Beam Therapeutics Announces Multiple Scientific Advances in its Base Editing Technology Platforms
New Research Describes Novel Base Editors with Enhanced Editing Efficiency, Specificity, and
Keynote presentation at The 1st
International Conference on Base Editing– Enzymes and Applications titled “ABE8: Superior adenine base editors with expanded targeting range, higher activity, and therapeutic application” presented by Nicole Gaudelli, Ph.D., senior scientist and head of DNA Editing Platform at Beam.
Nature Biotechnologypaper titled “Evaluation and Minimization of Cas9-Independent Off-Target DNA Editing by Cytosine Base Editors” by co-lead authors
Jordan L. Domanand Aditya Raguram, with corresponding author David R. Liu, Ph.D., professor at Broad Instituteand Harvard University, Howard Hughes Medical Instituteinvestigator, and co-founder of Beam.
Nature Biotechnologypaper titled “Continuous Evolution of SpCas9 Variants Compatible with Non-G PAMs” by co-lead authors
Shannon M. Millerand Tina Wang, Ph.D., also with Dr. Liu as a corresponding author.
“Base editing is an emerging and highly differentiated new editing technology, and Beam is committed to further enhancing our base editing platform to potentially enable a wide range of therapeutic applications,” said
Beam’s base editors represent a potentially new class of precision genetic medicines that enable precise, predictable and efficient single base changes in the genome. ABEs and CBEs can make precisely-targeted A-to-G and C-to-T DNA base changes, respectively, with high efficiency and without the introduction of double-stranded breaks in the DNA, which can result in unwanted DNA insertions, deletions and rearrangements.
In the first publication, Dr. Gaudelli’s presentation highlighted the continued evolution of Beam’s adenine base editors (ABEs). Dr. Gaudelli used a bacterial selection strategy to identify ABEs with enhanced properties in mammalian cells. These proprietary “ABE8” variants demonstrated increased therapeutic potential relative to previously-published versions, including increased on-target activity while maintaining undetectable levels of off-target editing.
In the second publication, Dr. Liu’s Nature Biotechnology paper describes novel cytosine base editors (CBEs), which diminish or eliminate the low levels of off-target editing observed with the first published version of CBEs (known as BE3). Off-target editing rates observed with BE3 are very low and are within mutation rates that naturally occur with no consequence in somatic cells. Dr. Liu’s laboratory identified certain novel mutations in the deaminase domain that yielded base editors that retained high on-target editing while eliminating detectable off-target editing.
Finally, an additional publication from Dr. Liu’s laboratory, also in Nature Biotechnology, outlined novel variants of base editors that are able to bind a wider range of genetic targets, expanding their therapeutic potential. These editors feature modifications that allow them to bind to different “PAM” sequences in the genome. PAM sequences are short sequences of DNA bases that define where the targeting domain of the base editor can bind. The newly developed variants could potentially target up to 95% of all point mutations correctable with base editors and were shown to be compatible with both ABEs and CBEs.
This press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the ability of base editors to target up to 95% of all point mutations correctable with base editors. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks identified under the heading “Risk Factors” and elsewhere in the final prospectus dated