THE NOW & FUTURE OF RNA THERAPEUTICS
Awaji, Japan
Monday, June 19– Friday, June 23, 2023
Monday 5:30 pm – 8:00 pm
Monday 8:00 pm – 10:00 pm
Tuesday 9:30 am – 12:10 pm
Tuesday Lunchtime
Tuesday 2:00 pm – 6:15 pm
Tuesday 7:15 pm – 9:30 pm
Tuesday 9:30 pm – 10:30 pm
Wednesday 9:30 am – 12:15 pm
Wednesday
Wednesday
Thursday
Thursday
Thursday 6:30 pm –
1:30 pm – 6:00 pm
7:00 pm – 9:4
0 pm
9:30 am – 12:00 pm
2:00 pm – 4:45 pm
9:30 pm
Friday
Awaji Yumebutai Conference Center
Meeting venue: Main Hall, 2
nd
floor of the Conference Center
Poster session: Reception Hall B Foyer, 2
nd
floor of Conference Center
CSHA office: Room 202
Breakfast*: Coccolare, 2
nd
floor of Grand Nikko Awaji
Dinner (June 19 - 21): Reception Hall B, 2
nd
floor of Conference Center
Lunch (June 20-22): Reception Hall B, 2
nd
floor of Conference Center
Cocktails (June 22): Cielo, 1
st
floor of Grand Nikko Awaji
Banquet (June 22): Stella, 1
st
floor of Grand Nikko Awaji
*
Welcome Remarks
1 Opening Keynote Session
Dinner & Sake Barrel Breaking
2 RNA Targeting Mechanisms and
Applications
Parallel Mentoring Workshops for
Graduate Students and Post Docs
3 RNAs and Disease Targets or Drugs
Poster Session
Social Hour
4 Development of Small RNA (siRNA,
LNA, PNA, ASO) Drugs
Excursion to Hemeji Castle
5 RNA Delivery
6 Genome Editing
7 RNA Chemistry
Closing Remarks
Cocktails and Banquet
Departure
Only available for guests staying at Grand Nikko Awaji
Abstracts are the responsibility of the author(s) and publication of an
abstract does not imply endorsement by Cold Spring Harbor Asia of the
studies reported in the abstract.
These abstracts should not be cited in bibliographies. Material herein
should be treated as personal communications and should be cited as
such only with the consent of the author.
Please note that ANY photography or video/audio recording of oral
presentations or individual posters is strictly prohibited except with the
advance permission of the author(s), the organizers, and Cold Spring
Harbor Asia.
v
PROGRAM
MONDAY, June 19—5:30 PM
Welcome Remarks
CSH-Asia and Meeting Organizers
Marco Mangone
Arizona State University, USA
SESSION 1 KEYNOTE SESSION: RNA FROM BIRTH TO DEATH /
FROM BASIC TO TRANSLATIONAL
Chairperson: Mikiko Siomi, University of Tokyo, Tokyo, Japan
Introduction to the CSHA Ken-Ichi Arai auditing program
Hisao Masai [35’+10’]
Presenter affiliation: Tokyo Metropolitan Institute of Medical Science,
Tokyo, Japan.
KEYNOTE SPEAKERS
Molecular pathogenesis and potential therapy for RNA
modopathy
Tsutomu Suzuki [35’+10’]
Presenter affiliation: University of Tokyo, Tokyo, Japan.
1
Nonsense-mediated mRNA decay is misregulated in Fragile X
Syndrome
Lynne E. Maquat [35’+10’]
Presenter affiliation: University of Rochester, Rochester, New York.
2
MONDAY, June 19—8:00 PM
Dinner and Sake Barrel Breaking
vi
TUESDAY, June 20—9:30 AM
SESSION 2 RNA TARGETING MECHANISMS AND APPLICATIONS
China
Ligang Wu, Shanghai Institute of Biochemistry and Cell
Biology, Shanghai, Ch
Chairpersons: Yuanchao Xue, Chinese Academy of Sciences, Beijing,
ina
Mapping RNA interactome to annotate the molecular function of
risk variants
Yuanchao Xue [20’+10’]
Presenter affiliation: Institute of Biophysics, Chinese Academy of
Sciences, Beijing, China.
3
Regulation of immune cell differentiation and activation by ASOs
targeting mRNA structures recognized by Regnase-1-related
RNases
Osamu Takeuchi [20’+10’]
Presenter affiliation: Kyoto University, Kyoto, Japan.
4
Break
gRNA dependent and gRNA independent off-target binding
activities of dCas13
Huanhuan Feng,
Zhixue Li, Hongdao Zhang, Ligang Wu [20’+10’]
Presenter affiliation: Shanghai Institute of Biochemistry and Cell
Biology, Shanghai, China.
5
In vivo self-assembled small RNAs as a new generation of RNAi
therapeutics
Chen-Yu Zhang [20’+10’]
Presenter affiliation: Nanjing University, Nanjing, China.
6
The piRNA pathway in Drosophila—How does the molecular
machinery protect the genome from transposon invasion in the
germline?
Mikiko C. Siomi [15’+5’]
Presenter affiliation: The University of Tokyo, Tokyo, Japan.
7
Parallel Mentoring Workshops
for Gra
TUESDAY, June 20—Lunchtime
duate Students and Post Docs
vii
TUESDAY, June 20—2:00 PM
SESSION 3 RNAs AS DISEASE TARGETS OR DRUGS
Chairpersons: Vinod Balachandran, Memorial Sloan Kettering Cancer
Pancreatic cancer—Exceptional survivors to RNA vaccines
Vinod P. Balachandran [20’+10’]
Presenter affiliation: Memorial Sloan Kettering Cancer Center, New
York, New York
Center, New York, New York, USA
Jinzhong Lin, Fudan University, Shanghai, China
.
8
KEYNOTE SPEAKER
Targeting RNA modification and RNA-RNA interaction for future
therapies
Chuan He [35’+10’]
Presenter affiliation: The University of Chicago, Chicago, Illinois.
9
RNA regulation in spermatogenesis and male infertility
Mo-Fang Liu [15’+5’]
Presenter affiliation: Shanghai Institute of Biochemistry and Cell
Biology, Shanghai, China.
10
Contribution and therapeutic implications of retroelement
insertions in genetic diseases
Boxun Zhao,
E. Alice Lee, Timothy W. Yu [15’+5’]
Presenter affiliation: Boston Children's Hospital, Boston,
Massachusetts.
11
Break
Single-cell stereo-seq reveals induced progenitor cells involved
in axolotl brain regeneration
Ying Gu [20’+10’]
Presenter affiliation: BGI, Shenzhen, China.
12
Splice modulating RNA targeting therapies for brain disorders:
from treating many to treating one
Willeke M. van Roon-Mom [20’+10’]
Presenter affiliation: Leiden University Medical Center, Leiden, the
Netherlands.
13
viii
Transcriptome changes and new therapeutic targets during the
initiation and progression of Duchenne Muscular Dystrophy in C.
elegans
Heather C. Hrach,
Shannon O'Brien, Hannah S. Steber, Jason
Newbern, Alan Rawls, Marco Mangone [15’+5’]
Presenter affiliation: The Biodesign Institute at Arizona State
University, Tempe, Arizona.
14
Nonsense-mediated RNA decay Is a unique vulnerability of
cancer cells with spliceosome gene mutations
Abigael Cheruiyot,
Claudia Cabrera, Shan Li, Zheng Yang, Sridhar N.
Srivatsan, Tanzir Ahmed, Yiwei Fu, Julie M. Bailis, Matthew J. Walter,
Zhongsheng You [15’+5’]
Presenter affiliation: Washington University in St. Louis, St. Louis,
Missouri.
15
mRNA selective regulation of translation nominates eIF3 as a
drug target in cancer
Haoran Duan,
Dieter A. Wolf [15’+5’]
Presenter affiliation: Westlake University, Hangzhou, China.
16
TUESDAY, June 20—7:15 PM
POSTER SESSION
Odd numbers present 7:15 pm – 8:15 pm
Even numbers present 8:15 pm – 9:30 pm
Reducing off-target effects of siRNA by introducing 2'-formamide
modification in the seed region
Seongjin An,
Yoshiaki Kobayashi, Kohei Nomura, Yasuaki Kimura,
Hiroshi Abe, Dai Akase, Misako Aida, Kumiko Ui-Tei
Presenter affiliation: The University of Tokyo, Chiba, Japan.
17
Characterization of circular RNAs in the regulation of
inflammatory responses
Shuya Hiroki,
Daisuke Ori, Taro Kawai
Presenter affiliation: Nara Institute of Science and Technology
(NAIST), Ikoma, Japan.
18
ix
Synthetic circular mRNA engineering for cell-type-specific
translational regulation
Shigetoshi Kameda,
Hirohisa Ohno, Hiroki Tanaka, Hidetaka Akita,
Hirohide Saito
Presenter affiliation: Center for iPS Cell Research and Application,
Kyoto University, Kyoto, Japan.
19
DNA damage stress-induced translocation of mutant FUS
proteins into cytosolic granules and screening for translocation
inhibitors
Masahiro Nogami,
Osamu Sano, Keiko Adachi-Tominari, Yoshika
Hayakawa-Yano, Takako Furukawa, Hidehisa Iwata, Kazuhiro Ogi,
Hideyuki Okano, Masato Yano
Presenter affiliation: Takeda Pharmaceutical Company Limited,
Fujisawa, Japan.
20
Development of single nucleotide mutation-distinguishable siRNA
for PIK3CA (SNPD-siPIK3CA) and its effects on cancer cells
Toshinori Ohyama,
Yoshiaki Kobayashi, Susumu Goyama, Kumiko Ui-
Tei
Presenter affiliation: The University of Tokyo, Tokyo, Japan.
21
Welcome to Join Zhejiang University RNA Medical Center
Hak Park,
Xiaoning Sun, Xin Li
Presenter affiliation: Zhejiang University School of Medicine,
Yiwu,Zhejiang, China.
22
Identification of the factors required for nuclear retention of
overexpressed mRNAs
Xiangchuan Wang,
Hongjie Zhang
Presenter affiliation: University of Macau, Macau, China.
23
Integrated database for RNA-targeting drug discovery
Chao Zeng,
Michiaki Hamada
Presenter affiliation: Waseda University, Tokyo, Japan; National
Institute of Advanced Industrial Science and Technology, Tokyo,
Japan.
24
DecoyFinder—Identification of decoy sequences in sets of
homologous RNA sequences
Mingyi Zhu,
Jeffrey Zuber, Zhen Tan, Gaurav Sharma, David H.
Mathews
Presenter affiliation: University of Rochester Medical Center,
Rochester, New York.
25
Social Hour
x
WEDNESDAY, June 21—9:30 AM
SESSION 4 DEVELOPMENT OF SMALL RNA (siRNA, LNA, PNA,
ASO) DRUGS
Chairperson: Isabel Aznarez, Stoke Therapeutics, Bedford,
Massachusetts
A novel RNA-based approach to treat genetic diseases via
upregulation of protein expression
Isabel Aznarez [20’+10’]
Presenter affiliation: Stoke Therapeutics, Bedford, Massachusetts.
26
Development of new molecular technologies for siRNA
therapeutics
Takeshi Wada [20’+10’]
Presenter affiliation: Tokyo University of Science, Noda, Japan.
27
A framework for individualized splice-switching oligonucleotide
therapy
Jinkuk Kim,
Timothy Yu [15’+5’]
Presenter affiliation: KAIST, Daejeon, South Korea.
28
Break
KEYNOTE SPEAKER
Designer DNA drug therapy for neurodegenerative disease
Don W. Cleveland [35’+10’]
Presenter affiliation: University of California at San Diego, La Jolla,
California.
29
Leveraging the REST-PTB axis to develop a therapeutic strategy
for brain repair
Xiang-Dong Fu [15’+5’]
Presenter affiliation: Westlake University, Hangzhou, China.
30
WEDNESDAY, June 21—1:30 PM
Excursion to Hemeji Castle
xi
WEDNESDAY, June 21—7:00 PM
SESSION 5 RNA DELIVERY
Chairperson: Zicai Liang, Ribo Life Science Co. Ltd., Beijing, China
siRNA therapeutics as the third wave of modern pharmaceutical
industry may profoundly change treatment of many chronic
diseases
Zicai Liang,
Sam Ma, Shan Gao, Li-Ming Gan, Hongyan Zhang
[20’+10’]
Presenter affiliation: Ribo Life Science Co. Ltd., Beijing, China.
31
An impact of helper lipids on extrahepatic delivery of nucleic
acids based on ionizable cationic lipids
Hideyoshi Harashima, Mahmoud Younis, Seigo Kimura, Yusuke Sato
[20’+10’]
Presenter affiliation: Hokkaido University, Sapporo, Japan.
32
Revolutionary gene therapy—AAV9 delivers hope for SMA and
ALS treatment through miRNA delivery
Jun-An Chen [15’+5’]
Presenter affiliation: Academia Sinica, Taipei, Taiwan.
33
Broadly protective COVID-19 mRNA vaccine—Beyond the spike
protein
Renee Hajnik,
Haitao Hu [15’+5’]
Presenter affiliation: University of Texas Medical Branch, Galveston,
Texas.
34
Break
The potential role of mRNA decay in transcription
Hanae Sato,
Robert H. Singer [15’+5’]
Presenter affiliation: Kanazawa University, Kanazawa, Japan; Albert
Einstein College of Medicine, Bronx, New York.
35
Broad-spectrum mRNA vaccine candidates against SARS-Cov-2
variants
Jinzhong Lin [15’+5’]
Presenter affiliation: Fudan Center for mRNA Translational Research,
Shanghai, China.
36
xii
THURSDAY, June 22—9:30 AM
SESSION 6 GENOME EDITING
Chairperson: Qiangfeng Cliff Zhang, Tsinghua University, Beijing,
China
SmrtNet: Predicting RNA-targeting small molecules using deep
neural network
Yuhan Fei, Jiasheng Zhang, Pengfei Wang, Jianbo Ma,
Zhuoer Dong,
Zilin Cai, Qiangfeng C. Zhang [20’+10’]
Presenter affiliation: Tsinghua University, Beijing, China; Tsinghua-
Peking Center for Life Sciences, Beijing, China.
37
Structure, function and engineering of the type III-E CRISPR
nuclease-protease complex
Hiroshi Nishimasu [20’+10’]
Presenter affiliation: University of Tokyo, Tokyo, Japan.
38
Single-base precision Cas13 enables systematic silencing of
oncogenic fusions
Wenxin Hu,
Amit Kumar, Teresa Sadras, Joseph Trapani, Paul G.
Ekert, Mohamed Fareh [15’+5’]
Presenter affiliation: Peter MacCallum Cancer Centre, Melbourne,
Australia; The University of Melbourne, Melbourne, Australia.
39
Break
How CRISPR-Cas system defend against RNA bacteriophage
Jiuyu Wang,
Liang Liu, Lilan You, Yanli Wang [20’+10’]
Presenter affiliation: Institute of Biophysics, Chinese Academy of
Sciences, Beijing, China.
40
Targeting the oncogenic protein YB-1 in glioblastoma by RNA
decoy oligonucleotides
Jin-Zhu Wang,
Hong Zhu, Pu You, Hui Liu, Wei-Kang Wang, Liusong
Yang, Zefeng Wang, Jingyi Hui [15’+5’]
Presenter affiliation: Chinese Academy of Sciences, Shanghai, China.
41
xiii
THURSDAY, June 22—2:00 PM
SESSION 7
Chairperson:
RNA CHEMISTRY
Chengqi Yi, Peking University, Beijing, China
CRISPR-free programmable RNA pseudouridylation to suppress
premature termination codons
Chengqi Yi [20’+10’]
Presenter affiliation: Peking University, Beijing, China.
42
Pum1 and Pum2 in embryonic stem cells are essential for neural
spatial patterning and germ cell inhibition
Ting Lu,
Shuo Shi [15’+5’]
Presenter affiliation: Shanghai Advanced Institute of Immunochemical
Studies, Shanghai, China.
43
Centromere transcription and centromere RNAs—Their roles,
regulation and separation of function during centromere
establishment and maintenance
Jing Zhu,
Hiroshi KIMURA, Karen Wing Yee YUEN [15’+5’]
Presenter affiliation: The University of Hong Kong, Hong Kong.
44
Break
High throughput discovery of ligandable RNA structures in the
human transcriptome
Lee E. Vandivier,
Luis Soares, Lu Han, Alexandra East-Seletsky,
Helene Chamberlin, Lindsay Boudreau, Anthony Montibello, Alex
Sexton, Herschel Mukherjee, Jennifer C. Petter, Dave Mauger, J.
Craig Blain, Kathleen E. McGinness [15’+5’]
Presenter affiliation: Arrakis Therapeutics, Waltham, Massachusetts.
45
How the tRNA m
5
C methyltranferases-NSUN2 and NSUN6-
recoginze and catalyze mRNA substrates
Cai-Tao Li,
Yuan-Yuan Zhang, Ru-Juan Liu [15’+5’]
Presenter affiliation: ShanghaiTech University, Shanghai, China.
46
Closing Remarks
Organizing Committee
THURSDAY, June 22—6:30 PM
COCKTAILS and BANQUET
