Pfizer and Beam Therapeutics announced an exclusive four-year research collaboration focused on in vivo base editing programs for rare diseases, marking Pfizer's foray in the CRISPR space.
The deal will leverage Beam’s proprietary in vivo delivery technologies, which use mRNA and lipid nanoparticles to deliver base editors to target organs. The Cambridge, Massachusetts start-up will conduct all research activities through development candidate selection for three undisclosed targets, which Pfizer says focus on rare genetic diseases of the liver, muscle and central nervous system.
Pfizer, backed by an established history in developing gene replacement therapies for rare diseases, sees the collaboration as an opportunity to advance the next generation of gene editing therapies — potentially leading to transformative treatments for rare genetic diseases.
Per the terms of the deal, 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