Molecular therapy : the journal of the American Society of
`’ Gene Therapy.
`RB155.8 .M656__—siv. 18, suppl. 1 (2010 May)
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`Miltenyi Ex. 1028 Page 1
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`Miltenyi Ex. 1028 Page 1
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`

`

`
`D SPONSOR
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`Miltenyi Ex. 1028 Page 3
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`Miltenyi Ex. 1028 Page 3
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`

`

`rr
`Volume 18, Supplement 1, May 2010
` The Journal of the American Society of Gene & Cell Therapy
`
`MoLEcuLaR
`"THERAPY
`
`
`
`
`
`AMERICAN
`SOCIETY OF GENE
`& CELL THERAPY
`
`43"" Annual Meeting
`May 19 - May 22, 2010
`Washinton, DC
`
`Abstract Review
`Committee
`
`Committee Chair:
`John F. Engelhardt, PhD
`
`RNA Virus Vectors
`Coordinating Reviewer:
`Arthur W. Nienhuis, MD
`
`Reviewers:
`Christopher Baum, MD
`Fabio Candotti, MD
`Robert G. Hawley, PhD
`David W. Russell, MD, Ph
`
`AAV Vectors
`Coordinating Reviewer:
`John A. Chiorini, PhD
`
`Reviewers:
`Douglas M. McCarty, PhD
`David V. Schaffer, PhD
`Matthew D. Weitzman, PhD
`Xiao Xiao, PhD
`
`Adenovirus and Other
`DNA Virus Vectors
`Coordinating Reviewer:
`Philip Ng, PhD
`
`Reviewers:
`Anja Ehrhardt, PhD
`Joseph C. Glorioso, Ill, PhD
`Florian Kreppel, PhD
`Robin J. Parks, PhD
`
`DNA Vectorology &
`Gene Targeting
`Coordinating Reviewer:
`Michele P. Calos, PhD
`
`Reviewers:
`Deborah Gill, PhD
`Perry B. Hackett, PhD
`Jakob Giehm Mikkelsen, PhD
`Matthew H. Porteus, MD, PhD
`
`American Society of Gene & Cell Therapy
`13Annual Meeting
`
`May 19-May 22, 2010
`Washington, DC
`
`Wednesday, May 19, 2010
`
`Scientific Symposium 100, 8:30 AM-1:30 AM
`Cell Therapy BioProcessing Symposium
`Co-Chairs: Boro Dropulic, PhD, ThomasTillett
`Speakers: David L. Urdal, PhD, Gary C. du Moulin, PhD, MPH, Jane S. Lebkowski, PhD,
`Madhusudan V. Peshwa, PhD, Mitchell Finer, PhD, Michelle Williams, PhD
`
`Scientific Symposium 101, 8:30 AM-11:30 AM
`A Global Gene Therapy Renaissance
`Chair: John Rasko, MBBS, PhD
`Speakers; De-Chao Yu, PhD, Yasutomo Nasu, MD, PhD, Chae-Ok Yim, PhD, Seppo Yld-Herttuala,
`MD, PhD, FESC, Alessandro Aiuti, MD, PhD
`
`Lunch Break, 11:30 AM-1:00 PM
`
`Scientific Symposium 110, 1:00 PM—3:00 PM
`Geneand Cell Therapy Translational Studies
`Chair; J. Kevin Donahue, MD
`Speakers: J. Kevin Donahue, MD, Douglas Losordo, MD, Barry J. Byrne, MD, PhD,
`Patrick Most, MD
`
`Scientific Symposium 111, 1:00 PM—3:00 PM
`Ethical Issues with Embryonic Stem Cells and Induced Pluripotent
`Stem Cells
`Chair: Jonathan Kimmelman, PhD
`Speakers: Jonathan D. Moreno, PhD, Insoo Hyun, PhD, Jeremy Sugarman, MD, MPH
`Panel Discussion: James Ellis, PhD, Roger H. Bertolotti, PhD
`
`Scientific Symposium 112, 1:00 PM—3:00 PM
`Barriers to HSC Gene Therapy
`Co-Chairs: Manuel Grez, PhD, Harry L. Malech, MD
`Speakers: Mary C. Dinauer, MD, PhD, Luigi M. Naldini, MD, PhD, C. Anthony Blau, MD,
`Christopher Baum, MD
`
`Scientific Symposium 113, 1:00 PM—3:00 PM
`Delivery of Oligonucleotide Therapeutics
`Speakers: Joseph DeSimone, PhD, Steven F. Dowdy, PhD, Joshua Mendell, MD, PhD
`
`Break, 3:00 PM - 3:30 PM
`
`Scientific Symposium 120, 3:30 PM—5:00 PM
`Emerging Field Review: Genomic Basis of Human Disease
`Chair: Paul B. McCray, Jr, MD
`Speakers: Eric D, Green, MD, PhD, Leslie G. Biesecker, MD, William A. Gahl, MD, PhD
`
`SSSSSSSSSN
`Molecutae TherapyVolume 18, Supplement |, May 2010
`i
`PMizht © The American Society of Gene & Cell Therapy
`
`Miltenyi Ex. 1028 Page 4
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`

`7a
`
`
`
`oo
`
`Education Session 121, 3:30 PM—5:00 PM
`Emerging Field Review: Regulatory RNAs
`Co-Chairs: Alfred Lewin, PhD, Bernhard Gentner, MD
`Speakers: Brian D. Brown, PhD, Ryan L. Boudreau, PhD, Bernhard Gentner, MD
`
`Education Session 122, 3:30 PM—5:00 PM
`Topical Review: Cell Therapy
`Chair: Edwin M. Horwitz,MD, PhD
`Speakers: Edwin M. Horwitz, MD, PhD, Peter Johnston, MD, FACC, Dao Pan, PhD
`
`Education Session 123, 3:30 PM—5:00 PM
`Topical Review: Gene Therapy for Eye Diseases
`Co-Chairs: Peter A. Campochiaro, MD, Tal Kafri, PhD
`Speakers: Peter A. Campochiaro, MD, John G. Flannery, PhD, Bill Hauswirth, PhD
`
`Education Session 124, 3:30 PM—5:00 PM
`Topical Review: Stem Cell Basics
`Chair: William F. Goins, PhD
`Speakers: G. lan Gallicano, PhD, Ming Zhan, PhD
`
`Break, 5:00 PM-—5:30 PM
`
`Education Session 130, 5:30 PM-7:00 PM
`Emerging Field Review: Progress Towards Using ES/iPS Cells for
`Regenerative Medicine
`Chair: Matthew H. Porteus,MD, PhD
`Speakers; Ronald D.G. McKay, PhD, Elias T. Zambidis, MD, PhD, EvanY.
`Snyder,MD, PhD, FAAP
`
`Education Session 131, 5:30 PM—7:00 PM
`Emerging Field Review: Targeted Integration
`Chair: Fulvio Mavilio, PhD
`Speakers: J. Keith Joung, MD PhD, Andrew M. Scharenberg, MD, David W. Russell, MD, PhD
`
`Education Session 132, 5:30 PM—7:00 PM
`Topical Review: Gene Delivery to the Heart
`Chair: Dongsheng Duan, PhD
`Speakers: Dongsheng Duan, PhD, Roger J. Hajjar, MD, Charles R, Bridges, MD, ScD
`
`Education Session 133, 5:30 PM—7:00 PM
`Topical Review: Immunotherapy of Cancer
`Chair: Chiara Bonini,MD
`Speakers: Richard A. Morgan, PhD, Gianpietro Dotti, MD, Chiara Bonini, MD
`
`Education Session 134, 5:30 PM—7:00 PM
`Topical Review: Pre-existing Immunity to Gene Transfer Vectors
`Chair: Roland W. Herzog, PhD
`Speakers: Michael Cancro, PhD, Luk Vandenberghe, PhD, Katherine A. High, MD
`
`Exhibit Hall Welcome Reception, 7:00 PM-9:00 PM
`
`Physical Methods of p
`Coordinating Reviewer.Clivery
`Nancy Smyth Templeton, Php
`Reviewers:
`MichaelA, Barry, PhD
`Jeffrey Hughes, PharmD, Php
`Christian Plank, Php
`Chun Wang, PhD
`
`Chemical and
`Molecular Conjugates
`Coordinating Reviewer:
`Suzie H. Pun, PhD
`
`Reviewers:
`Hideyoshi Harashima, PhD
`Theresa M. Reineke, PhD
`Tatiana Segura, PhD
`Millicent Sullivan, PhD
`
`|
`
`Genetic and Metabolic
`|Diseases Gene & Cell Therapy
`Coordinating Reviewer:
`|
`Cary Harding, MD
`
`Reviewers:
`Dwight Koeberl, MD, PhD
`Brendan Lee, MD, PhD
`Jon A. Wolff, MD
`
`Cardiovascular Gene &
`Cell Therapy
`Coordinating Reviewer:
`Barry J. Byrne, MD, PhD
`
`Reviewers:
`David A. Dichek, MD
`Roger J. Hajjar, MD
`Cathryn S. Mah, PhD
`Christina A. Pacak, PhD
`
`|
`
`|
`|
`
`ic
`
`Neurologic & Ophthalm
`Gene & Cell Therapy
`Coordinating Reviewer:
`Michael Kaplitt, MD, PhD
`
`Reviewers:
`Krystof Bankiewicz, MD, PhD
`Nicholas Boulis, MD
`Miguel Sena-Esteves, PhD
`Deborah J. Watson, PhD
`
`sj)
`Lt)
`SEnT
`ment | May 201
`+) herp
`Molecular Therapy Volume. 18, Suppkemer
`Copynghe © The AmericanSociety of Gene & 1
`Miltenyi Ex. 1028 Page 5
`
`Miltenyi Ex. 1028 Page 5
`
`

`

` Thursday, May 20, 2010
`
`Scientific Symposium 214, 10:30 AM-—12:30 PM
`Geneand Cell Therapeutic Approaches for Musculo-Skeletal Diseases
`Co-Chairs; Paula Clemens, MD, Alan J. Nixon, DVM, MS
`Speakers: Edward M. Schwarz, PhD, Steven C. Ghivizzani, PhD, Joel R. Chamberlain, PhD, Carmen
`Bertoni, PhD
`
`Scientific Symposium 215, 10:30 AM-—12:30 PM
`Novel Cell and Gene Therapy Interventions for the Lung
`Chair: Bruce Bunnell, PhD
`Speakers: Daniel Weiss,MD, PhD, Paul B. McCray, Jr, MD, Peter I. Letkes, PhD, John F:
`Engelhardt, PhD
`
`Scientific Symposium 216, 10:30 AM—12:30 PM
`NIH Resourcesfor Cell and Gene Therapy Investigators
`Speakers; Cheryl L. McDonald, MD, Daniel Rosenblum, MD, Catherine McKeon, PhD, DavidG.
`Badman, PhD, Roy S. Wu, PhD, Danilo A, Tagle, PhD, Traci Heath Mondoro, PhD, Sonia Skarlatos,
`PhD, Ying Huang, PhD
`
`Individual speaker
`Presentation titles will be
`included in the 13"
`Annual Final Program,
`which will be distributed
`at the meeting in
`Washington, DC. Please
`See the Final Program for
`Comprehensive program
`information, including room
`assignments, speaker order
`and individual presentation
`titles,
`
`Molecular TherapyVolume. 18, Supplement |, May 2010
`iil
`Copyright © The American Society of Gene & Cell Therapy
`
`Lunch Break, 12:30 PM—2:00 PM
`
`Miltenyi Ex. 1028 Page 6
`
`
`
`George Stamatoyannopoulos Lecture 200, 8:00 AM—9:15 AM
`Chair; Barrie J. Carter. PhD
`Speaker: Hal E. Broxmeyer, PhD
`
`Exhibit Hall Coffee Social, 9:15 AM—10:30 AM
`
`Scientific Symposium 210, 10:30 AM-12:30 PM
`Clinical Trials
`Chair, R. Jude Samulski, PhD
`Speakers; Steven A. Rosenberg, MD, PhD, fan MacLachlan, PhD, Bill Hauswirth, PhD, R. Jude
`Samulski, PhD
`
`Scientific Symposium 211, 10:30 AM-12:30 PM
`Tissue Specific Stem Cells
`Chair: Carolyn Lutzko, PhD
`Speakers: Markus Grompe, MD, Carolyn Lutzko, PhD, Mervin C. Yoder,MD
`
`Scientific Symposium 212, 10:30 AM—12:30 PM
`Cell and Gene Therapy Approaches for Genetic Diseases: Progress and
`Challenges
`Chair, Charles P. Venditti, MD, PhD
`Speakers: Nathalie Cartier-Lacave, MD, Alessandra Biff, MD, Ronald D.G. McKay, PhD
`Robert D. Steiner, MD
`
`Scientific Symposium 213, 10:30 AM-—12:30 PM
`How Viruses Cause Disease: Implications for Vector and Vaccine
`Development
`Chair; Roberto Cattaneo, PhD
`Speakers; Lynn W. Enquist, PhD, Jeffrey Bergelson, MD, Bernard Moss, MD, PhD, Roberto
`Cattaneo, PhD
`
`= M
`
`usculo-skeletal Gene &
`Cell Therapy
`Coordinating Reviewer:
`Alan R. Davis, PhD
`
`Reviewers:
`Jeffrey Chamberlain, PhD
`Paula Clemens, MD
`Scott Q. Harper, PhD
`Terri A. Zachos, DVM, PhD
`
`Infectious Diseases
`and Vaccines
`Coordinating Reviewer:
`John A, Zaia, MD
`
`Reviewers:
`Ann M. Leen, PhD
`Bruce E. Torbett, PhD, MSPH
`Grant D. Trobridge, PhD
`Dorothee von Laer, MD
`
`Cancer- Immunotherapy
`Coordinating Reviewer:
`Helen E. Heslop, MD
`
`Reviewers:
`Ronald Alvarez, MD
`Jeffrey J. Molldrem, MD
`James J. Mulé, PhD
`David B. Weiner, PhD
`
`Cancer — Oncolytic Viruses
`Coordinating Reviewer:
`Ronald Rodriguez, MD, PhD
`
`Reviewers:
`Winald Gerritsen, MD, PhD
`Chinghai Kao, PhD
`David H. Kirn, MD
`Masato Yamamoto, MD, PhD
`
`Miltenyi Ex. 1028 Page 6
`
`

`

`
`
`Meet-the-Investigator 220, 12:30 PM—1:45 PM
`Musculo-Skeletal
`Speakers: Jeffrey Chamberlain, PhD, Paula Clemens, MD
`
`Meet the Investigator Session 221, 12:30 PM—2:00 PM
`Cancer Stem Cells
`Speakers. Timothy P. Cripe, MD, PhD, Samuel D. Rabkin, PhD
`
`Oral Abstract Session 230, 2:00 PM-4:00 PM
`AAVVectors: Vector Trafficking .cccetsessenesssecctecssecneessesscrsetssenuseensssneansaseestiareasatersiversteetens PooL
`Co-Chairs: David W. Schaffer, Xiao Xiao
`+
`2:00 PM
`5
`3:00PM
`2
`2:15 PM
`6
`3:15 PM
`3
`2:30 PM
`7
`3:30PM
`4
`2:45PM
`8
`3:45PM
`
`.
`.
`.
`Oral Abstract Session 231, 2:00 PM—4:00 PM
`DNA Vectorology & Gene Targeting .ceccessscssesessesessesnssossessrenisseensnsscersnsteennnssnesrsstseessartsessests Prot
`Co-Chairs: Perry Hackett, Matthew Porteus
`9
`2:00 PM
`13 3:00 PM
`10 2:15PM
`14 3:15PM
`11.
`2:30 PM
`15 3:30 PM
`12 2:45 PM
`16 3:45 PM
`
`Oral Abstract Session 232, 2:00 PM—4:00 PM
`Neurologic & Ophthalmic Gene & Cell Therapverssserersserertrserserinnsnnessnenrsnsenereseeseseneearsnene Pol
`Co-Chairs: Michael Kaplitt, Deborah J. Watson
`17 2:00 PM
`21
`3:00 PM
`18 2:15PM
`22 3:15PM
`19 2:30 PM
`23 3:30 PM
`20 2:45 PM
`24 3:45 PM
`
`Oral Abstract Session 233, 2:00 PM—4:00 PM
`Cancer — Immunotherapy T Cell Studies... ices cesnees esis esieseeeeessiesnessnessieseneentesneds pSll
`Co-Chairs: Dean Lee, Chiara Bonini
`25 2:00 PM
`29 3:00 PM
`26 2:15 PM
`30 3:15 PM
`27 2:30 PM
`31
`3:30 PM
`28 2:45PM
`32 3:45 PM
`
`Cancer- Targeted Gene &
`“
`Cell Therapy
`coordinating Reviewer:
`William S.M. Wold, Php
`
`Reviewers:
`Karen S. Aboody, MD
`Frank Marini, PhD
`Stephen J. Russell, MD, PhD
`Richard G. Vile, PhD
`Hematologic and
`Immunologic Ge
`Cell Therapy &
`Coordinating Reviewer:
`.
`rn
`Mich
`ichel Sadelain, MD, PhD
`
`Reviewers:
`Gay Crooks. MD
`David W. Emery, PhD
`David Klatzmann, MD, PhD
`Adrian J. Thrashey.
`MD
`rasher, MD, PhD
`.
`Lung and Respiratory
`Disease Gene &
`Cell Therapy
`Coordinating Reviewer:
`Terence R. Flotte, MD
`
`Reviewers:
`Dongsheng Duan, PhD
`Larry G, Johnson, MD
`Maria Limberis, PhD
`Patrick L. Sinn, PhD
`
`Immunologic & Host
`Responsesin Gene &
`Cell Therapy
`Coordinating Reviewer:
`Pedro Lowenstein, MD, PhD
`
`Reviewers:
`Renier J. Brentjens, MD, PhD
`Oral Abstract Session 234, 2:00 PM—4:00 PM
`Evanthia Galanis, MD, DSc
`p.sl4
`Cancer-Oncolytic Viruses 1.........
`Go-Chalre: David Me Kim win EEBARORENTREEETEEE Maciej Lesniak, MD. FACS
`33 2:00 PM
`37 3:00 PM
`Lily Wu, MD, PhO
`34 2:15PM
`38 3:15PM
`35 2:30 PM
`39 3:30 PM
`36 2:45 PM
`40 3:45PM
`
`Gene Regulation
`Coordinating Reviewer.
`Olivier Danos, PhD
`
`Oral Abstract Session 235, 2:00 PM-4:00 PM
`Immunologic & Host Responses in Gene & Cell Therapy
`Co-Chairs: Evanthia Galanis,Maciej Lesniak
`41
`2:00 PM
`45 3:00 PM
`42 2:15PM
`46 3:15PM
`43 2:30PM
`47 3:30PM
`44 2:45PM
`48 3:45PM
`
`P
`
`...cccccscesesceecsssseerssseeerereeeseertenes
`
`DOL 7
`
`P
`
`Reviewers:
`Michael N. Antoniou, PhD
`Giuliana Ferrari, PhD
`Dirk M. Nettelbeck, PhD
`Paul D, Robbins. PhD
`
`Oral Abstract Session 236, 2:00 PM—4:00 PM
`Oligonucleotide & RNAI Therapeutics [vicececesencecesenenesesieseseiesenneseeeees Pro
`Co-Chairs: Dieter Gruenert, Faye Rogers
`49 2:00 PM
`53 3:00 PM
`50 2:15PM
`54 3:15 PM
`51
`2:30 PM
`55 3:30 PM
`52 2:45 PM
`56 3:45 PM
`
`(a
`:
`M
`5
`§
`Sa
`aplement
`1.
`MAN
`C)
`oe
`C
`.
`Sone & !
`on
`lv
`olecular TherapyVolume. 18. Supplemes
`pe itl
`Copyright & The American Sociery of Gene &
`Miltenyi Ex. 1028 Page 7
`
`Miltenyi Ex. 1028 Page 7
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`

`

`Exhibit Hall Networking Reception, 6:15 PM—8:15 PM
`
`Poster Session I, 6:15 PM-8:15 PM
`
`AAVVectots 1 vsesecrecee
`(Abstracts 57 through72)
`
`Adenovirus and Other DNA Vitus Vectors I veccesssscseesessseeeesesssecssnesenevsneneeseenaeeneeaggaetersatnense
`(Abstracts 73 through 86)
`
`Targeting Gene Modification and Integration srscssssessesssecsesnssnsesesnesecsesscessssssneanesnesnssaneates
`(Abstracts 87 through 99)
`
`Physical Methods of Delivery sissssssessscscsssvassossssssssecsssecsecssesersnscenetesserseesserssesatsecssstaccoestastice
`(Abstracts 100 through 116)
`
`pS23
`
`p.S29
`
`p.s34
`
`p.S38
`
` Outstanding New Investigator Symposium 240, 4:30 PM—6:15 PM
`
`
`
`Oligonucleotide &
`RNAi Therapeutics
`Coordinating Reviewer:
`Peter Glazer, MD, PhD
`
`Reviewers:
`Alan Gewirtz, MD
`Paloma H. Giangrande, PhD
`Dieter C. Gruenert, PhD
`Mark A, Kay, MD, PhD
`
`Stem Cell Therapies
`Coordinating Reviewer
`Donald B. Kohn, MD
`
`Reviewers
`Emanuela M. Bruscia, PhD
`R. Keith Humphries,
`MD, PhD
`Carolyn Lutzko, PhD
`Alice F. Tarantal, PhD
`
`Cell Processing and
`Vector Production
`Coordinating Reviewer:
`Isabelle Riviére, PhD
`
`Reviewers:
`Adrian P. Gee, PhD
`Stephen M. Kaminsky, PhD
`Bruce L. Levine, PhD
`J. Fraser Wright, PhD
`
`Special thanksto the abstract
`reviewersfortheir time and
`expertise.
`
`Chemical and Molecular Conjugates [oo cccececscceees cece ceneneescenenseseessnenensreanes
`(Abstracts 117 through 132)
`
`Genetic and Metabolic Diseases Gene & Cell Therapy T wcscicccseeseseseessnereen
`(Abstracts 133 through 147
`
`beeen p.S44
`
`dtateessenes p.S50
`
`Musculo-skeletal Gene & Cell TherapyI ,.cesssssessessrresssnrsnsnssssersersecrsssseseessseeenseteneetensearaneenses
`(Abstracts 148 through 160)
`
`.p-S57
`
`Infectious Diseases and Vaccines: Vaccine Related Gene Transfer Research .....esserreces
`(Abstracts 161 through 174)
`
`p-862
`
`Cancer — Immunotherapy [ vccccccecseceeeseeseessesssiesesnesssneseeseenssssnasensaeensssensaeenieas
`(Abstracts 175 through 190)
`
`ssesseseeses p.S67
`
`Cancer-Oncolytic Viruses 1 jecssscsseesseesessseseesssseesesssssnesnesavesneestecenesstersasensenseneersssenteceesensenss
`(Abstracts 191 through 204)
`
`Cancer-Targeted Gene & Cell Therapy I sessssessscssessssssssesectsersesesneecerssersesstenssresnesntaneeneenreeneenas
`(Abstracts 205 through 219)
`
`Hematologic and Immunologic Gene & Cell Therapy 1 seeccccsescscseeseeseecesneeneeneaneaeengnaneaneenne
`(Abstracts 220 through 233 and Abstract 869 which has
`been moved from Poster Session III)
`
`p-573
`
`p.S78
`
`p.S84
`
`Gene Regulation. ccssissncarscessnsorssesuvennaniaaseatnanrvosnusetnnsseannrnseonyrapsennnnssenenacaunccspesenconerenannseanegnieertrees ProOD
`(Abstracts 234 through 244)
`
`Oligonucleotide & RNAi Therapeutics ] ceccccsecssesecesesneenesneeescntsnenmsentsnesnersneesneeeneraes p.S93
`(Abstracts 245 through 259)
`
`Stem Cell Biologyasssevcitsviavsvincsianansonneainnacreancccrieonnasnnieabircuinnsaldinnanatenin Goamnarmamaanseees P99
`(Abstracts 260 through 273)
`
`Cell Processing and Vector Production T sesssessssessneessrecerseseennessnsssntesneetinesnseereeentesntssereeeteees PO LOF
`(Abstracts 274 through 282)
`
`eee
`Vv
`Molecular Therapy Volume. 18, Supplement L, May 2010
`Copyright © The American Society of Gene & Cell Therapy
`
`Miltenyi Ex. 1028 Page 8
`
`Miltenyi Ex. 1028 Page 8
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`

`

`
`
`Foundation Symposium 250, 7:00 PM-10:00 PM
`Expanding Frontiers in Spinal Muscular Atrophy Gene Therapy
`Chair, Christian L. Lorson, PhD
`Speakers: Christian L. Lorson, PhD, C. Firank Bennett, PhD, Marco Passini, PhD, Brian K.
`Kaspar, PhD, Petra Kaufman, MD
`
`Friday, May 21, 2010
`Top Abstracts Plenary Session 300, 8:00 AM-10:00 AM
`Plenary Abstract S@ssiOn.s.csccssesssecsecsseesneseseceerasiesnsstssitasersseesseenesnseasencnscanentantecaantiny p.S107
`Co-Chairs: Stanton L. Gerson, John J. Rossi
`283 8:05AM
`286 9:05 AM
`284 8:25AM
`287 9:25AM
`285 8:45AM
`288 9:45AM
`
`|
`
`Break, 10:00 AM—10:30 AM
`
`Scientific Symposium 310, 10:30 AM-12:30 PM
`Personalized Cell and Gene Therapy
`Chair: John J. Nemunaitis, MD
`Speakers: John J. Nemunaitis, MD, Lilia Bi, PhD, Carl H. June, MD, Jeffrey Schlom, PhD
`
`Scientific Symposium 311, 10:30 AM-—12:30 PM
`Stem Cells and Malignancy
`Chair: Evan Y. Snyder,MD, PhD, FAAP
`Speakers: Inder Verma, PhD, RogerJ. Packer, MD, Evan Y. Snyder, MD, PhD, FAAP, Carl Bart
`Rountree,MD
`
`Scientific Symposium 312, 10:30 AM-12:30 PM
`Current Status of Genetic Vaccines
`Chair: Stephen Gottschalk, MD
`Speakers: Si-Yi Chen, MD, PhD, Jerald C. Sadoff, MD, Sattva S. Neelapu, MD, Stephen
`Gottschalk, MD
`
`Scientific Symposium 313, 10:30 AM—12:30 PM
`Precise Genetic and Post-transcriptional Modification of Stem Cells
`Chair: Matthew H. Porteus,MD, PhD
`Speakers: Linzhao Cheng, PhD, Dieter C. Gruenert, PhD, Robert Blelloch, MD, PhD
`
`Scientific Symposium 314, 10:30 AM-12:30 PM
`New Physical Gene Delivery System and Vectorology
`Chair; Shulin Li, PhD
`Speakers: Christian Plank, PhD, Declan M. Soden, PhD, Kate E. Broderick, PhD, Laurence JN
`Cooper, MD, PhD
`
`Scientific Symposium 315, 10:30 AM—12:30 PM
`Genetic Modification of Stem Cells for Therapeutic Applications
`Chair; Frederic D. Bushman, PhD
`Speakers: Christofvon Kalle, MD, Matthew H. Porteus, MD, PhD, Frederic D. Bushman, PhD, Ko
`Mitani, PhD
`
`Lunch Break, 12:30 PM—2:00 PM
`
`:
`VI
`
`cae
`:
`Molecular Therapy Volume.18. Supplemen
`Copyright © The American Society of Gene <
`Miltenyi
`Ex. 1028 Page 9
`
`fA 20M
`Cell Thea
`
`Miltenyi Ex. 1028 Page 9
`
`

`

`
`
`Oral Abstract Session 320, 2:00 PM—4:00 PM
`Clinical Gene & Cell Therapy Oral Abstract SeSsion...ccccccceciseeeseseseseseenecntsnsenetneseneeees p.S110
`Co-Chairs: Guangping Gao, Matthew Porteus
`289 2:00 PM
`293 3:00 PM
`290 2:15PM
`294 3:15 PM
`291 2:30PM
`295 3:30PM
`292 2:45 PM
`296 3:45 PM
`
`Oral Abstract Session 321, 2:00 PM-—4:00 PM
`RNAVitus VeCtors.ccccccccsccccssesscescesteccscssseetsnecrenenecteceeec serene siene cise eceigeenieracimessieciescenenseneeeey p.S113
`Co-Chairs: Fabio Candotti, Robert G. Hawley
`297 2:00PM
`301
`3:00 PM
`298 2:15PM
`302 3:15PM
`299 2:30PM
`303 3:30 PM
`300 2:45 PM
`304 3:45PM
`
`Oral Abstract Session 322, 2:00 PM—4:00 PM
`AAVVectors: Vector Biology...
`suceasacsaeeeaseesarenetitsueneeenearenenseearaneaseenseaees Pood L LT
`Co-Chairs: Douglas M. McCarty, MatthewDz ‘Weitzman.
`305 2:00 PM
`309 3:00 PM
`306 2:15PM
`310 3:15 PM
`307 2:30 PM
`311
`3:30 PM
`308 2:45PM
`312 3:45 PM
`
`Oral Abstract Session 323, 2:00 PM—4:00 PM
`Gene Therapy for Lysosomal Storage Diseases and Other Genetic Disorders... p.S120
`Co-Chairs: Cary Harding, Chester B. Whitely
`313 2:00 PM
`317 3:00 PM
`314 2:15PM
`318 3:15 PM
`315 2:30 PM
`319 3:30 PM
`316 2:45 PM
`320 3:45 PM
`
`Oral Abstract Session 324, 2:00 PM—4:00 PM
`Physical Methods of Delivery
`aedcramaanEnaemeotte eeeiE ee ieee
`325 3:00 PM
`321
`2:00 PM
`326 3:15PM
`322 2:15PM
`327 3:30 PM
`323 2:30 PM
`328 3:45 PM
`324 2:45PM
`
`p.S123
`
`Oral Abstract Session 325, 2:00 PM—4:00 PM
`Cancer-Targeted Gene & Cell Therapy ccsssssenescneessenssnsensentsentennsentenesvenseenvecseenneenstnnsennnsnees
`Co-Chairs: David Dingli, Frank Marini
`329 2:00 PM
`333 3:00 PM
`330 2:15PM
`334 3:15 PM
`331
`2:30 PM
`335 3:30 PM
`332 2:45PM
`336 3:45 PM
`
`p.S127
`
`Oral Abstract Session 326, 2:00 PM—4:00 PM
`Hematologic/Immunologic: Basic...cece nines irsseriesiceseeey p.S130
`Co-Chairs: David W. Emery, David Klatzmann
`337 2:00 PM
`341
`3:00 PM
`338 2:15 PM
`342 3:15PM
`339 2:30 PM
`343 3:30 PM
`340 2:45 PM
`344 3:45 PM
`
`
`
`Break, 4:00 PM—4:30 PM
`
`Presidential Symposium 330, 4:30 PM-6:30 PM
`Chair; Kenneth Cornetta,MD
`Speakers: Katherine A, High, MD, Jean Bennett, MD, PhD, David A. Williams, MD
`
`Exhibit Hall Networking Reception, 6:30 PM—8:30 PM
`
`“ —
`Molecular Therapy Volume. 8, Supplement 1, May 2010
`Vil
`Copyright © The American Society of Gene & Cell Therapy
`
`Miltenyi Ex. 1028 Page 10
`
`Miltenyi Ex. 1028 Page 10
`
`

`

`
`
`Poster Session II, 6:30 PM-8:30 PM
`
`RNAVirus Vectors I...
`(Abstracts 345 through361).
`
`(Abstracts 3362 through377).
`
`wup.S134
`
`wae p-S140
`
`Adenovirus and Other DNA Virus Vectors I] v.ceccssesssessessseesseasenesesrenssassaneersnsseetereasesees
`(Abstracts 378 through 391)
`
`p-S147
`
`Chemical and Molecular Conjugates ID eccentrics
`(Abstracts 392 through 407)
`
`Genetic and Metabolic Diseases Gene & Cell Therapy I] cecccccssssssnssensseseenteneesnenneenes
`(Abstracts 408 through 422 and Abstract 764 which has
`been moved from Poster Session Il!)
`
`p.S151
`
`p.S157
`
`Cardiovascular Gene & Cell Therapy crecsssserecctesesssnsensenesseeesnessennananmearentsansnnentenesrsneeagssess
`(Abstracts 423 through 440)
`
`p.S164
`
`Ophthalmic Disorders and Technological Advances ...ecsseecstsssesnnerensesssiscuensseesenensnecsnnees
`(Abstracts 441 through 453)
`
`.pS171
`
`Musculo-skeletal Gene & Cell Therapy HD veessessessessessecssessensesesessessssstsansssieanerbsnesarseanenrenes
`(Abstracts 454 through 468)
`
`Cancer — Immunotherapy ID ssssssssesesssnsesnesnssssssieiersssnisccsssssecstesseessstenarseeuveneeusenvaneenepnaannenes
`(Abstracts 469 through 487)
`
`Cancer-Oncolytic Viruses TD ciceccsisssssssesecsesseseeseeeassnensenseetsseeiseneeesesnenen
`(Abstracts 488 through 506)
`
`p.S176
`
`p.s181
`
`p.S189
`
`Cancet-Targeted Gene & Cell TherapyII..
`wa
`(Abstracts 507 through 520; Abstract 519 withdrawn)
`
`a p.8196
`
`Hematologic and Immunologic Gene & Cell Therapy TH ccssessssescessssessereeseensessenseseenn
`(Abstracts 521 through 534)
`
`-p.S201
`
`Immunologic & Host Responses in Gene & Cell Therapysuisse
`(Abstracts 535 through 554)
`
`p-S206
`
`Oligonucleotide & RNAI Therapeutics TD ssscecsesceecrsssseesseeeseesnerensetsnennsnmnensneranmnnnerennrsennecany
`(Abstracts 555 through 569)
`
`p.S214
`
`Cell Processing and Vector Production LD yisssussessssssesesssenessesenesseneeneeniesnisneennssteenmsniesseens
`(Abstracts 570 through 579)
`
`p-S220
`
`Foundation Symposium 340, 7:00 PM-10:00 PM
`Progress and Prospects for Gene Therapy for CGD and Other Primary
`Immunodeficiencies
`Chair: Fabio Candotti, MD
`Speakers: Mary C. Dinauer, MD, PhD, Alessandro Aiuti, MD, PhD, Harry Malech, MD, Manual
`Grez, PhD, Adrian Thrasher,MD, PhD, Toni Cathomen, PhD, Linzhao Cheng, PhD
`
`Individual speaker
`presentation titles will be
`included in the 13"
`Annual Final Program,
`whichwill be distributed
`at the meeting in
`Washington, DC. Please
`see the Final Programfor
`comprehensive program
`information, including
`room assignments,
`speaker order and
`individual presentation
`titles.
`
`vill
`
`(ys mith
`Molecular Therapy Volume. 18, Supplem:
`Gene &Cal
`THER
`Copyright © ‘The American Society of
`Miltenyi Ex. 1028 Page 11
`
`Miltenyi Ex. 1028 Page 11
`
`

`

`ASGCTBusiness Meeting (with continental breakfast),
`8:00 AM-—8:30 AM
`
`Scientific Symposium 400, 8:30 AM-10:30 AM
`Late Stage Industry ClinicalTrials
`Co-Chairs: Douglas J. Jolly, PhD, Thomas Tillett
`Speakers; Robert Sims, MD, Ryuichi Morishita, MD, PhD, Sander van Deventer, MD, PhD
`
`Scientific Symposium 401, 8:30 AM—10:30 AM
`Local Delivery of Chemical Gene Therapy - Mechanisms, Quantitative
`Understanding and Applications of Biodegradable Carriers
`Chair: Ernst Wagner, PhD
`Speakers: Lonnie D. Shea, PhD, Aliasger K. Salem, PhD, David M. Lynn, PhD, Daniel G.
`Anderson, PhD
`
`Scientific Symposium 402, 8:30 AM-—10:30 AM
`EngineeredT Cells in the Clinic: Emerging Clinical and Regulatory Issues
`Chair: Jacqueline Corrigan-Curay, MD, JD
`Speakers; Daniel M. Takefman, PhD, Joy Cavagnaro, PhD, DABT, RAC, Carl H. June, MD
`Panel Discussion: Helen E. Heslop, MD, Michel Sadelain, MD, PhD, Steven A.
`Rosenberg, MD, PhD
`
`Scientific Symposium 403, 8:30 AM-—10:30 AM
`MechanismsofInnate Immunity Influencing Gene Therapy
`Chair: David B. Weiner. PhD
`Speakers: Valder Arruda, MD, PhD, Yiping Yang, MD, PhD, David B. Weiner, PhD, Michael T.
`Lotze, MD
`
`Scientific Symposium 404, 8:30 AM—10:30 AM
`MicroRNA and siRNA Rolesin Disease Progression and Development of
`Novel
`Chair: Maria G, Castro, PhD
`Speakers: Anna M. Krichevsky, PhD, M. Maral Mouradian, MD, Sean E. Lawler, PhD,
`Al Charest, PhD
`
`Scientific Symposium 405, 8:30 AM—10:3