By Alex Keown – Jan 10, 2022 – biospace.com (link to article)

Bayer and Mammoth Biosciences forged a strategic collaboration potentially valued at more than $1 billion. The partnership grants the Germany-based pharma giant access to Mammoth’s CRISPR technology to develop in vivo gene-editing therapies.

The companies plan to harness the power of the CRISPR technology across five preselected in vivo indications. While the companies did not disclose all of the indications they intend to target, Bayer said their first focus is liver-targeted diseases.

Bayer described privately-held Mammoth’s CRISPR program as a “key enabling technology” expected to enhance its efforts to develop new therapeutic approaches and bolster its recently-established cell and gene therapy capabilities. Bayer has quickly built a solid cell and gene therapy platform due to the acquisition of BlueRock in 2019 and AskBio in 2020.

In 2020, following those acquisitions, Bayer announced its cell and gene therapy strategy that aims to maximize the capabilities of its acquisitions. When the company announced the strategy, Bayer said the role of its Cell and Gene Therapy Platform would serve as a strategic guide to ensure complementary support and progress. Bayer is already harnessing its gene and cell therapy programs to tackle diseases like Parkinson’s. Bayer’s gene therapy subsidiaries are each taking different approaches toward the condition.

In its announcement this morning, Bayer said that cell and gene therapies are the next step in the evolution of drug development. The company could address the root cause of diseases and potentially reverse the disease state with a one-time treatment through cell and gene therapy approaches.

“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,” Stefan Oelrich, president of the Bayer Pharmaceuticals Division said in a statement. “Partnering with Mammoth’s cutting edge scientific team is a fundamental pillar for our company to improve the lives of patients suffering from conditions that are currently still difficult to treat.”

With the collaboration, Bayer will harness Mammoth’s ultra-small Cas enzymes, including Cas14 and Casɸ, which are expected to provide expanded high-fidelity gene-editing combined with targeted systemic delivery. Through an in vivo approach, Bayer said the company could use the technology to target a wide range of genetic diseases associated with high unmet medical needs.

Peter Nell, Ph.D., chief business officer and head of Therapeutic Strategy at Mammoth, said the company is excited to work with Bayer on this project. Nell said the partnership would build on Mammoth’s “technology leap” with its CRISPR system, combined with Bayer’s expertise in successful drug development.

“This joint effort has the potential to benefit patients by developing CRISPR-based approaches for the clinic with the appropriate urgency while ensuring scientific excellence and safety,” Nell said in a statement.

Under terms of the deal, Bayer will make an upfront payment of $40 million to California-based Mammoth Biosciences. When potential milestone and commercial payments are factored into the deal, Mammoth could rake in more than $1 billion. The agreement includes Bayer funding the research. Additionally, the two companies intend to explore ex vivo projects on what was described as a “nonexclusive basis.”