Pfizer and Beam Collaborate on Base Editing Programs for Rare Diseases

Cambridge-based Beam Therapeutics has entered into an exclusive four-year research collaboration with Pfizer focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system.  The base editing programs to be evaluated as part of the collaboration will leverage Beam’s proprietary in vivo delivery technologies, which use messenger RNA (mRNA) and lipid nanoparticles (LNP) to deliver base editors to target organs.

Beam’s proprietary base editing technologies are designed to enable a new class of precision genetic medicines that 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 terms of the collaboration agreement, 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, worldwide 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 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: Beam Therapeutics, 10 January, 2022)