What will Pfizer and Beam Therapeutics do?
American giant Pfizer has entered into a $ 1.35 billion deal with Beam Therapeutics. All the details on their plans for rare genetic diseases
Pfizer announced a $ 1.35 billion deal with Beam Therapeutics, a US biotech company specializing in the development of precision genetic drugs. Their partnership is expected to yield three drug candidates for rare genetic diseases.
THE AGREEMENT
The $ 1.35 billion agreement between Pfizer and Beam Therapeutics, according to the two companies , provides that Beam will receive $ 300 million upfront as part of the agreement, which has an initial term of four years and can be extended up to an additional year.
The project will allow Pfizer to develop up to three drug candidates for rare genetic diseases of the liver, muscles and central nervous system.
Under the terms of the agreement, yes in the press release, Beam will conduct all research activities through the selection of the candidate for development. Pfizer may opt for exclusive and worldwide licensing for each candidate, after which Pfizer will be responsible for all development activities, as well as potential regulatory and marketing approvals, for each such candidate.
However, Beam has the right to opt, at the end of Phase 1/2 studies, for a fee, for a co-development and co-commercialization agreement with respect to a program under which Pfizer and Beam would share the net profits. and development and marketing costs in a ratio of 65% / 35% (Pfizer / Beam).
WHAT BEAM DOES
Beam Therapeutics, based in Massachusetts, is a biotechnology company launched in 2018. It specializes in the use of technology based editing [editing basics, ed], a technique for making changes permanent and specific to individual bases in the DNA and in 'Rna, without cutting the filaments.
Gene editing, as opposed to gene therapy which adds healthy genes alongside one or more defective genes, “treats” the problem gene.
Using base editing allows you to change the individual letters of the genetic code in a way that, the company says, can produce extremely precise changes in the instructions for making cells and tissues. According to Beam, being such a targeted technique, it carries less risk of side effects than genome-editing approaches such as the Crispr technique, which led to the 2020 Nobel Prize.
THE PARTNERSHIP
Combining Beam's core editing technology with Pfizer's mRna and lipid nanoparticle (LNP) technology is aimed at advancing advances in potentially transformative therapies for patients with rare diseases.
"At Pfizer, we believe in the incredible potential of mRna and LNP technologies to address the greatest unmet needs of patients, as evidenced by the beneficial impact our mRna / LNP-based Covid-19 vaccine is having on the pandemic," he said. Pfizer Chief Scientific Officer Mikael Dolsten .
"We have a strong history in developing gene replacement therapies for rare diseases," he continued, "and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies – an exciting scientific frontier – that potentially it can lead to real change for people suffering from rare genetic diseases ”.
This is a machine translation from Italian language of a post published on Start Magazine at the URL https://www.startmag.it/sanita/cosa-faranno-pfizer-e-beam-therapeutics-insieme/ on Sun, 23 Jan 2022 16:01:26 +0000.