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BEAM Beam Therapeutics

APP
Utility
Methods of Editing Single Nucleotide Polymorphism Using Programmable Base Editor Systems
9 Dec 21
The invention features compositions and methods for altering mutations associated with Rett Syndrome (RTT).
David Bryson, John Evans, Michael Packer, Jason Michael Gehrke, Natalie Petrossian
Filed: 11 May 19
APP
Utility
Methods of Suppressing Pathogenic Mutations Using Programmable Base Editor Systems
2 Dec 21
Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide.
John Evans, Yanfang Fu, Michael Packer
Filed: 11 May 19
GRANT
Utility
Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence
26 Oct 21
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.
Nicole Gaudelli, Michael Packer, Ian Slaymaker, Yi Yu, Bernd Zetsche, Jason Michael Gehrke, Natalie Petrossian, Angelica Messana, Yvonne Aratyn, Francine Gregoire, Genesis Lung, Shaunna Berkovitch, David A. Born, Seung-Joo Lee
Filed: 18 Dec 20
GRANT
Utility
Compositions and methods for treating hemoglobinopathies
12 Oct 21
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD).
Ian Slaymaker, Nicole Gaudelli, Yi Yu, Bernd Zetsche, David A. Born, Seung-Joo Lee, Michael Packer
Filed: 26 Mar 21
APP
Utility
Compositions and Methods for Delivering a Nucleobase Editing System
30 Sep 21
The invention provides compositions and methods for delivering first and second polynucleotides each encoding a fragment of an A-to-G Base Editor fusion protein comprising one or more deaminases (e.g., adenosine deaminases) and nCas9, wherein the first polynucleotide encodes an N-terminal fragment of nCas9 fused to an intein-N of a split intein pair and the second polynucleotide encodes a C-terminal fragment of nCas9 fused to an intein-C of a split intein pair, and methods for delivering these fragments together with an sgRNA to a cell (e.g., AAV delivery), where the fragments are spliced together by a split intein system, thereby reconstituting a functional base editing system in the cell.
David Bryson
Filed: 7 Sep 19
APP
Utility
Multi-effector Nucleobase Editors and Methods of Using Same to Modify a Nucleic Acid Target Sequence
9 Sep 21
The invention features a multi-effector nucleobase editor capable of inducing changes at multiple different bases within a target nucleic acid and methods of using such editors.
Nicole Gaudelli, John Evans
Filed: 2 Aug 19
APP
Utility
Compositions and Methods for Treating Hemoglobinopathies
26 Aug 21
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD).
Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER
Filed: 26 Mar 21
APP
Utility
Compositions and Methods for Treating Hemoglobinopathies
19 Aug 21
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD).
Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER
Filed: 26 Mar 21
APP
Utility
Adenosine Deaminase Base Editors and Methods of Using Same to Modify a Nucleobase In a Target Sequence
6 May 21
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.
Nicole GAUDELLI, Michael PACKER, Ian SLAYMAKER, Yi YU, Bernd ZETSCHE, Jason Michael GEHRKE, Natalie PETROSSIAN, Angelica MESSANA, Yvonne ARATYN, Francine GREGOIRE, Genesis LUNG, Shaunna BERKOVITCH, David A. BORN, Seung-Joo LEE
Filed: 18 Dec 20
APP
Utility
Uses of Adenosine Base Editors
23 Dec 20
The disclosure provides methods and compositions for treating blood diseases/disorders, such as sickle cell disease, hemochromatosis, hemophilia, and beta-thalassemia.
David R. Liu, Nicole Gaudelli, Michael S. Packer, Gregory Newby
Filed: 15 Oct 18
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