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Additional Technologies Precision genetic medicines

A key part of our strategy is to continue to build on the expertise of our rigorous and innovative research culture by exploring new and complementary technologies in base editing, gene editing, and genetic medicine.

Through a license agreement with the Broad Institute of MIT and Harvard, Beam has exclusively licensed the use of certain RNA base editing technology and Cas12b nuclease technology for all applications. Our licensed RNA base editing technologies include the REPAIR™ system for A-to-I editing, as well as the RESCUE™ system for C-to-U editing. RNA base editing may be useful for transient editing of the transcriptome. Access to the Cas12b nuclease technology allows us to create DNA base editors that can bind to different target sites in the genome, further expanding the range of sites that we can edit. In addition, access to the Cas12b nuclease technology allows us to make “cut” edits, which may be appropriate for some applications that require a double-stranded break.

Beam has also exclusively licensed the use of prime editing from Prime Medicine, Inc., in certain fields and for certain applications similar to those Beam is already pursuing with base editing. Prime editing is an emerging gene editing technology that uses a reverse transcriptase to rewrite short sequences of DNA at a CRISPR-directed location, without causing a double-stranded break, The license gives Beam the exclusive right to make any transition mutation (such as an A-to-G or C-to-T change) as well as to exploit any approach for the treatment of sickle cell disease using prime editing.

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Collectively, these additional technologies enable us to advance a broad portfolio of programs across a wide range of editing profiles and multiple delivery vehicles, in some cases exploring multiple editing approaches in parallel for our highest-priority therapeutic programs.