EDITAS MEDICINE’S NEWSROOM

R. Hanna, M.D., et al. Reni-cel, the first AsCas12a gene-edited cell therapy, led to hemoglobin normalization and increased fetal hemoglobin in severe sickle cell disease patients in an interim analysis of the RUBY trial. Presented at the European Hematology Association (EHA) Annual Congress. June 13-16, 2024.

H. Frangoul, M.D., M.S., et al. Reni-cel, the first AsCas12a gene-edited cell therapy, shows promising preliminary results in key clinical outcomes in transfusion-dependent beta thalassemia patients treated in the EdiThal trial. Presented at the European Hematology Association (EHA) Annual Congress. June 13-16, 2024.

MacLeod, Heather. LNP-Based Delivery of CRISPR/Cas12a for the Potential Treatment of Myocilin-Associated Glaucoma. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024.

Li, Hui, et al. Metagenomic Discovery and Screening of Novel Recombinase Proteins for Targeted Integration. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024.

Wrighton, Paul J., et al. Chemically Modified AsCas12a Guide RNAs Improve Lipid Nanoparticle–Mediated In Vivo Gene Editing in Different Tissues. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024.

H. Frangoul, MD, et al, AsCas12a gene editing of HBG1/2 promoters with EDIT-301 (reni-cel) results in rapid and sustained normalization of hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease and transfusion-dependent beta-thalassemia. Presented at the 2024 Tandem Meetings of ASTCT and CIBMTR. February 21-24, 2024.

L.I. Jansson-Fritzberg, et al. Characterizing a more efficient AsCas12a. Presented at the Precision Genome Engineering Keystone Symposia: Delivery of Nucleic Acid Therapeutics. January 22-25, 2024.

R. Hanna, M.D., et al. AsCas12a gene editing of HBG1/2 promoters with EDIT-301 results in rapid and sustained normalization of hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease and transfusion-dependent beta-thalassemia. Presented at the American Society of Hematology (ASH) Annual Meeting and Exposition. December 9-12, 2023.

EDIT-301 Program Update: RUBY and EdiTHAL Trial Data Update. Presented in a Company-sponsored Webinar. December 11, 2023.

JN Lemercier. Characterization of guide RNAs for CRISPR Applications. Presented at The 2023 CRISPR-Based Therapy Analytical Development Summit. October 30 – November 1, 2023.

EDIT-301 Program Update: RUBY and EdiTHAL Trial Data Update. Presented in a Company-sponsored Virtual Event. June 12, 2023.

R. Hanna, M.D., et al. EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of Patients with Severe Sickle Cell Disease Using Highly Specific and Efficient AsCas12a Enzyme. Presented at the European Hematology Association Hybrid Congress. June 8 -16, 2023.

Bright S. Ashimatey, et al. Twelve-month Natural History Study of CEP290-associated Retinal Degeneration. Presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. April 23 – 27, 2023.

Pavlina Wolf. PCR Methods for the Characterization of AAV-based Drug Product from Titer to Potency. Presented at Gene Therapy Analytical Development 2022. November 30, 2022.

Alexander G. Allen, et al. EDIT-202, a multiplexed AsCas12a-edited iPSC-differentiated iNK, displays a mature phenotype, high KIR expression, and ADCC towards multiple solid tumor lines. Presented at The Society for Immunotherapy of Cancer (SITC) Annual Meeting, November 8-12, 2022.

Michael Nehil. Genetic Editing of iNK Cell Therapies to Enhance Tumor Killing Capacity. Presented at The iPSC-Derived Immunotherapies Congress 2022. October 19, 2022.

Mariacarmela Allocca, et al. A Mutation-Independent CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa. Presented at The European Society of Gene and Cell Therapy (ESGCT) Annual Meeting, October 11-14, 2022.

Samia Q. Khan, et al. EDIT-202, an AsCas12a and SLEEK™ gene-edited iPSC-derived NK cell therapy maintains prolonged persistence, high cytotoxicity, and enhanced in vivo control of solid tumors. Presented at The European Society of Gene and Cell Therapy (ESGCT) Annual Meeting, October 11-14, 2022.

Ramya Viswanathan, et al. SLEEK—A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next-generation Cell-based Medicines. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 24-27, 2022.

Chrysa Latrick, et al. Elucidating the impact of RNA secondary structure on RNP behavior. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 24-27, 2022.

Bryant Chica, et al. Characterization of CRISPR RNPs by Intrinsic Fluorescence and Ion Exchange Chromatography. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 24-27, 2022.

Steven Wolk. Characterization of gRNAs and Ribonucleoproteins for CRISPR Applications. Presented at Gene Therapy Analytical Development Europe 2022, May 30 – June 1, 2022.

Zoe (Chi-Hsiu) Liu, et al. A Mutation-independent CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP). Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 16-19, 2022.

John Zuris, et al. SLEEK: A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next-Generation Cell-Based Medicines. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 16-19, 2022.

Samia Q. Khan, et al. EDIT-202, a Multiplexed CRISPR-Cas12a Gene-Edited iPSC-Derived NK Cell Therapy, has Prolonged Persistence, Promotes High Cytotoxicity, and Enhances In Vivo Tumor Killing. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 16-19, 2022.

Brian R. Duke, et al. Exploratory Immuno-Safety Profile of EDIT-101, a First-in-Human In Vivo CRISPR Gene Editing Therapy for CEP290-Related Retinal Degeneration. Presented at The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 16-19, 2022.

John Zuris. A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next-Generation Cell-Based Medicines. Presented at TIDES USA Oligonucleotide & Peptide Therapeutics. May 9 – 13, 2022.

Mark S. Shearman. Ongoing Progress in the Phase 1/2 BRILLIANCE Clinical Trial for Treatment of CEP-290 Retinal Degeneration: In vivo CRISPR gene editing therapies for ocular disease. Presented at TIDES USA Oligonucleotide & Peptide Therapeutics. May 9 – 13, 2022.

Zoe (Chi-Hsiu) Liu, et al. A Mutation-independent CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP). Presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-4, 2022

Michael C. Jaskolka, et al. Exploratory Safety Profile of EDIT-101, a First-in-Human in vivo CRISPR Gene Editing Therapy for CEP290-Related Retinal Degeneration. Presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-4, 2022

Alexander G. Allen, et al. AsCas12a gene-edited iPSC-derived NK cells constitutively expressing CD16 and membrane-bound IL-15 demonstrate prolonged persistence and robust anti-tumor activities in a solid tumor mouse model. Presented at The American Association for Cancer Research (AACR) Annual Meeting 2022, April 8 – 13, 2022.

Samia Q. Khan, Alexandra Gerew, et al. Knock-out of CISH and TGFβR2 or knock-in of CD16 and mbIL-15 in iPSC-derived NK cells promotes high cytotoxicity and enhances in vivo tumor killing. Presented at The 63rd Annual Meeting and Exposition of the American Society of Hematology. December 11-14, 2021.

Patricia Sousa, et al. Preclinical development of EDIT-301, an autologous cell therapy comprising AsCas12a-RNP-modified mobilized peripheral blood CD34+ cells for the potential treatment of transfusion-dependent beta thalassemia. Presented at The 63rd Annual Meeting and Exposition of the American Society of Hematology. December 11-14, 2021.

Alexander G. Allen, et al. GAPDH knock-in of high-affinity CD16 and mbIL-15 in iPSC-derived NK cells drives high-level expression and increased anti-tumor function. Presented at The Society for Immunotherapy of Cancer (SITC) Annual Meeting 2021, November 10-14, 2021.

John Zuris. An Engineered AsCas12a Nuclease Facilitates the Rapid Generation of Therapeutic Cell Medicines. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021.

Keith Jarvis. IND-enabling Small-Scale Guide RNA Production Under GMP for CRISPR Based Cell Therapies. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021.

Steve Wolk. Characterization of gRNAs and Ribonucleoproteins for CRISPR Applications. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021.

Jean-Noel Lemercier, et al. Characterization of CRISPR RNPs by Ion-Exchange Chromatography. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021.

Pranjali Ghude, et al. Understanding CRISPR RNA Secondary Structure Impact on Ribonucleoprotein (RNP) Behavior by SEC-PAGE. Presented at TIDES USA Oligonucleotide and Peptide Therapeutics, September 20-23, 2021.

J. A. Zuris. A Method for Highly Efficient Knock-in and Expression of Transgene Cargos for Next Generation Cell-based Medicine. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 18-20, 2021.

Eugenio Marco et al. CALITAS: a CRISPR-Cas-aware ALigner for In silico off-TArget Search. Presented at Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting, August 18-20, 2021.

Edouard De Dreuzy, et al. Preclinical Data Supporting the Initiation of the EDIT-301 Phase I/II RUBY Clinical Trial for the Potential Treatment of Sickle Cell Disease. Presented at the 26th Congress of the European Hematology Association (EHA), June 9 – 17, 2021.

M. Allocca, D. Reyon, et al. Advances Toward a Dual AAV CRISPR-Cas9-based “Knockout and Replace” Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa. The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 11 – 14, 2021

H. MacLeod. The development of CRISPR-based medicines for the treatment of ocular diseases. The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, May 11 – 14, 2021

N. Dilmac, et al. CRISPR Gene Editing Rescues Deficits in Human USH2A Mutant Retinal Organoids. The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-7, 2021

M. Allocca, et al. Advances Toward a Dual AAV CRISPR-Cas9-based “Knockout and Replace” Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa. The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, May 1-7, 2021

K. Wong, et al. CRISPR-Cas12a Gene Editing Enhances Functional Metabolism of Natural Killer Cells and Enables Tumor Cell Cytolysis in Metabolically Stressful Conditions That Inhibit Effector Cell Function. Presented at the American Association for Cancer Research (AACR) Annual Meeting, April 10 – 15, 2021

Christopher M. Borges, et al. A Multiplexed CRISPR-Cas12a Gene-Edited Healthy Donor-Derived NK Cell Therapy with Increased Granzyme B and Degranulation Supports Improved Serial Killing Capacity. Presented at the American Association for Cancer Research (AACR) Annual Meeting, April 10 – 15, 2021

Jung-Il Moon, et al. Generation of Natural Killer Cells with Enhanced Function from a CRISPR/Cas12a-Edited Induced Pluripotent Stem Cell Line. Presented at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH), December 5-8, 2020.

Edouard De Dreuzy, et al. Robust Pre-Clinical Results and Large-Scale Manufacturing Process for EDIT-301: An Autologous Cell Therapy for the Potential Treatment of SCD. Presented at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH), December 5-8, 2020.

Christopher M. Borges, et al. Preclinical Development of EDIT-201, a Multigene Edited Healthy Donor NK Cell with Enhanced Anti-Tumor Function and Superior Serial Killing Activity in an Immunosuppressive Environment. Presented at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH), December 5-8, 2020.

K. Wong, et al. Preclinical Development of EDIT-201, a Multiplexed CRISPR-Cas12a Gene Edited Healthy Donor Derived NK Cell Therapy Demonstrating Improved Persistence and Resistance to the Tumor Microenvironment. Presented at The Society for Immunotherapy of Cancer (SITC) 35th Annual Meeting, November 9-14, 2020.

Jack Heath, et al. EDIT-301: An Autologous Cell Therapy to Promote Fetal Hemoglobin Expression for the Potential Treatment of Sickle Cell Disease. Presented at the 25th Congress of the European Hematology Association (EHA), June 11-21, 2020.

J.A. Zuris, et al. Highly Efficient Multi-Gene Knockout and Transgene Knock-in using CRISPR-Cas12a in Induced Pluripotent Stem Cells for the Generation of Engineered Cell Immunotherapies. Presented at the 23rd ASGCT Annual Meeting, May 12-15, 2020.

Swati Mukherjee, et al. In Vivo Proof of Concept for EDIT-102: A CRISPR/Cas9-Based Experimental Medicine for USH2A-Related Inherited Retinal Degeneration Caused by Mutations in Exon 13. Presented at the 23rd ASGCT Annual Meeting, May 12-15, 2020.

Abhishek Dass, et al. Dual AAV-based ‘Knock-out-and-replace’ of RHO as a Therapeutic Approach to Treat RHO-associated Autosomal Dominant Retinitis Pigmentosa (RHO adRP). Presented at the 23rd ASGCT Annual Meeting, May 12-15, 2020.

Eugenio Marco, et al. CALITAS: a CRISPR/Cas-aware ALigner for In silico off-TArget Search. Presented at the 23rd ASGCT Annual Meeting, May 12-15, 2020.

J .A. Zuris. Best Practices for Achieving Optimal Ex Vivo Genome Editing in Your Research. Presented at the 23rd ASGCT Annual Meeting, May 12-15, 2020.

Richard A. Morgan. Ex vivo applications of Cas12a. Presented at the Keystone Symposia: Engineering the Genome, February 8-12, 2020.

Edouard De Dreuzy, et al. EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD. Presented at the 61st Annual Meeting and Exposition of the American Society of Hematology (ASH). December 7-10, 2019.

C.M. Margulies, et al. Developing a CRISPR/Cas9 Editing Approach for the Treatment of USH2A Related Inherited Retinal Degeneration. Presented at the European Society for Gene and Cell Therapy 27th Annual Congress (ESGCT), October 22-25, 2019.

Gregory Gotta, et al. SaCas9 and AsCas12a (AsCpf1) are as potent and more specific than SpCas9. Presented at Cold Spring Harbor Laboratory: Genome Engineering: Frontiers of CRISPR/Cas, October 10-13, 2019.

Jack Heath, et al. Genome Editing of BG1/2Promoter Leads to Robust HbF Induction In Vivo, While Editing of BCL11A Erythroid Enhancer Results in Erythroid Defects. Presented at the 24th Congress of the European Hematology Association (EHA), June 13 – 16, 2019.

J.A. Zuris, et al. Strategies for multi-gene editing and reduction of translocations with CRISPR-Cpf1 in T cells for the development of improved cell therapies. Presented at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting, April 29 – May 2, 2019.

Vic Myer. Controlling Rearrangement Frequencies in the Context of Multigene Genome Editing. Presented at Keystone Symposia on Molecular and Cellular Biology – Genome Engineering: From Mechanisms to Therapies, February 19-23, 2019.

Kai-Hsin Chang, et al. Genome Editing of HBG1/2 Promoter Leads to Robust HbF Induction In Vivo While Editing of BCL11A Erythroid Enhancer Shows Erythroid Defect. Presented at the 60th ASH Annual Meeting & Exposition, December 1 – 4.

John A. Zuris, et al. Highly Efficient Single and Multi-gene Knockout with CRISPR-Cpf1 in T Cells for the Development of Improved Cell Therapies. Presented at the European Society of Gene & Cell Therapy (ESGCT) 26th Annual Congress, October 16 – 19.

Michael Stefanidakis, et al. Preclinical Assessment of In Vivo Gene Editing Efficiency, Specificity, and Tolerability of EDIT-101, an Investigational CRISPR Treatment for Leber Congenital Amaurosis 10 (LCA10). Presented at the European Society of Gene & Cell Therapy (ESGCT) 26th Annual Congress, October 16 – 19.

Shen Shen, et al. Treatment of Herpetic Keratitis with CRISPR/Cas9 Gene Editing in a Rabbit Disease Model. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

G. Grant Welstead, et al. Improving Efficacy of CAR T Cells through CRISPR/Cas9 Mediated Knockout of TGF?RII. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Edouard de Dreuzy, et al. Potent HbF Induction Following ssODN-mediated Repair of Cas9-induced DSB at the HBG Promoter in CD34+ HSPC. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Kai-Hsin Chang, et al. Saturated Mutagenesis Surrounding Beta-globin Locus Identifies Novel Therapeutic Targets for Fetal Globin Induction and Treatment of Sickle Cell Anemia. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Carrie M. Margulies, et al. Efficient Targeted Integration in Human T Cells with CRISPR-Cas9 for the Treatment of X-Linked Hyper-IgM Syndrome. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Jiang H, et al. Evaluation of Tolerability and Immunogenicity of EDIT-101 Following Subretinal Injection in Non- human Primates. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Christopher J. Wilson, et al. Gene Editing Specificity Assessment for EDIT-101, an LCA10 Therapeutic Candidate. Presented at the American Society of Gene & Cell Therapy (ASGCT) 21st Annual Meeting, May 16 – 19.

Terence Ta. Development of High Quality CRISPR/Cas9 Agents. Presented at TIDES: Oligonucleotide and Peptide Therapeutics, May 7 – 10.

Vic Myer. gRNA Quality for CRISPR Medicines. Presented at TIDES: Oligonucleotide and Peptide Therapeutics, May 7 – 10.

Christopher M. Owens, et al. CRISPR/Cas9 Targeted Disruption of Herpes Simplex Virus type 1 in a Rabbit Latency Model Reduces Viral Reactivation and Associated Corneal Pathology. Presented at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting, April 29 – May 3, 2018

Michael Stefanidakis, et al. Efficient in vivo editing of CEP290 IVS26 by EDIT-101 as a novel therapeutic for the treatment of Leber Congenital Amaurosis 10. Presented at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting, April 29 – May 3, 2018.

Christopher Wilson. Methods for Gene Editing Measurement and Off-Target Discovery. Presented at NIST-FDA Genome Editing Workshop, April 23-24, 2018.

Charles Albright, Ph.D. Discovery of EDIT-101 for the Treatment of Leber’s Congenital Amaurosis Type 10. Presented at Keystone Symposia on Molecular and Cellular Biology: Precision Genome Editing with Programmable Nucleases, January 28 – February 1, 2018.

John Zuris. Highly efficient editing with CRISPR-Cpf1 in primary T cells and HSCs. Presented at Keystone Symposia on Molecular and Cellular Biology: Precision Genome Editing with Programmable Nucleases, January 28 – February 1, 2018.

Aditi Chalishazar, et al. Expanding CRISPR Genome Editing Strategies in Hematopoietic Stem and Progenitor Cells for the Treatment of Hematologic Diseases. Presented at the 59th ASH Annual Meeting & Exposition, December 9-12, 2017.

Cecilia Fernandez. CRISPR Genome Editing: Considerations for Therapeutic Applications. Presented at EuroTIDES, Vienna, Austria, November 7-10, 2017.

Michael Stefanidakis, et al. Development of a Subretinally Delivered CEP290-Specific CRISPR Medicine for the Treatment of Leber Congenital Amaurosis 10 (LCA10), October 19, 2017.

Junxia Wang. Development of Multiplex Sensitive Anti-Drug Antibody Assays for CRISPR/Cas9 Gene Therapies, September 27, 2017.

Grant Welstead. The Development of CRISPR Based Medicines for the Treatment of Hematological Diseases, September 20, 2017.

Cecilia Cotta-Ramusino. Characterization of Genomic Rearrangements in Response to CRISPR/Cas9-Induced Double-Stranded Breaks. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017.

Maxwell N. Skor. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017.

Eugenio Marco, et al. UDiTasTM: A streamlined genome editing detection method for on- and off-target edits, large deletions, and translocations. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017.

Eric Tillotson, et al. An in vitro method for benchmarking of CRISPR-associated endonucleases. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017.

Barrett Steinberg, et al. Directed evolution platforms and applications for engineering RNA-guided nucleases. Presented at Synthetic Biology: Engineering Evolution & Design (SEED) 2017, Vancouver, BC, June 22, 2017.

Hari Jayaram, et al. Towards predictable editing: influence of target sequence, cell type, and choice of type II nuclease on desired repair outcomes. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017.

Barrett Steinberg, et al. Directed evolution of Cas9 to reduce identified off-target cleavage. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017.

John Zuris, et al. Comparison of RNP-mediated editing by Type V Cpf1variants across multiple cell types. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017.

Hari Jayaram. Defining and characterizing the components of a CRISPR-Cas9 genomic medicine. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017.

Shannon Boye, et al. Efficient In Vivo Gene Editing of Inherited Retinal Disease Genes in Mice and Non-Human Primates. Presented at the ASGCT 20th Annual Meeting, May 13, 2017.

Georgia Giannoukos, et al. UDiTaS(TM): A streamlined genome editing detection method for on- and off-target edits, large deletions, and translocations. Presented at the ASGCT 20th Annual Meeting, May 12, 2017.

Jennifer Gori. CRISPR-mediated Editing of Hematopoietic Stem Cells for the Treatment of Beta-Hemoglobinopathies. Presented at the ASGCT 20th Annual Meeting, May 11, 2017.

Carrie M. Margulies, et al. Characterization of Targeted Integration with Viral and Non-Viral DNA Donors. Presented at the ASGCT 20th Annual Meeting, May 11, 2017.

P. Singhal, et al. Friedland. Self-inactivating Cas9: a method for reducing exposure while maintaining efficacy in virally-delivered Cas9 applications. Presented at the ASGCT 20th Annual Meeting, May 11, 2017.

Shen Shen, et al. Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice. Presented at the ASGCT 20th Annual Meeting, May 10, 2017.

Haiyan Jiang. Opportunities and Challenges in Development of CRISPR Medicines. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017.

Derek Cerchione, et al. Directed evolution of targeted Cas9 cleavage to the LCA10 splice donor mutation. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017.

Eric Tillotson, et al. Assessing Cas9-gRNA ribonucleoprotein complex formation for development of ex-vivo therapeutics. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017.

Guillaume Harmange, et al. Probing the Quality of Cas9 Ribonucleoprotein Complex Using Biochemistry and Biophysics. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017.

Ramya Viswanathan, et al. Comparison of RNP-mediated editing by Type V Cpf1 variants. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017.

Christopher Wilson. Advancing CRISPR Technologies for Therapeutic Application. Presented at the TIDES Oligonucleotide & Peptide Therapeutics conference, May 3, 2017.

Cecilia Fernandez. Genome Editing: Considerations for Therapeutic Applications. Presented at the TIDES Oligonucleotide & Peptide Therapeutics conference, May 3, 2017.

Christopher Wilson, et al. A Genome Editing Lead Finding Platform for Therapeutics. Cambridge, MA.

Anne Bothmer, et al. DNA Repair Factor Overexpression Screen Identifies Factors Required for Repair Pathway Choice. Presented at Keystone Symposia on Molecular and Cellular Biology: Genomic Instability and DNA Repair, April 2-6, 2017.

Cecilia Cotta-Ramusino, et al. Effect of Different CRISPR/Cas9 Variants on Repair Pathway Choice. Presented at Keystone Symposia on Molecular and Cellular Biology: Genomic Instability and DNA Repair, April 2-6, 2017.

Anne Bothmer, et al. Characterization of the Interplay Between DNA Repair and CRISPR/Cas9-Induced DNA Lesions. Presented at Keystone Symposia on Molecular and Cellular Biology: Precision Genome Engineering, January 8—12, 2017.

Vic Myer. Advancing CRISPR Technologies for Therapeutic Application. Presented at the Keystone Genome Editing Meeting, January 5, 2017.

J.M. Heath, et al. Efficient CRISPR/Cas9 Mediated Gene Editing in Long-Term Engrafting Human Hematopoietic Stem Progenitor Cells. Presented at the American Society of Hematology Annual Meeting, December 3-6, 2016 in San Diego, CA.

J.M Heath, et al. Highly Efficient CRISPR/Cas9 Mediated Gene Editing in Long-Term Engrafting Human Hematopoietic Stem Progenitor CellsA. Presented at the European Society of Gene and Cell Therapy Annual Meeting, October 18-21, 2016 in Florence, Italy.

David Bumcrot. Advancing CRISPR Medicines: Challenges and Solutions. Presented at the Oligonucleotide Therapeutics Society Annual Meeting, September 25-28 in Montreal, Canada.

Morgan Maeder. Envisioning a Gene Editing Approach to Treat Inherited Blindness. Presented at the Federation of American Societies For Experimental Biology, June 5-10 in Lisbon, Portugal.

Grant Welstead, et al. Successful Generation of CAR+PD-1- Primary T Cells Using Cas9-Mediated Genome Editing. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C.

Tanushree Phadke, et al. DNA Ends Matter: The Impact of Using CRISPR/Cas9 Variants on DNA Repair Pathway Choice and Editing Profiles at The HBB Locus. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C.

Josh Tycko, et al. Screening S. aureus CRISPR-Cas9 Paired Guide RNAs for Efficient Targeted Deletion in Duchenne Muscular Dystrophy. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C.

Morgan L. Maeder, et al. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C.

J. M. Heath, et al. Highly Efficient CRISPR/Cas9 Gene Editing and Long-Term Engraftment of Human Hematopoietic Stem and Progenitor Cells. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C.

Morgan L. Maeder, et al. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at the Association for Research in Vision and Ophthalmology (ARVO), May 1-5, 2016 in Seattle, WA.

C. Cotta-Ramusino, et al. Therapeutic Editing of the HBB Locus Using the Endogenous HBD Locus as a Donor Template. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 15, 2015.

A.E. Friedland, et al. Staphyloccocus aureus Cas9 an alternative Cas9 for genome editing applications. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 15, 2015.

G. Grant Welstead, et al. Characterization of Cas9-mediated genome editing in human T cells. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 14, 2015.

William Selleck, et al. Biophysical Characterization and Direct Delivery of S. pyogenes Cas9 Ribonucleoprotein Complexes. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 13, 2015.

J.L. Gori, et al. Cas9-mediated genome editing in hematopoietic stem/progenitor cells. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 13, 2015.

C. Cotta-Ramusino, et al. Gene targeting of the HBB locus by Crispr/Cas9 to investigate repair pathway choice in response to different types of DNA lesions. Presented at Keystone Symposium for Genomic Instability and DNA Repair, March 2, 2015.

A.E. Friedland, et al. Staphyloccocus aureus Cas9: An alternative Cas9 for genome editing applications. Presented at Keystone Symposium for Precision Genome Engineering and Synthetic Biology, January 12, 2015.

A.E. Friedland, et al. Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications. Published in Genome Biology (2015) 16:257.

Konermann, S., et al. Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex. NCBI, December 10, 2014. doi:10.1038/nature14136. [Epub ahead of print]

Zuris, J.A., et al. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nature Biotechnology, October 30, 2014. doi: 10.1038/nbt.3081. [E-pub ahead of print]

Swiech, L., et al. In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nature Biotechnology, October 19, 2014. doi: 10.1038/nbt.3055. [E-pub ahead of print]

Platt, R.J., et al. CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling. Cell, October 9, 2014. doi: http://dx.doi.org/10.1016/j.cell.2014.09.014. [E-pub ahead of print]

O’Connell, M.R., et al. Programmable RNA recognition and cleavage by CRISPR/Cas9. Nature, September 28, 2014. doi: 10.1038/nature13769. [E-pub ahead of print]

Sanjana, N.E., et al. Improved vectors and genome-wide libraries for CRISPR screening. Nature Methods, August 11, 2014. doi:10.1038/nmeth.3047. [E-pub ahead of print]

Guell, M., et al. Genome editing assessment using CRISPR Genome Analyzer (CRISPR-GA). Bioinformatics, July 1, 2014. doi: 10.1093/bioinformatics/btu427. [E-pub ahead of print]

Hsu, P.D., et al. Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell, June 6, 2014. doi: http://dx.doi.org/10.1016/j.cell.2014.05.010. [E-pub ahead of print]

Guilinger, J.P., et al. Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification. Nature Biotechnology, April 25, 2014. doi:10.1038/nbt.2909. [E-pub ahead of print]

Tsai, S.Q., et al. Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nature Biotechnology, April 25, 2014. doi:10.1038/nbt.2908. [E-pub ahead of print]

Sander, J.D., et al. CRISPR-Cas systems for editing, regulating and targeting genomes. Nature Biotechnology, March 2, 2014. doi:10.1038/ntb.2842. [E-pub ahead of print]

Nishimasu, H., et al. Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA. Cell, Feb. 13, 2014. DOI: 10.1016/j.cell.2014.02.001. [E-pub ahead of print]

Jinek, M., et al. Structures of Cas9 endonucleases reveal RNA-mediated conformational activation. Science, Feb. 6, 2014. DOI: 10.1126/science.1247997. [Epub ahead of print]

Fu Y., et al. Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nature Biotechnology, 2014 Jan 26; doi: 10.1038/nbt2808. [Epub ahead of print]

Sternberg, et al. DNA interrogation by the CRISPR RNA-guided endonuclease Cas9. Nature (2014) doi:10.1038/nature13011. [Epub ahead of print]

O. Shalem, et al. Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells. Science. doi: 10.1126/science.1247005. Epub 2013 Dec 12.

K.M. Esvelt, et al. Church. Orthogonal Cas9 proteins for RNA-guided gene regulation and editing. Nat Methods. 2013 Nov;10(11):1116-21.

O. Niewoehner, et al. Evolution of CRISPR RNA recognition and processing by Cas6 endonucleases. Nucleic Acids Res. 2013 Oct 22

P. Mali, et al. Cas9 as a versatile tool for engineering biology. Nat Methods. 2013 Oct;10(10):957-63.

M.L. Maeder, et al. Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins. Nat Biotechnol. 2013 Dec;31(12):1137-42. doi: 10.1038/nbt.2726. Epub 2013 Oct 9.

Y. Qi, et al. Targeted deletion and inversion of tandemly arrayed genes in Arabidopsis thaliana using zinc finger nucleases. G3 (Bethesda). 2013 Oct 3;3(10):1707-15.

E.M. Mendenhall, et al. Locus-specific editing of histone modifications at endogenous enhancers. Nat Biotechnol. 2013 Dec;31(12):1133-6. doi: 10.1038/nbt.2701. Epub 2013 Sep 8.

F.A. Ran, et al. Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity. Cell. 2013 Aug 29. doi:10.1016/j.cell.2013.08.021.

Y.B. Tzur, et al. Heritable Custom Genomic Modifications in Caenorhabditis elegans via a CRISPR–Cas9 System. Genetics. Advance online publication. 2013 Aug 26. doi:10.1534/genetics.113.156075.

Z. Feng, et al. Efficient genome editing in plants using a CRISPR/Cas system. Cell Research. 2013 Aug 20. doi:10.1038/cr.2013.114.

J.D. Sander, et al. In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites. Nucleic Acids Research. 2013 Aug 14. doi:10.1093/nar/gkt716.

V. Pattanayak, et al. High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nature Biotechnology. 2013 Aug 11. doi:10.1038/nbt.2673.

T. Lo, et al. Precise and Heritable Genome Editing in Evolutionarily Diverse Nematodes Using TALENs and CRISPR/Cas9 to Engineer Insertions and Deletions. Genetics. Advance online publication. 2013 Aug 9. doi:10.1534/genetics.113.155382.

J. Li, et al. Multiplex and homologous recombination–mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9. Nature Biotechnology. 2013 Aug 8. doi:10.1038/nbt.2654.

P. Mali, et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nature Biotechnology. 2013 Aug 1. doi:10.1038/nbt.2675.

L. Yang, et al. Optimization of scarless human stem cell genome editing. Nucleic Acids Research. 2013 July 31. doi: 10.1093/nar/gkt555.

M.L. Maeder, et al. CRISPR RNA–guided activation of endogenous human genes. Nature Methods. 2013 July 25. doi:10.1038/nmeth.2598.

S. Konermann, et al. Optical control of mammalian endogenous transcription and epigenetic states. Nature. 2013 July 23. doi:10.1038/nature12466.

P.D. Hsu, et al. DNA targeting specificity of RNA-guided Cas9 nucleases. Nature Biotechnology. 2013 July 21. doi:10.1038/nbt.2647.

L.A. Gilbert, et al. CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes. Cell. 2013 July 11.10.1016/j.cell.2013.06.044.

W.Y. Hwang, et al. Heritable and Precise Zebrafish Genome Editing Using a CRISPR-Cas System. PLOS One. 2013 July 9. doi: 10.1371/journal.pone.0068708.

D. Reyon, et al. Engineering Customized TALE Nucleases (TALENs) and TALE Transcription Factors by Fast Ligation-Based Automatable Solid-Phase High-Throughput (FLASH) Assembly. Current Protocols in Molecular Biology. 2013 July 1. doi:10.1002/0471142727.mb1216s103.

A.E. Friedland, et al. Heritable genome editing in C. elegans via a CRISPR-Cas9 system. Nature Methods. 2013 June 30. doi:10.1038/nmeth.2532.

Y. Fu, et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nature Biotechnology. 2013 June 23. doi:10.1038/nbt.2623.

X. Li, et al. piggyBac transposase tools for genome engineering. Proc Natl Acad Sci U S A. 2013 Jun 18;110(25):E2279-87.

D. Bikard, et al. Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system. Nucleic Acids Research. 2013 June 12. doi: 10.1093/nar/gkt520.

H.Wang, et al. One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering. 2013 May 2. doi:10.1016/j.cell.2013.04.025.

M.J. Osborn, et al. TALEN-based Gene Correction for Epidermolysis Bullosa. Molecular Therapy. 2013 April 2. doi:10.1038/mt.2013.56.

H.Y. Lee, et al. RNA-protein analysis using a conditional CRISPR nuclease. Proc Natl Acad Sci U S A. 2013 Apr 2;110(14):5416-21.

M.L. Maeder, et al. Robust, synergistic regulation of human gene expression using TALE activators. Nat Methods. 2013 March 10. doi: 10.1038/nmeth.2366.

E. Charpentier, et al. Biotechnology: Rewriting a genome. Nature. 2013 Mar 7;495(7439):50-1.

J.E. DiCarlo, et al. Genome engineering in Saccharomyces cerevisiae using CRISPR-Cas systems. Nucleic Acids Research. 2013 March 4. doi: 10.1093/nar/gkt135.

L.S. Qi, et al. Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression. Cell. 28 February 2013. 10.1016/j.cell.2013.02.022.

W.Y. Hwang, et al. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nature Biotechnology. 2013 Jan 29. doi:10.1038/nbt.2501.

W. Jiang, et al. RNA-guided editing of bacterial genomes using CRISPR-Cas systems. Nature Biotechnology. 2013 January 29. doi:10.1038/nbt.2508.

P. Mali, et al. RNA-Guided Human Genome Engineering via Cas9. Science. 2013 Jan 3. doi:10.1126/science.1232033.

L. Cong, et al. Multiplex Genome Engineering Using CRISPR/Cas Systems. Science. 2013 Jan 3. doi:10.1126/science.1231143.

J.K. Joung, et al. TALENs: a widely applicable technology for targeted genome editing. Nature Reviews Molecular Cell Biology. 2013 Jan. doi:10.1038/nrm3486.

M. Jinek, et al. RNA-programmed genome editing in human cells. Elife. 2013;2:e00471.

D. Reyon, et al. Engineering Designer Transcription Activator–Like Effector Nucleases (TALENs) by REAL or REAL-Fast Assembly. Current Protocols in Molecular Biology. 2012 Oct 1. doi: 10.1002/0471142727.mb1215s100.

A.S. Khalil, et al. A synthetic biology framework for programming eukaryotic transcription functions. Cell. 2012 Aug 3;150(3):647-58.

C.L. Ramirez, et al. Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects. Nucleic Acids Res. 2012 Jul;40(12):5560-8.

M. Jinek, et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012 Aug 17;337(6096):816-21. doi: 10.1126/science.1225829. Epub 2012 Jun 28.