Patents Assigned to Beam Therapeutics, Inc.
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Publication number: 20240167008Abstract: The present invention provides novel systems, methods and compositions for making and using recombinantly engineered novel Cas9 enzymes optimized for human cells, for nucleic acid targeting and manipulation. The present invention is based on the discovery of novel Cas9 enzymes from Streptococcus constellatus, Sharpen spp. isolate RUG017, Veillonella parvula, Ezakiella peruensis, Lactobacillus fermentum strain AF15-40LB strain and Peptoniphilus sp. Marseille-P3761 bacteria that were codon-optimized and recombinantly produced for use in human cells. In some embodiments, novel Cas9 enzymes can be used for base editing. In some embodiments, the novel engineered Cas9 enzymes are used to treat human diseases.Type: ApplicationFiled: March 23, 2022Publication date: May 23, 2024Applicant: Beam Therapeutics Inc.Inventors: Bernd ZETSCHE, Luis BARRERA, David A. BORN, Ming SUN
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Publication number: 20240158775Abstract: The invention provides adenosine deaminase variants that are capable of deaminating adenine and/or cytosine in a target polynucleotide (e.g., DNA). The disclosure also provides multi-molecular complexes, fusion proteins, base editors, and base editor systems comprising the adenosine deaminase variants with adenine and cytosine deaminase activity and/or cytosine deaminase specificity, and methods of use thereof.Type: ApplicationFiled: September 26, 2023Publication date: May 16, 2024Applicant: Beam Therapeutics Inc.Inventors: Nicole Gaudelli, Seung-Joo Lee, Patricia Rosa Feliciano, Dieter Ka Yeung Lam
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Publication number: 20240132867Abstract: 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.Type: ApplicationFiled: October 13, 2023Publication date: April 25, 2024Applicant: Beam Therapeutics Inc.Inventors: 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
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Publication number: 20240132868Abstract: The invention features polynucleotides that encode bases editors having a heterologous intron for self-inactivation, compositions comprising such polynucleotides, and methods of inactivating a base editor encoded by such polynucleotides.Type: ApplicationFiled: November 27, 2023Publication date: April 25, 2024Applicant: Beam Therapeutics Inc.Inventors: David BRYSON, Jack SULLIVAN
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Publication number: 20240124866Abstract: The disclosure provides methods and compositions for treating blood diseases/disorders, such as sickle cell disease, hemochromatosis, hemophilia, and beta-thalassemia. For example the disclosure provides therapeutic guide RNAs that target the promotor of HBG1/2 to generate point mutations that increase expression of fetal hemoglobin. As another example, the disclosure provides therapeutic guide RNAs that target mutations in HBB, Factor VIII, and HFE to treat sickle cell disease, beta-thalassemia, hemophilia and hemochromatosis. The disclosure also provides fusion proteins comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA. In some embodiments, the fusion proteins are in complex with nucleic acids, such as guide RNAs (gRNAs), which target the fusion proteins to a DNA sequence (e.g., an HBG1 or HBG2 protmoter sequence, or an HFE, GBB, or F8 gene sequence).Type: ApplicationFiled: September 1, 2023Publication date: April 18, 2024Applicants: The Broad Institute, Inc., President and Fellows of Harvard College, Beam Therapeutics Inc.Inventors: David R. Liu, Nicole Marie Gaudelli, Michael S. Packer, Gregory Newby
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Publication number: 20240124536Abstract: Provided herein are compositions for gene modification related to base editor systems, and methods of using the same to treat or prevent conditions associated with the extracellular deposition in various tissues of amyloid fibrils formed by the aggregation of misfolded transthyretin (TTR) proteins. Such conditions include, but are not limited to, polyneuropathy due to hereditary transthyretin amyloidosis (hATTR-PN) and hereditary cardiomyopathy due to transthyretin amyloidosis (hATTR-CM), both associated with autosomal dominant mutations of the TTR gene, and an age-related cardiomyopathy associated with wild-type TTR proteins (ATTRwt), also known as senile cardiac amyloidosis.Type: ApplicationFiled: November 20, 2023Publication date: April 18, 2024Applicant: Beam Therapeutics Inc.Inventors: Alexandra C. CHADWICK, Kallanthottathil G. RAJEEV
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Publication number: 20240117345Abstract: The invention features compositions and methods for editing a transthyretin polynucleotide sequence to treat amyloidosis.Type: ApplicationFiled: November 13, 2023Publication date: April 11, 2024Applicant: Beam Therapeutics Inc.Inventors: Michael PACKER, Lo-I CHENG, Tanggis BOHNUUD
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Publication number: 20230383277Abstract: Described and provided are adenosine base editors and compositions comprising adenosine base editors that have increased efficiency. Also described and provided are methods of using base editors comprising adenosine deaminase variants for altering mutations associated with Glycogen Storage Disease Type 1a (GSD1a).Type: ApplicationFiled: October 14, 2021Publication date: November 30, 2023Applicant: Beam Therapeutics Inc.Inventors: Brian CAFFERTY, Tanggis BOHNUUD, Lo-I CHENG, Michael PACKER, Yvonne ARATYN-SCHAUS
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Publication number: 20230348359Abstract: The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.Type: ApplicationFiled: July 12, 2023Publication date: November 2, 2023Applicant: Beam Therapeutics Inc.Inventors: Mina Fawzy Shehata, Gregory Lawrence Hamilton, Neeraj Narendra Patwardhan, Cory Dane Sago, Milloni Balwantkumar Chhabra
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Patent number: 11795443Abstract: The disclosure provides methods and compositions for treating blood diseases/disorders, such as sickle cell disease, hemochromatosis, hemophilia, and beta-thalassemia. For example the disclosure provides therapeutic guide RNAs that target the promotor of HBG1/2 to generate point mutations that increase expression of fetal hemoglobin. As another example, the disclosure provides therapeutic guide RNAs that target mutations in HBB, Factor VIII, and HFE to treat sickle cell disease, beta-thalassemia, hemophilia and hemochromatosis. The disclosure also provides fusion proteins comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA. In some embodiments, the fusion proteins are in complex with nucleic acids, such as guide RNAs (gRNAs), which target the fusion proteins to a DNA sequence (e.g., an HBG1 or HBG2 protmoter sequence, or an HFE, GBB, or F8 gene sequence).Type: GrantFiled: October 16, 2018Date of Patent: October 24, 2023Assignees: The Broad Institute, Inc., President and Fellows of Harvard College, Beam Therapeutics, Inc.Inventors: David R. Liu, Nicole Marie Gaudelli, Michael S. Packer, Gregory Newby
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Patent number: 11752202Abstract: 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.Type: GrantFiled: April 13, 2022Date of Patent: September 12, 2023Assignee: Beam Therapeutics Inc.Inventors: Ian Slaymaker, Nicole Gaudelli, Yi Yu, Bernd Zetsche, David A. Born, Seung-Joo Lee, Michael Packer
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Publication number: 20230242884Abstract: The invention 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 and methods of using same to treat genetical disorder or conditions, e.g. sickle cell disease, with engraftment.Type: ApplicationFiled: February 12, 2021Publication date: August 3, 2023Applicant: Beam Therapeutics Inc.Inventors: Sarah SMITH, Dana LEVASSEUR, Jonathan YEN
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Publication number: 20230159956Abstract: 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. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.Type: ApplicationFiled: May 11, 2019Publication date: May 25, 2023Applicant: Beam Therapeutics Inc.Inventors: David Bryson, John Evans, Michael Packer, Jason Michael Gehrke, Natalie Petrossian
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Publication number: 20230140953Abstract: The invention provides compositions comprising novel programmable adenosine base editor systems (e.g., ABE8) that provide methods of treating a disease or disorder, (e.g., Parkinson's disease, Hurler syndrome, Rett syndrome, or Stargardt disease) in a subject by administering to the subject a programmable adenosine base editor system (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a disease-associated gene.Type: ApplicationFiled: February 13, 2020Publication date: May 11, 2023Applicant: BEAM THERAPEUTICS INC.Inventors: Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER, Jason Michael GEHRKE, Natalie PETROSSIAN, Angelica MESSANA, Shaunna BERKOVITCH
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Publication number: 20230128472Abstract: 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.Type: ApplicationFiled: July 26, 2022Publication date: April 27, 2023Applicant: Beam Therapeutics Inc.Inventors: Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER
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Publication number: 20230101597Abstract: The present invention features compositions and methods for editing deleterious mutations associated with alpha-1 anti-trypsin (A1AT) deficiency. In particular embodiments, the invention provides methods for correcting mutations in an A1AT polynucleotide using an adenosine deaminase base editor, ABE8, having unprecedented levels of efficiency.Type: ApplicationFiled: February 13, 2020Publication date: March 30, 2023Applicant: BEAM THERAPEUTICS INC.Inventors: Nicole GAUDELLI, Michael PACKER, Bernd ZETSCHE, Ian SLAYMAKER, Yi YU, David A. BORN, Seung-Joo LEE
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Publication number: 20230080198Abstract: The present invention features genetically modified immune cells comprising novel adenosine base editors (e.g., ABE8) having enhanced anti-neoplasia activity, resistance to immune suppression, and decreased risk of eliciting a graft-versus-host reaction or host-versus-graft reaction, or a combination thereof. The present invention also features methods for producing and using these modified immune effector cells.Type: ApplicationFiled: February 13, 2020Publication date: March 16, 2023Applicant: BEAM THERAPEUTICS INC.Inventors: Nicole GAUDELLI, Michael PACKER, Ian SLAYMAKER, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Jason M. GEHRKE
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Publication number: 20230075877Abstract: The invention features novel programmable nucleobase editors comprising adenosine deaminase domains and methods of using the same for polynucleotide editing. In some embodiments, programmable nucleobase editors edit a pathogenic mutation associated with a genetic disease.Type: ApplicationFiled: September 9, 2020Publication date: March 9, 2023Applicant: Beam Therapeutics Inc.Inventors: Nicole GAUDELLI, Michael PACKER
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Publication number: 20230070861Abstract: The invention features compositions and methods for introducing mutations into the hepatitis B virus (HBV) genome.Type: ApplicationFiled: May 8, 2020Publication date: March 9, 2023Applicant: Beam Therapeutics Inc.Inventors: Elena SMEKALOVA, Michael PACKER, Francine GREGOIRE, Luis BARRERA, Giuseppe CIARAMELLA
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Publication number: 20230021636Abstract: As described below, the present invention features genetically modified immune cells having enhanced anti-neoplasia activity, resistance to immune suppression, and decreased risk of eliciting a graft versus host reaction, or a combination thereof. The present invention also features methods for producing and using these modified immune effector cells.Type: ApplicationFiled: September 25, 2020Publication date: January 26, 2023Applicants: Beam Therapeutics Inc., UCL BUSINESS LTD.Inventors: Jason Michael GEHRKE, Aaron D. EDWARDS, Ryan MURRAY, Waseem QASIM