Rare Daily Staff

Beam Therapeutics has entered into a collaboration and license agreement with newly formed Prime Medicine to research and develop a novel gene editing technology called prime editing to develop treatments for sickle cell disease and disorders of single-base transition mutations.

Under the terms of their agreement, Beam has the exclusive right to develop prime editing technology for the creation or correction of any single-base transition mutations, as well as for the treatment of sickle cell disease, both of which Beam is already pursuing with its base editing technology. Transition mutations (for example A to G, C to T) are the largest single class of disease-associated genetic mutations, and are also potentially treatable with base editing. Beam plans to evaluate prime editing technology for potential use in future programs. Prime editing was recently developed by one of Beam’s co-founders, David Liu, and his group at the Broad Institute of Harvard and MIT.

“This partnership enables both companies to advance the technology in distinct spaces, with Beam focusing on the kinds of edits that are most similar to our base editing technology,” said John Evans, CEO of Beam.

As part of the collaboration, Beam is providing initial interim leadership to Prime Medicine for the first year of the collaboration, and will have the right to designate a member on Prime Medicine’s board. The parties will also grant each other non-exclusive licenses to certain CRISPR technology and delivery technology to enable the development of prime editing products.

In early October, Liu and his team published a paper in the journal Nature that described prime editing as “a versatile and precise genome editing method that directly writes new genetic information into a specified DNA site using a catalytically impaired Cas9 fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit.”

The researchers said they were able to efficiently correct the primary genetic causes of sickle cell disease and Tay-Sachs disease in human cells without creating too many unwanted effects.

In September, Beam filed with the Securities and Exchange Commission for an initial public.

Photo: John Evans, CEO of Beam Therapeutics

X