Pfizer, Beam Focus on Precision Medicine for Rare Diseases

Pfizer and Beam Therapeutics recently entered into a four-year collaboration focused on in vivo base editing programs to generate precision medicine for rare diseases of the liver, muscle, and central nervous system.

The companies will leverage Beam Therapeutics’ in vivo delivery technologies, which use messenger RNA (mRNA) and lipid nanoparticles (LNP) to deliver base editors to target organs.

These base editing technologies will enable a new class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA, a

Pfizer spokesperson explained. This approach creates a more specific and efficient edit.

Combining these technologies with Pfizer’s experience in developing and manufacturing medicines and vaccines will help advance potentially transformative therapies for patients living with rare diseases.

“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,” Mikael Dolsten, MD, PhD, chief scientific officer and president of worldwide research development, and medical at Pfizer, said in the announcement.

“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,” Dolsten continued.

Under the terms of the collaboration, Beam Therapeutics will conduct all research activities through development candidate selection for three undisclosed targets.

Notably, the targets are not included in Beam Therapeutics’ existing programs.

Pfizer will be responsible for all development activities, potential regulatory approvals, and commercialization’s for each candidate. In addition, the company may opt for an exclusive, global license as well.

Beam Therapeutics will receive an upfront payment of $300 million and is eligible for development, regulatory, and commercial milestone payments for potential total deal considerations of up to $1.35 billion.

John Evans, chief executive officer of Beam Therapeutics’, stated that the collaboration will provide a unique opportunity to create transformative base editing technology and expand overall delivery capabilities.

“We look forward to working together with Pfizer to advance these technologies and potentially expand out impact for people suffering from serious disease,” Evans concluded.