MEDICAL SCIENCE

China, US race to develop ‘gene editing’ technology that could, if approved, revolutionise medicine

PUBLISHED : Wednesday, 24 February, 2016, 2:25pm
UPDATED : Wednesday, 24 February, 2016, 2:25pm

US companies racing to develop a promising gene editing technology are up against a formidable competitor - the Chinese government.

China has long set its heart on building an expertise in genomics and its government is pouring funds into a new and sometimes controversial tool called Crispr, encouraging its researchers to advance the technology. Chinese scientists say they were among the first in using Crispr to make wheat resistant to a common fungal disease, dogs more muscular and pigs with leaner meat.

The scientific research bankrolled by the Chinese government could eventually be tapped by agricultural and pharmaceutical companies. Programmes funded by Beijing are, among other things, working on disease-resistant tomatoes, breast cancer treatments and increasing the oil content in soy beans.

Researchers in Guangzhou who received government funds went a step further, sparking an international ethical debate last year after tweaking the genetic make up of human embryos using Crispr for the first time.

READ MORE: Chinese scientists genetically modify human embryos for the first time

The genetic editing tool acts like a pair of low-cost and highly precise molecular scissors that can cut out unwanted sections of DNA and insert desired ones.

It has revolutionised what was once a time consuming, pricey and inaccurate process, and scientists and businesses worldwide are seeking to capitalise on this emerging technique.

The expertise the Chinese groups are developing positions them to eventually challenge US gene-editing companies, some of which have raised millions in venture capital.

“I would rank the US and China as first and second Crispr-Cas9 research countries, respectively, at this time. Both countries have much strength in this area,” said Paul Knoepfler, an associate professor of cell biology and human anatomy at the UC Davis School of Medicine in California.

“The US currently gets the edge in high-profile papers, Crispr biotech and intellectual property. China has published a lot in Crispr animals.”

The Boston Consulting Group estimated in September that US gene editing companies had attracted more than US$1 billion in investment since 2013. Editas Medicine, Intellia Therapeutics and Poseida Therapeutics are among the US biotech companies researching Crispr to tackle health disorders.

Crispr has yet to be proven effective in creating new treatments, but pharmaceutical companies worldwide see it as a potent tool. The hope is that it could potentially generate therapies for conditions from cancer to blood diseases.

To use that technology to make your child run faster or jump higher is uniformly frowned upon. The technology to do that, however, will soon be in place
Eric Hendrickson, gene editing expert

A Chinese team at Sun Yat-sen University in Guangzhou last year became the first to report Crispr work in human embryos, attempting to edit a gene that causes the blood disorder, thalassemia. The study was at least partly funded by two government-run organisations, the National Natural Science Foundation of China and another called the National Basic Research Programme.

The research caused an international stir, even though the scientists only used “non-viable” human embryos, or those obtained through fertility clinics and without any potential for live births. Professor Huang Junjiu, who led the study, declined to comment.

The Chinese researchers said in their findings that Crispr needs to be better understood and more accurate before being used in human testing.

While there is still little private investment in gene editing in China, a publicly traded Chinese company called Shenzhen Jinjia Color Printing Group said in a December statement that it would provide 3 million yuan (HK$3.6 million) in funding to the university. The company, which prints cigarette boxes, has identified the health industry as a key area of growth and hopes to eventually share the rights to the Crispr-based thalassemia treatment technology, it said via e-mail.

The US federal government doesn’t fund work that destroys or creates human embryos for research purposes. The UK’s Human Fertilisation and Embryology Authority gave a group of researchers permission this month to conduct Crispr research on human embryos, although they still need an ethics committee to approve their plans.

Using Crispr to cure disease “is probably ethical”, said Eric Hendrickson, a professor at the University of Minnesota Medical School, whose research uses Crispr techniques for DNA repair. “To use that technology to make your child run faster or jump higher is uniformly frowned upon. The technology to do that, however, will soon be in place.”

Most of China’s funding for Crispr research is coming from the government with very few private companies putting money into gene modification work, said Lai Liangxue, deputy director at the Southern China Institute of Stem Cell Biology and Regenerative Medicine. “Whether it’s animal or plant, our country has special funds for this aspect of work.”

READ MORE: Gene editing used by Chinese researchers on human DNA is Science magazine’s breakthrough of 2015

The National Natural Science Foundation of China, a prominent government backed institution that funds research, awarded more than 23 million yuan last year to at least 42 Crispr projects, more than double the previous year.

It is just one of several government institutions providing Crispr funding in China. China is also aided by a large pool of internationally trained scientists, many of whom have returned home after working overseas. The Chinese Ministry of Science and Technology didn’t respond to a fax seeking comment.

Backed partly by grants from the Ministry of Science and Technology, Lai, the researcher in southern China, has focused on biomedical applications of Crispr. He has given pigs genes that allow them to contract human diseases and serve as trial subjects for new treatments, or even act as potential sources of organs for human transplant.

I would rank the US and China as first and second ... research countries, respectively, at this time
Paul Knoepfler, associate professor of cell biology

Lai’s team also snipped a gene that inhibits muscle growth in beagles, enabling the modified dogs to have stronger muscles, run faster and jump higher than normal ones. The same technology could potentially benefit the police and military in the future if applied to canine breeds commonly used by law enforcement agencies, he said.

Part of the challenge for Chinese companies will be getting licenses to use Crispr commercially once there are patents awarded internationally. Several academic institutions in the US and elsewhere have already filed patent applications for Crispr-Cas9 technology. “Without obtaining licenses from these parties, commercial applications in China or elsewhere will be hampered,” Jin-Soo Kim, a professor with the Centre for Genome Engineering at Seoul National University.

Crispr has already boosted a new industry in China that supplies genetically edited animals to foreign research labs and pharmaceutical companies. Researchers in the US and China also see its potential in agriculture to potentially create disease resistant grains or better quality meat.

Raising a genetically engineered pig with Crispr technology is already cheaper in China at about 700,000 yuan, while in the US it could cost four or five times as much, said Lai. Labour and other costs are lower in China.

Still, Crispr-edited animals and crops in China so far remain off the market, he said, as their safety needs to be studied. In agriculture, too, Chinese scientists or their commercial partners will need to negotiate with the holders of the related patents and probably pay a fee to use them.

READ MORE: Britain approves controversial gene-editing technique after Chinese scientists condemned for modifying human embryos

The Iowa-based seed company DuPont Pioneer entered into a licensing pact with Lithuania’s Vilnius University in June, one of the first groups to discover the gene-editing functions of the Cas9 protein.

“If in the future we think they don’t cause any harm to the human body and can indeed increase output, they will be very useful to the development of our country’s agriculture sector,” said Lai, referring to edited animals and crops.