Pfizer and Beam Therapeutics, a Cambridge, Massachusetts-based bio/pharma company developing precision genetic medicines through base editing, have entered into an exclusive four-year research collaboration for rare genetic diseases in a deal in a deal worth up to $1.35-billion ($350 million upfront and up to $1.05 billion in milestone payments).

The base editing programs will use Beam’s in-vivo delivery technologies, which use messenger RNA and lipid nanoparticles to deliver base editors to target organs. The pact is focused on rare genetic diseases of the liver, muscles, and central nervous system. Beam’s proprietary base editing technologies are designed to target a single base in the genome without making a double-stranded break in the DNA. This approach aims to create a more precise and efficient edit compared to traditional gene-editing methods, which operate by creating targeted double-stranded breaks in the DNA, resulting in potential challenges associated with unwanted DNA modifications.

Under the collaboration, Beam will conduct all research activities through development candidate selection for three undisclosed targets, which are not included in Beam’s existing programs. Pfizer may opt in to exclusive, global licenses to each development candidate, after which, it will be responsible for all development activities as well as potential regulatory approvals and commercialization for each such candidate. Beam has a right to opt in, at the end of Phase I/II studies, into a global co-development and co-commercialization agreement for one program. For that program, Pfizer and Beam would share net profits as well as development and commercialization costs in a 65%/35% ratio (Pfizer/Beam).

Beam will receive an upfront payment of $300 million and, assuming Pfizer exercises its opt-in license rights for all three targets, is eligible for development, regulatory and commercial milestone payments for potential total deal consideration of up to $1.35 billion. Beam is also eligible to receive royalties on global net sales for each licensed program. The collaboration has an initial term of four years and may be extended up to one additional year.

Source: Pfizer and Beam Therapeutics