ASGCT 27th Annual Meeting Abstracts
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General Session
B3 - Lysosomal Storage Diseases
Valeria Calbi1, Francesca Fumagalli1, Fabiola De Mattia1, Alberto Zambon1, Vera Gallo1, Salvatore Recupero1, Elena Sophia Fratini2, Alessia Ippolito1, Francesca Ciotti1, Maddalena Fraschini1, Marina Sarzana1, Stefano Scarparo1, Eugenio Montini1, Sara Locatelli1, Marcella Facchini1, Alessandra Clerici1, Francesco Morena3, Sabata Martino3, Muska Yarzi4, Sean L. Moro5, Andrew Shenker6, Jean Brooks4, Alan Richardson4, Laura Campbell4, Alessandro Aiuti2
1San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy,2San Raffaele Telethon Institute for Gene Therapy (SR-Tiget); Vita Salute San Raffaele University, Milan, Italy,3University of Perugia, Perugia, Italy,4Orchard Therapeutics, London, United Kingdom,5Orchard Therapeutics, Boston, MA,6Clinical Consultant, Pennington, NJ"
General Session
G2 - Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies (Including CAR-T, CAR-NK, TCR editing)
Karla F. Mullen, Daniel Grubaugh, Kayla R. Welsh, Jeffery Foss, Julia L. A. Nguyen, Nishi Gheewala, Leticia Kuri-Cervantes, James I. Andorko, Bruce C. Schnepp, Timothy D. Culp, Philip R. Johnson
Interius BioTherapeutics, Philadelphia, PA"
General Session
A1 - Viral Vectors (excluding AAV – including RNA, adenovirus, herpesvirus, bocavirus, and chimetics)
Jeff Moffit, Salvatore Iovino, Graham Casey, Leila Malkoc, Gillian Payne, Josh Merritt, Robert M. Kotin, Marc Abrams, Joel Schneider
Carbon Biosciences, Waltham, MA"
Oral Abstract Session
J2 - Cell Therapy and Cell-Based Gene Therapy Trails
Ami Shah1,2,3, Julián Sevilla4,5, Jóse Luis López Lorenzo6,7, Susana Navarro5,7,8, Lucía Llanos6,7, Belgoña Pérez de Camino Gaisse6,7, Sol Sanchez6,7, Josune Zubicaray4, Bert Glader2,3, May Chien2,3, Oscar Quintana Bustamante5,7,8, Marta Gonzalez Vicent4,5, June Iriondo4,5, Michael Rothe9, Philipp John-Neek9, Antonella Bastone9, Axel Schambach9, Miriam Zeini10, Grace Choi10, Eileen Nicoletti10, Gayatri Rao10, Maria-Grazia Roncarolo1,2,3, Juan A. Bueren5,7,8, Jonathan Schwartz10, Jose-Carlos Segovia5,7,8
1Ctr Definitive and Curative Med, Stanford Univ, Stanford, CA,2Dept Pediatrics, Div of Hematol Oncol Stem Cell Transplant Regenerative Med, Stanford Univ Sch Med, Stanford, CA,3Lucile Packard Children’s Hosp, Palo Alto, CA,4Hematol y Hemoterapia, Fundación Invest Bioméd, Hosp Infantil Univ Niño Jesús (HIUNJ), Madrid, Spain,5Ctr Invest Bioméd en Red de Enfermedades Raras (CIBERER), Madrid, Spain,6Hosp Univ Fundación Jiménez Díaz, Inst Invest Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain,7Unidad Mixta Terapias Avanzadas, Inst Invest Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain,8Unidad Innovación Bioméd, Ctr Invest Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain,9Hannover Med Sch, Hannover, Germany,10Rocket Pharmaceuticals, Cranbury, NJ"
Oral Abstract Session
F1 - Cancer - Immunotherapy and Cancer Vaccines
Renzo Perales1, Jian Yan2, Mark Yarchoan3, Edward Gane4, Thomas Marron5, Neil Cooch1, Daniel Shu6, Elana Fertig7, Luciane Kagohara8, Gabor Bartha9, Josette Northcott9, John Lyle9, Sarah Rochestie1, Joann Peters1, Jason Connor10, Elizabeth Jaffee11, Alfredo Perales-Puchalt1, David B. Weiner12, Ildiko Csiki1, Niranjan Y. Sardesai1
1Geneos Therapeutics, Philadelphia, PA,2Research & Development, Geneos Therapeutics, Philadelphia, PA,3Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD,4New Zealand Liver Transplant Unit, University of Auckland, Auckland, New Zealand,5Early Phase Trials Unit, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY,6Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD,7Department of Applied Mathematics and Statistics, Johns Hopkins University Whiting School of Engineering, Baltimore, MD,8Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD,9Personalis, Inc., Fremont, CA,10ConfluenceStat, LLC, Orlando, FL,11The Johns Hopkins University School of Medicine, Baltimore, MD,12The Wistar Institute, Philadelphia, PA"
Oral Abstract Session
J2 - Cell Therapy and Cell-Based Gene Therapy Trails
Leonid S. Metelitsa, Amy N. Courtney, Carlos A. Ramos
Baylor College of Medicine, Houston, TX"
Oral Abstract Session
J2 - Cell Therapy and Cell-Based Gene Therapy Trails
Bernhard Rudolf Gentner1, Matteo Barcella2, Francesca Farina3, Marica Eoli4, Alessia Capotondo2, Filippo Gagliardi5, Silvia Snider5, Quintino Giorgio D'Alessandris6, Valeria Cuccarini7, Elena Anghileri4, Mariagrazia Garramone2, Valentina Brambilla8, Francesco Di Meco9, Paolo Ferroli9, Valeria Ferla3, Federico Legnani9, Stefania Mazzoleni8, Alessandro Olivi6, Roberto Pallini6, Marina Grisoli7, Gaetano Finocchiaro10, Carlo Russo11, Fabio Ciceri3, Luigi Naldini2
1CHUV, Lausanne, Switzerland,2SR-Tiget, Milan, Italy,3Hematology and Bone Marrow Transplant Unit-HSR, Milan, Italy,4Neuro-Oncology Unit - INCB, Milan, Italy,5Neurosurgery Unit - HSR, Milan, Italy,6Neurosurgery Unit - Policlinico Gemelli, Rome, Italy,7Neuroradiology Unit - INCB, Milan, Italy,8Genenta Science, Milan, Italy,9Neurosurgery Unit - INCB, Milan, Italy,10Neuro-Oncology Unit - HSR, Milan, Italy,11Genenta Science, New York, NJ"
Oral Abstract Session
G2 - Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies (Including CAR-T, CAR-NK, TCR editing)
Frederic D. Bushman1, Joseph Fraietta1,2, Amy Marshall2, Jan Joseph Melenhorst3, Aoife M. Roche1, John K. Everett1, Sonja Zolnoski1, Walter Rogal2, Cory Czuczman2, The CART Therapy Team2, Carl H. June2,4, Elizabeth Hexner2, Julie Jadlowsky2
1Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA,2Center for Cellular Immunotherapies, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA,3Cleveland Clinic Lerner College of Medicine, Cleveland, OH,4Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA"
Oral Abstract Session
J1 - Gene Therapy Trials - In-Vivo Modification
Luke Roberts1, Timothy Henry2, Eugene Chung2, Monica Alvisi1, Ferzin Sethna1, David Murray3, Jay Traverse4, Lothar Roessig1, Tugba Ozkan1, Stacy Webb1, Monika Mittal1, Leigh Ervin1, Kaitlyn Walker1, Hesham Sadek5, Sheila Mikhail6, Kobra Haghighi7, Canwen Jiang1, R. Jude Samulski6, Evangelia G. Kranias7, Anna Tretiakova1, Roger Hajjar8
1Asklepios BioPharmaceutical, Inc, Durham, NC,2Christ Hospital, Cincinnati, OH,3University of Wisconsin, Madison, WI,4The Minneapolis Heart Institute, Minneapolis, MN,5UT Southwestern Medical Center, Dallas, TX,6Asklepios BioPharmaceutical, Inc., Durham, NC,7University of Cincinnati, Cincinnati, OH,8Massachusetts General Brigham, Cambridge, MA"
Oral Abstract Session
B6 - Ophthalmic and Auditory Diseases
Lawrence Lustig1, Manohar Bance2, Akira Ishiyama3, Robert Nash4, Ruben Polo5, Angel Ramos6, Manuel Jesus Manrique Rodriguez7, Jay T Rubinstein8, Tera Quigley9, Jason Riggs9, Eduardo Corrales9, Jeffery Anderson9, Vassili Valayannopoulos9
1Department of Otolaryngology - Head & Neck Surgery, Columbia University Medical Center, New York, NY,2Cambridge University, Cambridge, United Kingdom,3UCLA, Los Angeles, CA,4Great Ormond Street Hospital, London, United Kingdom,5Hospital Universitario Ramon y Cajal, Madrid, Spain,6Las Palmas University, Las Palmas Gran Canaria, Spain,7Clinica Universidad de Navarra, Pamplona, Spain,8University of Washington, Seattle, WA,9Regeneron Pharmaceuticals, New York, NY"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Michelle E. LeBlanc, Chuanqi Peng, Shashank Shukla, Mitchell R. Kopacz, Kelsey A. Rush, Jungyeon Hwang, Michael J. Crowley, Subhadeep Dutta, Hugues Bernard, Ria Vashishth, Jonathan T. Lu, Sandeep Nema, Joseph J. Higgins, Joseph J. Senn
SalioGen Therapeutics, Lexington, MA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Kartika Venugopal, Michael Magee, Alberto De Iaco, Jason Rodriguez, James Rottman, Donghui Li, Giulia Schiroli, Yu Cao, Rebecca Levy, Travis Schute, Mohit Gupta, Christina Ferren, Michael Monte, Jane Wang, Bill Querbes, Cecilia Cotta-Ramusino, Madhusudan V. Peshwa, Michael C. Holmes
Tessera Therapeutics, Somerville, MA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Jeffrey A. Ruffolo, Stephen Nayfach, Aadyot Bhatnagar, Joseph Gallagher, Joel Beazer, Jennifer Yip, Riffat Hussain, Jordan Russ, Alexander J. Meeske, Peter Cameron, Ali Madani
Profluent Bio, Berkeley, CA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Kyong-Rim Kieffer-Kwon, Jacob Layer, Zhongxia Yi, Socheata Ly, Elise Keston-Smith, Shailesh Gurung, Elizabeth Narayanan, Sierra Harken, Abril Fleitas, Aisha A. Aljanahi, Dawn Ciulla, Matthew Pandelakis, Hari Jayaram, Vlad Presnyak, Inna Shcherbakova
ReNAgade Therapeutics, Cambridge, MA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Lucas B. Harrington, Janice Chen
Mammoth Biosciences, Brisbane, CA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Amrutha Pattamatta1, Mohammad Samie2, Awdhoot Godbole3, Chen Ting-Wen1, Anagha Sawant1, Susan Ping4, Alexandra Srnka1, Siobhan Ford4, Lujain Al-Sowaimel1, Ricardos Tabet4, Robert Bell1, Lisa R. Keyes5, Sheila Rao-Dayton5, Rob Moccia3, Claudia H. Huichalaf4, David Shivak6, Bryan Zeitler2, Amy Pooler2, Christine Bulawa1
1Previous affiliation, Pfizer Inc, Cambridge, MA,2Sangamo Therapeutics, Inc., Richmond, CA,3Pfizer Inc, Cambridge, MA,4Genomic Medicine, Alexion AstraZeneca Rare Disease, Cambridge, MA,5Genomic Medicine, Alexion AstraZeneca Rare Disease, Durham, NC,6Previous affiliation, Sangamo Therapeutics Inc, Richmond, CA"
Oral Abstract Session
C6 - Gene Targeting and Gene Correction New Technologies
Le Cong1, Di Yin2, Guangxue Xu3, Yuanhao Jerry Qu1, Chengkun Wang1, Xiaotong Wang2, William Arthur Johnson4, Gabriel Filsinger2, Tim Wannier5, George M. Church6, Lai Yee Phoon7, Boya Gao7, Li Lan7
1Pathology, Genetics, Stanford University, Stanford, CA,2Stanford University, Stanford, CA,3Stanford University, Palo Alto, CA,4Stanford University Laboratory for Cell & Gene Medicine - Palo Alto, CA, Stanford, CA,5WildMicrobes, NA, CA,6Harvard Medical School, Boston, MA,7Duke University, Durham, NC"
Oral Abstract Session
A4 - AAV Vectors - Preclinical and Proof-of-Concept In-Vivo Studies (Excluding Non-Human Primates)
Daniella Munezero1, Jingting Zhu1, Arun Paripati1, Russell Chuah1, Lauren Taylor1, Afrooz Rashnonejad1,2
1Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH,2Department of Pediatrics, The Ohio State University, Columbus, OH"
Oral Abstract Session
A4 - AAV Vectors - Preclinical and Proof-of-Concept In-Vivo Studies (Excluding Non-Human Primates)
Brooke Babineau, Sheila Sears, Jennifer Su, Chao Tai, Keiko Takahashi, Adriana Gonzalez-Sandoval, Annie Tanenhaus, Pingping Jia, Rangoli Aeran, Tselmeg Amarlkhagva, Ming Chen, Dixon Hoffelt, Jason Le, Keith Place, Sammy Liang, Mark Elegue, Suresh Poda, Stephanie Tagliatela
Encoded Therapeutics, South San Francisco, CA"
Oral Abstract Session
A4 - AAV Vectors - Preclinical and Proof-of-Concept In-Vivo Studies (Excluding Non-Human Primates)
Henry Lee1, Gabrielle McGinty1, Amanda Liebhardt1, Bjoern Welzel1, Alex Zhang1, Clifford Woolf1, Guangping Gao2, Alexander Rotenberg1
1Neurology, Boston Children's Hospital, Boston, MA,2UMass Chan Medical School, Worcester, MA"
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