Posters and Presentations Our scientific advances
We are excited to share updates on our scientific progress in translating gene editing into a potentially innovative new class of precision genetic medicines.
Robust autologous CD34+ HSPC manufacturing with a closed and automated process optimized for patients with sickle cell disease
Brent Coisman, Corrina Lucini, Lillian Ye, Margaret Zhang, John Jamieson, Jordan Kim, Nate Goff, Brianna McCuaig, Kathryn Goldner, Jack Mai, Jessica Hoyer, Alexandra Shay, Jenny Olins, Ajit Kamath, Kelly Fitzpatrick, Tao Bai, Kangjian Qiao, Chris Oesterling, Paul Kopesky
Read MoreBEAM-302: Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1)
Nicolas Currier, M.D., Ph.D.
Read MoreBEAM-302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha-1 Antitrypsin Deficiency
Ayan Banerjee, Lo-I Cheng, Dominique Leboeuf, Brianna Bannister, Steven Boule, Courtney Sawyer, Valerie Winton, Jerry Decker, Carrie Curtis, Dongyu Chen, Karen Cao, Delai Chen, Bo Yan, Ronak Shah, Yi Yu, Sarah Smith, Michael Packer, Giuseppe Ciaramella
Read MoreA Single, Systemic Administration of BEAM-301 Mitigated Fasting Hypoglycemia One Year after Dosing in a Transgenic Mouse Model of Glycogen Storage Disease Type-Ia
Yvonne Aratyn-Schaus
Read MoreAdenine Base Editing enables sustained reduction of HBsAg expression and inhibition of Hepatitis B viral replication in preclinical models
Smekalova E.M., Kumar A., Combe E., Dejene S., Chen C-Y., Leboeuf D., Martinez M.G., Rees H., Barrera L.A., Ciaramella G., Packer M.S., Testoni B., Gregoire F., Zoulim F.
Read MorePaired HSC Epitope Engineering of CD117 (cKIT) for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for The Potential Treatment of Hemoglobinopathies
Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Archita Menon, Adam Wolin, Moriah White, Faith Musenge, Adam Camblin, Cristina Baricordi, Brent Coisman, Corrina Lucini, Tao Bai, Kangjian Qiao, Wayne Austin, Michelle DeLelys, Hugh Kromer, Lisa Hardy, Megan Law, Raffi Manoukian, Kiera Fallon, Mudra Patel, Antoinette Cozier, Colin Lazzara, Jeremy Decker, Tanggis Bohnuud, Seung-Joo Lee, Paul Kopesky, Sarah Smith, Charlotte McDonagh, Nicole M. Gaudelli, S. Haihua Chu, Adam Hartigan, Giuseppe Ciaramella
Read MoreIn Vivo Genetic Eye Disease Correction Using Split AAV-Mediated Adenine Base Editing
Jack Sullivan
Read MoreImproved cytosine base editors generated from TadA variant
Dieter K. Lam, Patricia R. Feliciano, Amena Arif, Tanggis Bohnuud, Thomas P. Fernandez, Jason M. Gehrke, Phil Grayson, Kin D. Lee, Manuel A. Ortega, Courtney Sawyer, Noah D. Schwaegerle, Leila Peraro, Lauren Young, Seung-Joo Lee, Giuseppe Ciaramella & Nicole M. Gaudell
Read MoreEngineered 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
Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Conrad Rinaldi, Jenny Olins, Kara Hoar, Archita Venugopal Menon, Moriah White, Adam Wolin, Faith Musenge, Adam Camblin, Brent Coisman, Tao Bai, Kangjian Qiao, Mudra Patel, Jeremy Decker, Bob Gantzer, Thomas Leete, Yi Yu, Tanggis Bohnuud, Seung-Joo Lee, Sarah Smith, Charlotte Mcdonagh, Nicole M. Gaudelli, S. Haihua Chu, Adam Hartigan, Giuseppe Ciaramella
Read MoreESCAPE-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
S. Haihua Chu, Elizabeth Budak, Nandini Mondal, Kathy Zhang, Alexander Harmon, Jeffrey Wong, Dieter Lam, Corrina Lucini, Brent Coisman, Kangjian Qiao, Tao Bai, Jeremy Decker, Bob Gantzer, Kindo Lee, Maya Sen, Tanggis Bohnuud, Luis Barrera, Brian Cafferty, Seung-Joo Lee, Paul Kopesky, Nicole M. Gaudelli, Charlotte F. McDonagh, Giuseppe Ciaramella, and Adam J. Hartigan.
Read MoreCytosine base editing inhibits Hepatitis B Virus replication and reduces HBsAg expression in vitro and in vivo
Elena Smekalova, Maria Guadalupe Martinez, Emmanuel Combe, Selam Dejene, Michael Packer, Dominique LeBoeuf, Anuj Kumar, Luis Barrera, Robert Dorkin, Rosie Chen, Giuseppe Ciaramella, Francine Gregoire, Barbara Testoni, and Fabien Zoulim
Read MoreDirected evolution and engineering of CBE-T: next-generation cytosine base editors utilizing TadA
Nicole Gaudelli, PhD
Read moreNon-genotoxic antibody-based conditioning paired with multi-plex base edited HSCs for the potential treatment of sickle cell disease
Nicole Gaudelli, PhD
Read moreEfficient LNP delivery of mRNA in vivo and ex vivo to T and NK cells
Lindsey Coholan, Elizabeth Campbell, Briana Lutz, Daniel Lee, Amanda Costa, Robert Dorkin, Cory Sago, Jason Gehrke, Guiseppe Ciaramella and Yinmeng Yang.
Read MoreSingle, Systemic Administration of BEAM 301 Mitigates Fasting Hypoglycemia and Restores Metabolic Function in a Transgenic Mouse Model of Glycogen Storage Disease Type Ia
Yvonne Aratyn Schaus
Read MoreUse of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency
Michael S Packer, Dominique Leboeuf, Lo-I Cheng, Raymond Yang, Jeremy Decker, Monique Otero, Luis A Barrera, Brian J Cafferty, Valerie J Winton, Yvonne Aratyn-Schaus, Sarah Smith, Giuseppe Ciaramella, Francine M Gregoire
Read MoreScreening of Chemically Distinct Lipid Nanoparticle In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells
Cory Sago, Elizabeth Campbell, Brianna Lutz, Neeraj Patwardhan, Gregory Hamilton, Courtney Wong, Daniel Lee, Shayon Keating, Kathryn Murray, Manmohan Singh, and Giuseppe Ciaramella
Read MoreConversion of HbS to Hb G-Makassar by adenine base editing is compatible with normal hemoglobin function
S. Haihua Chu, Manuel Ortega, Patricia Feliciano, Valerie Winton, Chavonna Xu, Daniel Haupt, Teresa McDonald, Salette Martinez, Alexander Liquori, Jeffrey Marshall, Daisy Lam, Jenny Olins, Conrad Rinaldi, Kyle Rehberger, Colin Lazarra, Jeremy Decker, Bob Gantzer, Ming Sun, Tanggis Bohnuud, David Born, Luis Barrera, Bo Yan, Ian Slaymaker, Michael S. Packer, Sarah Smith, Carlo Zambonelli, Seung-Joo Lee, Nicole M. Gaudelli, Adam J. Hartigan and Giuseppe Ciaramella.
Read MoreCD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 investigational CAR T-cell therapy for the treatment of T-cell malignancies
Yinmeng (Amy) Yang, Lindsey Coholan, Adam Camblin, Ryan Murray, Amanda Costa, David Sweezy, Faith Musenge, Mark Naniong, Hui Wu, Geoffrey Burns, Lauren Young, Yingying Zhang, Scott Haskett, Moriah White, Alden Ladd, Luis Barrera, Lisa Schlehuber, Sarah Smith, Yeh-Chuin Poh, Giuseppe Ciaramella and Jason Gehrke
Read Morein vivo Base Editing Corrects Metabolic Defects
in Glycogen Storage Disease Type-Ia by Yvonne Aratyn-Schaus
Read MorecccDNA inactivation using cytosine base editors
Elena Smekalova* 1, Maria 2, Emmanuel Combe 2, Michael Packer 1, Luis Barrera 1, Selam Dejene 1, Yvonne Aratyn-Schaus 1, Giuseppe Ciaramella 1, Barbara Testoni 2, Francine Gregoire 1, Fabien Zoulim 2 5
Read moreLC MS Confirmation of Single Amino Acid Correction by Base
Bo Yan, Valerie J Winton, S. Haihua Chu, Michael S Packer, Calvin Lee, Sarah Smith, Adam Hartigan, Carlo Zambonelli, Francine M Gregoire, Manmohan Singh, Giuseppe Ciaramella
Read moreUsing Base Editing and LNP Delivery to Correct Disease-Causi
Delai Chen,* Shailendra Sane, Raymond Yang, Robert Dorkin, Eric Zhang, Dongyu Chen, Xiao Luo, Yvonne Aratyn-Schaus, Brian Cafferty, James Tam, Krishna Ramanan, Jeffrey Marshall, Aaron Larsen, Michael S Packer, Sarah Smith, Carlo Zambonelli, Francine M Gregoire, Giuseppe Ciaramella, Manmohan Singh
Read moreAdenine Base Editing of the Sickle Allele in CD34 Hem
S. Haihua Chu, Daisy Lam, Michael S Packer, Jenny Olins , Alexander Liquori , Kyle Rehberger , Conrad Rinaldi, Jeffrey Marshall, Calvin Lee, Bo Yan, Jeremy Decker, Bob Gantzer , Scott Haskett , Tanggis Bohnuud , David Born, Luis Barrera, Ian Slaymaker , Nicole Gaudelli , Sarah Smith, Adam Hartigan and Giuseppe Ciaramella
Read moreAdenine Base Editing of Gamma Globin Gene Promoters Shows no
Ling Lin*, Lauren Young*, Jenny Olins *, Jeremy Decker, Alexander J. Liquori , S. Yingying Zhang, Haihua Chu, Daisy Lam, Conrad Rinaldi, Adrian P. Rybak, Michael S. Packer, Nicole M. Gaudelli , Luis Barrera, Jeffery Marshall, Matt Humes , Bob Gantzer , Brian Cafferty, Aaron Larson, Carlo Zambonelli , Yeh Chuin Poh , Manmohan Singh, Adam J. Hartigan, Giuseppe Ciaramella
Read moreBase-Editing as a Therapeutic Approach for the Direct Cor
Yvonne Aratyn-Schaus*,Thomas P. Fernandeza, Michael S. Packera, Lo-I Chenga, Genesis Lunga, Sarah E. Smitha, Krishna Ramanana, J. Robert Dorkina, Delai Chena, Giuseppe Ciaramellaa, Francine M Gregoirea*
Read moreA Novel Base Editing Approach to Directly Edit the Causativ
S. Haihua Chu, Michael S Packer, Jenny Olins, Genesis Lung, Lo-I Cheng, Alexander Liquori, Calvin Lee, Jeffrey Marshall, Bo Yan, Jeremy Decker, Bob Gantzer, Ling Lin, Tanggis Bohnuud, Luis Barrera, Daisy Lam, Conrad Rinaldi, Adrian Rybak, Minerva Sanchez, Yeh-Chuin Poh, Sarah Smith, Scott J Haskett, Ian Slaymaker, Nicole Gaudelli, Carlo Zambonelli, Manmohan Singh, Adam Hartigan and Giuseppe Ciaram
Read moreBase Editing of Gamma Globin Gene Promoters Generates Durabl
Adrian P. Rybak , Elsie Zahr Akrawi, Conrad Rinaldi, Scott J. Haskett, Ling Lin, Jeffrey Marshall, Alexander Liquori, Luis Barrera, Jenny Olins, S. Haihua Chu, Jeremy Decker, Minerva Sanchez, Yeh Chuin Poh, Matt Humes, Michael S. Packer, Nicole M. Gaudelli, Sarah Smith, Adam Hartigan and Giuseppe Ciaramella.
Read morein vivo Base Editing Corrects Metabolic Defects
in Glycogen Storage Disease Type-Ia by Yvonne Aratyn-Schaus
Read MoreUse of Adenine Base Editors to Precisely Correct the Dis
Michael Packera*, Vivek Chowdharyb*, Genesis Lunga, Lo-I Chenga, Yvonne Aratyn-Schaus, Sarah Smitha, Aalok Shaha, Delai Chena, Marina Ziegerb, Brian Caffertya, Bo Yana, Giuseppe Ciaramellaa, Christian Muellerb*, Francine M Gregoirea*
Read moreComplementary Base Editing Approaches for the Treatmen
Ling Lin, Adrian P. Rybak, Conrad Rinaldi, S. Haihua Chu, Jonathan Yen, Yanfang Fu, Elsie Zahr Akrawi, Sarah Smith, Scott J. Haskett, Alexander J. Liquori, Minerva E. Sanchez, Yeh-Chuin Poh, Michael S. Packer, Nicole M. Gaudelli, Jeffery Marshall, Carlo Zambonelli, Manmohan Singh, Dana N. Levasseur, Adam J. Hartigan, Giuseppe Ciaramella
Read moreCytosine base editors with minimized unguided DNA and R
Authors: Yu, Y., Leete, T.C., Born, D.A. et al
Read moreDirected evolution of adenine base editors with increased ac
Authors: Gaudelli, N.M., Lam, D.K., Rees, H.A. et al.
Read moreCytosine and adenine base editing of the brain, liver, ret
Authors: Jonathan M. Levy, Wei-Hsi Yeh, Nachiket Pendse, Jessie R. Davis, Erin Hennessey, Rossano Butcher, Luke W. Koblan, Jason Comander, Qin Liu & David R. Liu.
Read moreComplementary Base Editing Approaches for the Treatment of
Authors: Ling Lin, PhD , Adrian P. Rybak, PhD , Conrad Rinaldi, M.S. , Jonathan Yen, PhD , Yanfang Fu, PhD , Elsie Akrawi , Sarah Smith , Scott J....
Read moreBase editing: precision chemistry on the genome and transcri
Authors: Holly A. Rees and David R. Liu.
Read moreProgrammable base editing of A•T to G•C in genomic DNA with
Authors: Nicole M. Gaudelli, Alexis C. Komor, Holly A. Rees, Michael S. Packer, Ahmed H. Badran, David I. Bryson, and David R. L
Read moreScience Improved base excision repair inhibition and bact
Alexis C. Komor, Kevin T. Zhao, Michael S. Packer, Nicole M. Gaudelli, Amanda L. Waterbury, Luke W. Koblan, Y. Bill Kim, Ahmed H. Badran, and David R. Liu.
Read moreImproving the DNA specificity and applicability of base
Authors: Holly A. Rees, Alexis C. Komor, Wei-Hsi Yeh, Joana Caetano-Lopes, Matthew Warman, Albert S. B. Edge, and David R. Liu.
Read moreIncreasing the genome-targeting scope and precision of base
Authors: Y. Bill Kim, Alexis C. Komor, Jonathan M. Levy, Michael S. Packer, Kevin T. Zhao, and David R. Liu.
Read moreProgrammable editing of a target base in genomic DNA withou
Authors: Alexis C. Komor, Y. Bill Kim, Michael S. Packer, John A. Zuris, and David R. Liu.
Read moreSearch-and-replace genome editing without double-strand
Andrew V. Anzalone, Peyton B. Randolph, Jessie R. Davis, Alexander A. Sousa, Luke W. Koblan, Jonathan M. Levy, Peter J. Chen, Christopher Wilson, Gregory A. Newby, Aditya Raguram & David...
Read moreA cytosine deaminase for programmable single-base RNA editin
Omar O. Abudayyeh, Jonathan S. Gootenberg, Brian Franklin, Jeremy Koob, Max J. Kellner, Alim Ladha, Julia Joung, Paul Kirchgatterer, David B. T. Cox, Feng Zhang
Read moreRNA editing with CRISPR-Cas13
Authors: David B. T. Cox, Jonathan S. Gootenberg, Omar O. Abudayyeh, Brian Franklin, Max J. Kellner, Julia Joung, and Feng Zhang.
Read moreEngineering of CRISPR-Cas12b for human genome editing
Jonathan Strecker, Sara Jones, Balwina Koopal, Jonathan Schmid-Burgk, Bernd Zetsche, Linyi Gao, Kira S. Makarova, Eugene V. Koonin & Feng Zhang
Read more