Chinese scientists become first to test CRISPR in humans, as ‘Sputnik 2.0’ begins

China has beaten out U.S. academics and biotechs to become the first in testing new gene editing tech known as CRISPR-Cas9 in humans.

Reported in the Nature journal, Chinese scientists led by oncologist Lu You at Sichuan University in Chengdu, injected modified cells using CRISPR into a patient with a form of lung cancer at the West China Hospital.

Initially it looked like a U.S. biotech would be the first to start human trials using what was until last month only preclinical; then a team funded by the billionaire co-founder of Napster Sean Parker stepped up and threw his weight and wallet behind T-cell biology specialist Dr. Carl June, and a series of major U.S. medical academic centers led by the University of Pennsylvania, in an effort to get their team to run the first-ever CRISPR trial.

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But in the end, it was Chinese researchers who will now have the title of starting the first-ever human trial using CRISPR gene editing.

Speaking to Nature, Dr. June said: “I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product.” He now eyes his own team’s clinical trial to start early next year, he told the journal.

The Chinese team plans to treat 10 people in all with a series of injections, and the primary focus is on safety, given how early-stage the research is. There are long-running concerns about whether this gene-editing tech could produce some severe side effects (notably, an unwanted autoimmune response), so showing safety will be paramount in these early days.

This follows on from China being the first in using CRISPR-edited human embryos, as well as CRISPR-edited monkeys.

Each group is using CRISPR in slightly different ways, but the fundamental aspect sees the tech used to edit genes by leveraging an RNA guide molecule to enter in specific cells.

A protein called Cas9 then attaches to the DNA and essentially cuts it, all of which either gets rid of, completely removes or replaces a gene with a better strand of DNA. It could be used in a host of diseases, but oncology appears to be the favored early target.

According to the report in Nature, the team removed immune cells from the recipient’s blood and then disabled a gene in them using CRISPR-Cas9.

Lu’s team then cultured the edited cells, increasing their number, and injected them back into the patient, who has metastatic non-small-cell lung cancer. The hope is that, without PD-1, the edited cells will attack and kill the cancer.

Editas, Intellia and most recently CRISPR Therapeutics have all this year got off an IPO based on their preclinical work on CRISPR, but perhaps surprisingly have not been first into the clinic for human testing. Caribou Biosciences, which raised $30 million in a Series B financing round back in May, remains one of the last big CRISPR biotechs to not go public.

There are however still lingering questions over who owns the patents to these technologies and how, should this change, it will impact those biotechs and academic groups who have signed up to use these tools.