Under the four-year research collaboration, the companies will focus on in vivo base editing programmes for three targets for rare genetic diseases of the liver, muscle and central nervous system
Pfizer has announced a collaboration with biotechnology company Beam Therapeutics to develop therapies for rare genetic diseases in a potential deal worth up to $1.35bn.
Under the four-year research collaboration, the companies will focus on in vivo base editing programmes for three targets for rare genetic diseases of the liver, muscle and central nervous system.
As part of the collaboration, Beam will carry out all research activities through development candidate selection for three undisclosed targets, which are not part of its existing programmes.
Under the terms of the agreement, the gene-editing specialist will receive an upfront payment of $300m.
Beam’s in vivo delivery technologies will be leveraged for the evaluation of the base editing programmes. Using messenger RNA (mRNA) and lipid nanoparticles (LNP), the technologies deliver base editors to target organs.
By combining the technologies with Pfizer’s expertise in developing medicines and vaccines, the collaboration intends to advance potentially transformative therapies for rare diseases.
Pfizer worldwide research, development and medical president and chief scientific officer Mikael Dolsten said: “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.
“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.”
The collaboration enables Pfizer to opt in to exclusive, worldwide licences to each development candidate. Upon exercising the option, the company will be responsible for all development activities, regulatory approvals and commercialisation for each such candidate.
If Pfizer chooses for opt-in license rights for all three targets, Beam will be eligible to receive development, regulatory and commercial milestone payments worth up to $1.35bn.
In addition, Beam will be eligible for royalties on global net sales for each liceced programme.
Recently, Pfizer has partnered with BioNTech to research, develop and commercialise what they claim is the world’s first mRNA vaccine against shingles (herpes zoster virus/HZV).