Volume 24, Supplement 1, May 2016

The Journal of the American Society of Gene & Cell Therapy

AMERICAN American Society of Gene & Cell Therapy

SOCIETY OF GENE 19th Annual Meeting
& CELL THERAPY
May 4-7, 2016
19th Annual Meeting Washington, DC
May 4 - 7, 2016
Washington, DC, USA
Wednesday, May 4, 2016
Scientific Symposium 100, 8:00 AM to 10:00 AM
Abstract Reviewers
Amyotrophic Lateral Sclerosis (ALS): Potential Therapeutic Targets in Novel
Abstract Committee Chair Disease Mechanisms
Andre Lieber, MD, PhD Chair(s): John H. Wolfe, VMD, PhD
University of Washington Speaker(s): Nicholas Boulis, MD, Don W. Cleveland, PhD, Ronald L. Klein, PhD, Philip C.
Seattle, WA Wong, PhD

A1-RNA Virus Vectors

Abstract Review Chair Scientific Symposium 101, 8:00 AM to 10:00 AM
Grant D. Trobridge, PhD Frontiers in Cardiovascular Gene and Cell Therapy
Reviewers Chair(s): Seppo Yla-Herttuala, MD, PhD, FESC
Carol H. Miao, PhD Speaker(s): Charles R. Bridges, MD, ScD, Marinee K.L. Chuah, PhD, Michael Regnier,
Axel Schambach, MD, PhD PhD, Lior Zangi, PhD
John F. Tisdale, MD
Bruce E. Torbett, PhD, MSPH
Andrew C. Wilber, PhD
Scientific Symposium 102, 8:00 AM to 10:00 AM
Gene Therapy Product Development in the Modern Era: Current Regulatory
A2-AAV Vectors Issues
Abstract Review Chair Chair(s): Patrick Au, PhD, Joy Cavagnaro, PhD, DABT, ATS
R. Jude Samulski, PhD Speaker(s): Wilson W. Bryan, MD, Denise K. Gavin, PhD, Maura C. O’Leary, MD,
Reviewers Mercedes Serabian, MS, DABT
John F. Engelhardt, PhD
Douglas M. McCarty, PhD
Philippe Moullier, MD, PhD Scientific Symposium 103, 8:00 AM to 10:00 AM
Nicholas Muzyczka, PhD* Integrating Combination Approaches to Modulate the Immune Response to
David V. Schaffer, PhD* Cell and Gene Therapies
Chair(s): Conrad Russell Y. Cruz, MD, PhD, Renata Stripecke, PhD
A3-Adenovirus and Other
DNA Virus Vectors
Speaker(s): Maria G. Castro, PhD, Hildegund CJ Ertl, MD, Roland W. Herzog, PhD
Abstract Review Chair
Andrew P. Byrnes, PhD Scientific Symposium 104, 8:00 AM to 10:00 AM
Reviewers Targeting the Liver with Gene and Cell Therapeutics
Julia Davydova, MD, PhD* Chair(s): Federico Mingozzi, PhD, Dao Pan, PhD
Anja Ehrhardt, PhD Speaker(s): Alberto Auricchio, MD, Federico Mingozzi, PhD, Charles P. Venditti, MD, PhD,
Florian Kreppel, PhD Thomas Wechsler, PhD
Hiroyuki Mizuguchi, PhD*
Xiangyang Zhou, PhD
Special Symposium 105, 8:00 AM to 10:00 AM
B-Gene Targeting and Gene Special Symposium on Concepts and Clinical Applications of Genome
Correction
Abstract Review Chair
Editing - Part I: Science
Andrew M. Scharenberg, MD* Chair(s): J. Keith Joung, MD, PhD
Reviewers Speaker(s): Jennifer A. Doudna, PhD, Jennifer A. Doudna, PhD, Michael C. Holmes, PhD,
Marguerite V. Evans- Michael C. Holmes, PhD, J. Keith Joung, MD, PhD, J. Keith Joung, MD, PhD, Duanqing
Galea, PhD* Pei, PhD, Duanqing Pei, PhD
Charles A. Gersbach, PhD
Dirk Grimm, PhD Special Symposium 105, 10:15 AM to 12:15 PM
Punam Malik, MD
Rita Perlingeiro, PhD
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. Cook-
Deegan, MD, Robert M. Cook-Deegan, MD, Thomas H. Murray, PhD, Thomas H. Murray,
PhD

Molecular Therapy Volume 24, Supplement 1, May 2016 i

Copyright © The American Society of Gene & Cell Therapy
 PROGRAM

Oral Abstract Session 110, 10:30 AM to 12:00 PM C-Oligonucleotide

Therapeutics
RNA Virus Vectors............................................................................................................................. S1
Abstract Review Chair
Chair(s): Carol H. Miao, PhD, Bruce E. Torbett, PhD, MSPH
1 10:30 AM Bruce Sullenger, PhD
2 10:45 AM Reviewers
3 11:00 AM Maxim V. Berezovski, PhD*
4 11:15 AM Paloma H. Giangrande, PhD
5 11:30 AM Ryszard Kole, PhD*
6 11:45 AM Anton P. McCaffrey, PhD
Marco S. Weinberg, PhD
Oral Abstract Session 111, 10:30 AM to 12:00 PM D- Chemical/Molecular
AAV Vector Biology........................................................................................................................... S4 Conjugates and Physical
Chair(s): Jeffrey S. Bartlett, PhD, Guangping Gao, PhD Methods for Delivery of Gene
7 10:30 AM Therapeutics
8 10:45 AM Abstract Review Chairs
9 11:00 AM Dexi Liu, PhD
10 11:15 AM Ernst Wagner, PhD*
11 11:30 AM Reviewers
12 11:45 AM Julie Gehl, MD, Dr.Med.Sci.*
Stephen L. Hart, PhD
Oral Abstract Session 112, 10:30 AM to 12:00 PM Richard Heller, PhD
Kenya Kamimura, MD, PhD*
Adenoviruses....................................................................................................................................... S7
Olivia Merkel, PhD*
Chair(s): Andrew P. Byrnes, PhD, Julia Davydova, MD, PhD
13 10:30 AM Millicent O. Sullivan, PhD
14 10:45 AM
E1-Diabetes, Metabolic and
15 11:00 AM
16 11:15 AM Genetic Diseases
17 11:30 AM Abstract Review Chair
18 11:45 AM Gerald S. Lipshutz, MD
Reviewers
Nicola Brunetti-Pierri, MD
Oral Abstract Session 113, 10:30 AM to 12:00 PM Randy J. Chandler, PhD, MB*
Cardiovascular and Pulmonary Diseases......................................................................................... S9 Stephanie Cherqui, PhD
Chair(s): Carolyn Lutzko, PhD, Christian Mueller, PhD Cary O. Harding, MD
19 10:30 AM Dwight Koeberl, MD, PhD
20 10:45 AM
21 11:00 AM E2-Cardiovascular and
22 11:15 AM Pulmonary Diseases
23 11:30 AM Abstract Review Chair
24 11:45 AM Dieter C. Gruenert, PhD
Reviewers
Oral Abstract Session 114, 10:30 AM to 12:00 PM Bruce A. Bunnell, PhD
David A. Dean, PhD
Delivery Questions for Neural Applications................................................................................ S12 Karin M.L. Gaensler, MD*
Chair(s): Alberto Auricchio, MD, Andrea Contestabile, PhD Mauro Giacca, MD, PhD*
25 10:30 AM
William B. Guggino, PhD
26 10:45 AM
Carolyn Lutzko, PhD
27 11:00 AM
28 11:15 AM
E3-Neurologic Diseases
29 11:30 AM
(including Ophthalmic and
30 11:45 AM
Auditory Diseases)
Abstract Review Chair
Oral Abstract Session 115, 10:30 AM to 12:00 PM Jean Bennett, MD, PhD
Pre-clinical Development Activities for Gene Therapy Trials................................................... S14 Reviewers
Chair(s): Gwendolyn K. Binder-Scholl, PhD, Susan C. Stevenson, PhD Alberto Auricchio, MD
31 10:30 AM Beverly L. Davidson, PhD
32 10:45 AM John Forsayeth, PhD
33 11:00 AM Guangping Gao, PhD
34 11:15 AM Luk H. Vandenberghe, PhD
35 11:30 AM
36 11:45 AM

ii Molecular Therapy Volume 24, Supplement 1, May 2016

Copyright © The American Society of Gene & Cell Therapy
 PROGRAM

E4-Musculo-skeletal Lunch Break (On Own – Not Provided), 12:00 PM to 1:30 PM

Diseases
Abstract Review Chair
Terence A. Partridge, PhD Industry Lunch Symposium 120, 12:00 PM to 1:30 PM
Reviewers Industry Lunch Symposium
Joel R. Chamberlain, PhD
Dongsheng Duan, PhD
Education Session 130, 1:30 PM to 3:00 PM
Paul Gregorevic, PhD*
Scott Q. Harper, PhD Emerging Field Review: Towards Therapeutic Applications with
Peter L. Jones, PhD* Nanoparticle Formulations of Nucleic Acids
Chair(s): Rajagopal Ramesh, PhD, Millicent O. Sullivan, PhD
E5-Cancer – Immunotherapy, Speaker(s): Muthiah Manoharan, PhD, W. Mark Saltzman, PhD, Assem G. Ziady, PhD
Cancer Vaccines
Abstract Review Chair
Malcolm K. Brenner, MD, PhD* Education Session 131, 1:30 PM to 3:00 PM
Reviewers Topical Review: AAV and Adenovirus Gene Transfer
Karen S. Aboody, MD Chair(s): Aravind Asokan, PhD, Dirk Grimm, PhD
Chiara Bonini, MD Speaker(s): Aravind Asokan, PhD, Douglas M. McCarty, PhD, Dmitry M. Shayakhmetov,
Ann M. Leen, PhD PhD
Marcela V. Maus, MD, PhD
Barbara Savoldo, MD, PhD
Education Session 132, 1:30 PM to 3:00 PM
E6-Cancer – Oncolytic Topical Review: Advances/Alternatives in Genome Therapies
Viruses Chair(s): Richard P. Harbottle, PhD
Abstract Review Chair Speaker(s): Richard P. Harbottle, PhD, Zoltan Ivics, PhD, Natalay Y. Kouprina, PhD
Samuel D. Rabkin, PhD
Reviewers
John C. Bell, PhD Education Session 133, 1:30 PM to 3:00 PM
Evanthia Galanis, MD, DSc Topical Review: Gene Therapy for Non Malignant Diseases
Kah-Whye Peng, PhD* Chair(s): John F. Tisdale, MD, PhD
Stephen H. Thorne, PhD* Speaker(s): Daniel E. Bauer, MD, PhD, Gerd A. Blobel, MD, PhD, Giuliana Ferrari, PhD
Hiroaki Wakimoto, MD, PhD*

E7-Cancer - Targeted Gene Education Session 134, 1:30 PM to 3:00 PM

and Cell Therapy Topical Review: Orphan Diseases
Abstract Review Chair Chair(s): Bobby Gaspar, MD, PhD
Maria C. Castro, PhD Speaker(s): Nathalie Cartier-Lacave, MD, Paul Lasko, PhD, Rachel Salzman, DVM
Reviewers
Irina V. Balyasnikova, PhD*
Oral Abstract Session 140, 3:30 PM to 5:30 PM
Juan Fueyo, MD
G. Yancey Gillespie, PhD Targeted Genome Editing: Gene Editing in Hematopoietic Cells........................................... S16
Paola Grandi, PhD Chair(s): Marguerite V. Evans-Galea, PhD, Punam Malik, MD
Balveen Kaur, PhD 37 3:30 PM
38 3:45 PM
39 4:00 PM
40 4:15 PM
41 4:30 PM
42 4:45 PM
43 5:00 PM
44 5:15 PM

Oral Abstract Session 141, 3:30 PM to 5:30 PM

Diabetes, Metabolic and Genetic Diseases................................................................................... S20
Chair(s): Randy J. Chandler, PhD, MB, Cary O. Harding, MD
Individual speaker 45 3:30 PM
presentation titles will 46 3:45 PM
be included in the 19th 47 4:00 PM
48 4:15 PM
Annual Meeting Program 49 4:30 PM
Guide, which will be 50 4:45 PM
distributed at the meeting 51 5:00 PM
in Washington, DC. 52 5:15 PM
Please see the Program
Guide for comprehensive
information, including room
assignments, speaker order
and individual presentation
titles.
Molecular Therapy Volume 24, Supplement 1, May 2016 iii
Copyright © The American Society of Gene & Cell Therapy
 PROGRAM

Oral Abstract Session 142, 3:30 PM to 5:30 PM E8-Hematologic and

Gene Therapy for CNS Diseases................................................................................................... S23 Immunologic Diseases
Abstract Review Chair
Chair(s): Jean Bennett, MD, PhD, Guangping Gao, PhD
53 3:30 PM Harry L. Malech, MD
54 3:45 PM Reviewers
55 4:00 PM Jennifer E. Adair, PhD
56 4:15 PM Alessandro Aiuti, MD, PhD
57 4:30 PM Gay Crooks, MD
58 4:45 PM John T. Gray, PhD
59 5:00 PM Karin Pike-Overzet, PhD
60 5:15 PM
F-Immunological Aspects of
Gene Therapy
Oral Abstract Session 143, 3:30 PM to 5:30 PM Abstract Review Chair
Cancer-Oncolytic RNA Viruses..................................................................................................... S27 Michael A. Barry, PhD
Chair(s): Kah-Whye Peng, PhD, Samuel D. Rabkin, PhD Reviewers
61 3:30 PM Pedro Lowenstein, MD, PhD
62 3:45 PM Ashley T. Martino, PhD*
63 4:00 PM Dmitry M. Shayakhmetov, PhD
64 4:15 PM Renata Stripecke, PhD
65 4:30 PM David B. Weiner, PhD
66 4:45 PM
67 5:00 PM G- Cell Therapies
68 5:15 PM
Abstract Review Chair
David W. Russell, MD, PhD
Oral Abstract Session 144, 3:30 PM to 5:30 PM Reviewers
Cancer-Targeted Gene and Cell Therapy...................................................................................... S30 Magali Cucchiarini, PhD*
Chair(s): Irina V. Balyasnikova, PhD, Paola Grandi, PhD Andre Larochelle, MD, PhD*
69 3:30 PM Jan A. Nolta, PhD
70 3:45 PM Eirini P. Papapetrou, MD, PhD
71 4:00 PM Tim Townes, PhD*
72 4:15 PM
73 4:30 PM H1- Vector and Cell
74 4:45 PM Engineering, Production or
75 5:00 PM Manufacturing
76 5:15 PM Abstract Review Chair
Boro Dropulic, PhD
Reviewers
Oral Abstract Session 145, 3:30 PM to 5:30 PM Bryan T. Butman, PhD*
Immunological Aspects of Gene Therapy I: AAV Vectors....................................................... S34 Maria Mercedes Segura, PhD*
Chair(s): Ashley T. Martino, PhD, Pedro Lowenstein, MD, PhD Vladimir A. Slepushkin, PhD*
77 3:30 PM J. Fraser Wright, PhD
78 3:45 PM
79 4:00 PM H2-Pharmacology/
80 4:15 PM Toxicology Studies or Assay
81 4:30 PM Development
82 4:45 PM Abstract Review Chair
83 5:00 PM
Gene Liau, PhD*
84 5:15 PM
Reviewers:
Gwendolyn K. Binder-Scholl,
PhD*
Linda B. Couto, PhD
Susan C. Stevenson, PhD*
Gabor Veres, PhD
Teresa Wright, PhD*

iv Molecular Therapy Volume 24, Supplement 1, May 2016

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 PROGRAM
H3-Clinical Protocol
Development, Regulatory Exhibit Hall Welcome Reception & Poster Session I, 5:30 PM to 7:30 PM
Interactions, and Ethics
Abstract Review Chair
Phillip B. Maples, PhD* AAV Vectors I................................................................................................................................... S37
Reviewers (Abstracts 85 through 102; Abstract 96 moved to Poster Session II after Abstract 310)
Dale G. Ando, MD
Marina Cavazzana-Calvo, MD, Adenovirus Vectors and Other DNA Virus Vectors.................................................................. S44
PhD (Abstracts 103 through 117)
Rimas J. Orentas, PhD*
Isabelle Riviere, PhD
David Stroncek, MD* Targeted Genome Editing I............................................................................................................ S49
(Abstracts 118 through 142; Abstract 120 moved to Oral Abstract Session 411 after Abstract
Special thanks to the abstract 732)
reviewers for their time and
expertise!
Oligonucleotide Therapeutics I...................................................................................................... S58
*Indicates first time reviewer (Abstracts 143 through 156; Abstracts 147 & 151 have been withdrawn)
for ASGCT.
To volunteer to review Diabetes, Metabolic and Genetic Diseases I................................................................................. S61
abstracts for the ASGCT 20th (Abstracts 157 through 171)
Annual Meeting, visit the
ASGCT Membership booth.
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 v

Copyright © The American Society of Gene & Cell Therapy
 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 8:00 AM
248 8:20 AM
249 8:40 AM
250 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 Academic-
Industry 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

vi Molecular Therapy Volume 24, Supplement 1, May 2016

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 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 4:00 PM
252 4:15 PM
253 4:30 PM
254 4:45 PM
255 5:00 PM
256 5:15 PM
257 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 4:00 PM
259 4:15 PM
260 4:30 PM
261 4:45 PM
262 5:00 PM
263 5:15 PM
264 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 4:00 PM
266 4:15 PM
267 4:30 PM
268 4:45 PM
269 5:00 PM
270 5:15 PM
271 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 4:00 PM
273 4:15 PM
274 4:30 PM
275 4:45 PM
276 5:00 PM
277 5:15 PM
278 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 4:00 PM
280 4:15 PM
281 4:30 PM
282 4:45 PM
283 5:00 PM
284 5:15 PM
285 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 4:00 PM
287 4:15 PM
288 4:30 PM
289 4:45 PM
290 5:00 PM
291 5:15 PM
292 5:30 PM

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Copyright © The American Society of Gene & Cell Therapy
 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)

viii Molecular Therapy Volume 24, Supplement 1, May 2016

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 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 2:30 PM
479 2:50 PM
480 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 4:00 PM
482 4:15 PM
483 4:30 PM
484 4:45 PM
485 5:00 PM
733 5:15 PM (Moved from Oral Session 411)
487 5:30 PM
488 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 4:00 PM
490 4:15 PM
491 4:30 PM
492 4:45 PM
493 5:00 PM
494 5:15 PM
495 5:30 PM
496 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 4:00 PM
498 4:15 PM
499 4:30 PM
500 4:45 PM
501 5:00 PM
502 5:15 PM
503 5:30 PM
504 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 4:00 PM
506 4:15 PM
507 4:30 PM
508 4:45 PM
509 5:00 PM
510 5:15 PM
511 5:30 PM
512 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 4:00 PM
514 4:15 PM
515 4:30 PM
516 4:45 PM
517 5:00 PM
518 5:15 PM
519 5:30 PM
520 5:45 PM

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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 4:00 PM
522 4:15 PM
523 4:30 PM
524 4:45 PM
525 5:00 PM
526 5:15 PM
527 5:30 PM
528 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)

Molecular Therapy Volume 24, Supplement 1, May 2016 xi

Copyright © The American Society of Gene & Cell Therapy
 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

xii 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 10:15 AM
722 10:30 AM
723 10:45 AM
724 11:00 AM
725 11:15 AM
726 11:30 AM
727 11:45 AM
728 12:00 PM

Molecular Therapy Volume 24, Supplement 1, May 2016 xiii

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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 10:15 AM
730 10:30 AM
731 10:45 AM
732 11:00 AM
120 11:15 AM (Moved from Poster Session I)
734 11:30 AM
735 11:45 AM
736 12:00 PM

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 10:15 AM
738 10:30 AM
739 10:45 AM
740 11:00 AM
741 11:15 AM
742 11:30 AM
743 11:45 AM
744 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 10:15 AM
746 10:30 AM
747 10:45 AM
748 11:00 AM
749 11:15 AM
750 11:30 AM
751 11:45 AM
752 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 10:15 AM
754 10:30 AM
755 10:45 AM
756 11:00 AM
757 11:15 AM
758 11:30 AM
759 11:45 AM
760 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 10:15 AM
762 10:30 AM
763 10:45 AM
764 11:00 AM
765 11:15 AM
766 11:30 AM
767 11:45 AM
768 12:00 PM

xiv Molecular Therapy Volume 24, Supplement 1, May 2016

Copyright © The American Society of Gene & Cell Therapy