The drama surrounding CRISPR patents unfolded like a telenovela for the scientific community. The intellectual property linked to CRISPR was so impressive that the novel gene-editing tool led to a high stakes patent battle that is still being sorted out. But that has not stopped startups, biotechs and pharmaceutical companies from applying the tool.
CRISPR works like the keyboard shortcut CTRL F, followed by the delete key: By coupling a protein capable of cutting through DNA – our genetic blueprint – with an RNA molecule that hones in on genes, bits can be sliced away as needed to treat disease. This technology means genes, the biological essence that makes each living plant or animal unique, are no longer predetermined. They are malleable and controllable – like putty in human hands.
Researchers are excited about the potential applications: Deleting genes that cause chronic diseases, growing human organs in pigs, curing blindness and maybe, one day, creating geneticially modified humans.
During the mad rush to file patents after the research was published in 2012, a co-discoverer and a researcher close to the CRISPR technology, Emmanuelle Charpentier and Rodger Novak, took their know-how and founded Crispr Therapeutics. The Swiss startup is high profile and was among the four biggest IPOs related to CRISPR research, with a capital increase of $56 million (€47 million). The others in that group — Editas Medicine, Intellia Therapeutics and Juno Therapeutics — are all based in the United States.
But tough international competition has not kept Germany on the sidelines. German pharmaceutical company Bayer jumped on board in 2015, backing Crispr Therapeutics with $335 million over a five-year period. Bayer acquired a $35 million stake in the company. And in 2016, Germany’s Merck and Co. took its CRISPR–related work to Hamburg-based Evotec, known for their disease screening abilities. In both cases, drug discovery is the end goal.
And earlier this month, CureVac, a Tübigen-based biotech working with messenger RNA (mRNA) technology, another recent scientific breakthrough, teamed up with Crispr Therapeutics and Bayer to see how mRNA and CRISPR can work together. CureVac is the German poster-child of the biotech industry, having been backed in 2015 with $52 million for a 4-percent stake by the Bill and Melinda Gates foundation.
Franz Werner Haas, CureVac’s chief corporate officer, does not know of any collaborations of this kind in Germany. “CRISPR takes advantage of what mRNA can do and how gene editing can be controlled,” Mr. Haas said. “We are going to revolutionize medicine by making the body produce its own drugs,” he continued. The research focus is rare liver diseases, and the end goal is to move to human trials. The timeline, however, is confidential.
This partnership might help boost Germany’s CRISPR reputation, as the country has yet to use the technology in clinical trials. The first human trials using CRISPR started last year in China and the US. And in early August, a team of US scientists published a study in the journal Nature on how they successfully removed a gene that codes for a debilitating heart condition in human embryos.
CRISPR technology, as one can imagine, raises a number of moral and ethical questions. Should pigs be genetically modified to create a never-ending supply of organs that are then harvested for human use? Are researchers obligated to sterilize mosquitoes – ensuring their demise as a species – in order to stop the spread of malaria? Can parents have their child’s genetic code altered, so that the child’s muscles one day grow as big as Arnold Schwarzenegger’s?
As troubling as these thoughts are, the potential benefits are extraordinary. “Now is the time for lots of clinical studies,” said Reinhard Dengler, doctor and vice-chairman of the German Society for Musculoskeletal Diseases. In a few years, people confined to wheelchairs because of a disease like Duchenne muscular dystrophy could be given an injection that deploys billions of CRISPR “agents” to search and destroy the genes that keep muscle cells defective. Furthermore, this same principle could be applied to cut out HIV from human cells, effectively curing the condition, or at least that’s what the founders of the US company Excision BioTherapeutics hope.
In China, researchers are developing a CRISPR gel that stops uterine cancer caused by the commonly-spread sexually transmitted infection HPV, which affects 79 million Americans, according to the Centers for Disease Control and Prevention. Researchers at Editas Medicine in the US are looking at the application of CRISPR for treating Sickle Cell Anemia, and Juno Therapeutics, another US company, is trying to modify the body’s T-cells, the cells that act as an army protecting the body from outside invaders, to target different types of cancer. The possibilities seem endless.
However, Ms. Charpentier, the co-founder of Crispr Therapeutics, thinks using the technology to try to control human evolution or create designer babies is wrong. “We should not touch the human germline,” she said, meaning humans’ genetic material should be passed unaltered from generation to generation. Any genetic changes should be done solely for the purpose of treating existing diseases.
This ethical line in the sand on how to use gene editing also works out well for pharmaceutical companies and biotech firms. They can treat, even cure, individual patients, but not eradicate the conditions themselves – a perfect business model.
This article was originally published in Handelsblatt’s sister publication Wirtschafts Woche. Christine Coester edited this article for Handelsblatt Global. To contact the author: email@example.com