Mammoth Biosciences' 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 agreement, the two companies will start their collaboration with a focus on liver-targeted diseases. By addressing the root cause of diseases, it is possible to permanently reverse 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 ultrasmall Cas enzymes, including Cas14 and Casɸ, allows for expanded high-fidelity gene editing to be combined with targeted systemic delivery. With Bayer's new access to this novel gene editing technology, the companies have the potential to advance in vivo applicability with the ultracompact size of these novel CRISPR systems.

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 of more than $1 billion upon successful achievement of certain research, development, and commercial milestones across five preselected in vivo indications, with an initial focus on liver-targeted diseases. In addition, Bayer will pay research funding and tiered royalties up to a low double-digit percentage of net sales. The companies are also exploring work on ex vivo projects on a nonexclusive basis.

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