Re-scissors are entering the medical mainstream

So-called gene cuts have become famous and infamous around discussions of designer babies and super soldiers. But many consider Crispr/Cas9 to be one of the technologies that can revolutionize medicine. About ten years after the scientific breakthrough – by the way, much of it was in Vienna – the Crispr technology is starting to make the leap into everyday medical life.

Because the American biotech Vertex and the Swiss biotech company Crispr Therapeutics will submit the first product based on the technology to the European Medicines Agency EMA. In addition, the two companies expect the submission to the US agency FDA, which is also responsible for medicine in addition to food, to be completed in the first quarter of 2023. If both authorities give the green light, it will be the commercial breakthrough for the first Crispr application.

Crispr is the way DNA strands can be precisely cut to replace part of the genome – and has made genetic engineering easy and cheap. Vertex and Crispr Therapeutics now want to use a gene therapy called “Exa-cel” against the hereditary diseases sickle cell anemia and beta-thalassemia. “We continue to work diligently to bring the first Crispr therapy to market for a genetic disease,” Vertex executive vice president Nia Tatsis said in a statement. The collaboration with Crispr Therapeutics from Switzerland has existed since 2015. The two companies share the development costs and profits worldwide in the ratio 60:40.

The CRISPR co-inventor in Vienna: “We are not far from Jurassic Park”

Patent disputes and mistakes along the way

Crispr/Cas9 was mainly developed by Emmanuelle Charpentier and Jennifer Doudna, they have now received the Nobel Prize in Chemistry for it. But there is a dispute over who “invented” the technology and who ultimately gets to use it. According to estimates, more than 11,000 patent families have now been registered for the technology. The main line of conflict runs between a group of universities (University of California, Berkeley, University of Vienna) Charpentier on the one hand and the Harvard-MIT Broad Institute on the other. But the Nobel Prize for Charpentier and Doudna does not mean a legal right to patent revenues estimated at up to ten billion dollars, as decided by the US Patent Office in 2022 (more on this here).

More and more success is being achieved with Crispr. As reported, an American research team gave sight to a patient using the technology. Australian researchers also showed that Crispr can be used to prevent viruses from multiplying in infected cells – including the SARS-CoV-2 pandemic virus. However, many Crispr treatments are still in the testing stage. The “Brilliance” study, well known in the medical world, was stopped for the time being because the first application of a CRISPR/Cas9-based therapy in the human body did not quite produce the hoped-for results – only 3 out of 14 patients experienced a relevant improvement in the sight. To get from “proof of concept” to further development, you now need a financially strong business partner.

If the gene therapy from Vertex and Crispr Therapeutics is approved in the US and EU, it will send a signal to many other Crispr-based gene therapies. In 2022, the market is expected to grow to 1.327 billion dollars, which is an increase of about 25 percent compared to 2021. Although there are still great reservations about genetic engineering in many regions of the world (and thus also against relatively new genetic engineering such as . such as Crispr), more and more innovation is taking place. In Switzerland, for example, the Federal Council intends to present a proposal for “risk-based approval” for plants that have been modified using CRISPR/Cas genetic scissors by 2024. China and India also want to simplify the approval of genome-edited plants . In the EU, genetic scissors remain subject to strict genetic engineering laws.

CRISPR: According to the European Court of Justice, “genetic scissors” fall under the strict laws on genetic engineering

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