Frankfurt, Düsseldorf In the search for new opportunities in the fight against diseases, this technology is considered to be one of the greatest hopes: By editing genes, certain sections of the human genome are to be switched off or renewed. This targeted change in DNA could become the key to new therapies – and is therefore attracting the large pharmaceutical companies.
This was shown twice on Monday. Bayer AG and the US pharmaceutical company Pfizer have each announced partnerships with smaller biotech companies, which at best could reach a volume in the billions. Both are about gene therapy – albeit with technological differences.
Bayer has long made cell and gene therapy the core of its ambitious growth plans in the pharmaceutical sector and is expanding this with the new, potentially multi-billion dollar biotech alliance. Together with the US company Mammoth Biosciences, the Leverkusen company wants to develop so-called “in vivo gene editing therapies”.
In this process, the Crispr / Cas gene scissors are to be used as an instrument for innovative therapies, particularly in the case of liver diseases. The technology enables gene segments to be cut out or exchanged relatively precisely. With this, the researchers want to directly address the root cause of a disease.
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This offers the chance to achieve therapeutic success with just a single treatment instead of months of treatment with drugs. The concept differs fundamentally from common active pharmaceutical ingredients that intervene in the metabolism at the protein or enzyme level.
The targeted gene changes can take place outside the body (“ex vivo”), but also directly in the body. From Bayer’s point of view, “in vivo” is particularly promising for hereditary diseases.
The agreement gives Bayer access to Mammoth’s advanced gene editing technology. The company was only founded in 2018 and was co-founded by molecular biologist Jennifer Doudna. In 2020, she and Emmanuelle Charpentier received the Nobel Prize in Chemistry for the discovery of Crispr / Cas.
The winner of the Nobel Prize in Chemistry is one of the founders of Mammoth Biosciences.
(Photo: Getty Images)
As part of the cooperation, Mammoth will receive $ 40 million in advance from Bayer and is also entitled to success-based payments of up to a billion dollars.
For Bayer, the alliance is another important step in the expansion of gene and cell therapy research. The group significantly expanded its position in this field in 2020 with the takeover of the US company Ask Bio for up to four billion dollars. The group is also represented in the field of cell therapies through the company Blue Rock Therapeutics, which was acquired in 2019.
The two companies and other research activities in the field were combined in a separate platform for cell and gene therapy within Bayer’s pharmaceuticals division last year. The declared goal is to expand the “increasingly leading position” in the field. Several Bayer product candidates in this area are already being clinically tested.
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Now the group wants to use the combination with the Crispr system from Mammoth to “exploit the full technological potential of our cell and gene therapy strategy,” said the head of Bayer’s pharmaceuticals division, Stefan Oelrich, about the new alliance.
Bayer first got involved in gene editing in 2016 through an alliance with Crispr Therapeutics, a company founded by Emmanuelle Charpentier. However, Bayer later withdrew from the Casebia joint venture founded at the time.
Crispr / Cas-based gene therapies are based on a novel concept: In the laboratory, viruses are equipped with genetic codes for enzymes that are necessary to intervene in the DNA. The viruses bring the codes into human cells as transport vehicles, where the enzymes are then formed.
The technology developed by Mammoth is characterized by the fact that its Cas enzymes are particularly small. This, in turn, should make it easier to transport with the help of the viruses, as their absorption capacity is limited.
The US pharmaceutical company Pfizer also relies on the targeted modification of DNA through gene editing. On Monday, the Americans announced an alliance with the biotech company Beam Therapeutics, which is based in Cambridge, Massachusetts – the heart of American biotech research.
Pfizer expands mRNA expertise
Pfizer is initially providing the young company with $ 300 million. Depending on the achievement of certain milestones, the alliance can also reach a volume of more than one billion dollars. The aim is to find new approaches to combating rare diseases. As part of the deal, Pfizer can further develop three drug candidates for diseases of the liver, muscles and the central nervous system.
Beam uses a modified form of the common Crispr-Cas system. Only individual bases in the DNA strand are exchanged in a very targeted manner. From the biotech company’s point of view, this technology promises fewer side effects than when using conventional gene editing.
The Americans also use the messenger substance mRNA to transport the code in the body. It has become world famous thanks to the success of the Covid 19 vaccines. In the vaccine from Pfizer and Biontech, mRNA smuggles the blueprint for parts of the coronavirus into human cells. This is where the parts of the virus are produced, which triggers the body’s immune response.
With the Beam Alliance, Pfizer is expanding its research network in the highly promising mRNA technology. In the future, it will also be used, for example, in the fight against cancer.
More: In the shadow of the pandemic: How the oncology leader gives hope to millions of cancer patients.