Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration

Pfizer and Beam Therapeutics, a biotechnology company developing precision genetic medicines through base editing, announced an exclusive four-year research collaboration on Jan. 10, 2022. The collaboration, worth up to $1.35 billion, will use Beam’s in-vivo base editing programs to conduct research on three undisclosed targets for rare genetic diseases of the liver, muscle, and central nervous system.

The base editing program 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. These technologies are designed to enable precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. According to a Pfizer press release, this would create an efficient edit when compared to traditional gene editing methods.

“At Pfizer, we believe in the powerful potential of mRNA and LNP technologies to address the greatest unmet needs for patients, as evidenced by the beneficial impact our mRNA/LNP-based COVID-19 vaccine is having on the pandemic,” said Mikael Dolsten, chief scientific officer and president, Worldwide Research, Development and Medical, Pfizer, in the press release. “We have a strong history in developing gene replacement therapies for rare diseases, and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies – an exciting scientific frontier – potentially leading to transformation for people living with rare genetic diseases.”

“We are thrilled to partner with Pfizer, a global leader in the design, development, and commercialization of novel medicines,” said John Evans, CEO of Beam, in the press release. “Our leading platform for precision genetic medicine has greatly evolved over the last few years, and we are committed to ensuring the broadest reach of these potentially life-changing technologies. This collaboration will provide a unique opportunity to create potentially transformative base editing programs for indications with critical unmet needs, leveraging our proprietary base editing technology and expanding delivery capabilities. We look forward to working together with Pfizer to advance these technologies and potentially expand our impact for people suffering from serious diseases.”

Pfizer will pay Beam $300 million upfront for their services in this collaboration. Should Pfizer exercise its opt-in license rights for all three targets, they will be eligible for development, regulatory, and commercial milestone payments for potential total deal consideration of up to $1.35 billion, in addition to royalties on global net sales.

Source: Pfizer