Presentations and Publications

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July 6, 2026 | Beam-304

BEAM-304: a platform of base editing therapeutics for patients with phenylketonuria

FASEB Genome Engineering: Research and Applications

June 15, 2026 | Risto-cel

Ristoglogene autogetemcel treatment restored red blood cell health and function in patients with sickle cell disease, with sickling and rheology parameters comparable to sickle cell trait

European Hematology Association 2026 Congress

May 18, 2026 | Beam-302

The potential for base editing to treat diseases impacting the lungs: Ongoing data from the BEAM-302 clinical trial in alpha-1 antitrypsin deficiency (AATD)

American Thoracic Society International Conference

April 1, 2026 | Risto-cel

Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

The New England Journal of Medicine

December 8, 2025 | Risto-cel

Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (ristoglogene autogetemcel; BEAM-101) in the ongoing BEACON study

2025 ASH Annual Meeting

December 6, 2025 | Risto-cel

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: safety and efficacy findings from the ongoing BEACON study

2025 ASH Annual Meeting

June 13, 2025 | Risto-cel

Base editing for sickle cell disease: ongoing results from the BEACON study evaluating the safety and efficacy of BEAM-101, the first base-edited autologous CD34+ HSPC one-time cell therapy

European Hematology Association 2025 Congress

June 13, 2025 | Risto-cel

Integrated Editing and Manufacturing Process Design for BEAM-101, an Autologous CD34+ HSPC Therapy for Sickle Cell Disease, Results in Robust Process Yield and Increased HbF Induction

European Hematology Association 2025 Congress

June 13, 2025 | Risto-cel

Red blood cell (RBC) health and function post BEAM-101 treatment: multiple exploratory biomarkers demonstrate rheology and sickling parameters comparable to sickle cell trait (SCT)

European Hematology Association 2025 Congress

May 18, 2025 | Beam-302

Evaluating the Economic Burden: Lifetime Direct and Indirect Costs of Managing Alpha-1 Antitrypsin Deficiency for Patients with the Pi*ZZ Genotype Receiving Augmentation Therapy in the United States

American Thoracic Society 2025 International Conference

April 5, 2025 | Beam-302

BEAM-302: A potential base editing therapeutic for alpha-1 antitrypsin deficiency (Alpha-1)

2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress

December 9, 2024 | Beam-201

BEAM-201 for the Treatment of Relapsed and/or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL)

2024 ASH Annual Meeting

December 9, 2024 | Risto-cel

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

2024 ASH Annual Meeting

December 8, 2024 | Escape

CD117 antibody conditioning and multiplex base editing enable rapid and robust fetal hemoglobin reactivation in a rhesus autologous transplantation model

2024 ASH Annual Meeting

December 7, 2024 | Risto-cel

Initial Results from the Beacon Clinical Study

2024 ASH Annual Meeting

November 10, 2024 | Beam-301

Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia

Nature Communications

June 14, 2024 | Risto-cel

Robust autologous CD34+ HSPC manufacturing with a closed and automated process optimized for patients with sickle cell disease

2024 EHA Congress

June 8, 2024 | Beam-302

BEAM-302: Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1)

Alpha-1 National Conference

October 25, 2023 | Beam-301

A Single, Systemic Administration of BEAM-301 Mitigated Fasting Hypoglycemia One Year after Dosing in a Transgenic Mouse Model of Glycogen Storage Disease Type-Ia

Alpha-1 Antitrypsin Deficiency 2023 Meeting

October 25, 2023 | Beam-302

BEAM-302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha-1 Antitrypsin Deficiency

2023 ESGCT Congress

September 20, 2023 | Research

Adenine Base Editing enables sustained reduction of HBsAg expression and inhibition of Hepatitis B viral replication in preclinical models

2023 Inserm

September 7, 2023 | Beam-302

BEAM-302: Targeting AATD-related Liver and Lung Disease with Base Editing

Alpha-1 Antitrypsin Deficiency 2023 Meeting

May 19, 2023 | Escape

Paired HSC Epitope Engineering of CD117 (cKIT) for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for The Potential Treatment of Hemoglobinopathies

ASGCT 26th Annual Meeting

May 19, 2023 | Research

In Vivo Genetic Eye Disease Correction Using Split AAV-Mediated Adenine Base Editing

ASGCT 26th Annual Meeting

January 9, 2023 | Research

Improved cytosine base editors generated from TadA variant

Nature Biotechnology

December 12, 2022 | Escape

Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired HSC epitope engineering and upregulation of fetal hemoglobin for antibody-mediated autologous hematopoietic stem cell therapy conditioning for the potential treatment of hemoglobinopathies

2022 ASH Annual Meeting

December 10, 2022 | Escape

ESCAPE-2: Paired HSC CD117-epitope engineering and direct editing of sickle allele through base editing for antibody-mediated autologous HSC therapy conditioning for potential treatment of sickle cell anemia

2022 ASH Annual Meeting

September 19, 2022 | Research

Cytosine base editing inhibits Hepatitis B Virus replication and reduces HBsAg expression in vitro and in vivo

HBV International Meeting

August 27, 2022 | Research

Directed evolution and engineering of CBE-T: next-generation cytosine base editors utilizing TadA

Genome engineering: CRISPR frontiers

June 27, 2022 | Escape

Non-genotoxic antibody-based conditioning paired with multi-plex base edited HSCs for the potential treatment of sickle cell disease

FASEB Genome Engineering Conference

May 17, 2022 | Research

Efficient LNP delivery of mRNA in vivo and ex vivo to T and NK cells

ASGCT 25th Annual Meeting

May 16, 2022 | Beam-301

Single, Systemic Administration of BEAM 301 Mitigates Fasting Hypoglycemia and Restores Metabolic Function in a Transgenic Mouse Model of Glycogen Storage Disease Type Ia

ASGCT 25th Annual Meeting

May 16, 2022 | Beam-302

Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency

ASGCT 25th Annual Meeting

December 13, 2021 | Research

Screening of Chemically Distinct Lipid Nanoparticle In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells

2021 ASH Annual Meeting

December 13, 2021 | Research

Applied Base Editing to Treat Beta Hemoglobinopathies

2021 ASH Annual Meeting

November 23, 2021 | Beam-102

Conversion of HbS to Hb G-Makassar by adenine base editing is compatible with normal hemoglobin function

Blood

November 12, 2021 | Beam-201

CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 investigational CAR T-cell therapy for the treatment of T-cell malignancies

SITC 36th Annual Meeting

October 20, 2021 | Beam-301

in vivo Base Editing Corrects Metabolic Defects in Glycogen Storage Disease Type-Ia

ESGCT 2021 Virtual Congress

September 27, 2021 | Research

cccDNA inactivation using cytosine base editors

2021 HBV International Meeting

September 23, 2021 | Research

Optimization of LNP for in vivo base editing

TIDES 2021

May 21, 2021 | Research

Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases

ASGCT 24th Annual Meeting

May 21, 2021 | Research

LC MS Confirmation of Single Amino Acid Correction by Base Editing

ASGCT 24th Annual Meeting

December 21, 2020 | Research

Adenine Base Editing of Gamma Globin Gene Promoters Shows no Detectable Off-Target RNA or DNA Editing

2020 ASH Annual Meeting

December 21, 2020 | Research

Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

2020 ASH Annual Meeting

December 21, 2020 | Risto-cel

Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

2020 ASH Annual Meeting

November 21, 2020 | Beam-301

Base-Editing as a Therapeutic Approach for the Direct Correction of Disease Causing Mutations underlying Glycogen Storage Disease Type Ia

2020 AASLD Annual Meeting

May 13, 2020 | Beam-302

Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency

ASGCT 23rd Annual Meeting

May 13, 2020 | Risto-cel

Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease

ASGCT 23rd Annual Meeting

May 13, 2020 | Risto-cel

A Novel Base Editing Approach to Directly Edit the Causative Mutation in Sickle Cell Disease

ASGCT 23rd Annual Meeting

May 11, 2020 | Beam-302

Translating Base Editing Technology into a Potential Treatment for Alpha-1 Antitrypsin Deficiency

ASGCT 23rd Annual Meeting

April 28, 2020 | Research

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

Nature

April 13, 2020 | Research

Directed evolution of adenine base editors with increased activity and therapeutic application

Nature

January 14, 2020 | Research

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

Nature Biomedical Engineering

December 19, 2019 | Risto-cel

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and b-thalassemia

2019 ASH Annual Meeting

November 13, 2019 | Risto-cel

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Blood

October 29, 2019 | Research

Engineering of CRISPR-Cas12b for human genome editing

Nature Communications

October 29, 2019 | Research

Search-and-replace genome editing without double-strand breaks or donor DNA

Nature

October 19, 2019 | Research

A cytosine deaminase for programmable single-base RNA editing

Science

October 15, 2018 | Research

Base editing: precision chemistry on the genome and transcriptome of living cells

Nature Review Genetics

October 25, 2017 | Research

RNA editing with CRISPR-Cas13

Science

October 25, 2017 | Research

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

Nature

August 30, 2017 | Research

Improved base excision repair inhibition and bacteriophage

Science Advances

June 6, 2017 | Research

Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery

Nature Communications

February 13, 2017 | Research

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

Nature Biotechnology

May 19, 2016 | Research

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

Nature

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Presentations and Publications Our scientific advances

Filter By:

BEAM-304: a platform of base editing therapeutics for patients with phenylketonuria

Ristoglogene autogetemcel treatment restored red blood cell health and function in patients with sickle cell disease, with sickling and rheology parameters comparable to sickle cell trait

The potential for base editing to treat diseases impacting the lungs: Ongoing data from the BEAM-302 clinical trial in alpha-1 antitrypsin deficiency (AATD)

Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (ristoglogene autogetemcel; BEAM-101) in the ongoing BEACON study

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: safety and efficacy findings from the ongoing BEACON study

Base editing for sickle cell disease: ongoing results from the BEACON study evaluating the safety and efficacy of BEAM-101, the first base-edited autologous CD34+ HSPC one-time cell therapy

Integrated Editing and Manufacturing Process Design for BEAM-101, an Autologous CD34+ HSPC Therapy for Sickle Cell Disease, Results in Robust Process Yield and Increased HbF Induction

Red blood cell (RBC) health and function post BEAM-101 treatment: multiple exploratory biomarkers demonstrate rheology and sickling parameters comparable to sickle cell trait (SCT)

Evaluating the Economic Burden: Lifetime Direct and Indirect Costs of Managing Alpha-1 Antitrypsin Deficiency for Patients with the Pi*ZZ Genotype Receiving Augmentation Therapy in the United States

BEAM-302: A potential base editing therapeutic for alpha-1 antitrypsin deficiency (Alpha-1)

BEAM-201 for the Treatment of Relapsed and/or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL)

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

CD117 antibody conditioning and multiplex base editing enable rapid and robust fetal hemoglobin reactivation in a rhesus autologous transplantation model

Initial Results from the Beacon Clinical Study

Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia

Robust autologous CD34+ HSPC manufacturing with a closed and automated process optimized for patients with sickle cell disease

BEAM-302: Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1)

A Single, Systemic Administration of BEAM-301 Mitigated Fasting Hypoglycemia One Year after Dosing in a Transgenic Mouse Model of Glycogen Storage Disease Type-Ia

BEAM-302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha-1 Antitrypsin Deficiency

Adenine Base Editing enables sustained reduction of HBsAg expression and inhibition of Hepatitis B viral replication in preclinical models

BEAM-302: Targeting AATD-related Liver and Lung Disease with Base Editing

Paired HSC Epitope Engineering of CD117 (cKIT) for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for The Potential Treatment of Hemoglobinopathies

In Vivo Genetic Eye Disease Correction Using Split AAV-Mediated Adenine Base Editing

Improved cytosine base editors generated from TadA variant

Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired HSC epitope engineering and upregulation of fetal hemoglobin for antibody-mediated autologous hematopoietic stem cell therapy conditioning for the potential treatment of hemoglobinopathies

ESCAPE-2: Paired HSC CD117-epitope engineering and direct editing of sickle allele through base editing for antibody-mediated autologous HSC therapy conditioning for potential treatment of sickle cell anemia

Cytosine base editing inhibits Hepatitis B Virus replication and reduces HBsAg expression in vitro and in vivo

Directed evolution and engineering of CBE-T: next-generation cytosine base editors utilizing TadA

Non-genotoxic antibody-based conditioning paired with multi-plex base edited HSCs for the potential treatment of sickle cell disease

Efficient LNP delivery of mRNA in vivo and ex vivo to T and NK cells

Single, Systemic Administration of BEAM 301 Mitigates Fasting Hypoglycemia and Restores Metabolic Function in a Transgenic Mouse Model of Glycogen Storage Disease Type Ia

Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency

Screening of Chemically Distinct Lipid Nanoparticle In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells

Applied Base Editing to Treat Beta Hemoglobinopathies

Conversion of HbS to Hb G-Makassar by adenine base editing is compatible with normal hemoglobin function

CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 investigational CAR T-cell therapy for the treatment of T-cell malignancies

in vivo Base Editing Corrects Metabolic Defects in Glycogen Storage Disease Type-Ia

cccDNA inactivation using cytosine base editors

Optimization of LNP for in vivo base editing

Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases

LC MS Confirmation of Single Amino Acid Correction by Base Editing

Adenine Base Editing of Gamma Globin Gene Promoters Shows no Detectable Off-Target RNA or DNA Editing

Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

Base-Editing as a Therapeutic Approach for the Direct Correction of Disease Causing Mutations underlying Glycogen Storage Disease Type Ia

Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency

Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease

A Novel Base Editing Approach to Directly Edit the Causative Mutation in Sickle Cell Disease

Translating Base Editing Technology into a Potential Treatment for Alpha-1 Antitrypsin Deficiency

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

Directed evolution of adenine base editors with increased activity and therapeutic application

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and b-thalassemia

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Engineering of CRISPR-Cas12b for human genome editing

Search-and-replace genome editing without double-strand breaks or donor DNA

A cytosine deaminase for programmable single-base RNA editing

Base editing: precision chemistry on the genome and transcriptome of living cells

RNA editing with CRISPR-Cas13

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

Improved base excision repair inhibition and bacteriophage

Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage