12 January 2022 - - US-based pharmaceutical company Pfizer Inc. (NYSE: PFE) and US-based biotechnology company Beam Therapeutics Inc. (NASDAQ: BEAM) have entered an exclusive four-year research collaboration focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system, the company said.

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 and lipid nanoparticles to deliver base editors to target organs.

Combining these technologies with Pfizer's proven experience in developing and manufacturing medicines and vaccines, this collaboration seeks to advance potentially transformative therapies for patients living with rare diseases.

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 has a right to opt in, at the end of Phase 1/2 studies, upon the payment of an option exercise fee, to a global co-development and co-commercialization agreement with respect to one program licensed under the collaboration pursuant to which 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 USD 300m 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 USD 1.35bn.

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.