Pfizer Joins Beam in $1.4 Billion Genetic Therapy Alliance

(Bloomberg) — Pfizer Inc. will pay as much as $1.35 billion to partner with Beam Therapeutics Inc. on developing drugs for rare genetic diseases using base editing, a technology for manipulating DNA. 

Beam will get $300 million upfront under the four-year agreement, the companies said Monday in a statement. The deal allows Pfizer to develop up to three drug candidates for rare genetic diseases of the liver, muscle and central nervous system that Beam will select.

Base editing is a technique for changing individual letters of genetic code, an approach that Cambridge, Massachusetts-based Beam says can yield extremely precise modifications in the instructions for making cells and tissues. Because it alters DNA in such a targeted way, Beam says base editing carries less risk of side effects than genome-editing approaches like Crispr.

The development of Crispr won the 2020 Nobel Prize for two prominent researchers and launched a bevy of biotech companies seeking their first approved drug based on the technology. While Crispr can remove and replace specific DNA sequences, it’s prone to off-target effects that may lead to unwanted results in therapies. 

“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,” Pfizer’s Chief Scientific Officer Mikael Dolsten said in a statement. 

Beam uses messenger RNA and lipid nanoparticles to deliver its technology into the body. Pfizer brings expertise with both mRNA and nanoparticles, which are components of Covid-19 vaccines developed and produced with its German partner BioNTech SE. 

Pfizer’s Covid-19 vaccine has been a success story unlike any seen in the biopharma industry. The New York-based drugmaker expects 2021 global sales of the shot were $36 billion, the most ever by a pharmaceutical product in a single year. Sales are projected to be $31 billion this year.

Now, Pfizer is looking to expand its mRNA franchise through external business development. The company’s top leadership is particularly interested in early-stage RNA assets that would have benefits beyond the field of infectious disease, in areas such as rare diseases and cancer.

Shares of Pfizer rose about 60% in 2021 as its Covid vaccine sales boomed. Shares of Beam fell 2.4% in the same year. 

The deal was announced on the first day of the 2022 JPMorgan Health Care Conference. Usually held in San Francisco, the meeting is being held online amid the spread of the omicron variant. 

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