Pfizer will invest up to $ 1.35 billion in a partnership with Beam Therapeutics to develop drugs for rare genetic diseases, all through the use of basic editing, a technique for manipulating DNA. Beam will get $ 300 million upfront under the four-year deal, the companies said. The agreement 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.
The base editor is a technique that changes the individual letters of the genetic code, an approach that American Beam says can produce extremely precise changes in the instructions for making cells and tissues. Because it alters DNA in such a targeted way, Beam says basic editing carries less risk of side effects than genome-editing approaches like the Crispr technique. The development of Crispr led to the 2020 Nobel Prize. Crispr can remove and replace specific DNA sequences, but can lead to undesirable 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 with the potential to lead to transformation for people living with rare genetic diseases,” said Mikael Dolsten. Pfizer’s chief scientific officer in a statement. Beam uses messenger Rna and lipid nanoparticles to bring its technology into the body. Pfizer brings experience with both mRNA and nanoparticles, components of Covid-19 vaccines developed and produced with German partner BioNTech.
Pfizer’s Covid-19 vaccine represents success story unlike any other in the biopharmaceutical industry. The New York-based pharmaceutical company expects 2021 global sales of the vaccine to be $ 36 billion, the highest ever achieved by a pharmaceutical product in just one year. Pfizer is looking to expand its mRNA franchise through external business development. The company’s top management is particularly interested in early stage RNA techniques that would have benefits beyond the infectious disease field, in areas such as rare diseases and cancer. Pfizer’s shares were up about 60% in 2021 as sales of its Covid vaccine 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. The meeting, usually held in San Francisco, is being held online this year to address the explosion of the Omicron variant.