Bayer, Mammoth announce novel gene editing technology partnership

German pharma and crop sciences major Bayer AG on Monday announced a strategic partnership with privately held Mammoth Biosciences to develop therapeutic tools based on CRISPR/CAS9 gene editing as part of its move to widen its cell and gene therapy development efforts.

The strategic collaboration agreement includes an option for the use of Mammoth’s CRISPR systems to develop in vivo gene-editing therapies, which supports Mammoth’s vision of unlocking the full potential of novel CRISPR systems, Bayer stated in a joint release.
Mammoth Biosciences’ groundbreaking gene-editing technology is a key enabling technology, as well as a stand-alone therapeutic modality. It will significantly enhance Bayer’s efforts to develop transformative therapies for patients faster and strengthen the company’s recently established new cell and gene therapy platform.
Under the terms of the agreement the two companies will start their collaboration with a focus on liver-targeted diseases.
“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,” said Stefan Oelrich, member of the board of management, Bayer AG and president of the Bayer’s Pharmaceuticals Division. 
“We’re excited to be working together with Bayer, building on the technology leap of our novel CRISPR systems, along with Bayer’s expertise in successful drug development,” said Dr Peter Nell, chief business officer and head of therapeutic Strategy at Mammoth. 
Cell and gene therapies are the next step in the evolution of drug development. By addressing the root cause of diseases, they are potentially capable of permanently reversing diseases with a one-time treatment. Gene editing serves as a key enabler for cell therapies when used outside the living body (ex vivo) and allows therapeutic targeting of a wide range of genetic diseases with a high unmet medical need when used inside the living body (in vivo).
Mammoth Biosciences’ proprietary toolkit of ultra-small Cas enzymes, including Cas14 and Cas?, allows for expanded high-fidelity gene editing to be combined with targeted systemic delivery. Under the agreement, Bayer gains access to this novel gene-editing technology, which offers the potential of an advanced in vivo applicability due to the ultra-compact size of these novel CRISPR systems.
Under the terms of the agreement, Mammoth Biosciences will receive an upfront payment of $40 million and is eligible to receive target option exercise fees as well as potential future payments in the magnitude of more than $1 billion upon successful achievement of certain research, development, and commercial milestones across five preselected in vivo indications with a first focus on liver-targeted diseases. In addition, Bayer will pay research funding and tiered royalties up to low double-digit percentage of net sales. The companies are also exploring work on ex vivo projects on a nonexclusive basis.