Volume 24, Supplement 1, May 2016 The Journal of the American Society of Gene & Cell Therapy
American Society of Gene & Cell Therapy 19th Annual Meeting
AMERICAN SOCIETY OF GENE & CELL THERAPY 19th Annual Meeting May 4 - 7, 2016 Washington, DC, USA
Abstract Reviewers Abstract Committee Chair Andre Lieber, MD, PhD University of Washington Seattle, WA A1-RNA Virus Vectors Abstract Review Chair Grant D. Trobridge, PhD Reviewers Carol H. Miao, PhD Axel Schambach, MD, PhD John F. Tisdale, MD Bruce E. Torbett, PhD, MSPH Andrew C. Wilber, PhD A2-AAV Vectors Abstract Review Chair R. Jude Samulski, PhD Reviewers John F. Engelhardt, PhD Douglas M. McCarty, PhD Philippe Moullier, MD, PhD Nicholas Muzyczka, PhD* David V. Schaffer, PhD* A3-Adenovirus and Other DNA Virus Vectors Abstract Review Chair Andrew P. Byrnes, PhD Reviewers Julia Davydova, MD, PhD* Anja Ehrhardt, PhD Florian Kreppel, PhD Hiroyuki Mizuguchi, PhD* Xiangyang Zhou, PhD B-Gene Targeting and Gene Correction Abstract Review Chair Andrew M. Scharenberg, MD* Reviewers Marguerite V. EvansGalea, PhD* Charles A. Gersbach, PhD Dirk Grimm, PhD Punam Malik, MD Rita Perlingeiro, PhD
May 4-7, 2016 Washington, DC
Wednesday, May 4, 2016 Scientific Symposium 100, 8:00 AM to 10:00 AM Amyotrophic Lateral Sclerosis (ALS): Potential Therapeutic Targets in Novel Disease Mechanisms Chair(s): John H. Wolfe, VMD, PhD Speaker(s): Nicholas Boulis, MD, Don W. Cleveland, PhD, Ronald L. Klein, PhD, Philip C. Wong, PhD
Scientific Symposium 101, 8:00 AM to 10:00 AM Frontiers in Cardiovascular Gene and Cell Therapy
Chair(s): Seppo Yla-Herttuala, MD, PhD, FESC Speaker(s): Charles R. Bridges, MD, ScD, Marinee K.L. Chuah, PhD, Michael Regnier, PhD, Lior Zangi, PhD
Scientific Symposium 102, 8:00 AM to 10:00 AM Gene Therapy Product Development in the Modern Era: Current Regulatory Issues Chair(s): Patrick Au, PhD, Joy Cavagnaro, PhD, DABT, ATS Speaker(s): Wilson W. Bryan, MD, Denise K. Gavin, PhD, Maura C. O’Leary, MD, Mercedes Serabian, MS, DABT
Scientific Symposium 103, 8:00 AM to 10:00 AM Integrating Combination Approaches to Modulate the Immune Response to Cell and Gene Therapies Chair(s): Conrad Russell Y. Cruz, MD, PhD, Renata Stripecke, PhD Speaker(s): Maria G. Castro, PhD, Hildegund CJ Ertl, MD, Roland W. Herzog, PhD
Scientific Symposium 104, 8:00 AM to 10:00 AM Targeting the Liver with Gene and Cell Therapeutics
Chair(s): Federico Mingozzi, PhD, Dao Pan, PhD Speaker(s): Alberto Auricchio, MD, Federico Mingozzi, PhD, Charles P. Venditti, MD, PhD, Thomas Wechsler, PhD
Special Symposium 105, 8:00 AM to 10:00 AM Special Symposium on Concepts and Clinical Applications of Genome Editing - Part I: Science
Chair(s): J. Keith Joung, MD, PhD Speaker(s): Jennifer A. Doudna, PhD, Jennifer A. Doudna, PhD, Michael C. Holmes, PhD, Michael C. Holmes, PhD, J. Keith Joung, MD, PhD, J. Keith Joung, MD, PhD, Duanqing Pei, PhD, Duanqing Pei, PhD
Special Symposium 105, 10:15 AM to 12:15 PM Special Symposium on Concepts and Clinical Applications of Genome Editing - Part II: Ethics
Chair(s): Theodore Friedmann, MD, Nelson A. Wivel, MD Speaker(s): George J. Annas, JD, MPH, George J. Annas, JD, MPH, Robert M. CookDeegan, MD, Robert M. Cook-Deegan, MD, Thomas H. Murray, PhD, Thomas H. Murray, PhD
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
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PROGRAM Oral Abstract Session 110, 10:30 AM to 12:00 PM RNA Virus Vectors............................................................................................................................. S1 Chair(s): Carol H. Miao, PhD, Bruce E. Torbett, PhD, MSPH 1 2 3 4 5 6
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 111, 10:30 AM to 12:00 PM AAV Vector Biology........................................................................................................................... S4 Chair(s): Jeffrey S. Bartlett, PhD, Guangping Gao, PhD 7 8 9 10 11 12
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 112, 10:30 AM to 12:00 PM Adenoviruses....................................................................................................................................... S7 Chair(s): Andrew P. Byrnes, PhD, Julia Davydova, MD, PhD 13 14 15 16 17 18
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 113, 10:30 AM to 12:00 PM Cardiovascular and Pulmonary Diseases......................................................................................... S9 Chair(s): Carolyn Lutzko, PhD, Christian Mueller, PhD 19 20 21 22 23 24
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 114, 10:30 AM to 12:00 PM Delivery Questions for Neural Applications................................................................................ S12 Chair(s): Alberto Auricchio, MD, Andrea Contestabile, PhD 25 26 27 28 29 30
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 115, 10:30 AM to 12:00 PM Pre-clinical Development Activities for Gene Therapy Trials................................................... S14 Chair(s): Gwendolyn K. Binder-Scholl, PhD, Susan C. Stevenson, PhD 31 32 33 34 35 36
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10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
C-Oligonucleotide Therapeutics Abstract Review Chair Bruce Sullenger, PhD Reviewers Maxim V. Berezovski, PhD* Paloma H. Giangrande, PhD Ryszard Kole, PhD* Anton P. McCaffrey, PhD Marco S. Weinberg, PhD D- Chemical/Molecular Conjugates and Physical Methods for Delivery of Gene Therapeutics Abstract Review Chairs Dexi Liu, PhD Ernst Wagner, PhD* Reviewers Julie Gehl, MD, Dr.Med.Sci.* Stephen L. Hart, PhD Richard Heller, PhD Kenya Kamimura, MD, PhD* Olivia Merkel, PhD* Millicent O. Sullivan, PhD E1-Diabetes, Metabolic and Genetic Diseases Abstract Review Chair Gerald S. Lipshutz, MD Reviewers Nicola Brunetti-Pierri, MD Randy J. Chandler, PhD, MB* Stephanie Cherqui, PhD Cary O. Harding, MD Dwight Koeberl, MD, PhD E2-Cardiovascular and Pulmonary Diseases Abstract Review Chair Dieter C. Gruenert, PhD Reviewers Bruce A. Bunnell, PhD David A. Dean, PhD Karin M.L. Gaensler, MD* Mauro Giacca, MD, PhD* William B. Guggino, PhD Carolyn Lutzko, PhD E3-Neurologic Diseases (including Ophthalmic and Auditory Diseases) Abstract Review Chair Jean Bennett, MD, PhD Reviewers Alberto Auricchio, MD Beverly L. Davidson, PhD John Forsayeth, PhD Guangping Gao, PhD Luk H. Vandenberghe, PhD
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM E4-Musculo-skeletal Diseases Abstract Review Chair Terence A. Partridge, PhD Reviewers Joel R. Chamberlain, PhD Dongsheng Duan, PhD Paul Gregorevic, PhD* Scott Q. Harper, PhD Peter L. Jones, PhD* E5-Cancer – Immunotherapy, Cancer Vaccines Abstract Review Chair Malcolm K. Brenner, MD, PhD* Reviewers Karen S. Aboody, MD Chiara Bonini, MD Ann M. Leen, PhD Marcela V. Maus, MD, PhD Barbara Savoldo, MD, PhD E6-Cancer – Oncolytic Viruses Abstract Review Chair Samuel D. Rabkin, PhD Reviewers John C. Bell, PhD Evanthia Galanis, MD, DSc Kah-Whye Peng, PhD* Stephen H. Thorne, PhD* Hiroaki Wakimoto, MD, PhD* E7-Cancer - Targeted Gene and Cell Therapy Abstract Review Chair Maria C. Castro, PhD Reviewers Irina V. Balyasnikova, PhD* Juan Fueyo, MD G. Yancey Gillespie, PhD Paola Grandi, PhD Balveen Kaur, PhD
Lunch Break (On Own – Not Provided), 12:00 PM to 1:30 PM Industry Lunch Symposium 120, 12:00 PM to 1:30 PM Industry Lunch Symposium Education Session 130, 1:30 PM to 3:00 PM Emerging Field Review: Towards Therapeutic Applications with Nanoparticle Formulations of Nucleic Acids
Chair(s): Rajagopal Ramesh, PhD, Millicent O. Sullivan, PhD Speaker(s): Muthiah Manoharan, PhD, W. Mark Saltzman, PhD, Assem G. Ziady, PhD
Education Session 131, 1:30 PM to 3:00 PM Topical Review: AAV and Adenovirus Gene Transfer
Chair(s): Aravind Asokan, PhD, Dirk Grimm, PhD Speaker(s): Aravind Asokan, PhD, Douglas M. McCarty, PhD, Dmitry M. Shayakhmetov, PhD
Education Session 132, 1:30 PM to 3:00 PM Topical Review: Advances/Alternatives in Genome Therapies
Chair(s): Richard P. Harbottle, PhD Speaker(s): Richard P. Harbottle, PhD, Zoltan Ivics, PhD, Natalay Y. Kouprina, PhD
Education Session 133, 1:30 PM to 3:00 PM Topical Review: Gene Therapy for Non Malignant Diseases
Chair(s): John F. Tisdale, MD, PhD Speaker(s): Daniel E. Bauer, MD, PhD, Gerd A. Blobel, MD, PhD, Giuliana Ferrari, PhD
Education Session 134, 1:30 PM to 3:00 PM Topical Review: Orphan Diseases
Chair(s): Bobby Gaspar, MD, PhD Speaker(s): Nathalie Cartier-Lacave, MD, Paul Lasko, PhD, Rachel Salzman, DVM
Oral Abstract Session 140, 3:30 PM to 5:30 PM Targeted Genome Editing: Gene Editing in Hematopoietic Cells........................................... S16 Chair(s): Marguerite V. Evans-Galea, PhD, Punam Malik, MD 37 38 39 40 41 42 43 44
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 141, 3:30 PM to 5:30 PM Diabetes, Metabolic and Genetic Diseases................................................................................... S20 Individual speaker presentation titles will be included in the 19th Annual Meeting Program Guide, which will be distributed at the meeting in Washington, DC. Please see the Program Guide for comprehensive information, including room assignments, speaker order and individual presentation titles.
Chair(s): Randy J. Chandler, PhD, MB, Cary O. Harding, MD 45 46 47 48 49 50 51 52
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
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PROGRAM Oral Abstract Session 142, 3:30 PM to 5:30 PM Gene Therapy for CNS Diseases................................................................................................... S23 Chair(s): Jean Bennett, MD, PhD, Guangping Gao, PhD 53 54 55 56 57 58 59 60
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 143, 3:30 PM to 5:30 PM Cancer-Oncolytic RNA Viruses..................................................................................................... S27 Chair(s): Kah-Whye Peng, PhD, Samuel D. Rabkin, PhD 61 62 63 64 65 66 67 68
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 144, 3:30 PM to 5:30 PM Cancer-Targeted Gene and Cell Therapy...................................................................................... S30 Chair(s): Irina V. Balyasnikova, PhD, Paola Grandi, PhD 69 70 71 72 73 74 75 76
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 145, 3:30 PM to 5:30 PM Immunological Aspects of Gene Therapy I: AAV Vectors....................................................... S34 Chair(s): Ashley T. Martino, PhD, Pedro Lowenstein, MD, PhD 77 78 79 80 81 82 83 84
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3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
E8-Hematologic and Immunologic Diseases Abstract Review Chair Harry L. Malech, MD Reviewers Jennifer E. Adair, PhD Alessandro Aiuti, MD, PhD Gay Crooks, MD John T. Gray, PhD Karin Pike-Overzet, PhD F-Immunological Aspects of Gene Therapy Abstract Review Chair Michael A. Barry, PhD Reviewers Pedro Lowenstein, MD, PhD Ashley T. Martino, PhD* Dmitry M. Shayakhmetov, PhD Renata Stripecke, PhD David B. Weiner, PhD G- Cell Therapies Abstract Review Chair David W. Russell, MD, PhD Reviewers Magali Cucchiarini, PhD* Andre Larochelle, MD, PhD* Jan A. Nolta, PhD Eirini P. Papapetrou, MD, PhD Tim Townes, PhD* H1- Vector and Cell Engineering, Production or Manufacturing Abstract Review Chair Boro Dropulic, PhD Reviewers Bryan T. Butman, PhD* Maria Mercedes Segura, PhD* Vladimir A. Slepushkin, PhD* J. Fraser Wright, PhD H2-Pharmacology/ Toxicology Studies or Assay Development Abstract Review Chair Gene Liau, PhD* Reviewers: Gwendolyn K. Binder-Scholl, PhD* Linda B. Couto, PhD Susan C. Stevenson, PhD* Gabor Veres, PhD Teresa Wright, PhD*
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM H3-Clinical Protocol Development, Regulatory Interactions, and Ethics Abstract Review Chair Phillip B. Maples, PhD* Reviewers Dale G. Ando, MD Marina Cavazzana-Calvo, MD, PhD Rimas J. Orentas, PhD* Isabelle Riviere, PhD David Stroncek, MD* Special thanks to the abstract reviewers for their time and expertise! *Indicates first time reviewer for ASGCT. To volunteer to review abstracts for the ASGCT 20th Annual Meeting, visit the ASGCT Membership booth.
Exhibit Hall Welcome Reception & Poster Session I, 5:30 PM to 7:30 PM AAV Vectors I................................................................................................................................... S37 (Abstracts 85 through 102; Abstract 96 moved to Poster Session II after Abstract 310)
Adenovirus Vectors and Other DNA Virus Vectors.................................................................. S44 (Abstracts 103 through 117)
Targeted Genome Editing I............................................................................................................ S49
(Abstracts 118 through 142; Abstract 120 moved to Oral Abstract Session 411 after Abstract 732)
Oligonucleotide Therapeutics I...................................................................................................... S58 (Abstracts 143 through 156; Abstracts 147 & 151 have been withdrawn)
Diabetes, Metabolic and Genetic Diseases I................................................................................. S61 (Abstracts 157 through 171)
Cardiovascular and Pulmonary Diseases....................................................................................... S67 (Abstracts 172 through 183; Abstract 175 has been withdrawn)
Gene Therapy for Neurosensory Diseases................................................................................... S72 (Abstracts 184 through 189)
Cancer-Immunotherapy, Cancer Vaccines I.................................................................................. S74 (Abstracts 190 through 206)
Cancer-Targeted Gene and Cell Therapy I................................................................................... S80 (Abstracts 207 through 219)
Hematologic & Immunologic Diseases I...................................................................................... S86 (Abstracts 220 through 233)
Cell Therapies I................................................................................................................................. S91 (Abstracts 234 through 246; Abstract 237 has been withdrawn)
Reconnection and Mentoring Events, 7:30 PM to 9:30 PM Industry Symposium 150, 7:30 PM to 9:30 PM Industry Symposium NIH Symposium 151, 7:30 PM to 9:30 PM The Interface of Academia, the NIH, Foundations, and Biopharm/Biotech: Best Practices to Ensure the Continued Growth and Success of Gene and Cell Therapies
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
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PROGRAM
Thursday, May 5, 2016 Scientific Symposium 200, 8:00 AM to 10:00 AM AAV, Adenovirus, and Lentiviral Vectors Updates
Chair(s): Jeffrey A. Medin, PhD, Stephen J. Russell, MD, PhD Speaker(s): Christian J. Buchholz, PhD, Kenneth Cornetta, MD, Clodagh O’Shea, PhD, Arun Srivastava, PhD
Scientific Symposium 201, 8:00 AM to 10:00 AM Clinical Trials Spotlight ................................................................................................................... S97 Chair(s): Jean Bennett, MD, PhD, Donald B. Kohn, MD 247 248 249 250
8:00 AM 8:20 AM 8:40 AM 9:00 AM
Scientific Symposium 202, 8:00 AM to 10:00 AM Gene Therapies for Inherited and Metabolic Diseases
Chair(s): Stephanie Cherqui, PhD, Hiroyuki Nakai, MD, PhD Speaker(s): Alessandra Biffi, MD, Beverly L. Davidson, PhD, Christian Mueller, PhD, Dao Pan, PhD
Scientific Symposium 203, 8:00 AM to 10:00 AM Recent Advances in Human Stem Cell Research and Therapy
Chair(s): Hans-Peter Kiem, MD Speaker(s): Jason Butler, PhD, Danwei Huangfu, PhD, Carolyn Lutzko, PhD, Frank McKeon, PhD, Renee Reijo Pera, PhD
Scientific Symposium 204, 8:00 AM to 10:00 AM Translational Research and Product Development through AcademicIndustry Partnerships: Models for Success
Chair(s): Gwendolyn K. Binder-Scholl, PhD, H. Trent Spencer, PhD Speaker(s): Gwendolyn K. Binder-Scholl, PhD, Katherine A. High, MD, Carl H. June, MD, Fulvio Mavilio, PhD
Exhibit Hall Coffee Social – with Oral Poster Session, 9:45 AM – 10:45 AM George Stamatoyannopoulos Lecture 210, 10:45 AM to 12:00 PM George Stamatoyannopoulos Lecture & Presentation of the Excellence in Research Awards Chair(s): Cynthia Dunbar, MD Speaker(s): David R. Liu, PhD
Lunch Break (On Own – Not Provided), 12:00 PM to 1:30 PM Industry Lunch Symposium 220, 12:00 PM to 1:30 PM Industry Lunch Symposium Outstanding New Investigator Symposium 230, 1:30 PM to 3:30 PM Outstanding New Investigator Symposium
Chair(s): Terence R. Flotte, MD Speaker(s): Jordan Green, PhD, Marcin Kortylewski, PhD, Eirini P. Papapetrou, MD, PhD, Junghae Suh, PhD
Exhibit Hall Coffee Social, 3:30 PM to 4:15 PM
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM Oral Abstract Session 240, 4:00 PM to 5:45 PM Evolving Better Parvoviral Vectors for Gene Therapy............................................................... S99 Chair(s): John F. Engelhardt, PhD, David V. Schaffer, PhD 251 252 253 254 255 256 257
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 241, 4:00 PM to 5:45 PM Oligonucleotide Therapeutics I....................................................................................................S102 Chair(s): Ryszard Kole, PhD, William H. Thiel, PhD 258 259 260 261 262 263 264
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 242, 4:00 PM to 5:45 PM Gene Therapy for Neurosensory Diseases.................................................................................S105 Chair(s): Beverly L. Davidson, PhD, Luk H. Vandenberghe, PhD 265 266 267 268 269 270 271
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 243, 4:00 PM to 5:45 PM Cancer-Immunotherapy, Cancer Vaccines I................................................................................S108 Chair(s): Karen S. Aboody, MD, Malcolm K. Brenner, MD, PhD 272 273 274 275 276 277 278
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 244, 4:00 PM to 5:45 PM Hematologic & Immunologic Diseases I....................................................................................S111 Chair(s): John T. Gray, PhD, Karin Pike-Overzet, PhD 279 280 281 282 283 284 285
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 245, 4:00 PM to 5:45 PM Vector and Cell Engineering/Manufacturing.............................................................................S115 Chair(s): Vladimir Slepushkin, PhD, J. Fraser Wright, PhD 286 287 288 289 290 291 292 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
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PROGRAM Exhibit Hall Networking Reception & Poster Session II (including Tools and Technology Forum), 5:45 PM to 7:45 PM AAV Vectors II................................................................................................................................S118 (Abstracts 293 through 310; Abstract 96 moved from Poster Session I)
Targeted Genome Editing II.........................................................................................................S125 (Abstracts 311 through 334)
Non-Viral Gene Transfer and Therapy I....................................................................................S133 (Abstracts 335 through 345)
Diabetes, Metabolic and Genetic Diseases II.............................................................................S138 (Abstracts 346 through 360)
Gene Therapy for CNS Diseases.................................................................................................S144 (Abstracts 361 through 376)
Musculo-Skeletal Diseases I..........................................................................................................S150 (Abstracts 377 through 390; Abstract 384 has been withdrawn; Abstract 622 has been moved from Poster Session III)
Cancer-Immunotherapy, Cancer Vaccines II..............................................................................S155 (Abstracts 391 through 408)
Cancer-Oncolytic Viruses I...........................................................................................................S162 (Abstracts 409 through 423)
Immunological Aspects of Gene Therapy I...............................................................................S168 (Abstracts 424 through 441)
Cell Therapies II..............................................................................................................................S175 (Abstracts 442 through 454; Abstract 450 has been withdrawn)
Vector and Cell Engineering/Manufacturing I..........................................................................S180 (Abstracts 455 through 466)
Pharmacology/Toxicology Studies or Assay Development.....................................................S184 (Abstracts 467 through 477)
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM
Friday, May 6, 2016 Scientific Symposium 300, 8:00 AM to 10:00 AM Basic Biology of Nanoparticles and Interaction with Cells
Chair(s): Stephen L. Hart, PhD, Assem G. Ziady, PhD Speaker(s): Jordan Green, PhD, Judy Lieberman, MD, PhD, Joachim Rädler, PhD, Millicent O. Sullivan, PhD
Scientific Symposium 301, 8:00 AM to 10:00 AM Cancer Therapeutics: Targeting the Target
Chair(s): John C. Bell, PhD, Evanthia Galanis, MD, DSc Speaker(s): Renier J. Brentjens, MD, PhD, Michael Caligiuri, MD, Naoto Hirano, MD, PhD, Lili Yang, PhD
Scientific Symposium 302, 8:00 AM to 10:00 AM Cell Therapy to Improve the Outcome of Hemopoietic Stem Cell Transplant
Chair(s): Helen E. Heslop, MD, Elizabeth J. Shpall, MD Speaker(s): David E. Avigan, MD, Austin J. Barrett, MD, Colleen Delaney, MD, MSC, Katy Rezvani, MD, PhD
Scientific Symposium 303, 8:00 AM to 10:00 AM Emerging and Transnational Clinical Trials
Chair(s): Ian E. Alexander, MBBS, PhD, Thierry C. VandenDriessche, PhD Speaker(s): Luigi M. Naldini, MD, PhD, Waseem Qasim, MRCP, PhD, Masayo Takahashi, MD, PhD, Takanori Yamagata, MD, PhD
Scientific Symposium 304, 8:00 AM to 10:00 AM Evolution of Gene & Cell Therapy Enabling Technologies - Challenges for Clinical Applications
Chair(s): Robert E. Sobol, MD, Gabor Veres, PhD Speaker(s): Jean Bennett, MD, PhD, Dan S. Kaufman, MD, PhD, Howard L. Kaufman, MD, Matthew H. Porteus, MD, PhD
Exhibit Hall Coffee Social – with Oral Poster Session, 9:45 AM – 10:45 AM Outstanding Achievement Award Lecture 310, 10:45 AM to 11:45 AM Outstanding Achievement Award Lecture with Sonia Skarlatos Public Service Award Presentation Chair(s): Terence R. Flotte, MD Speaker(s): Seppo Yla-Herttuala, MD, PhD, FESC
Lunch Break (On Own – Not Provided), 11:45 AM to 1:15 PM Presidential Symposium 320, 1:15 PM to 3:30 PM Presidential Symposium & Presentation of the Top Abstracts...............................................S189 Chair(s): Michel Sadelain, MD, PhD Speaker(s): Alain Fischer, MD, Theodore Friedmann, MD
Presentation of Top Abstracts: 478 479 480
2:30 PM 2:50 PM 3:10 PM
Exhibit Hall Coffee Social, 3:30 PM to 4:15 PM
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PROGRAM Oral Abstract Session 330, 4:00 PM to 6:00 PM Targeted Genome Editing: In Vivo Genome Editing..............................................................S190 Chair(s): Rita Perlingeiro, PhD, Andrew M. Scharenberg, MD 481 482 483 484 485 733 487 488
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM (Moved from Oral Session 411) 5:30 PM 5:45 PM
Oral Abstract Session 331, 4:00 PM to 6:00 PM Chemical and Physical Methods for Delivery of Gene Therapeutics....................................S194 Chair(s): Stephen L. Hart, PhD, Ernst Wagner, PhD 489 490 491 492 493 494 495 496
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 332, 4:00 PM to 6:00 PM Musculo-skeletal Diseases..............................................................................................................S197 Chair(s): Scott Q. Harper, PhD, Terence A. Partridge, PhD 497 498 499 500 501 502 503 504
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 333, 4:00 PM to 6:00 PM Cancer-Immunotherapy, Cancer Vaccines II..............................................................................S201 Chair(s): Attilio Bondanza, MD, Philip D. Gregory, DPhil 505 506 507 508 509 510 511 512
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 334, 4:00 PM to 6:00 PM Cancer-Oncolytic DNA Viruses...................................................................................................S204 Chair(s): Stephen H. Thorne, PhD, Hiroaki Wakimoto, MD PhD 513 514 515 516 517 518 519 520
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4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM Oral Abstract Session 335, 4:00 PM to 6:00 PM Cell Therapies..................................................................................................................................S208 Chair(s): Andre Larochelle, MD, PhD, Eirini P. Papapetrou, MD, PhD 521 522 523 524 525 526 527 528
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Exhibit Hall Networking Reception & Poster Session III (including Tools and Technology Forum), 6:00 PM to 8:00 PM RNA Virus Vectors.........................................................................................................................S211 (Abstracts 529 through 538)
AAV Vectors III..............................................................................................................................S215 (Abstracts 539 through 556)
Targeted Genome Editing III.......................................................................................................S223 (Abstracts 557 through 579; Abstract 578 has been withdrawn)
Oligonucleotide Therapeutics II...................................................................................................S231 (Abstracts 580 through 592)
Non-Viral Gene Transfer and Therapy II...................................................................................S234 (Abstracts 593 through 603)
Delivery Questions for Neural Applications..............................................................................S239 (Abstracts 604 through 621)
Musculo-Skeletal Diseases II.........................................................................................................S246 (Abstracts 623 through 636; Abstract 622 has been moved to Poster Session II after Abstract 390)
Cancer-Immunotherapy, Cancer Vaccines III............................................................................S252 (Abstracts 637 through 653)
Cancer-Oncolytic Viruses II..........................................................................................................S259 (Abstracts 654 through 667)
Cancer-Targeted Gene and Cell Therapy II...............................................................................S264 (Abstracts 668 through 680)
Hematologic & Immunologic Diseases II..................................................................................S269 (Abstracts 681 through 693)
Immunological Aspects of Gene Therapy II: AAV Vectors..................................................S274 (Abstracts 694 through 702)
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PROGRAM Vector and Cell Engineering/Manufacturing II.........................................................................S278 (Abstracts 703 through 714)
Clinical Protocol Development, Regulatory Interactions, and Ethics....................................S282 (Abstracts 715 through 720)
Closing Night Reception at the Newseum – Ticket Required, 8:00 PM to 10:00 PM
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM
Saturday, May 7, 2016 ASGCT Business Meeting and Coffee Break, 7:30 AM to 8:00 AM Education Session 400, 8:00 AM to 10:00 AM Emerging Field Review: The Basics of Genome Editing
Chair(s): Thomas Wechsler, PhD Speaker(s): Charles A. Gersbach, PhD, Miguel Gonzalez-Blanco, PhD, Andrew M. Scharenberg, MD
Education Session 401, 8:00 AM to 10:00 AM Topical Review: How to Fund Gene Therapy Research
Chair(s): Terry R. Bishop, PhD Speaker(s): Terry R. Bishop, PhD, Hal E. Broxmeyer, PhD, Scott Q. Harper, PhD
Education Session 402, 8:00 AM to 10:00 AM Topical Review: T cell Immunotherapy
Chair(s): Hans-Peter Kiem, MD Speaker(s): Catherine M. Bollard, MD, Helen E. Heslop, MD, Shannon L. Maude, MD, PhD, Pin Wang, PhD
Scientific Symposium 403, 8:00 AM to 10:00 AM Advances in Musculoskeletal Gene and Cell Therapy
Chair(s): Gloria Matthews, DVM, PhD, DACVS Speaker(s): Jeffrey S. Chamberlain, PhD, James D. Kang, MD, Rita Perlingeiro, PhD, Bing Wang, MD, PhD
Scientific Symposium 404, 8:00 AM to 10:00 AM Advances in Oligonucleotide Diagnostics and Therapy
Chair(s): Paloma H. Giangrande, PhD, Martin J. Hicks, PhD Speaker(s): Elizabeth Ackermann, PhD, Brian D. Adams, PhD, Edward M. Kaye, MD, Amy Simon, MD
Scientific Symposium 405, 8:00 AM to 10:00 AM Physical Delivery Driving the Development of Innovative Molecular Therapeutics
Chair(s): Ernst Wagner, PhD, Matthew H. Wilson, MD, PhD Speaker(s): Daniel G. Anderson, PhD, Julie Gehl, MD, Dr.Med.Sci., Alexander Marson, MD, PhD, Michael J. Passineau, PhD
Oral Abstract Session 410, 10:15 AM to 12:15 PM AAV Vectors and Pre-clinical Analysis........................................................................................S284 Chair(s): Aravind Asokan, PhD, Eduard Ayuso 721 722 723 724 725 726 727 728
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
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PROGRAM Oral Abstract Session 411, 10:15 AM to 12:15 PM Targeted Genome Editing: Methods and Technology..............................................................S287 Chair(s): Charles A. Gersbach, PhD, Dirk Grimm, PhD 729 730 731 732 120 734 735 736
10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
(Moved from Poster Session I)
Oral Abstract Session 412, 10:15 AM to 12:15 PM Oligonucleotide Therapeutics II...................................................................................................S290 Chair(s): John Burnett, PhD, Marco S. Weinberg, Ph.D. 737 738 739 740 741 742 743 744
10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
Oral Abstract Session 413, 10:15 AM to 12:15 PM Cancer-Immunotherapy, Cancer Vaccines III............................................................................S294 Chair(s): Marcela V. Maus, MD, PhD, Barbara Savoldo, MD, PhD 745 746 747 748 749 750 751 752
10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
Oral Abstract Session 414, 10:15 AM to 12:15 PM Hematologic & Immunologic Diseases II..................................................................................S297 Chair(s): Jennifer E. Adair, PhD, Gay Crooks, MD 753 754 755 756 757 758 759 760
10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
Oral Abstract Session 415, 10:15 AM to 12:15 PM Immunological Aspects of Gene Therapy II.............................................................................S301 Chair(s): Renata Stripecke, PhD, David B. Weiner, PhD 761 762 763 764 765 766 767 768
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10:15 AM 10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM 12:00 PM
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
American Society of Gene & Cell Therapy 19th Annual Meeting
AMERICAN SOCIETY OF GENE & CELL THERAPY 19th Annual Meeting May 4 - 7, 2016 Washington, DC, USA
Abstract Reviewers Abstract Committee Chair Andre Lieber, MD, PhD University of Washington Seattle, WA A1-RNA Virus Vectors Abstract Review Chair Grant D. Trobridge, PhD Reviewers Carol H. Miao, PhD Axel Schambach, MD, PhD John F. Tisdale, MD Bruce E. Torbett, PhD, MSPH Andrew C. Wilber, PhD A2-AAV Vectors Abstract Review Chair R. Jude Samulski, PhD Reviewers John F. Engelhardt, PhD Douglas M. McCarty, PhD Philippe Moullier, MD, PhD Nicholas Muzyczka, PhD* David V. Schaffer, PhD* A3-Adenovirus and Other DNA Virus Vectors Abstract Review Chair Andrew P. Byrnes, PhD Reviewers Julia Davydova, MD, PhD* Anja Ehrhardt, PhD Florian Kreppel, PhD Hiroyuki Mizuguchi, PhD* Xiangyang Zhou, PhD B-Gene Targeting and Gene Correction Abstract Review Chair Andrew M. Scharenberg, MD* Reviewers Marguerite V. EvansGalea, PhD* Charles A. Gersbach, PhD Dirk Grimm, PhD Punam Malik, MD Rita Perlingeiro, PhD
May 4-7, 2016 Washington, DC
Wednesday, May 4, 2016 Scientific Symposium 100, 8:00 AM to 10:00 AM Amyotrophic Lateral Sclerosis (ALS): Potential Therapeutic Targets in Novel Disease Mechanisms Chair(s): John H. Wolfe, VMD, PhD Speaker(s): Nicholas Boulis, MD, Don W. Cleveland, PhD, Ronald L. Klein, PhD, Philip C. Wong, PhD
Scientific Symposium 101, 8:00 AM to 10:00 AM Frontiers in Cardiovascular Gene and Cell Therapy
Chair(s): Seppo Yla-Herttuala, MD, PhD, FESC Speaker(s): Charles R. Bridges, MD, ScD, Marinee K.L. Chuah, PhD, Michael Regnier, PhD, Lior Zangi, PhD
Scientific Symposium 102, 8:00 AM to 10:00 AM Gene Therapy Product Development in the Modern Era: Current Regulatory Issues Chair(s): Patrick Au, PhD, Joy Cavagnaro, PhD, DABT, ATS Speaker(s): Wilson W. Bryan, MD, Denise K. Gavin, PhD, Maura C. O’Leary, MD, Mercedes Serabian, MS, DABT
Scientific Symposium 103, 8:00 AM to 10:00 AM Integrating Combination Approaches to Modulate the Immune Response to Cell and Gene Therapies Chair(s): Conrad Russell Y. Cruz, MD, PhD, Renata Stripecke, PhD Speaker(s): Maria G. Castro, PhD, Hildegund CJ Ertl, MD, Roland W. Herzog, PhD
Scientific Symposium 104, 8:00 AM to 10:00 AM Targeting the Liver with Gene and Cell Therapeutics
Chair(s): Federico Mingozzi, PhD, Dao Pan, PhD Speaker(s): Alberto Auricchio, MD, Federico Mingozzi, PhD, Charles P. Venditti, MD, PhD, Thomas Wechsler, PhD
Special Symposium 105, 8:00 AM to 10:00 AM Special Symposium on Concepts and Clinical Applications of Genome Editing - Part I: Science
Chair(s): J. Keith Joung, MD, PhD Speaker(s): Jennifer A. Doudna, PhD, Jennifer A. Doudna, PhD, Michael C. Holmes, PhD, Michael C. Holmes, PhD, J. Keith Joung, MD, PhD, J. Keith Joung, MD, PhD, Duanqing Pei, PhD, Duanqing Pei, PhD
Special Symposium 105, 10:15 AM to 12:15 PM Special Symposium on Concepts and Clinical Applications of Genome Editing - Part II: Ethics
Chair(s): Theodore Friedmann, MD, Nelson A. Wivel, MD Speaker(s): George J. Annas, JD, MPH, George J. Annas, JD, MPH, Robert M. CookDeegan, MD, Robert M. Cook-Deegan, MD, Thomas H. Murray, PhD, Thomas H. Murray, PhD
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
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PROGRAM Oral Abstract Session 110, 10:30 AM to 12:00 PM RNA Virus Vectors............................................................................................................................. S1 Chair(s): Carol H. Miao, PhD, Bruce E. Torbett, PhD, MSPH 1 2 3 4 5 6
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 111, 10:30 AM to 12:00 PM AAV Vector Biology........................................................................................................................... S4 Chair(s): Jeffrey S. Bartlett, PhD, Guangping Gao, PhD 7 8 9 10 11 12
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 112, 10:30 AM to 12:00 PM Adenoviruses....................................................................................................................................... S7 Chair(s): Andrew P. Byrnes, PhD, Julia Davydova, MD, PhD 13 14 15 16 17 18
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 113, 10:30 AM to 12:00 PM Cardiovascular and Pulmonary Diseases......................................................................................... S9 Chair(s): Carolyn Lutzko, PhD, Christian Mueller, PhD 19 20 21 22 23 24
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 114, 10:30 AM to 12:00 PM Delivery Questions for Neural Applications................................................................................ S12 Chair(s): Alberto Auricchio, MD, Andrea Contestabile, PhD 25 26 27 28 29 30
10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
Oral Abstract Session 115, 10:30 AM to 12:00 PM Pre-clinical Development Activities for Gene Therapy Trials................................................... S14 Chair(s): Gwendolyn K. Binder-Scholl, PhD, Susan C. Stevenson, PhD 31 32 33 34 35 36
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10:30 AM 10:45 AM 11:00 AM 11:15 AM 11:30 AM 11:45 AM
C-Oligonucleotide Therapeutics Abstract Review Chair Bruce Sullenger, PhD Reviewers Maxim V. Berezovski, PhD* Paloma H. Giangrande, PhD Ryszard Kole, PhD* Anton P. McCaffrey, PhD Marco S. Weinberg, PhD D- Chemical/Molecular Conjugates and Physical Methods for Delivery of Gene Therapeutics Abstract Review Chairs Dexi Liu, PhD Ernst Wagner, PhD* Reviewers Julie Gehl, MD, Dr.Med.Sci.* Stephen L. Hart, PhD Richard Heller, PhD Kenya Kamimura, MD, PhD* Olivia Merkel, PhD* Millicent O. Sullivan, PhD E1-Diabetes, Metabolic and Genetic Diseases Abstract Review Chair Gerald S. Lipshutz, MD Reviewers Nicola Brunetti-Pierri, MD Randy J. Chandler, PhD, MB* Stephanie Cherqui, PhD Cary O. Harding, MD Dwight Koeberl, MD, PhD E2-Cardiovascular and Pulmonary Diseases Abstract Review Chair Dieter C. Gruenert, PhD Reviewers Bruce A. Bunnell, PhD David A. Dean, PhD Karin M.L. Gaensler, MD* Mauro Giacca, MD, PhD* William B. Guggino, PhD Carolyn Lutzko, PhD E3-Neurologic Diseases (including Ophthalmic and Auditory Diseases) Abstract Review Chair Jean Bennett, MD, PhD Reviewers Alberto Auricchio, MD Beverly L. Davidson, PhD John Forsayeth, PhD Guangping Gao, PhD Luk H. Vandenberghe, PhD
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM E4-Musculo-skeletal Diseases Abstract Review Chair Terence A. Partridge, PhD Reviewers Joel R. Chamberlain, PhD Dongsheng Duan, PhD Paul Gregorevic, PhD* Scott Q. Harper, PhD Peter L. Jones, PhD* E5-Cancer – Immunotherapy, Cancer Vaccines Abstract Review Chair Malcolm K. Brenner, MD, PhD* Reviewers Karen S. Aboody, MD Chiara Bonini, MD Ann M. Leen, PhD Marcela V. Maus, MD, PhD Barbara Savoldo, MD, PhD E6-Cancer – Oncolytic Viruses Abstract Review Chair Samuel D. Rabkin, PhD Reviewers John C. Bell, PhD Evanthia Galanis, MD, DSc Kah-Whye Peng, PhD* Stephen H. Thorne, PhD* Hiroaki Wakimoto, MD, PhD* E7-Cancer - Targeted Gene and Cell Therapy Abstract Review Chair Maria C. Castro, PhD Reviewers Irina V. Balyasnikova, PhD* Juan Fueyo, MD G. Yancey Gillespie, PhD Paola Grandi, PhD Balveen Kaur, PhD
Lunch Break (On Own – Not Provided), 12:00 PM to 1:30 PM Industry Lunch Symposium 120, 12:00 PM to 1:30 PM Industry Lunch Symposium Education Session 130, 1:30 PM to 3:00 PM Emerging Field Review: Towards Therapeutic Applications with Nanoparticle Formulations of Nucleic Acids
Chair(s): Rajagopal Ramesh, PhD, Millicent O. Sullivan, PhD Speaker(s): Muthiah Manoharan, PhD, W. Mark Saltzman, PhD, Assem G. Ziady, PhD
Education Session 131, 1:30 PM to 3:00 PM Topical Review: AAV and Adenovirus Gene Transfer
Chair(s): Aravind Asokan, PhD, Dirk Grimm, PhD Speaker(s): Aravind Asokan, PhD, Douglas M. McCarty, PhD, Dmitry M. Shayakhmetov, PhD
Education Session 132, 1:30 PM to 3:00 PM Topical Review: Advances/Alternatives in Genome Therapies
Chair(s): Richard P. Harbottle, PhD Speaker(s): Richard P. Harbottle, PhD, Zoltan Ivics, PhD, Natalay Y. Kouprina, PhD
Education Session 133, 1:30 PM to 3:00 PM Topical Review: Gene Therapy for Non Malignant Diseases
Chair(s): John F. Tisdale, MD, PhD Speaker(s): Daniel E. Bauer, MD, PhD, Gerd A. Blobel, MD, PhD, Giuliana Ferrari, PhD
Education Session 134, 1:30 PM to 3:00 PM Topical Review: Orphan Diseases
Chair(s): Bobby Gaspar, MD, PhD Speaker(s): Nathalie Cartier-Lacave, MD, Paul Lasko, PhD, Rachel Salzman, DVM
Oral Abstract Session 140, 3:30 PM to 5:30 PM Targeted Genome Editing: Gene Editing in Hematopoietic Cells........................................... S16 Chair(s): Marguerite V. Evans-Galea, PhD, Punam Malik, MD 37 38 39 40 41 42 43 44
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 141, 3:30 PM to 5:30 PM Diabetes, Metabolic and Genetic Diseases................................................................................... S20 Individual speaker presentation titles will be included in the 19th Annual Meeting Program Guide, which will be distributed at the meeting in Washington, DC. Please see the Program Guide for comprehensive information, including room assignments, speaker order and individual presentation titles.
Chair(s): Randy J. Chandler, PhD, MB, Cary O. Harding, MD 45 46 47 48 49 50 51 52
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
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PROGRAM Oral Abstract Session 142, 3:30 PM to 5:30 PM Gene Therapy for CNS Diseases................................................................................................... S23 Chair(s): Jean Bennett, MD, PhD, Guangping Gao, PhD 53 54 55 56 57 58 59 60
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 143, 3:30 PM to 5:30 PM Cancer-Oncolytic RNA Viruses..................................................................................................... S27 Chair(s): Kah-Whye Peng, PhD, Samuel D. Rabkin, PhD 61 62 63 64 65 66 67 68
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 144, 3:30 PM to 5:30 PM Cancer-Targeted Gene and Cell Therapy...................................................................................... S30 Chair(s): Irina V. Balyasnikova, PhD, Paola Grandi, PhD 69 70 71 72 73 74 75 76
3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
Oral Abstract Session 145, 3:30 PM to 5:30 PM Immunological Aspects of Gene Therapy I: AAV Vectors....................................................... S34 Chair(s): Ashley T. Martino, PhD, Pedro Lowenstein, MD, PhD 77 78 79 80 81 82 83 84
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3:30 PM 3:45 PM 4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM
E8-Hematologic and Immunologic Diseases Abstract Review Chair Harry L. Malech, MD Reviewers Jennifer E. Adair, PhD Alessandro Aiuti, MD, PhD Gay Crooks, MD John T. Gray, PhD Karin Pike-Overzet, PhD F-Immunological Aspects of Gene Therapy Abstract Review Chair Michael A. Barry, PhD Reviewers Pedro Lowenstein, MD, PhD Ashley T. Martino, PhD* Dmitry M. Shayakhmetov, PhD Renata Stripecke, PhD David B. Weiner, PhD G- Cell Therapies Abstract Review Chair David W. Russell, MD, PhD Reviewers Magali Cucchiarini, PhD* Andre Larochelle, MD, PhD* Jan A. Nolta, PhD Eirini P. Papapetrou, MD, PhD Tim Townes, PhD* H1- Vector and Cell Engineering, Production or Manufacturing Abstract Review Chair Boro Dropulic, PhD Reviewers Bryan T. Butman, PhD* Maria Mercedes Segura, PhD* Vladimir A. Slepushkin, PhD* J. Fraser Wright, PhD H2-Pharmacology/ Toxicology Studies or Assay Development Abstract Review Chair Gene Liau, PhD* Reviewers: Gwendolyn K. Binder-Scholl, PhD* Linda B. Couto, PhD Susan C. Stevenson, PhD* Gabor Veres, PhD Teresa Wright, PhD*
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM H3-Clinical Protocol Development, Regulatory Interactions, and Ethics Abstract Review Chair Phillip B. Maples, PhD* Reviewers Dale G. Ando, MD Marina Cavazzana-Calvo, MD, PhD Rimas J. Orentas, PhD* Isabelle Riviere, PhD David Stroncek, MD* Special thanks to the abstract reviewers for their time and expertise! *Indicates first time reviewer for ASGCT. To volunteer to review abstracts for the ASGCT 20th Annual Meeting, visit the ASGCT Membership booth.
Exhibit Hall Welcome Reception & Poster Session I, 5:30 PM to 7:30 PM AAV Vectors I................................................................................................................................... S37 (Abstracts 85 through 102; Abstract 96 moved to Poster Session II after Abstract 310)
Adenovirus Vectors and Other DNA Virus Vectors.................................................................. S44 (Abstracts 103 through 117)
Targeted Genome Editing I............................................................................................................ S49
(Abstracts 118 through 142; Abstract 120 moved to Oral Abstract Session 411 after Abstract 732)
Oligonucleotide Therapeutics I...................................................................................................... S58 (Abstracts 143 through 156; Abstracts 147 & 151 have been withdrawn)
Diabetes, Metabolic and Genetic Diseases I................................................................................. S61 (Abstracts 157 through 171)
Cardiovascular and Pulmonary Diseases....................................................................................... S67 (Abstracts 172 through 183; Abstract 175 has been withdrawn)
Gene Therapy for Neurosensory Diseases................................................................................... S72 (Abstracts 184 through 189)
Cancer-Immunotherapy, Cancer Vaccines I.................................................................................. S74 (Abstracts 190 through 206)
Cancer-Targeted Gene and Cell Therapy I................................................................................... S80 (Abstracts 207 through 219)
Hematologic & Immunologic Diseases I...................................................................................... S86 (Abstracts 220 through 233)
Cell Therapies I................................................................................................................................. S91 (Abstracts 234 through 246; Abstract 237 has been withdrawn)
Reconnection and Mentoring Events, 7:30 PM to 9:30 PM Industry Symposium 150, 7:30 PM to 9:30 PM Industry Symposium NIH Symposium 151, 7:30 PM to 9:30 PM The Interface of Academia, the NIH, Foundations, and Biopharm/Biotech: Best Practices to Ensure the Continued Growth and Success of Gene and Cell Therapies
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
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PROGRAM
Thursday, May 5, 2016 Scientific Symposium 200, 8:00 AM to 10:00 AM AAV, Adenovirus, and Lentiviral Vectors Updates
Chair(s): Jeffrey A. Medin, PhD, Stephen J. Russell, MD, PhD Speaker(s): Christian J. Buchholz, PhD, Kenneth Cornetta, MD, Clodagh O’Shea, PhD, Arun Srivastava, PhD
Scientific Symposium 201, 8:00 AM to 10:00 AM Clinical Trials Spotlight ................................................................................................................... S97 Chair(s): Jean Bennett, MD, PhD, Donald B. Kohn, MD 247 248 249 250
8:00 AM 8:20 AM 8:40 AM 9:00 AM
Scientific Symposium 202, 8:00 AM to 10:00 AM Gene Therapies for Inherited and Metabolic Diseases
Chair(s): Stephanie Cherqui, PhD, Hiroyuki Nakai, MD, PhD Speaker(s): Alessandra Biffi, MD, Beverly L. Davidson, PhD, Christian Mueller, PhD, Dao Pan, PhD
Scientific Symposium 203, 8:00 AM to 10:00 AM Recent Advances in Human Stem Cell Research and Therapy
Chair(s): Hans-Peter Kiem, MD Speaker(s): Jason Butler, PhD, Danwei Huangfu, PhD, Carolyn Lutzko, PhD, Frank McKeon, PhD, Renee Reijo Pera, PhD
Scientific Symposium 204, 8:00 AM to 10:00 AM Translational Research and Product Development through AcademicIndustry Partnerships: Models for Success
Chair(s): Gwendolyn K. Binder-Scholl, PhD, H. Trent Spencer, PhD Speaker(s): Gwendolyn K. Binder-Scholl, PhD, Katherine A. High, MD, Carl H. June, MD, Fulvio Mavilio, PhD
Exhibit Hall Coffee Social – with Oral Poster Session, 9:45 AM – 10:45 AM George Stamatoyannopoulos Lecture 210, 10:45 AM to 12:00 PM George Stamatoyannopoulos Lecture & Presentation of the Excellence in Research Awards Chair(s): Cynthia Dunbar, MD Speaker(s): David R. Liu, PhD
Lunch Break (On Own – Not Provided), 12:00 PM to 1:30 PM Industry Lunch Symposium 220, 12:00 PM to 1:30 PM Industry Lunch Symposium Outstanding New Investigator Symposium 230, 1:30 PM to 3:30 PM Outstanding New Investigator Symposium
Chair(s): Terence R. Flotte, MD Speaker(s): Jordan Green, PhD, Marcin Kortylewski, PhD, Eirini P. Papapetrou, MD, PhD, Junghae Suh, PhD
Exhibit Hall Coffee Social, 3:30 PM to 4:15 PM
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM Oral Abstract Session 240, 4:00 PM to 5:45 PM Evolving Better Parvoviral Vectors for Gene Therapy............................................................... S99 Chair(s): John F. Engelhardt, PhD, David V. Schaffer, PhD 251 252 253 254 255 256 257
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 241, 4:00 PM to 5:45 PM Oligonucleotide Therapeutics I....................................................................................................S102 Chair(s): Ryszard Kole, PhD, William H. Thiel, PhD 258 259 260 261 262 263 264
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 242, 4:00 PM to 5:45 PM Gene Therapy for Neurosensory Diseases.................................................................................S105 Chair(s): Beverly L. Davidson, PhD, Luk H. Vandenberghe, PhD 265 266 267 268 269 270 271
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 243, 4:00 PM to 5:45 PM Cancer-Immunotherapy, Cancer Vaccines I................................................................................S108 Chair(s): Karen S. Aboody, MD, Malcolm K. Brenner, MD, PhD 272 273 274 275 276 277 278
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 244, 4:00 PM to 5:45 PM Hematologic & Immunologic Diseases I....................................................................................S111 Chair(s): John T. Gray, PhD, Karin Pike-Overzet, PhD 279 280 281 282 283 284 285
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
Oral Abstract Session 245, 4:00 PM to 5:45 PM Vector and Cell Engineering/Manufacturing.............................................................................S115 Chair(s): Vladimir Slepushkin, PhD, J. Fraser Wright, PhD 286 287 288 289 290 291 292 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM
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PROGRAM Exhibit Hall Networking Reception & Poster Session II (including Tools and Technology Forum), 5:45 PM to 7:45 PM AAV Vectors II................................................................................................................................S118 (Abstracts 293 through 310; Abstract 96 moved from Poster Session I)
Targeted Genome Editing II.........................................................................................................S125 (Abstracts 311 through 334)
Non-Viral Gene Transfer and Therapy I....................................................................................S133 (Abstracts 335 through 345)
Diabetes, Metabolic and Genetic Diseases II.............................................................................S138 (Abstracts 346 through 360)
Gene Therapy for CNS Diseases.................................................................................................S144 (Abstracts 361 through 376)
Musculo-Skeletal Diseases I..........................................................................................................S150 (Abstracts 377 through 390; Abstract 384 has been withdrawn; Abstract 622 has been moved from Poster Session III)
Cancer-Immunotherapy, Cancer Vaccines II..............................................................................S155 (Abstracts 391 through 408)
Cancer-Oncolytic Viruses I...........................................................................................................S162 (Abstracts 409 through 423)
Immunological Aspects of Gene Therapy I...............................................................................S168 (Abstracts 424 through 441)
Cell Therapies II..............................................................................................................................S175 (Abstracts 442 through 454; Abstract 450 has been withdrawn)
Vector and Cell Engineering/Manufacturing I..........................................................................S180 (Abstracts 455 through 466)
Pharmacology/Toxicology Studies or Assay Development.....................................................S184 (Abstracts 467 through 477)
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM
Friday, May 6, 2016 Scientific Symposium 300, 8:00 AM to 10:00 AM Basic Biology of Nanoparticles and Interaction with Cells
Chair(s): Stephen L. Hart, PhD, Assem G. Ziady, PhD Speaker(s): Jordan Green, PhD, Judy Lieberman, MD, PhD, Joachim Rädler, PhD, Millicent O. Sullivan, PhD
Scientific Symposium 301, 8:00 AM to 10:00 AM Cancer Therapeutics: Targeting the Target
Chair(s): John C. Bell, PhD, Evanthia Galanis, MD, DSc Speaker(s): Renier J. Brentjens, MD, PhD, Michael Caligiuri, MD, Naoto Hirano, MD, PhD, Lili Yang, PhD
Scientific Symposium 302, 8:00 AM to 10:00 AM Cell Therapy to Improve the Outcome of Hemopoietic Stem Cell Transplant
Chair(s): Helen E. Heslop, MD, Elizabeth J. Shpall, MD Speaker(s): David E. Avigan, MD, Austin J. Barrett, MD, Colleen Delaney, MD, MSC, Katy Rezvani, MD, PhD
Scientific Symposium 303, 8:00 AM to 10:00 AM Emerging and Transnational Clinical Trials
Chair(s): Ian E. Alexander, MBBS, PhD, Thierry C. VandenDriessche, PhD Speaker(s): Luigi M. Naldini, MD, PhD, Waseem Qasim, MRCP, PhD, Masayo Takahashi, MD, PhD, Takanori Yamagata, MD, PhD
Scientific Symposium 304, 8:00 AM to 10:00 AM Evolution of Gene & Cell Therapy Enabling Technologies - Challenges for Clinical Applications
Chair(s): Robert E. Sobol, MD, Gabor Veres, PhD Speaker(s): Jean Bennett, MD, PhD, Dan S. Kaufman, MD, PhD, Howard L. Kaufman, MD, Matthew H. Porteus, MD, PhD
Exhibit Hall Coffee Social – with Oral Poster Session, 9:45 AM – 10:45 AM Outstanding Achievement Award Lecture 310, 10:45 AM to 11:45 AM Outstanding Achievement Award Lecture with Sonia Skarlatos Public Service Award Presentation Chair(s): Terence R. Flotte, MD Speaker(s): Seppo Yla-Herttuala, MD, PhD, FESC
Lunch Break (On Own – Not Provided), 11:45 AM to 1:15 PM Presidential Symposium 320, 1:15 PM to 3:30 PM Presidential Symposium & Presentation of the Top Abstracts...............................................S189 Chair(s): Michel Sadelain, MD, PhD Speaker(s): Alain Fischer, MD, Theodore Friedmann, MD
Presentation of Top Abstracts: 478 479 480
2:30 PM 2:50 PM 3:10 PM
Exhibit Hall Coffee Social, 3:30 PM to 4:15 PM
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PROGRAM Oral Abstract Session 330, 4:00 PM to 6:00 PM Targeted Genome Editing: In Vivo Genome Editing..............................................................S190 Chair(s): Rita Perlingeiro, PhD, Andrew M. Scharenberg, MD 481 482 483 484 485 733 487 488
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM (Moved from Oral Session 411) 5:30 PM 5:45 PM
Oral Abstract Session 331, 4:00 PM to 6:00 PM Chemical and Physical Methods for Delivery of Gene Therapeutics....................................S194 Chair(s): Stephen L. Hart, PhD, Ernst Wagner, PhD 489 490 491 492 493 494 495 496
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 332, 4:00 PM to 6:00 PM Musculo-skeletal Diseases..............................................................................................................S197 Chair(s): Scott Q. Harper, PhD, Terence A. Partridge, PhD 497 498 499 500 501 502 503 504
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 333, 4:00 PM to 6:00 PM Cancer-Immunotherapy, Cancer Vaccines II..............................................................................S201 Chair(s): Attilio Bondanza, MD, Philip D. Gregory, DPhil 505 506 507 508 509 510 511 512
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Oral Abstract Session 334, 4:00 PM to 6:00 PM Cancer-Oncolytic DNA Viruses...................................................................................................S204 Chair(s): Stephen H. Thorne, PhD, Hiroaki Wakimoto, MD PhD 513 514 515 516 517 518 519 520
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4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM Oral Abstract Session 335, 4:00 PM to 6:00 PM Cell Therapies..................................................................................................................................S208 Chair(s): Andre Larochelle, MD, PhD, Eirini P. Papapetrou, MD, PhD 521 522 523 524 525 526 527 528
4:00 PM 4:15 PM 4:30 PM 4:45 PM 5:00 PM 5:15 PM 5:30 PM 5:45 PM
Exhibit Hall Networking Reception & Poster Session III (including Tools and Technology Forum), 6:00 PM to 8:00 PM RNA Virus Vectors.........................................................................................................................S211 (Abstracts 529 through 538)
AAV Vectors III..............................................................................................................................S215 (Abstracts 539 through 556)
Targeted Genome Editing III.......................................................................................................S223 (Abstracts 557 through 579; Abstract 578 has been withdrawn)
Oligonucleotide Therapeutics II...................................................................................................S231 (Abstracts 580 through 592)
Non-Viral Gene Transfer and Therapy II...................................................................................S234 (Abstracts 593 through 603)
Delivery Questions for Neural Applications..............................................................................S239 (Abstracts 604 through 621)
Musculo-Skeletal Diseases II.........................................................................................................S246 (Abstracts 623 through 636; Abstract 622 has been moved to Poster Session II after Abstract 390)
Cancer-Immunotherapy, Cancer Vaccines III............................................................................S252 (Abstracts 637 through 653)
Cancer-Oncolytic Viruses II..........................................................................................................S259 (Abstracts 654 through 667)
Cancer-Targeted Gene and Cell Therapy II...............................................................................S264 (Abstracts 668 through 680)
Hematologic & Immunologic Diseases II..................................................................................S269 (Abstracts 681 through 693)
Immunological Aspects of Gene Therapy II: AAV Vectors..................................................S274 (Abstracts 694 through 702)
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PROGRAM Vector and Cell Engineering/Manufacturing II.........................................................................S278 (Abstracts 703 through 714)
Clinical Protocol Development, Regulatory Interactions, and Ethics....................................S282 (Abstracts 715 through 720)
Closing Night Reception at the Newseum – Ticket Required, 8:00 PM to 10:00 PM
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
PROGRAM
Saturday, May 7, 2016 ASGCT Business Meeting and Coffee Break, 7:30 AM to 8:00 AM Education Session 400, 8:00 AM to 10:00 AM Emerging Field Review: The Basics of Genome Editing
Chair(s): Thomas Wechsler, PhD Speaker(s): Charles A. Gersbach, PhD, Miguel Gonzalez-Blanco, PhD, Andrew M. Scharenberg, MD
Education Session 401, 8:00 AM to 10:00 AM Topical Review: How to Fund Gene Therapy Research
Chair(s): Terry R. Bishop, PhD Speaker(s): Terry R. Bishop, PhD, Hal E. Broxmeyer, PhD, Scott Q. Harper, PhD
Education Session 402, 8:00 AM to 10:00 AM Topical Review: T cell Immunotherapy
Chair(s): Hans-Peter Kiem, MD Speaker(s): Catherine M. Bollard, MD, Helen E. Heslop, MD, Shannon L. Maude, MD, PhD, Pin Wang, PhD
Scientific Symposium 403, 8:00 AM to 10:00 AM Advances in Musculoskeletal Gene and Cell Therapy
Chair(s): Gloria Matthews, DVM, PhD, DACVS Speaker(s): Jeffrey S. Chamberlain, PhD, James D. Kang, MD, Rita Perlingeiro, PhD, Bing Wang, MD, PhD
Scientific Symposium 404, 8:00 AM to 10:00 AM Advances in Oligonucleotide Diagnostics and Therapy
Chair(s): Paloma H. Giangrande, PhD, Martin J. Hicks, PhD Speaker(s): Elizabeth Ackermann, PhD, Brian D. Adams, PhD, Edward M. Kaye, MD, Amy Simon, MD
Scientific Symposium 405, 8:00 AM to 10:00 AM Physical Delivery Driving the Development of Innovative Molecular Therapeutics
Chair(s): Ernst Wagner, PhD, Matthew H. Wilson, MD, PhD Speaker(s): Daniel G. Anderson, PhD, Julie Gehl, MD, Dr.Med.Sci., Alexander Marson, MD, PhD, Michael J. Passineau, PhD
Oral Abstract Session 410, 10:15 AM to 12:15 PM AAV Vectors and Pre-clinical Analysis........................................................................................S284 Chair(s): Aravind Asokan, PhD, Eduard Ayuso 721 722 723 724 725 726 727 728
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PROGRAM Oral Abstract Session 411, 10:15 AM to 12:15 PM Targeted Genome Editing: Methods and Technology..............................................................S287 Chair(s): Charles A. Gersbach, PhD, Dirk Grimm, PhD 729 730 731 732 120 734 735 736
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(Moved from Poster Session I)
Oral Abstract Session 412, 10:15 AM to 12:15 PM Oligonucleotide Therapeutics II...................................................................................................S290 Chair(s): John Burnett, PhD, Marco S. Weinberg, Ph.D. 737 738 739 740 741 742 743 744
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Oral Abstract Session 413, 10:15 AM to 12:15 PM Cancer-Immunotherapy, Cancer Vaccines III............................................................................S294 Chair(s): Marcela V. Maus, MD, PhD, Barbara Savoldo, MD, PhD 745 746 747 748 749 750 751 752
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Oral Abstract Session 414, 10:15 AM to 12:15 PM Hematologic & Immunologic Diseases II..................................................................................S297 Chair(s): Jennifer E. Adair, PhD, Gay Crooks, MD 753 754 755 756 757 758 759 760
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Oral Abstract Session 415, 10:15 AM to 12:15 PM Immunological Aspects of Gene Therapy II.............................................................................S301 Chair(s): Renata Stripecke, PhD, David B. Weiner, PhD 761 762 763 764 765 766 767 768
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Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
