USA – Pfizer has announced a four-year research partnership with Beam Therapeutics, a high-profile biotech company that specializes in base editing and the mRNA components used to deliver such treatments into the body.
The two biotech companies will engage in a four-year research agreement worth up to US$1.3 billion on focusing on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle, and central nervous system.
Pfizer is paying Beam US$300 million up front, with the latter eligible for future milestone payments of up to US$1.05 billion if Pfizer exercises its opt-in rights for all three targets.
“We see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies,” said Pfizer’s chief scientific officer, Mikael Dolsten.
Beam’s in vivo delivery technologies, which use mRNA and lipid nanoparticles (LNP) to deliver base editors to target organs, will be used in all three programs.
Beam will conduct all research activities under the agreed-upon terms through development candidate selection.
Pfizer will then be able to option exclusive, global licenses to each of them, at which point it will assume responsibility for all development activities.
Beam will also receive royalties based on global net sales for each licensed program. Beam will have the option to enter into a global co-development and co-commercialization agreement on one of the programs at the conclusion of Phase I/II studies.
The announcement of the transaction comes in during the J.P. Morgan Healthcare conference, which is typically a forum for major licensing or acquisition agreements in the biotech and pharmaceutical industries. Because of COVID-19, the meeting is being held virtually.
Beam was founded five years ago on the basis of research from Harvard University’s David Liu’s laboratory that demonstrated a more precise way to edit genes.
Base editing technology
Base editing, as opposed to the first generation of CRISPR-based gene editing, which cuts both strands of DNA, is intended to change a single DNA “letter” into another without causing a double-stranded break.
Its potential, like that of CRISPR, is enormous. The technology has the potential to be used to correct disease-causing mutations, silence or activate genes, and, in theory, has a lower risk of causing unwanted genetic changes.
The Beam collaboration is also Pfizer’s second major deal in a short period of time. The company, whose mRNA-based COVID-19 vaccine Comirnaty has generated record sales, recently agreed to buy Arena Pharmaceuticals for approximately US$6.7 billion.
Pfizer also strengthened its ties with BioNTech, its Comirnaty partner, last week, agreeing to focus their efforts on developing an mRNA-based vaccine for shingles.
Pfizer has also announced two additional agreements to expand its use of mRNA. The company is collaborating with Codex DNA to use that biotech’s technology, which could lead to more efficient
The Beam collaboration is Pfizer’s most significant foray into gene editing. Until now, the pharmaceutical company has invested more heavily in gene replacement therapy, and it now has two treatments in late-stage clinical trials for hemophilia and Duchenne muscular dystrophy.
And, by selecting Beam, Pfizer foregoes first-generation CRISPR editing in favor of base editing, as well as focusing on inside-the-body treatment rather than therapies comprised of lab-modified cells.
The latter type of therapy, such as those developed by CRISPR Therapeutics and Vertex, is further along in clinical development, but it is logistically more difficult and carries additional risks.
If they choose to, the companies will split net profits as well as development and commercialization costs, with Pfizer receiving 65 percent and Beam receiving 35 percent. The collaboration could be extended for another year.