Abstracts

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 Aiuti<sup>2

1</sup>San 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 Shah^1,2,3, Julián Sevilla^4,5, Jóse Luis López Lorenzo^6,7, Susana Navarro^5,7,8, Lucía Llanos^6,7, Belgoña Pérez de Camino Gaisse^6,7, Sol Sanchez^6,7, Josune Zubicaray⁴, Bert Glader^2,3, May Chien^2,3, Oscar Quintana Bustamante^5,7,8, Marta Gonzalez Vicent^4,5, June Iriondo^4,5, Michael Rothe⁹, Philipp John-Neek⁹, Antonella Bastone⁹, Axel Schambach⁹, Miriam Zeini¹⁰, Grace Choi¹⁰, Eileen Nicoletti¹⁰, Gayatri Rao¹⁰, Maria-Grazia Roncarolo^1,2,3, Juan A. Bueren^5,7,8, Jonathan Schwartz¹⁰, Jose-Carlos Segovia<sup>5,7,8

1</sup>Ctr 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. Sardesai<sup>1

1</sup>Geneos 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 Naldini<sup>2

1</sup>CHUV, 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 Fraietta^1,2, Amy Marshall², Jan Joseph Melenhorst³, Aoife M. Roche¹, John K. Everett¹, Sonja Zolnoski¹, Walter Rogal², Cory Czuczman², The CART Therapy Team², Carl H. June^2,4, Elizabeth Hexner², Julie Jadlowsky<sup>2

1</sup>Microbiology, 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 Hajjar<sup>8

1</sup>Asklepios 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 Valayannopoulos<sup>9

1</sup>Department 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 Bulawa<sup>1

1</sup>Previous 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 Lan<sup>7

1</sup>Pathology, 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 Rashnonejad<sup>1,2

1</sup>Center 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 Rotenberg<sup>1

1</sup>Neurology, Boston Children's Hospital, Boston, MA,²UMass Chan Medical School, Worcester, MA"