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Is Genome-editing CRISPR Ready to Leave the Lab?

The 2017 Harvard China Forum hosted a panel on “Futuristic Technologies & Innovations in AI and Biotech”, where Feng Zhang was invited to share insights on genome-editing.



The 2017 Harvard China Forum hosted a panel on “Futuristic Technologies & Innovations in AI and Biotech” where Feng Zhang (second from the right) was invited to share insights on genome-editing.

If you are not familiar with CRISPR, you might be missing one of the most exciting breakthroughs in genome-editing since 2012. The genome-editing toolkit CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) allows the permanent modification of DNA, theoretically opening up the potential for lifespan extension and instant curing of diseases.

The strong potential of CRISPR is creating a boom in the genome-editing industry. According to a study from Research and Markets, the genome-editing market is expected to reach $5.54 billion by 2021, doubling its current size.

The question right now is whether this four-year-old genome-editing technique is mature enough for application. The pioneer of CRISPR, MIT’s Feng Zhang, recently shared his insights, and it looks like — despite the media hype — the frontier technology still has a long way to go.

Feng Zhang joined a panel at the 2017 Harvard China Forum, North America’s leading and 20-year-running student-organized conference on China. The 34-year-old molecular biologist at the Broad Institute of MIT and Harvard introduced three decision points to evaluate the technology – scalability, non-tech factors like regulatory processes, and resources like capital and collaborations required to test the tech.

Zhang explained that further developmental resources and human capital are still required, “and at that point we can take the technology into a commercial setting.”

So, where is CRISPR right now?

The young technique made its debut on disease treatment when a similar editing technique called TALENS developed a new solution for leukemia treatment in 2015. A one-year-old girl named Layla received cancer-fighting cells (T-cells) from a healthy donor. The cells were successfully modified by genome-editing techniques, and were injected into the little girl’s body to replace her fragile immune system.

One year later, Chinese scientists became the first to adopt the CRISPR technique to edit a set of immune cells, which were delivered into a patient suffering from lung cancer. According to the leading oncologist Lu You at Sichuan University in Chengdu, the procedure went smoothly, and the patient was monitored over the next six months. Meanwhile, the United States accelerated the introduction of clinical trials on CRISPR gene-editing by approving a proposal to adopt CRISPR in cancer immunotherapy, a kind of treatment that stimulates the immune response.

However, the application of technique is undoubtedly still in its early days. It is still unknown whether the technique will cause any adverse effects. Some experts also doubt whether it would show superior efficacy compared to existing cancer-fighting techniques like antibodies.

“Academic research in the laboratory funded by research grants is where you can take the time and risks to explore things that are challenging and uncertain,” said Zhang. “But it is hard to get commercial funding in this case because the capabilities of and return on those early ideas are hard to predict.”

While scientists are pushing forward on CRISPR testing, this has been fundamentally restrained by regulatory processes. At present, using CRISPR or any other genome-editing technique is legal for research purposes in the US, but this does not mean the government will always give the green light to involved scientists. The government is still looking at whether CRISPR will bring real changes. And it is worth noting that any possible safety issues emerging in clinical trials may negatively impact ongoing CRISPR research efforts.

Capital is always welcoming new techniques. Even before the genome–editing technique has made any promise to the market, venture capital has already substantially helped cultivate genome-editing companies to become the next medical Google or Facebook. Last year, funding to genome-editing companies surpassed $1 billion, a milestone for the industry, according to a CB insights report. Since its founding, CRISPR Therapeutics, a biopharmaceutical company focusing on translating CRISPR/Cas9 gene-editing technology into medicines for serious diseases, for example, has raised a total of $218 million, including $56 million through its IPO.

So back to the question: Where is CRISPR now? Unfortunately, although the market and media are enthusiastic, the technique remains some time away from any commercial use.


Author: Tony Peng, Synced Tech Journalist  | EditorMichael Sarazen

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