Mammoth Biosciences: Hope for Gene Therapy: Bayer agrees alliance with company of Nobel Prize winner in chemistry

Jennifer Doudna

The winner of the Nobel Prize in Chemistry is one of the founders of Mammoth Biosciences.

(Foto: Getty Images)

Frankfurt Bayer is stepping up its involvement in the field of cell and gene therapies with another biotech alliance, potentially worth billions. Together with the US company Mammoth Biosciences, the Leverkusen-based company wants to develop so-called “in vivo gene editing therapies”, as Bayer announced on Monday.

In this process, the Crispr / Cas gene scissors are to be used as an instrument for novel gene therapies, particularly in the case of liver diseases. “In vivo” means that chromosomes are changed in the patient’s body.

Mammoth Biosciences, which specializes in technologies in the Crispr / Cas area, will receive $ 40 million in advance from Bayer as part of the cooperation and is also entitled to success-based payments of up to a billion dollars.

The Crispr / Cas technology enables gene segments to be cut out or exchanged relatively precisely. Mammoth was founded in 2018 and, among other things, specializes in developing particularly small Cas enzymes. The co-founder is the molecular biologist Jennifer Doudna, who together with Emmanuelle Charpentier received the Nobel Prize in Chemistry in 2020 for the discovery of Crispr / Cas.

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For Bayer, the new 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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“By combining the novel Crispr systems from Mammoth Biosciences with our new platforms for gene augmentation and induced pluripotent stem cells (iPSC), we can exploit the full technological potential of our cell and gene therapy strategy,” said the head of Bayer’s pharmaceuticals division , Stefan Oelrich, on Monday about the new alliance.

Bayer first became involved in the Crispr / Cas area in 2016 through an alliance with the company Crispr Therapeutics founded by Emmanuelle Charpentier. However, Bayer later withdrew from the Casebia joint venture founded at the time with Crispr.

The technology developed by Mammoth is characterized by the fact that its Cas enzymes are particularly small. This in turn should facilitate the transport with the help of special vehicles, so-called adeno-associated viruses (AAV), which are usually used in gene therapies. Because the absorption capacity of these viruses is limited. In Crispr / Cas-based gene therapies, the basic concept is to equip the viruses with the genetic code for the Crispr enzymes, which are then formed in the infected cells.

Gene therapies are characterized by the fact that defective genes are switched off or replaced with new ones. Genetically modified viruses are usually used as transfer vehicles – so-called vectors. The concept differs fundamentally from common active pharmaceutical ingredients that intervene in the metabolism at the protein or enzyme level.

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