CRISPR in China
On 19 June 2017, Intellia Therapeutics (co-founder: Jennifer Doudna) and CRISPR Therapeutics (co‑founder: Emmanuelle Charpentier) announced that China’s State Intellectual Property Office (SIPO) has indicated that it will grant each of the University of California, Berkeley/University of Vienna a patent broadly covering CRISPR-Cas single-guide gene editing methods and compositions.
The patents include claims covering methods for editing DNA in non-cellular and cellular settings, including in eukaryotic cells such as human and mammalian cells. They also include CRISPR-Cas composition of matter and system claims for use in any setting, including claims covering the use of CRISPR-Cas in producing medicines for treating disease. Now, the SIPO says Charpentier’s company CRISPR Therapeutics and Doudna’s two CRISPR start-ups, Intellia Therapeutics and Caribou Biosciences, have the rights to apply licensing of their CRISPR-Cas technology in the country.
The EPO and the UKIPO have previously issued patents derived from the underlying international patent application. These applications are based on the same U.S. priority application that was involved in the interference proceeding at the USPTO with the Broad et al.
Clinical trials in China
China’s decision to award the patents comes as three Chinese clinical trials using CRISPR technology continue to recruit patients (several more have been announced but are not yet in recruitment phase), of which two have already started administering the experimental treatments. The trial led by researchers from Sichuan University’s West China Hospital in Chengdu, which began in October 2016, was the first CRISPR trial in the world to commence and involves the use of CRISPR on humans to modify genes in Chinese lung cancer patients. The second trial is being run by Nanjing University’s Nanjing Drum Tower Hospital last April, in which genes were removed, modified, then re-injected into 20 patients with late-stage nasopharyngeal carcinoma, gastric cancer and lymphoma. Notably, it seems one of the trials announced proposes to use CRISPR to achieve in vivo gene editing – i.e. using CRISPR to edit genes directly in a live patient’s body, rather than extracting cells and modifying them outside the body for reintroduction into the body (ex vivo editing).
The high level of CRISPR patenting activity and the rapid proliferation and progress of clinical trials in China are testament to the continuing rise of China as a real force in biotech. Legend Biotech Co recently caused waves as ASCO 2017, emerging as a dark horse to challenge established Western powers like Novartis, Kite and Juno by presenting impressive results from its phase I CAR-T trial for treating multiple myeloma (which achieved clinical remission in 33 of 35 patients and complete remission in 14 of 19 patients monitored for over four months). Genomics giant BGI similarly made a splash in 2015 with its creation of gene edited "micropigs" generated using older TALENs technology to knockout a copy of the growth hormone receptor gene in fetal cells, and in 2016 signalled its intent to use CRISPR to alter the size, colour and patterns of koi carp.
The different regulatory environment and strong government backing for CRISPR and other gene editing projects in China may have allowed Chinese companies and researchers to get a head start on their counterparts in the West, with the first CRISPR edited monkeys in 2014 and first CRISPR edited human embryos in 2015. However, with the first CRISPR editing of a human embryo in the US reportedly accomplished and soon to be published (and allegedly more successful at reducing off-target effects and mosaicism than the previous attempts in China), there are signs the rest of the world may be catching up. However, what is clear is that, in this modern age of biotechnology, China must not be ignored; and for a technology with the power of CRISPR (and inherent risks that entails) it there is urgent need for transparent and open discussion, promotion of awareness and, where possible, harmonisation of a regulatory regime in which society can ethically pursue the huge potential for CRISPR and other gene editing technologies.