GERMANY – Bayer and Mammoth Biosciences have announced an agreement to use the latter’s CRISPR systems to develop in vivo gene-editing therapies, with an initial focus on liver-targeted diseases.
Bayer will make an upfront payment of US$40 million as part of the agreement, with Mammoth eligible for target option exercise fees as well as potential future milestone payments of more than US$1 billion across five preselected in vivo indications.
The collaboration further strengthens Bayer’s push into cell and gene therapies following the launch of a dedicated unit in 2020.
The collaboration with Mammoth, according to Bayer, will “significantly enhance” its efforts to develop transformative therapies for patients more quickly.
“Bringing together Mammoth’s novel CRISPR systems with our existing gene augmentation and our induced pluripotent stem cell (iPSC) platforms will allow us to unleash the full potential of our cell and gene therapy strategy,” remarked Stefan Oelrich, president of Bayer’s pharmaceuticals division.
Bayer entered the cell therapy market with the acquisition of BlueRock Therapeutics in 2019, and has since expanded its position with the acquisition of Asklepios Biopharmaceutical and a licensing agreement with Atara Biotherapeutics to develop anti-tumour immune cell therapies.
The German pharmaceutical company will gain access to Mammoth’s toolkit of ultra-small Cas enzymes, including Cas14 and CasPhi, as part of the agreement, allowing for expanded high-fidelity gene editing to be combined with targeted systemic delivery.
Furthermore, Bayer will provide research funding as well as tiered royalties up to a low double-digit percentage of net sales.
The drugmakers also stated that they are looking into non-exclusive work on ex vivo projects.
Mammoth Biosciences’ gene-editing technology, in particular, is expected to accelerate Bayer’s efforts to develop transformative therapies for patients and to strengthen the company’s recently established new cell and gene therapy platform, according to Bayer executives.
Prior to the Bayer agreement, Mammoth signed its first similar agreement with Vertex in October, focusing on in vivo gene editing therapies for two unidentified genetic diseases. The biotech also has a diagnostic division that is developing COVID-19 tests.
Bayer, for its part, made a big bet on gene therapy in 2020 with the potential US$4 billion AskBio acquisition.
Bayer has also acquired three clinical-stage assets from the adeno-associated virus-based gene therapy specialist, targeting Pompe disease, congestive heart failure, and Parkinson’s disease.