Beam Therapeutics Inc.

David A Born

Yi Yu

Bernd Zetsche

Ian Slaymaker

Nicole Marie Gaudelli

Michael S Packer

Seung-Joo Lee

Jason Michael Gehrke

Michael Packer

Francine Gregoire

Genesis Lung

Natalie Petrossian

Shaunna Berkovitch

Angelica Messana

Yvonne Aratyn

Nicole Gaudelli

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed 'ABE8' having unprecedented levels (e.g., >60-70%) of efficiency.

Compositions and methods for treating hemoglobinopathies

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed 'ABE8' having unprecedented levels (e.g., &#x3e;60-70%) of efficiency.

The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence

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Cambridge, MA, United States of America

David A Born