Acuitas Therapeutics is a private biotechnology company that develops delivery systems for nucleic acid therapeutics based on lipid nanoparticles. Acuitas' LNP technology is used in the Pfizer-BioNTech COVID-19 vaccine.

Financial details of the agreement were not disclosed.

The base-editing programs to be evaluated as part of the collaboration between Pfizer and Beam will leverage Beam's proprietary in vivo delivery technologies, which use mRNA and LNP to deliver base editors to target organs. The collaboration seeks to advance potentially transformative therapies for patients living with rare diseases, both firms said.

Beam will conduct all research activities, including candidate selection, for three undisclosed targets that are not included in Beam's existing programs. Pfizer may opt into exclusive worldwide licenses for each candidate, after which it will be responsible for all development activities, as well as potential regulatory approvals and commercialization for each 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 a potential total of up to $1.35 billion. Beam is also eligible to receive royalties on global net sales of each licensed program.

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