To start the ball rolling, Bayer will make an upfront payment of $40 million to Mammoth, thereby getting a first glimpse into the biotech’s genome editing toolbox, which grew out of the work of CRISPR pioneer and Nobel laureate Jennifer Doudna. Potential future milestone payments could exceed $1 billion based on achievement of certain research, development and commercial milestones across five preselected in vivo indications. The initial focus will be on liver-targeted diseases.

Bayer will additionally pay research funding and tiered royalties up to a low double-digit percentage of net sales. “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, president of the Leverkusen-based group’s pharmaceuticals division.

Oelrich said the US partner’s “groundbreaking” gene technology is a key enabling tool as well as a stand-alone therapeutic modality. The cooperation, he said, will “significantly enhance Bayer’s own efforts to develop transformative therapies for patients faster and strengthen its recently established new cell and gene therapy platform.”

Mammoth is concentrating its efforts on novel CRISPR systems that have different Cas proteins as genetic scissors, including Cas14 and CasΦ systems. The goal, the company says, is to design smaller and more precise gene editing tools to have better editing efficiency and safety compared with the first-generation Cas9 system.

Peter Nell, the company’s chief business officer and head of therapeutic strategy, told US trade journal Fierce Biotech that the biotech’s CRISPR expertise means it will be tasked with finding the best system and the best design for Bayer’s projects during the research phase.

Mammoth is also working on in vivo gene editing therapies for two unidentified genetic diseases with Boston-headquartered Vertex Pharmaceuticals. In October 2020, Vertex agreed to pay Mammoth $41 million upfront and up to $650 million in milestone payments to establish the collaboration.

The deal with Mammoth will be Bayer’s third major investment in gene therapy. In an October 2020 deal potentially worth $4 billion, it bought Asklepios BioPharmaceutical (AskBio), based in the US state of North Carolina’s Research Triangle Park, and gained access to the biotech’s gene therapy platform and its CDMO base.

In 2019, Bayer paid $240 million upfront for the remaining stake in its cell therapy joint venture, BlueRock Therapeutics, thereby picking up an induced iPSC pipeline led by a late-preclinical Parkinson’s disease candidate.

Author: Dede Williams, Freelance Journalist