The next wave of genomic medicine is taking shape, including gene-editing therapies that work in patients, and Pfizer and Bayer have each formed new alliances that will expand their reach to include these in vivo approaches.
Pfizer announced a new partnership Partnership with Cambridge, Massachusetts-based biotech company Beam Therapeutics to develop therapies based on its base editing technology. To start the alliance, Pfizer made an upfront payment of $300 million to its new partner. Beam will process all studies up to candidate selection. The agreement is for four years and can be extended for another year.
Beam’s base-editing technology enables companies to change individual letters in the genetic code. The biotech’s approach uses messenger RNA and lipid nanoparticles to deliver their base editors to target organs. Edit without damaging double-stranded DNA. Base editing enables more precise editing than other methods, such as using CRISPR Cas9, which can cause double-strand breaks that increase the risk of unwanted changes to the genetic code.
Pfizer and Beam did not specify disease targets, other than to say that the alliance covers rare genetic diseases of the liver, muscle and central nervous system. Programs currently under development by Beam are not included in the new agreement. If Pfizer exercises its option to license drug candidates developed under the alliance, it will be responsible for further development efforts, including passing regulatory review and commercializing the therapies.
Beam could earn up to $1.35 billion in milestone payments if Pfizer exercises options on all three targets covered by the partnership. The biotech will also receive royalties from Pfizer’s sale of any commercialized products produced by the alliance. Beam does have the opportunity to share in the development and potential commercialization of the collaborative therapeutic candidate. The agreement grants the biotech the option to enter into a global co-development and co-commercialization agreement for one of the programs at the conclusion of the Phase 1/2 clinical trial. Beam must pay an option exercise fee; development, commercialization costs and profits are split between 65% for Pfizer and 35% for Beam.
“We have a long history of developing gene replacement therapies for rare diseases, and we see our collaboration with Beam as an opportunity to advance the next generation of gene editing therapies — an exciting frontier in science — that could lead to patients with rare genes The shift, Mikael Dolsten, Pfizer’s chief scientific officer and president of global research, development and medical, said in the deal announcement.
Pfizer joins other companies that have signed deals R&D alliance with Beam, including Apellis Pharmaceuticals and Shen Yun therapy.
At the same time, Bayer hopes to protocol In partnership with Mammoth Biosciences. Brisbane, California-based Mammoth originally developed CRISPR Cas9-based diagnostics, and this work remains part of its pipeline. But the company continues to add new CRISPR cutting enzymes. The company said the Cas14 and CasPhi enzymes allow it to explore therapeutic applications. CEO Trevor Martin told MedCity News last year.
Bayer announced Monday that it is paying $40 million upfront to begin a collaboration that will enable the German company to develop in vivo gene-editing therapies using Mammoth’s CRISPR technology. The protocol covers five preselected in vivo indications. The companies did not disclose indications, other than to say that the first focus of the agreement is liver disease. Depending on the progress of the affiliate program, Mammoth could earn more than $1 billion in milestone payments. Marianne De Backer, head of strategy, business development and licensing at Bayer, said in a presentation Monday at JPMorgan Chase’s annual healthcare conference that working with Mammoth and others has enabled her company to make a difference. A way to address unmet medical needs and with technologies not available within Bayer.
Bayer has agreed to pay research fees for the therapies covered by the agreement, as well as royalties from its new partners if any of the drugs in the agreement hit the market. Additionally, Bayer and Mammoth have agreed to explore extracorporeal therapy on a non-exclusive basis.
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