and born.

The latest saga over his visa application and revocation didn’t help. Joy Zhang, co-organizer of the BioGovernance event held on 11 February, said that regulatory gaps exposed by the event have been noticed by relevant Chinese authorities.

He’s media relations partner has not responded to our request for interview.

Gene Editing For DMD

Treating rare diseases with gene-editing technology is promising. In the US, Sarepta Therapeutics, Inc. has a pending application for the gene therapy SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy. SRP-9001’s user fee date is 29 May 2023, and Sarepta announced on 28 February that FDA does not plan to hold an advisory committee for the application. 

Eric Olson, professor and chairman of molecular biology at University of Texas Southwestern Medical Center, told the Pink Sheet that ethic oversight is vital for any gene-editing research on rare conditions such as DMD. “Hopefully, there’s more oversight and transparency than in his last trial on babies,” noted Olson, the author of a recent paper titled CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System.

Given the uncertainty, He’s research casts a further shadow due to a lack of ethnic oversight and rigorous scrutiny.

However, patients are eager to embrace the technology. So far, there is no drug approved in China for the devastating condition, and gene therapy and gene editing are considered to be a long-term cure instead of the current standard of care in the country, corticosteroids, which only delay the onset of the symptoms and include severe adverse effects which include stunted growth, weight gains and fragile bones and fractures.

Gene Editing In China

In China, gene editing is being studied on thalassemia, a rare blood disorder. Beijing-based EdiGene Inc. and Shanghai-based BRL Medicine are the only companies that have obtained the investigative new drug clearance for their gene-editing projects.

In February, China said it would ban the export of gene editing technology including CRISPR cas9, TALEN and ZFN. The move is a direct response to United States imposing bans on exporting advanced technology such as chip manufacturing to China (Also see "China Planning To Ban Gene Editing Technology Exports" - Pink Sheet, 7 Feb, 2023.). 

However, China is not a leading player in gene editing and the impact of the ban is expected to be limited.

If He does make a big breakthrough in the field, it might be revealed as dramatically as the gene-edited babies were. In a 26 February Twitter post, He said he will be stopping all social media updates and focusing on scientific research.