Preliminary Program

For more information, please visit www.kasbp.org

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WELCOME TO 2017 KASBP SPRING SYMPOSIUM

Korean American Society in Biotech and Pharmaceuticals (KASBP) welcomes you to 2017 KASBP Spring

Symposium, made possible by a generous support from Yuhan Co. Ltd. and Hanmi Pharmaceutical.

Built on a very successful event last year, 2017 KASBP Spring Symposium is coming back to Boston for the

second time. This event will promote and expand KASBP membership in the Greater Boston area and establish a

stronger and wider network amongst KASBP members in the northeast region. This symposium also provides an

opportunity for members to establish professional networks and share information and experiences in the pursuit

of excellence in pharmaceutical research and development.

The symposium organizing committee is also delighted to announce Dr. William C. Hahn as a keynote speaker.

Dr. Hahn is a world-renowned researcher in the field of cancer cell biology at Dana-Farber Institute and a

distinguished professor of medical oncology at Harvard University.

The committee is also proud and excited to present the outstanding list of speakers and panelists. Invited speakers

will share their experiences with cutting-edge science in early discovery research to the clinical trials in

biopharmaceutical research, which will be featured in detail during the three scientific sessions: 1) Immuno-

oncology / de novo protein design, 2) Korean industry innovation and translational research, 3) Diabetes research

and outcome trial as well as imaging technology for speech map. Invited panelist will participate in the discussion

with young scientists who are interested in pursuing a career in the industry. For the first time this year, the

committee is hosting a special event on Saturday night with three special guest presentations on biotechnology

venture opportunities and strategies. All sessions are carefully coordinated to enhance the understanding of topics

on biotechnology and pharmaceutical research, industry-academia alliance, and business development.

Continuing the tradition, KASBP-Yuhan and KASBP-Hanmi Fellowship Awards will be presented to young

scholars who will be selected among the graduate students and post-doctoral scholars in recognition for their

exceptional contribution to their respective research field.

We hope you to enjoy the cutting–edge science and have productive scientific exchanges with fellow scholars in

academia and members of pharmaceutical and biotechnology industries. More importantly, we hope the

symposium can serve as a fun and informative time to network with fellow Korean-American scientists.

With best wishes,

2017 KASBP Spring Symposium Organizing Committee

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2016-2017 KASBP OFFICERS

Title Name Affiliation

President JEONG, Jae Uk (정재욱) GSK

President Designated CHOE, Yun H. (최윤) Lucas & Mercanti

1st Vice President SUH, K. Stephen (서광순) Hackensack Med Center

2nd Vice President KIM, Sean (김승빈) Blueprint Medicines

Executive Director JIN, Yong Hwan (진용환) Samsung Biologics

Science Director Lee, Hyun-Hee (이현희) Merck

Program Director Hong, Peter (홍성원) Regeneron

Financial Director HWANG, Seongwoo (황성우) PTC Therapeutics

General Director CHOI, Suktae (최석태) Celgene

Web Director HEO, Jun Hyuk (허준혁) Merck

1st Membership Director KIM, Sahee (김사희) RevHealth, LLC

2nd Membership Director JEONG, Claire (정가영) GSK

Public Relations Director BANG, Hanseong (방한성) CMIC CMO USA Corp

YG Director YOU, Diana Dahea (유다혜) Rutgers University

Councilor MOON, Young-Choon (문영춘) PTC Therapeutics

Councilor LEE, Hak-Myung (이학명) Shire

Councilor KIM, Jae-Hun (김재훈) IFF

Councilor KIM, Youngsun (김영선) Adello Biologics

Councilor LIM, Sung Taek (임성택) Sanofi

Councilor KOH, Jong Sung (고종성) Genosco

Local Chapters

Boston Chapter President SHIN, Hyunjin (Gene) (신현진) Takeda

Boston Chapter Vice President LEE, Hyun-Hee (이현희) Merck

Boston Chapter General Director LEE, Dooyoung (이두영) Applied Biomath

Boston Chapter Treasurer KIM, Haley (김혜민) Takeda

Connecticut Chapter President KIM, Sung-Kwon (김성권) Alexion

DC Chapter President PARK, Sang Tae (박상태) Macrogen

DC Chapter Vice President SONG, Jeong Keun (송정근) L & J Biosciences

DC Chapter General Director KIM, Miha (김미하) Leidos Bimedical

DC Chapter Science Director LEE, Byung Ha (이병하) NeoImmuneTech

NJ Chapter President KIM, Youngsun (김영선) Adello Biologics

NJ Chapter Vice President JUNN, Eunsung (전은성) Rutgers University

Philadelphia Chapter President CHANG, KernHee (장건희) J&J

KASBP-SF

President MA, Sunghoon (마성훈) Exelixis

Vice President LIM, Hanjo (임한조) Genentech

Vice President YOO, Seung-Yun (유승연) Gilead

Executive Director JO, Hyunsun(조현선) Embedbio

Executive Director HWANG, Bum-Yeol (황범열) DuPont Pioneer

Science Director CHANG, Ji Hoon (장지훈) Amgen

Financial Director JEONG, Joon Won (정준원) Carmot

Public Relationship Director LIM, Min Young (임민영) ThermoFisher Scientific

General Director CHO, Hanna (조해나) Clovis Oncology

Web Director SOHN, Dongmin (손동민) UCSF

Councilor JOH, Danny (조현정) Sangamo

Councilor PARK, Chong Yon (박정연) UCSF

Councilor HAN, Wooseok (한우석) Novartis

Councilor KIM, Yong-Jae (김용재) Gilead

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SYMPOSIUM SCHEDULE AT A GLANCE

June 16 (Friday) June 17 (Saturday)

AM 7 7:30 – 8:30 AM Registration and

Breakfast

8:30 – 8:35 AM Opening Remarks 8

8:35 – 9:45 AM Scientific Session A

9

9:45 – 10:05 AM Sponsor Presentation

and Introduction of new KASBP chapters

10 10:05 –10:20 AM Coffee Break

10:20 –11:30 AM Scientific Session B

11

11:30 – 12:10 PM Fellowship Awards and

Presentations

PM 12 12:10 – 12:20 PM Group Photo

1 12:20 – 3:20 PM Lunch and Poster

Session 2

3 3:00-5:30 PM

Job Fair

-details will be available at

www.kasbp.org

3:20 – 5:05 PM Scientific Session C

4

5

5:30 – 6:30 PM

Registration and Networking

5:05– 5:10 PM Closing Remarks

6 Departure or Networking

Optional Dinner and Networking Session

(registeration required)

6:00 – 8:00 PM

6:30 – 7:50 PM

Opening & Congratulatory Remarks

and Dinner 7

8 7:50 – 8:45 PM

Keynote Presentation

8:45 – 9:00 PM

Sponsor Presentation

9 9:00 – 9:50 PM

Networking Session 1

10 9:50 – 10:40 PM

Networking Session 2 and Panel

Discussion: Pharma industry -

Academia

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SYMPOSIUM SCHEDULE IN DETAIL

June 16, 2017, Friday

Job Fair (details will be provided before the symposium at www. kasbp.org)

3:00 pm ~ 5:30 pm Contact: Suktae Choi (email: jobfairkasbp@gmail.com)

Registration & Networking

5:30 pm ~ 6:30 pm

Coordinators: Yun Choe (KASBP President-designated), Lucas & Mercanti

Sahee Kim, RevHealth, LLC

Opening & Congratulatory Remarks and Dinner

6:30 pm ~ 6:50 pm

Opening Remark

Jae Uk Jeong, KASBP President & GlaxoSmithKline

Congratulatory Remarks

Jong-Gyun Kim, Director, Head of Global Research Center, Yuhan Corporation

Jay S. Kim, School of Management, Boston University

Jung Hoon Woo, Director General of KHIDI USA

6:50 pm ~ 7:50 pm

Dinner

Toast: Tae-Ung Eom, President, Samyang Biopharmaceuticals Corp.

Keynote Presentation

7:50 pm ~ 8:45 pm

Coordinator: Sean Kim (2nd Vice president KASBP), Blueprint Medicines

Keynote Speaker:

William C. Hahn, Dana-Farber Cancer Institute, Harvard Medical School

“ Defining a Cancer Dependencies Map”

Sponsor Presentations

8:45 pm ~ 9:00 pm

Yuhan Corporation: Han-Joo Kim, Head, R&D Strategy and Partnering Team

Networking Sessions 1 & 2

9:00 pm ~ 9:50 pm & 9:50 pm ~ 10:40 pm

Organizer: Stephen Suh (1st Vice President, KASBP), Hackensack Med Center

Biology

A. Immuno-oncology/Autoimmune/Inflammatory

Moderators: Hyun-Hee Lee and Hyungwook Lim

B. Respiratory/metabolic/cardiovascular/Aging/mental/Neurogenerative

Moderators: Alex Yi and Kern Chang

C. Cell and Gene Therapy/Viral infection/Rare disease

Moderators: K. Stephen Suh and Hakryul Jo

Chemistry

Moderators: Sungtaek Lim and Mooje Sung

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PK/PD/pre-clinical/Clinical Science/CMC:

Moderators: Sean Kim and Peter Hong

BD/Legal/VC:

Moderators: Hanseong Bang and Yun Choe

Pharmacy/YG

Moderators: Dahea You and Sahee Kim

Pharma industry – Academia

9:50 pm ~ 10:40 pm

Moderator: Hyunjin (Gene) Shin, Takeda

Panelists:

Min-Kyu Cho, Novartis

Dann Huh, Biogen

Seunghee Jo, Agios

Dooyoung Lee, Applied Biomath

Elizabeth Paik, CRISPR Therapeutics

Young Generation (YG) group networking

10:40 pm ~

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June 17, 2017, Saturday

Registration & Light Breakfast

7:30 am ~ 8:30 am

Opening Remarks

8:30 am ~ 8:35 am

KASBP-Boston Chapter President: Hyunjin Shin, Takeda

Session A -------- Chair: Jaekyoo Lee, Genosco

8:35 am ~ 9:45 am

A-1: “Induction of Anti-Tumor Immunity by Targeting TIGIT in Solid Cancer”

Angie Park, OncoMed Pharmaceuticals

A-2: "Evolution of Complex Molecular Mechanism Dissected by De Novo Protein Design -

Computationally Designed Transmembrane Antiporter of Zinc and Proton"

Nathan Joh, Amgen (Former NIH Fellow at UCSF)

Sponsor Talk

9: 45 am ~ 9:55 am

Osong Medical Innovation Foundation: Tae Gyu Lee

Introduction of New KASBP Chapters

9: 55 am ~ 10:05 am

SF Chapter President: Sunghoon Ma, Exelixis

NJ Chapter President: Youngsun Kim, Adello Biologics

Coffee Break

10:05 am ~ 10:20 am

Session B ------- Chair: Mooje Sung, Novartis

10:20 am ~ 11:30 am

B-1: “Korea as Active Life Science Innovation Hub in Asia”

Jungkue Lee, Bridge Biotherapeutics

B-2: “Drug Discovery; Translating Science into Medicine”

Taeyoung Yoon, Dong-A Socio R&D Center

Fellowship Award Ceremony & Presentation ----- Chair: Hyun-Hee Lee, Merck

11:30 am ~ 12:10 pm

Group Photo Session

12:10 pm ~ 12:20 pm

Lunch & Poster Session

12:20 pm ~ 3:20 pm

Session C -------- Chair: Dooyoung Lee, Applied BioMath

3:20 pm ~ 5:05 pm

C-1: “The Role of Obesity-Induced Inflammation in The Development of Insulin

Resistance and Type 2 Diabetes”

Jongsoon Lee, Joslin Diabetes Center, Harvard Medical School

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C-2: “New Developments in Diabetes Outcomes Trials: The New Crosstalk between Cardiologists and

Diabetologists”

Alexander Yi, Novartis

C-3: “Speech Map: a New Imaging and Analysis Technique to Quantify Speech Using MRI”

Jonghye Woo, Harvard University

Closing Remarks

5:05 pm ~ 5:10 pm

KASBP President: Jae Uk Jeong, GlaxoSmithKline

Dinner & Networking (optional / registration required)

6:00 pm ~ 8:00 pm

Special Presentations

Jay S. Kim, Boston University

Sung Ho Park, SV Investment

Derek Yoon, AJU IB Investment

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KEYNOTE SPEAKER ABSTRACT

Defining a Cancer Dependencies Map

William C. Hahn, Harvard Medical School

Biography:

Professor of Medicine, Harvard Medical School; 2001-present; Chief of the Division of Molecular and Cellular Oncology

(2010-present), and Chair of the Executive Committee for Research (2015-present) at the Dana-Farber Cancer Institute;

Institute Member of the Broad Institute of Harvard and MIT (2004-present); Postdoctoral Fellow, Whitehead Institute for

Biomedical Research (1997-2001); Resident and Fellow, Massachusetts General Hospital and Dana-Farber Cancer Institute

(1996-1999); M.D., Harvard Medical School (1994); Ph.D. Harvard University (1994), A.B. Biochemical Sciences, Harvard

College (1987).

Abstract:

Although we now have a draft view of the genetic alterations that occur in human cancer, the number of mutations found

at low frequency and the molecular heterogeneity of most cancers makes identifying genes that contribute to cancer

phenotypes challenging. Determining the function of genes altered in cancer genomes is essential to develop new

therapeutic approaches. To complement these genome characterization studies, we have used genome scale gain and loss

of function approaches to identify genes required for cell survival and transformation. Specifically, we have performed

systematic studies to interrogate rare alleles found altered in cancer genomes and used advances in synthetic gene

synthesis to prospectively interrogate all possible alleles of known cancer genes. In parallel, we have performed both

genome scale RNAi and CRISRP-Cas9 screens in more than 500 cell lines to identify differentially essential genes and the

context that specifies gene dependency. These studies allow us to begin to define a global cancer dependencies map.

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SCIENTIFIC SESSION – SPEAKERS BIO AND ABSTRACTS

Session A

A-1: Induction of anti-Tumor Immunity by Targeting TIGIT in Solid Cancer

Angie Park, OncoMed Pharmaceuticals

Biography

Senior Director, OncoMed Pharmaceuticals, Inc, 2016 – present; Director, OncoMed Pharmaceuticals, Inc, 2013-2015;

Associate Director, OncoMed Pharmaceuticals, Inc, 2010-2012; Sr. Scientist, OncoMed Pharmaceuticals, Inc, 2005-2009;

Research Investigator, University of Michigan Cancer Center, MI, 2001-2005; Post-doc, University of Michigan Cancer

Center, MI, 1996-2000, Post-doc, Vollum Institute, OHSU, Portland, OR, 1994-1996; Ph.D., Indiana University School of

Medicine, Dept of Biochemistry and Molecular Biology, Indianapolis, IN, 1987-1994.

Abstract

Using OncoMed’s rabbit MAP Trap platform, we have developed antibodies against checkpoint inhibitor TIGIT. Anti-

TIGIT antibodies can block PVR ligand binding and inhibit TIGIT signaling. Anti-TIGIT antibody induced tumor specific

T-cell responses, particularly of the Th1 type, increased antigen-specific CD8 response, and promoted a reduction in Treg-

mediated immune-suppressive activity, leading to tumor growth suppression and generation of long-term immunological

memory against tumors.

A-2: Evolution of Complex Molecular Mechanism Dissected by De Novo Protein Design - Computationally Designed

Transmembrane Antiporter of Zinc and Proton

Nathan Joh, Amgen

Biography

Scientist, Amgen, Thousand Oaks CA 2016 – present; Development Scientist, Bayer HealthCare, Berkeley CA, 2014 –

2016; NIH Postdoctoral Fellow, UCSF Dpt of Pharmaceutical Chemistry, 2011 – 2014; NIH Postdoctoral Fellow, UPenn

Dpt of Biochemistry and Biophysics, 2009-2011; PhD, Biochemistry and Molecular Biology, UCLA Dpt. Of Chemistry and

Biochemistry (2009)

Abstract

De-novo computational design appertains to achieving the desired function by building the protein from scratch. So, this

approach allows active testing of principles governing the protein structure, function and dynamics, in contrast to the passive

investigations involving traditional biochemical approaches, such as mutagenesis and protein chain truncation. Here, I

designed a 25-residue-long peptide, dubbed Rocker, that self-assembles into a tetrameric helical bundle that harnesses proton

concentration gradient to actively transport Zn(II) ions, and vice versa, across the lipid bilayer in vesicles. X-ray crystal

structures show the the intended peptide-peptide interface. Nuclear magnetic resonance shows stable, conformationally

dynamic tetrameric bundle. Rocker illuminates the possible evolutionary pathway for natural transporter proteins. How

protein design can be implemented in therapeutics development will also be discussed.

Session B

B-1: Korea as Active Life Science Innovation Hub in Asia

Jungkue Lee, Bridge Biotherapeutics

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Biography

Mr. James Junkgue Lee has served Bridge Biotherapeutics as CEO since its inception in September 2015. Prior to Bridge,

he founded Rexbio, specialized in discovery of monoclonal antibody for the treatment of pancreatic cancer. He co-founded

CrystalGenomics in 2000 and had played key roles in financing and business development until 2017. He started industry

career at LG Chemical Ltd (currently LG Life Sciences) 1993 after earning MS degree at Department of Chemistry, Seoul

National University.

Abstract

Korea has made steady and robust progress in research capability in academia and development capabilities in industry.

Recent global partnering of Korean biopharma companies with global pharma companies has proven Korea’s such improved

capabilities.

At the same time, VC industry and equity stock market have grown steadily for last two decade, supporting R&D focused

biotech companies have spun out of universities and local pharma companies. This 20-minute talk will provide several

examples research intensive local pharma companies and VC-backed biotech companies which are competing with global

biopharma companies, to recruit talent scientist and entrepreneurs from around the world. 1st generation biotech

entrepreneurs who started biotech around 2000 have been looking around to find novel sciences and talents domestically

and internationally, which will provide different opportunities to KASBP communities..

B-2: Drug Discovery; Translating Science into Medicine

Taeyoung Yoon, Dong-A Socio R&D Center

Biography

Senior Vice President, Don-A Socio R&D Center, 2012 – present; Senior Investigator, Novartis, Cambridge, MA, 2004-

2012; Neurogen Corp, CT, 1996-2004; PhD, Chemistry, Yale University, CT, 1989-1994; M.S. (1987) and B.S. (1985),

Chemistry, Seoul National University, Korea

Abstract

First-in-class drug discovery is a process through which novel target hypotheses are translated into innovative clinical

applications. During such a process, identification and optimization of ‘tool compounds’ is intertwined with increasing

levels of validation of the target concept. Taking as an example the recent success in global license-out of the MER

Tyrosine Kinase program, the speaker will elaborate further on what it takes to build a ‘discovery engine’ that can form the

foundation upon which to grow competitive in the global bio-pharmaceutical market.

Session C

C-1: The Role of Obesity-Induced Inflammation in The Development of Insulin

Resistance and Type 2 Diabetes

Jongsoon Lee, Joslin Diabetes Center, Harvard Medical School

Biography:

Assistant Professor, Harvard Medical School, Boston, MA, 2009–present; Assistant Investigator, Joslin Diabetes Center,

Boston, MA, 2006–present: Instructor, Harvard Medical School, 1999–2009; Research Associate, Joslin Diabetes Center,

MA, 1999–2006; Postdoctoral Research Fellow, Joslin Diabetes Center/Harvard Medical School, Boston, MA, 1995–1999;

Postdoctoral Research Associate, Boston University, School of Medicine, Boston, MA, 1993–1995; Ph.D. Biochemistry,

Department of Biochemistry, Boston University, School of Medicine, Boston, MA (1993); Research Scientist, Genetic

Engineering Institute, Korean Institute of Science and Technology, Seoul, Korea, 1986–1988; M.S. Biochemistry,

Department of Zoology, College of Natural Science, Seoul National University, Seoul, Korea (1985); B.A. Physiology,

Department of Zoology, College of Natural Science, Seoul National University, Seoul, Korea (1983).

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Abstract

Obesity is the major cause of the development of insulin resistance and Type 2 Diabetes. Recently, the notion that obesity-

induced inflammation mediates the development of insulin resistance in animal models and humans has been gaining strong

support. Furthermore, numerous studies have also shown that immune cells in local tissues, in particular in visceral adipose

tissue, play a major role in the regulation of obesity-induced inflammation. It has been shown that obesity disrupts the

immune balance by suppressing anti-inflammatory cells (e.g., regulatory T cells [Tregs]) while simultaneously activating

pro-inflammatory cells (e.g., adipose tissue macrophages [ATMs]). Many studies from the classical immunology field show

that complex cross-regulating interactions between different immune cell types control inflammation. However, the roles

these interactions play have not been studied extensively in the metabolism field. We have recently shown that natural killer

(NK) cells play a critical role in the development of obesity-induced inflammation and insulin resistance, in part by

controlling ATM activation and adipose tissue inflammation. Hence, our studies may provide important preclinical evidence

for the notion that obesity-induced inflammation regulated by adipose NK cells could be a therapeutic target for the

treatment of insulin resistance and Type 2 Diabetes.

C-2: New Developments in Diabetes Outcomes Trials: The New Crosstalk between Cardiologists and Diabetologists

Alexander Yi, Novartis

Biography

Senior Investigator II, Translational Medicine Expert, Novartis Institutes for BioMedical Research, Cambridge, MA 2013

– present; Instructor of Medicine, Division of Cardiology, Massachusetts General Hospital, Boston, MA 2013-2013;

Research Fellow in Medicine, Division of Cardiology, Massachusetts General Hospital, Boston, MA 2008-2012; Cardiology

Fellow, Division of Cardiology, Massachusetts General Hospital, Boston, MA 2006-2012; Residency, Internal Medicine,

New York Presbyterian Hospital-Weill Cornell Medical College, New York, NY 2003-2006; M.D., University of California,

San Francisco (2003); Ph.D., University of California, San Francisco (2001); B.S., Biology, Massachusetts Institute of

Technology (1994)

Abstract

Cardiovascular disease is a major life-threatening complication of patients with type 2 diabetes mellitus. Thus, the

publication of meta-analyses suggesting an increased risk of CV events associated with the thiazolidinedione rosiglitazone

caused great concern and led to a 2008 FDA guidance that new antidiabetic agents must rule out excess cardiovascular risk

prior to approval. Nearly a decade later, recent results of large placebo-controlled studies of SGLT2 inhibitors and GLP-1

agonists have demonstrated for the first time the potential for antidiabetic drugs to reduce the risk of heart disease and heart

failure. These positive results have spurred interest among cardiologists in the potential for diabetes medicines to reduce the

risk of cardiovascular disease in high risk diabetes patients.

C-3: Speech Map: a New Imaging and Analysis Technique to Quantify Speech Using MRI

Jonghye Woo, Harvard University

Biography

Assistant Professor, Harvard Medical School and Massachusetts General Hospital, Boston, MA, 2015-Present; Postdoctoral

Research Fellow and Faculty member, University of Maryland and Johns Hopkins University, Baltimore, MD, 2010-2014;

Research Associate, Cedars-Sinai Medical Center, Los Angeles, CA, 2009-2010; Ph. D., Electrical Engineering, University

of Southern California, Los Angeles (2009), M.S., Electrical Engineering, University of Southern California, Los Angeles

(2007); B.S. Electrical Engineering, Seoul National University, Seoul, South Korea (2005).

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Abstract

Quantitative measurement of functional and anatomical traits of 4D tongue motion in the course of speech remains a major

challenge inscientific research and clinical applications. In this talk, I will introduce MRI techniques including high-

resolution, diffusion, cine- and tagged-MRI and associated image/motion analysis techniques to measure tongue anatomy

and motion. I will then introduce a statistical multimodal atlas of 4D tongue motion using healthy subjects that enables a

combined quantitative characterization of tongue motion in a reference anatomical configuration. This atlas framework,

termed speech map, combines cine- and tagged-MRI to provide both the anatomic reference and motion information during

speech. Using this framework, the anatomic configuration of the tongue appears motionless, while the motion fields and

associated strain measurements change over the time course of speech. In addition, to form a succinct representation of the

highdimensional and complex motion fields, machine learning techniques are carried out to characterize the central

tendencies and variations of motion fields of our speech tasks. Our framework provides a platform to quantitatively and

objectively explain the differences and variability of tongue motion by illuminating internal motion and strain that have so

far been intractable. The findings are used to understand how tongue function for speech is limited by abnormal internal

motion and strain in tongue cancer and ALS (Amyotrophic Lateral Sclerosis) patients.

SPECIAL BREAK-OUT SESSION – SPEAKER BIO AND ABSTRACT

Boston’s bio innovation ecosystem

Jay S. Kim, Boston Univeristy

Biography

Dr. Jay Kim is Associate Professor of Operations and Technology Management at Boston University's Questrom School of

Business, where he teaches courses in operations management, global operations strategy, supply chain management, and

product innovation. He was the department chair in 1995-97, and the faculty director of the School’s various international

programs in 1997-2006. He was the research director of Global Manufacturing Futures Project in 1994-97. Professor

Kim’s research is focused on developing and implementing global operations and value chain strategies. Particularly, he

is investigating the complementary effects from two dominant forces of the 21st century economy that radically change

competitive requirements for global companies – accelerating technological innovations in a wide range of sectors and

vigorous economic development in emerging market countries. He has given lectures on global operations strategy, quality

improvement, value chain enhancement, and innovative business models to managers of various technology-intensive

companies, such as IBM, Raytheon, Johnson & Johnson, Carrier, Sanyo and Toshiba of Japan, and Korean conglomerates

like Daewoo, LG, SK, KEPCO, and Samsung. In 1997, he served as a special advisor for Chairman Kim Woo-Choong of

Korea's Daewoo Group.

Abstract

The speech focus on Boston’s bio innovation ecosystem that facilitates the translational development of scientific discovery

into commercial innovation. Using the examples of LabCentral and IBE (Institute for Biomedical Entrepreneurship), the

speaker discusses the business-side stories of B2B (bench to bedside) or L2M (lab to market) challenges faced by bio

researchers and scientists and presents opportunities for future innovators and entrepreneurs.

IPO Processes in KOSDAQ

Sung Ho Park, SV Investment Corporation

Biography

Mr. Sung Ho Park is the CEO of SV Investment Corporation (SVI), a Korean venture capital firm he founded in 2006. He

was the CEO of Hanwha-SV Meister SPAC from 2010 to 2013. Sung Ho has also served as a non-executive member of

the IPO Review Board for KOSDAQ. SVI was established as an equity investment company by leveraging the heritage

of SV Partners (formerly S-IPO), a consulting company he founded in 2000 specializing in IPO advisory. Over the past

decade, SVI has become a leading private equity and venture capital firm by focusing on investments in life sciences,

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electronic components, and telecommunications sectors. SVI primarily invests in South Korean enterprises as a direct

investor and has broadened its geographical reach and access via secondary investments outside of Korea, (e.g. Korea-China

Joint Investment Funds). Prior to founding the SV Group, Sung Ho was a senior fund manager at Hyundai Investment &

Securities Co., Ltd. Before his tenure at Hyundai Group, Sung Ho was a member of Investment Banking groups at Tongyang

Securities and Dongsuh Securities, where he managed IPOs and M&A transactions. Before his career in investment banking

and venture capital, Sung Ho was a licensed KICPA at Samil PricewaterhouseCoopers Korea. He consulted more than 20

companies on IPO listing and managed more than 15 M&A transactions. Sung Ho holds BA in Business Administration

from Sogang University

Abstract

In 1996, the KOSDAQ Securities Exchange was established as a new stock exchange independent from the Korea Stock

Exchange. Since then KOSDAQ has rapidly grown not only in terms of its total market capitalization but also in quality of

listed companies in terms of their financial performance, level of transparency and increased liquidity. In 2005, the

KOSDAQ introduced a new IPO track for enterprises and entrepreneurs developing unique and cutting-edge technology.

This new IPO review program has allowed qualified technology-based growth companies to receive a P&L waiver, a critical

part of traditional IPO review processes in Korea. This waiver has enabled qualified companies to be listed on KOSDAQ

before they could generate revenues. Over the following decade, this new system has overcome the challenges of risk and

transparency issues. Moreover, it has contributed to creating a more business-friendly environment by providing

opportunities for qualified technology-based enterprises and their private-stage investors to finance growth and corporate

development. Since 2005, 33 of 37 companies listed on KOSDAQ via this tech-based IPO program have come from the

bio/life sciences sector. SVI & SVP have been the leading advocates and pioneers of this new IPO program. This lecture

will help attendees to gain a greater understanding and potential of KOSDAQ IPO processes. Details of the regulations will

be explained using recent examples of technology-based IPOs on KOSDAQ

US Life Science Ecosystem

Derek (Dong Min) Yoon, Aju IB Investment US

Biography

Mr. Yoon is Partner at Aju IB Investment, one of leading venture capital firms in Korea. Based in Boston, Massachusetts,

Mr. Yoon oversees Aju IB’s international investments with focus on U.S. based life science companies and early stage start-

ups with novel science. Mr. Yoon also leads multiple cross-border strategic partnerships by leveraging his relationship with

various pharmaceutical companies in Korea. Currently, he is involved in multiple overseas venture capital fundraising

projects by interacting with Korean governmental institutional funds as well as various private investors in Asia.

Additionally, Mr. Yoon is a board member of Clearside Biomedical and Trefoil Therapeutics, and a board observer at several

other private biotechnology companies. Prior to joining Aju IB Investment, he spent more than fifteen years in alternative

(VC/PE) investments and healthcare corporate banking where he took numerous responsibilities in portfolio management,

deal structuring, and fundraising. His previous experience includes venture investment management at Kibo Capital, the

oldest venture fund in Korea; portfolio management at RBS Citizens Healthcare Banking Group; and venture accelerator

management at Berwind Private Equity, a multi-generational family office located in Harvard, Massachusetts. Mr. Yoon

earned his M.B.A. from Babson College, M.S.F. from Boston College and B.S. in Chemical Engineering from Yonsei

University in Seoul, Korea.

Abstract

The speech will provide analysis on US life science ecosystem to define key components of the ecosystem as well as role

of each component. The speaker will also share his professional experience with US-based VC investors and scientists/

researchers, which will conclude benchmark points and recommendations to Korea.

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POSTER SESSION – AWARDEE ABSTRACTS

2016 SPRING FELLOWSHIP AWARDEES

KASBP-HANMI FELLOWSHIP

KASBP-YUHAN FELLOWSHIP

Min-Kyung Choo, Ph.D

Harvard Medical School

Soo Seok Hwang, Ph.D

Yale University

Heeoon Han, Ph.D

University of Pennsylvania

Hanseul Yang

Rockefeller Univeristy

Ji-Hoon Park, Ph.D

NIH

Hong-Yeoul Ryu, Ph.D.

Yale University

HANMI AND YUHAN FELLOWSHIP AWARDEE ABSTRACTS

TLR sensing of bacterial spore-associated RNA triggers host immune responses with detrimental effects

Min-Kyung Choo, Yasuyo Sano, Changhoon Kim, Kei Yasuda, Xiao-Dong Li, Xin Lin, Mary Stenzel-Poore, Lena

Alexopoulou, Sankar Ghosh, Eicke Latz, Ian R. Rifkin, Zhijian J. Chen, George C. Stewart, Hyonyong Chong and Jin Mo

Park

Cutaneous Biology Research Center, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA

02129, USA

The spores of pathogenic bacteria are involved in host entry and the initial encounter with the host immune system. How

bacterial spores interact with host immunity, however, remains poorly understood. Here, we show that the spores of Bacillus

anthracis (BA), the etiologic agent of anthrax, possess an intrinsic ability to induce host immune responses. This

immunostimulatory activity is attributable to high amounts of RNA present in the spore surface layer. RNA-sensing TLRs,

TLR7, and TLR13 in mice and their human counterparts, are responsible for detecting and triggering the host cell response

to BA spores, whereas TLR2 mediates the sensing of vegetative BA. BA spores, but not vegetative BA, induce type I IFN

(IFN-I) production. Although TLR signaling in itself affords protection against BA, spore RNA-induced IFN-I signaling is

disruptive to BA clearance. Our study suggests a role for bacterial spore-associated RNA in microbial pathogenesis and

illustrates a little known aspect of interactions between the host and spore-forming bacteria.

Transcription factor YY1 restrains differentiation and function of regulatory T cells by blocking Foxp3 expression

and activity

Hwang, Soo Seok

Department of Immunobiology, Yale University School of Medicine (P.I : Richard A. Flavell, Ph.D, FRS)

Regulatory T (Treg) cells are essential for maintenance of immune homeostasis. Foxp3 is the key transcription factor for

Treg cell differentiation and function; however, molecular mechanisms for its negative regulation are poorly understood.

Here we show that YY1 expression is lower in T reg cells than Tconv cells, and its overexpression causes a marked

reduction of Foxp3 expression and abrogation of suppressive function of T reg cells. YY1 is increased in Treg cells under

inflammatory conditions with concomitant decrease of suppressor activity in dextran sulfate-induced colitis model. YY1

inhibits Smad3/4 binding to and chromatin remodeling of the Foxp3 locus. In addition, YY1 interrupts Foxp3-dependent

target gene expression by physically interacting with Foxp3 and by directly binding to the Foxp3 target genes. Thus,

YY1 inhibits differentiation and function of T reg cells by blocking Foxp3.

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Anion Relay Chemistry: The Development of an Effective Diastereoselective [3+2] Annulation Tactic Exploiting an

Aldol/Brook Rearrangement/Cyclization Cascade

Heeoon Han, and Amos B. Smith III*

Department of Chemistry, University of Pennsylvania 231 S. 34th street, Philadelphia, PA 19104

An effective [3+2] annulation tactic for the construction of diverse bicyclic compounds possessing highly functionalized

cyclopentane rings has been developed exploiting ketone enolates as the initial nucleophile for Anion-Relay-Chemistry

(ARC). The protocol entails a highly diastereoselective Aldol/Brook rearrangement/cyclization cascade, thus providing

diverse bicyclic building blocks with high functional handles in one-pot to access biologically active natural products such

as capnellenes (4), lucinone (5), africanol (6) and rhodomollein (7) and analogues thereof.

Epithelial-Mesenchymal Micro-niches Govern Stem Cell Lineage Choices

Hanseul Yang,1 Rene C. Adam,1 Yejing Ge,1 Zhong L. Hua,1 and Elaine Fuchs1,2 1Robin Neustein Laboratory of Mammalian Development and Cell Biology2Howard Hughes Medical Institute,The

Rockefeller University, New York, NY 10065, USA

Adult tissue stem cells (SCs) reside in niches, which, through intercellular contacts and signaling, influence SC behavior.

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Once activated, SCs typically give rise to short-lived transit-amplifying cells (TACs), which then progress to differentiate

into their lineages. Here, using single-cell RNA-seq, we unearth unexpected heterogeneity among SCs and TACs of hair

follicles. We trace the roots of this heterogeneity to micro-niches along epithelial-mesenchymal interfaces, where

progenitors display molecular signatures reflective of spatially distinct local signals and intercellular interactions. Using

lineage tracing, temporal single-cell analyses, and chromatin landscaping, we show that SC plasticity becomes restricted in

a sequentially and spatially choreographed program, culminating in seven spatially arranged uni-lineage progenitors within

TACs of mature follicles. By compartmentalizing SCs into micro-niches, tissues gain precise control over morphogenesis

and regeneration: some progenitors specify lineages immediately, whereas others retain potency, preserving self-renewing

features established early while progressively restricting lineages as they experience dynamic changes in microenvironment.

Efficacy of a cell-permeable metformin analog on inhibiting mitochondrial respiration and preventing tumorigenesis

in a mouse model of Li-Fraumeni syndrome

Park, JH, Wang, P-y, and Hwang, PM

Laboratory of Cardiovascular and Cancer Genetics, NHLBI, NIH, Building 10-CRC, Room 5-5288, Bethesda, MD 20814

Rationale – Metformin, widely used to treat type 2 diabetes, has been associated with decreased incidence of cancer.

Mechanistically, metformin has pleiotropic cellular effects that include activation of AMPK and inhibition of mitochondrial

respiration. In a pilot study, we reported that patients with Li-Fraumeni syndrome, a hereditary cancer predisposition

disorder caused by germline p53 mutations, display evidence of increased mitochondrial metabolism (Wang PY et al, NEJM

2013). Moreover, we showed that both the genetic and metformin-mediated disruption of mitochondrial respiration in a

mouse model of LFS delayed tumorigenesis (Wang PY, …Park JH, et al, JCI 2017). Because metformin requires organic

cation transporters to cross the cell membrane, we reasoned that a membrane-permeable analog of metformin HL156A (Ju,

KD et al, AJPRP, 2016) that potently inhibits respiration and crosses the blood brain barrier merited further study to

determine its efficacy in preventing cancer.

Objective – To test whether the metformin analog HL156A would attenuate de novo tumorigenesis in a mouse model of

LFS at treatment doses that correspond to its inhibition of mitochondrial respiration.

Methods and results – We first examined the effect of HL156A on mitochondrial respiration in HCT116 human colon cancer

cells. Compared to metformin, HL156A caused relatively acute inhibition of respiration likely due to its cell permeable

nature. Lactate release was concomitantly increased, consistent with a compensatory increase in glycolysis associated with

inhibition of oxidative phosphorylation. The potencies of HL156A and metformin in inhibiting cell growth were assessed

by a viability assay in HCT116 cells. The IC50 of HL156A and metformin were ~50 µM and ~1200 uM, respectively,

representing a ~24-fold higher potency of HL156A compared with metformin. The cytostatic effect of these compounds

was dependent on mitochondrial function as non-respiring SCO2-/- HCT116 cells were relatively resistant to growth

inhibition.

To test the in vivo efficacy of HL156A in cancer prevention, a LFS mouse model with knockin of the p53 R172H mutation

was treated with HL156A in drinking water at 0.2 or 0.4 mM which corresponds to a daily dose of 15 or 30 mg/kg,

respectively. As positive control, LFS mice were also treated with metformin (7.6 mM) in drinking water which we

previously reported to increase their cancer-free survival. Because LFS mice develop thymic lymphomas, we measured

oxygen consumption in freshly dissociated thymic cells after 4 wk of treatment. Treatment with 0.4 mM HL156A resulted

in a similar degree of inhibition of mitochondrial respiration as 7.6 mM metformin. After confirming inhibition of respiration

in vivo, LFS mice were treated with HL156A at 0.4 or 0.6 mM in drinking water and monitored for cancer-free survival.

Interestingly, treatment with 0.4 mM HL156A increased the median survival time of LFS mice by 20%, comparable to the

effect of metformin, while the higher dose of 0.6 mM failed to improve survival likely due to some cytotoxicity.

Conclusions – In comparison to metformin, HL156A was more potent at inhibiting mitochondrial respiration both in vitro

and in vivo although it showed similar efficacy at preventing cancer in LFS mice. The membrane transporter-independent

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uptake and blood-brain barrier permeability of HL156A may confer additional advantages for chemoprevention in humans

that are not apparent using a mouse model that develops mostly thymic lymphomas. Further studies may be helpful to

determine the merits of HL156A or other derivatives of metformin in preventing cancer.

Acknowledgements - Compound HL156A was kindly provided by Sung-wuk Kim, ImmunoMet, Houston, TX.

Loss of the SUMO protease Ulp2 triggers a specific multichromosome aneuploidy

Hong-Yeoul Ryu (Hong Yeol Rhu),1 Nicole R. Wilson,1 Sameet Mehta,2 Soo Seok Hwang,3

and Mark Hochstrasser1 1Department of Molecular Biophysics and Biochemistry, 2Yale Center for Genome Analysis, 3Department of Immunobiology,

Yale University, New Haven, Connecticut 06520, USA

Post-translational protein modification by the small ubiquitin-related modifier (SUMO) regulates numerous cellular

pathways, including transcription, cell division, and genome maintenance. The SUMO protease Ulp2 modulates many of

these SUMO-dependent processes in budding yeast. From whole-genome RNA sequencing (RNA-seq), we unexpectedly

discovered that cells lacking Ulp2 display a twofold increase in transcript levels across two particular chromosomes:

chromosome I (ChrI) and ChrXII. This is due to the two chromosomes being present at twice their normal copy number. An

abnormal number of chromosomes, termed aneuploidy, is usually deleterious. However, development of specific

aneuploidies allows rapid adaptation to cellular stresses, and aneuploidy characterizes most human tumors. Extra copies of

ChrI and ChrXII appear quickly following loss of active Ulp2 and can be eliminated following reintroduction of ULP2,

suggesting that aneuploidy is a reversible adaptive mechanism to counteract loss of the SUMO protease. Importantly,

increased dosage of two genes on ChrI—CLN3 and CCR4, encoding a G1-phase cyclin and a subunit of the Ccr4–Not

deadenylase complex, respectively—suppresses ulp2Δ aneuploidy, suggesting that increased levels of these genes underlie

the aneuploidy induced by Ulp2 loss. Our results reveal a complex aneuploidy mechanism that adapts cells to loss of the

SUMO protease Ulp2.

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POSTER SESSION –ABSTRACTS

Genomic resistance patterns to second-generation androgen blockade in paired tumor biopsies of metastatic

castrate-resistant prostate cancer

G. Celine Han1,2,4#, Justin Hwang1,2#, Stephanie A. Mullane1,2,4, Zhenwei Zhang1, David Liu1,2, Carrie Cibulskis2, Glenn C.

Gaviola3, Varand Ghazikhanian3, Rana R. McKay5, Glenn J. Bubley6, Scott L. Carter1,2, Steven P. Balk6, William C.

Hahn1,2,3, Mary-Ellen Taplin1,3*, Eliezer M. Van Allen1,2,3,4*

1Dana-Farber Cancer Institute, Boston, MA, 2Broad Institute of Harvard and MIT, Cambridge, MA. 3Brigham and Women’s

Hospital, Boston, MA, 4Center for Cancer Precision Medicine, Dana-Farber Cancer Institute, Boston, MA, 5University of

California San Diego, La Jolla, CA, 6Beth Israel Deaconess Medical Center, Boston, MA; #Co-first author, *Co-senior

author

Background Recent “next generation” androgen deprivation therapies (ADT), such as abiraterone and enzalutamide, have

improved survival in patients with castrate-resistant prostate cancer (CRPC). Despite therapy, most patients develop

resistance to these agents. We investigated the genetic basis of tumor evolution and clinical resistance to next generation

ADT in CRPC by using whole exome sequencing (WES) on paired pretreatment and post-resistance biopsies from CRPC

patients.

Methods Matched “trios” of germline, pre-treatment and post-resistant tumor samples were obtained from 7 patients treated

with abiraterone (n=4) and enzalutamide (n=3) and WES was performed. Clinical data, including PSA and radiographic

measurements, was used to classify patients as intrinsically resistant or initially responsive to treatment. Quality control,

mutation and indel calling, copy number variation identification were performed using analytical pipelines at the Broad

Institute. Tumor purity and ploidy were inferred, and phylogenetic analysis was performed using ABSOLUTE and Phylogic,

respectively to identify resistance associated alterations in the context of clinical phenotypes.

Results We identified multiple putative mechanisms and genetic categories of resistance to next generation ADT in CRPC.

Abiraterone resistant tumors harbored alterations in AR and MYC, while enzalutamide resistant tumors had cell cycle

pathway alterations. Experimentally, overexpressing cell cycle kinases promoted enzalutamide resistance, which was

mitigated through CDK4/6 blockade.

Conclusion This study outlines an approach to identify clinical genetic resistance mechanisms of next generation ADT in

CRPC through integration of genomic data from serial biopsies, clinical patient outcomes, and preclinical functional

screening. These findings confirm previously known potential resistance mechanisms, such as AR and MYC activation, and

inform therapeutic sequence and combination strategies in genomically selected advanced CRPC.

Establishing a CRISPR-based platform to study schizophrenia-associated genes in human neurons

Seok-Man Ho1,3,5, Brigham J. Hartley1,5, Natalie Barretto1,2,5 and Kristen J. Brennand1,2,4,5#

Departments of 1Psychiatry, 2Neuroscience, 3Developmental and Stem Cell Biology, 4Genetics and Genomics, 5Friedman

Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY 10029

Schizophrenia (SZ) is a complex genetic neuropsychiatric disease inherited via both common and rare polygenic risk factors.

SZ genome wide association studies (GWAS) have identified many SZ-associated single nucleotide polymorphisms (SNPs)

positioned in the putative enhancer regions of neuronal genes, suggesting a link between these SNPs, their respective

neighboring gene(s), and SZ risk. Recently, the CommonMind Consortium (CMC) examined gene expression in post-

mortem brains, identifying five genes with the strongest correlation between genotype and brain expression levels: FURIN,

SNAP91, CLCN3, TSNARE1 and CNTN4 (herein referred to as the "CMC genes"); however, the functional role of these

five genes in post-mitotic human neurons remains unresolved. We recently adapted a scalable CRISPR activation and

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interference (CRISPRa and CRISPRi, respectively) platform to NGN2-induced excitatory neurons, enabling robust and

combinatorial manipulation of CMC gene expression in human excitatory neurons. Specifically, we generated CRISPRa

(dCas9-VPR) and CRISPRi (dCas9-KRAB) stable NPC lines to achieve consistent expression of dCas9-effectors, and then

validated lentiviral-expressed gRNAs for each gene in NGN2-neurons generated from 3 control individuals. RNAseq-based

comparison of excitatory neurons with altered expression of CMC genes is underway, facilitating a greater understanding

of how perturbed expression of these CMC genes may contribute to SZ risk. In parallel, multi-electrode array and calcium

imaging experiments are evaluating the functional effect of manipulating these CMC genes in excitatory neurons. Our goal

is to better understand the link between these CMC genes, neuronal activity and SZ risk.

Anti-PD-L1 VHHs as a monitoring and a cytokine-delivery reagent for dense pancreatic tumors

Hee-Jin Jeong

Department of Cancer Immunology and Virology, Dana Farber Cancer Institute, 450 Brookline Avenue, Boston, MA 02215,

USA

Treatment for pancreatic cancer is limited by the dense stroma surrounding tumors and an immunosuppressive tumor

microenvironment. To generate therapies more able to overcome these barriers, we developed single domain antibodies

against PD-L1, and fused these to IL-2 and IFNg. Targeted delivery of each cytokine reduced tumor burden by 50%, while

isotype control conjugated cytokines or blockade of PD-L1 alone showed little effect. Targeted delivery of IL-2 increased

intratumoral CD8 T cells, while IFNg reduced MDSCs and reprogrammed intratumoral macrophages. We exploit the near-

ubiquitous expression of PD-L1 by tumors to focus immune therapies on the tumor microenvironment, and propose that

this may be a general technique for delivering combination immunotherapy to the tumor microenvironment, while greatly

reducing the risk of systemic toxicities.

Structural Basis of Transcription Arrest by Coliphage HK022 Nun in an Escherichia coli RNA Polymerase

Elongation Complex

Jin Young Kang, Paul Dominic B Olinares, James Chen, Elizabeth A Campbell, Arkady Mustaev, Brian T Chait, Maxwell

E Gottesman, and Seth A Darst

The Rockefeller University, 1230 York Avenue, New York, NY10065, United States

Coliphage HK022 Nun blocks superinfection by coliphage λ by stalling RNA polymerase (RNAP) translocation specifically

on λ DNA. To understand how Nun blocks RNAP translocation in molecular view, we determined structures of Escherichia

coli RNAP ternary elongation complexes (TECs) with and without Nun by single particle cryo-electron microscopy. Nun

tightly binds to the TEC by taking advantage of gaps between the RNAP and the nucleic acids. The C-terminal segment of

Nun interacts with the RNAP β and β’ subunits inside the RNAP active site cleft as well as with nearly every element of the

nucleic-acid scaffold, essentially crosslinking the RNAP and the nucleic acids to prevent translocation, a mechanism

supported by the effects of Nun amino acid substitutions. The nature of Nun interactions inside the RNAP active site cleft

suggests that RNAP clamp opening is required for Nun to establish its interactions, explaining why Nun stalls transcription

on pause sites.

Pathway dependence and drug synergism in NF1-associated malignant peripheral nerve sheath tumors using the

zebrafish model

Dong Hyuk Ki, A.Thomas Look

Dana-Farber Cancer Institute and Boston Children’s Hospital

Background: Malignant peripheral nerve sheath tumors (MPNSTs) are very aggressive and often metastatic soft tissue

sarcomas, which are frequently found in patients who have neurofibromatosis type 1 (NF1). Currently, surgical excision is

the only curative therapy for MPNST, although many patients have unresectable or metastatic tumors at diagnosis and the

- 20 -

recurrence rate after surgery is high. Chemotherapy regimens are only partially effective and associated with significant

toxicity that can severely reduce quality of life.

Objective: To develop a faithful in vivo MPNST transplantation assay to determine drug efficacy and host toxicity to identify

promising drugs and drug combinations. Our long range goal is to define active combinations of three or more non-cross

resistant drugs that exhibit synergistic and sustained tumor cell cytotoxicity at dosages tolerable to the developing recipient

zebrafish.

Methods: Primary MPNSTs were harvested from sox10:mCherry;nf1a+/-;nf1b-/-;p53m/m zebrafish and the cells were

mechanically dispersed. Approximately 100-120 MPNST cells were implanted into embryos at 2 day post-fertilization (dpf),

either into the posterior aspect of the yolk cell or into the pericardium. Twenty-four hours after implantation, the

fluorescent tumor cross-sectional area was imaged and MEK, Hsp90, mTOR, topoisomerase inhibitors at various

concentrations, or DMSO vehicle were added to the fish water. The transplanted embryos were incubated for 4 days in

the vehicle or each individual drug. Quantitative assessment of the cross-sectional area of remaining fluorescent tumor

cellswas performed at 7 dpf and fish were raised in the absence of drug and imaged every 7days to assess the durability of

the response.

Results: The pericardial transplantation assay proved superior, because the tumor cells formed an easily quantifiable mass

in a region that lacks non-specific fluorescence and the tumor cells grew vigorously in vehicle treated fish over the 4-day

incubation period. Drugs in clinical use for MPNST such as PD-0325901, ganetespib, and AZD2014 showed drug responses

at their maximum tolerated doses in the pericardial implantation assay, and topotecan elicited the best cytotoxic response

against transplanted MPNST cells.

Conclusion: We developed a robust in vivo pericardial transplantation model to test drug efficacy using our zebrafish model

of MPNST. We identified topotecan as a promising anti-tumor drug for NF1-associated MPNST, and we are now assessing

FDA approved drugs to identify those that synergize with topotecan to induce sustained MPNST cell death at tolerable

dosages for the host.

Funding Source: Department of Defense Neurofibromatosis Research Program Investigator-Initiated Research Award, NF1

Research Consortium, CTF Drug Discovery Initiative (DDI) Award. The Latsis family fellowship of the Boston Children’s

Hospital Neurofibromatosis program

Bimodality-based top-down clustering of single-cell RNA sequencing data reveals hierarchical structure of the cell

type

Junil Kim1,2, Julia Wang1,2, Diana Stanescu1,4, Maria Golson1,2, Doris Stoffers1,3, Klaus Kaestner1,2, and Kyoung Jae Won1,2 1Institute for Diabetes, Obesity and Metabolism, Perelman School of Medicine, 2Department of Genetics, 3Department of

Medicine, University of Pennsylvania, 4Division of Endocrinology and Diabetes, The Children’s Hospital of Philadelphia

Single-cell RNA sequencing (scRNA-seq) is useful for identifying multiple cell types in heterogeneous cell composition.

Several clustering algorithms successfully visualize predefined cell types as representative marker genes from scRNA-seq

data, but it is still hard to classify cells without marker gene information. To address this problem, we developed a new

clustering algorithm adopting bimodality and top-down hierarchical approach. Our algorithm first identifies gene groups

which share consistent or contrasting bimodal membership, and then identifies cell clusters according to the membership

pattern of the gene group. We applied our algorithm to single-cell gene expression data of the 1,083 human pancreatic cells

and hierarchically clustered the pancreatic cells into seven cell types (alpha, beta, pp, delta, duct, acinar, and mesenchyme)

without marker gene information. We show that our clustering algorithm is more accurate than both bottom-up hierarchical

clustering and t-distributed stochastic neighbor embedding followed by density-based spatial clustering of applications with

noise.

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Flip-flop transcriptional regulation by FoxP3

Ho-Keun Kwon, Hui-Min Chen, Diane Mathis* and Christophe Benoist*

Division of Immunology, Department of Microbiology and Immunobiology, Harvard Medical School, and Evergrande

Center for Immunologic Diseases, Harvard Medical School and Brigham and Women’s Hospital, Boston MA 02115, USA

FoxP3, the key lineage-determining factor of Treg cells, activates or represses a range of transcriptional targets to determine

the many facets of Treg function, in concert with a number of transcriptional cofactors. To understand how FoxP3 juggles

these activities, we constructed a set of 130 mutants spanning the protein, evaluating impacts on DNA binding, cofactor

interactions, chromatin binding and transcriptional activation, and assessing relevance by CRISPR-based genome editing

in mice. Computational integration of binding and transcription results, supported by biochemical dissection of multi-

molecular complexes, showed that mutations affected these functions in a variegated and non-modular manner, with

different mutation footprints for classic targets of the Treg signature vs those typical of tissue Tregs. We demonstrated that

FoxP3 partakes in two dominant complexes of opposite function, activating when partnered with RelA, Ikzf2 and Kat5,

repressing when paired with Ezh2 and Ikzf3. Yet different interactions underpin transcriptional programs of tissue-Tregs.

Mutations with partial effects yielded mice with normal numbers of Tregs in the steady-state context, but of reduced fitness

under stress and autoimmune challenge, suggesting that similar mild FoxP3 variants may have unappreciated consequence

in human pathology.

Novel role of miR-29 in pancreatic cancer autophagy and its therapeutic potential

Jason J Kwon1, Jeffrey A Willy2, Ronald C Wek1,2, Murray Korc2,3,4,5, Xiao-Ming Yin6, and Janaiah Kota1,4,5

1Department of Medical and Molecular Genetics, Indiana University School of Medicine (IUSM), Indianapolis, IN, USA, 2Department of Biochemistry and Molecular Biology, IUSM, Indianapolis, IN, USA, 3Department of Medicine, IUSM,

Indianapolis, IN, USA, 4The Melvin and Bren Simon Cancer Center, IUSM, Indianapolis, IN, USA., 5Center for Pancreatic

Cancer Research, Indiana University and Purdue University-Indianapolis (IUPUI), Indianapolis, IN, USA, 6Department of

Pathology and Laboratory Medicine, IUSM, Indianapolis, IN, USA

Pancreatic Ductal Adenocarcinoma (PDAC) is one of the most lethal human malignancies with a five-year survival rate of

8% and is often undiagnosed until it has metastasized. These advanced tumors display resistance to current therapeutic

modalities. The lack of effective therapies and early detection reinforces the need to understand molecular mechanisms

associated with PDAC to develop novel therapies. We found consistent downregulation of miR-29 in pancreatic cancer cells,

and its restored expression sensitized chemotherapeutic resistant pancreatic cancer cell lines to gemcitabine, leading to

reduced cancer cell viability and increased cytotoxicity. Furthermore, reintroduction of miR-29 blocked autophagy flux,

evidenced by an accumulation of autophagosomes and autophagy substrate, p62, and decreased autophagosome-lysosome

fusion. In addition, miR-29 decreased TFEB and ATG9A expression, which are critical for lysosomal function and

autophagosome trafficking respectively. Subsequent knockdown of TFEB or ATG9A expression alone or in combination

resulted in inhibition of autophagy similar to miR-29 overexpression. Finally, miR-29 reduced migration, invasion, and

anchorage independent growth of pancreatic cancer cells. Collectively, our findings indicate that miR-29 functions as a

potent autophagy inhibitor that sensitizes pancreatic cancer cells to gemcitabine and decreases their invasive potential. Our

data provides evidence for the use of miR-29 as a novel therapeutic agent to target PDAC.

Electrophysiological and transcriptome profiling of hyperexcitable motor neurons derived from ALS patient iPSC

identifies downregulation of a potassium channel subtype

Seungkyu Lee1,2, Ole Wiskow3, Sulagna Ghosh3, Kasper Roet1,3, Xuan Hwang1,3, Bruce P. Bean2, Brian J. Wainger4, Kevin

Eggan3, and Clifford J. Woolf1,2

1 F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, MA 02115, USA, 2 Department of Neurobiology,

Harvard Medical School, Boston, MA 02115, USA, 3Department of Stem Cell and Regenerative Biology, Harvard University,

Cambridge, MA 02138, USA, 4 Department of Neurology, Massachusetts General Hospital, Charlestown, MA 02129, USA

- 22 -

Amyotrophic lateral sclerosis (ALS) is associated with motor neuron hyperexcitability. The hyperexcitability phenotype

was recapitulated in spinal motor neurons derived from ALS patient iPSC lines that harbor SOD1 and FUS mutations and

C9orf72 repeat expansions. The inhibition of this hyperexcitability using the potassium channel (Kv7) opener retigabine

reduces motor neuron cell death, supporting a pathogenic role (Wainger et al., Cell Reports, 2014). However, the

mechanisms involved in the hyperexcitability remain elusive. To identify the mechanism we have recently developed patch-

seq and patch-RT-qPCR techniques for linking neuronal excitability and gene expression at the single cell level, collecting

RNA after whole cell patch clamp recordings from motor neurons and performing single cell next generation sequencing

and high-throughput multiplex qPCR, respectively. Single cell gene expression profiling in functionally characterized

neurons was performed on HB9::GFP sorted iPSC-derived motor neurons from an ALS patient carrying the SOD1 A4V

mutation (39b) and isogenic control motor neurons with the mutation corrected (39b-cor). We found by whole cell current

clamp recording that 39b motor neurons have larger cell sizes and robust hyperexcitability compared to 39b-cor motor

neurons. Both single cell RNA-seq and ion channel targeted multiplexed high throughput qPCR analysis independently

show a significant down-regulation of a K channel in hyperexcitable ALS motor neurons from this patient in two

independent batches of motor neurons from the same line. These results suggest the reduced expression of a K channel as a

molecular mechanism for the increased excitability in familial ALS.

Targeting lytic and quiescent herpes simplex virus 1 genomes with CRISPR/Cas9

Hyung Suk Oh1, Magdalena Angelova1, Werner Neuhausser3, Kevin Eggan2, 3 and David M. Knipe1

1Department of Microbiology and Immunobiology, Harvard Medical School, Boston, Massachusetts, United States of

America, 2Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Harvard University, Cambridge,

Massachusetts, United States of America, 3Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard,

Cambridge, Massachusetts, United States of America

Herpes simplex virus (HSV) 1 can undergo lytic replication or establish latency following primary infection. Although there

are several anti-HSV drugs that target lytic infection, no treatment is available to target latent HSV genomes and prevent

reactivation. Here we evaluated the use of the CRISPR (Clustered regularly-interspaced short palindromic repeats)/Cas9

system to target HSV lytic and latent genomes. To study lytic infection, we established human foreskin fibroblast (HFF)

cell lines stably expressing Cas9 and guide RNAs (gRNAs) targeting essential HSV-1 genes, and infected the cells with

HSV-1. Cas9/gRNA expression reduced viral yield by 2-4 logs compared to a control cell line expressing Cas9 alone. We

showed that Cas9/gRNA induced mutations in HSV genomes earlier than 6 hpi, and the mutations accumulated over the

viral replication. Interestingly, replicating HSV genomes are more prone to editing than non-replicating HSV genomes. To

test the ability of CRISPR/Cas9 system to target latent genomes, we established a quiescent infection in HFFs with

replication-defective HSV-1 d109. We transduced quiescently infected cells with lentiviruses encoding Cas9/gRNA

targeting essential viral genes, followed by reactivation with wildtype HSV-1. We observed that reactivation of d109 from

quiescent infection was reduced by 2-5 logs by single gRNAs or combinations of gRNAs compared to the Cas9 control

without gRNA. Additionally, we showed by deep sequencing that quiescent HSV-1 genomes could be efficiently targeted

and edited by Cas9/gRNA. This study demonstrates that the CRISPR/Cas9 system can efficiently target lytic and latent

HSV genomes.

Enhanced Histone Acetylation Up-Regulates MDR1 and BCRP Transporters in Human Blood-Brain Barrier Cells

Dahea You1, Xia Wen2,3, Ayeshia Morris1, Jason R. Richardson4, Lauren M. Aleksunes2,3. 1Joint Graduate Program in Toxicology, Rutgers University, Piscataway, NJ, 2Environmental and Occupational Health

Sciences Institute, Rutgers University, Piscataway, NJ, 3Department of Pharmacology and Toxicology, Rutgers University,

Piscataway, NJ, 4Northeast Ohio Medical University, Rootstown, OH

Multidrug Resistance Protein 1 (MDR1, ABCB1) and the Breast Cancer Resistance Protein (BCRP, ABCG2) expressed at

the blood-brain barrier (BBB) are key efflux transporters that extrude chemicals from the BBB and regulate the efficacy

and/or toxicity of chemicals in the brain. Prior studies in cancer cells have pointed to the ability of histone deacetylase

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(HDAC) inhibitors to modulate the expression and function of MDR1 and BCRP. However, whether or not such regulation

occurs at the BBB is not known. Here we sought to test whether HDAC inhibitors could potentially alter expression and

function of MDR1 and BCRP at the BBB. To test this, we treated immortalized human brain capillary endothelial

(hCMEC/D3) cells, a model of the BBB, with six different HDAC inhibitors, valproic acid (VPA), sodium butyrate (NaB),

romidepsin, apicidin, suberoylanilide hydroxamic acid (SAHA), and trichostatin A (TSA), and assessed for expression and

function of MDR1 and BCRP. HDAC inhibition following treatment was confirmed by increased levels of acetylated histone

H3 protein. After 12 h of treatment, VPA, apicidin, SAHA, and TSA up-regulated MDR1 mRNA levels between 50% and

200%. All six HDAC inhibitors significantly induced BCRP mRNA levels between 100% and 270%. Similarly, the protein

expression of MDR1 and BCRP transporters was up-regulated about two-fold at 24 h. Interestingly, these effects have been

observed at clinically-relevant concentrations of HDAC inhibitors. Enhanced MDR1 expression corresponded with reduced

intracellular accumulation of the substrate rhodamine 123. Collectively, these results demonstrate that HDAC inhibitors up-

regulate MDR1 and BCRP transporters at the BBB by modifying histone acetylation. The clinical use of HDAC inhibitors

may enhance efflux transporter activity at the BBB and restrict access of xenobiotics to the brain.

Mechanisms of transcription factor-mediated direct reprogramming of mouse embryonic stem cells to trophoblast

stem-like cells

Catherine Rhee1,2, Samuel Beck1,2, Bum-Kyu Lee1,2, Lucy LeBlanc1,2, Haley O Tucker1,2, and Jonghwan Kim1,2,3,* 1Department of Molecular Biosciences, 2Institute for Cellular and Molecular Biology, 3Center for Systems and Synthetic

Biology, The University of Texas at Austin, Austin, TX, 78712

Direct reprogramming can be achieved by forced expression of master transcription factors. Yet how such factors mediate

repression of initial cell-type-specific genes while activating target cell-type-specific genes is unclear. Through embryonic

stem (ES) to trophoblast stem (TS)-like cell reprogramming by introducing individual TS cell-specific “CAG” factors (Cdx2,

Arid3a, Gata3), we interrogate their chromosomal target occupancies, modulation of global transcription, and chromatin

accessibility at the initial stage of reprogramming. From the studies, we uncover a sequential, two-step mechanism of

cellular reprogramming in which repression of pre-existing ES cell-associated gene expression program is followed by

activation of TS cell-specific genes by CAG factors. Therefore, we reveal that CAG factors function as both decommission

and pioneer factors during ES to TS-like cell fate conversion.

DNA aptamers that recognize the altered tertiary structure of mutant huntingtin modulate its activity

Baehyun Shin1, 2, Hyejin Oh1, 2, Gwen E. Owens3, Roy Jung1, 2, Hyeongseok Lee4, Seung Kwak5 , Ramee Lee5, Daniel J.

Lavery5, Susan L. Cotman1, 2, Jong-Min Lee1, 2, Marcy E. MacDonald1, 2, Ji-Joon Song4, Ravi Vijayvargia1, 2 and Ihn Sik

Seong1, 2 1Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114, USA, Department of Neurology,

Harvard Medical School, Boston, MA 02114, USA, 3Division of Biology and Biological Engineering, California Institute of

Technology, Pasadena, CA, USA, 4Department of Biological Sciences, KAIST Institute for the BioCentury, Korea Advanced

Institute of Science and Technology (KAIST), Daejeon 305-701, Republic of Korea, 5CHDI Foundation, Princeton NJ 08540,

USA.

Purpose: Huntington’s disease (HD) is a dominant CAG trinucleotide repeat expansion disorder, caused by an elongated

polyglutamine tract in huntingtin. We found its polyglutamine tract size influences both huntingtin’s structure and function,

including modulating its activity in stimulating polycomb repressive complex 2 (PRC2). These observations strongly imply

that small molecules that preferentially bind to mutant rather than normal huntingtin can be found and that, in doing so they

may selectively influence the impact of the longer polyglutamine tract on mutant huntingtin activity.

Materials & Methods: To provide an initial proof for the concept of small molecule-binding as a route to directly alter the

impact of the expanded polyglutamine tract on mutant huntingtin, we have screened a library of single stranded DNA

- 24 -

aptamers for mutant huntingtin specific binders, using highly purified human recombinant huntingtin with polyglutamine

tract lengths of either 23- or 78-residues. This biochemical strategy has yielded a specific set of aptamers that exhibit

differential binding affinities to mutant and normal huntingtin. In this study, we have evaluated the aptamers’ binding sites

and potential ability to modulate the effects of polyglutamine tract length on huntingtin structure and PRC2-stimulating

activity in HD human neural progenitor cells (hNPCs).

Results: We identified forty-five DNA aptamers that preferentially bind to Q78-huntingtin compared to Q23-huntingtin.

Four representative aptamers (MS1,2,3,4) comprise guanine rich sequences forming G-quadruplex structures and bind

predominantly to the CTD-II carboxyl-terminal sub-domain of mutant huntingtin. All four aptamers, particularly MS3 at

low concentration, were found to decrease the enhanced PRC2 stimulating activity conferred by the expanded polyglutamine

tract (Panel A). After incubating HD hNPCs (HD60) and normal hNPCs (HD17) lysates with biotinylated MS3 followed

by pull-down with streptavidin-conjugated beads, we observed a 2.5 fold more efficient pull-down of endogenous huntingtin

in HD60 compared to HD17 (Panel B). Moreover, MS3 transfection into HD60 showed the co-localization with cellular

mutant huntingtin (Panel C) and resulted in a significant decrease in the abnormally elevated PRC2 activity in mutant cells

without affecting the level in HD17 (Panel D).

Conclusion: Our DNA aptamers are a novel biochemical probe for detecting the subtle structural changes specific to mutant

huntingtin and provide a first proof-of-principle to directly modulate mutant huntingtin activity.

Supported by R01ES021800, P30ES005022.

- 25 -

Developing Computational Algorithms for Identifying Important Gene Sets for Thyroid Tumour from Genomic

Data

Yejin Esther Yun, Edwin Wang, Ralf Paschke, Markus Eszlinger

University of Calgary Cumming School of Medicine, Department of Biochemistry and Molecular Biology, Alberta

Children's Hospital Research Institute, Arnie Charbonneau Cancer Research Institute, Calgary, Alberta, Canada

Thyroid nodules are very commonly observed in adults but only a small percentage of the nodules is associated with

malignant tumour. With the advanced diagnostic technology and the enhanced surveillance, an accuracy of diagnostic

testing is of importance for thyroid cancer treatment. Although the fine needle aspiration biopsy is a standard diagnostic

tool to differentiate the malignant and benign tumour, it is invasive to perform such biopsy and still 20% of the results are

indeterminate. Therefore, patient genetic information guided diagnosis has become a necessity to improve patient outcome.

Several studies have been done to unravel driver mutations and to use them for diagnostic testing. However, there were

some cases that are not explained by known driver gene mutations. In order to overcome the challenge, a computational

algorithm will be developed to discover the relationship between the genetic variants and thyroid cancer by identifying

important gene sets that contribute in the background of the known driver mutations.

Funding agency: Alberta Innovates: Health Solutions

- 26 -

PAST FELLOWSHIP AWARDEES

KASBP-DAEWOONG FELLOWSHIP

2006 민재기 New York University, 김 한 Princeton University, 박혜진 Rutgers University

2007 문지숙 Harvard University, 박성연 Rutgers University, 이석근 Columbia University

2008 이흥규 Yale University, 김정환 Rutgers University, 강민식 Columbia University

2009 박진아 Harvard University, 최재민 Yale University, 김덕호 Johns Hopkins University

2010 기정민 Rockefeller University 김형욱 NIH, 안세진 Harvard University

2011 한무리 University of California, LA, 장환종 Boston College

2012 장정호 Columbia University, 최재우 Oregon State University

2013 JangEun Lee (University of Pennsylvania), Eun Chan Park (Rutgers University)

2014 Kimberly H. Kim (Harvard University), Seung Koo Lee (Weill Cornell Medical College), Min-Sik Kim (Johns

Hopkins University)

2015 Jiyeon Kim (UT Southwestern), Sun Mi Park (Memorial Sloan-Kettering Center), Byeong Seon Kim (University

of Pennsylvania)

2016 Sang Bae Lee (Columbia University), Junil Kim (University of Pennsylvania), Ho-Keun Kwon (Harvard Medical

School)

KASBP-GREEN CROSS FELLOWSHIP

2011 조한상 Harvard Medical School, 강성웅 Johns Hopkins University,

김미연 Columbia University, 소재영 Rutgers University, 황성용 NIEHS/NIH

2012 조원진 Drexel University, 강효정 Yale University, 이정현 Columbia University, 이용재 Yale University,

윤재현 NIH

2013 Yunjong Lee (Johns Hopkins University), Jun-Dae Kim (Yale University)

Bae-Hoon Kim (Yale University) Ja Young Kim-Muller (Columbia University)

2014 Catherine Rhee (University of Texas at Austin), Ji-Seon Seo (The Rockefeller University) Sehyun Kim (New York

University)

2015 Young-Su Yi (New York University), Hee-Woong Lim (University of Pennsylvania), Bloria Bora Kim (The

Pennsylvania State University)

2016 Eui Tae Kim (University of Pennsylvania), Kihyun Lee (Weill Cornell Medical Science)

KASBP-HANMI FELLOWSHIP

2011 안형진 Rockefeller University, 조창훈 Abramson Research Center

2012 김유나 University of North Carolina, 태현섭 Yale University, 이인혜 NIH

2013 이주희 Memorial Sloan-Kettering Cancer Center, 이경륜 Rutgers University 이만률 Indiana University

2014 Young Chan Cha (Wistar Institute), Min-Kyu Cho (New York University) Lark Kyun Kim (Yale University), Yu

Shin Kim (Johns Hopkins University)

2015 Seonil Kim (New York University), Peter B. Kim (Yale University)

2016 Sungwhan Oh (Harvard Medical School), Won-Gil Lee (Yale University), Hee-Jin Jeong (Harvard Medical School)

- 27 -

KASBP-YUHAN FELLOWSHIP

2011 김기영 Boston University, 심중섭 Johns Hopkins University

2012 허예민 University of Michigan, 방숙희 University of Pennsylvania, 백정호 Columbia University

2013 Dong Jun Lee (University of Chicago), Ingyu Kim (Yale University), Ja Yil Lee (Columbia University)

2014 Seouk Joon Kwon (Rensselaer Polytech Institute), Jeongmin Song (Yale University), Jae-Hyun Yang (Harvard

Medical School), Wan Seok Yang (Columbia University)

2015 Min-Joon Han (Harvard Medical School), Minjung Kang (Cornell University)

2016 Ki Su Kim (Harvard Medical School), Hongjae Sunwoo (Harvard Medical School), Seo-Young Park (University of

Massachusetts)

KASBP-ST PHARM FELLOWSHIP

2016 Jung-Eun Jang (New York University), Byungsu Kwon (MIT)

KASBP FELLOWSHIP

2009 최상호 NIH

2010 김상령 Columbia University, 윤태숙 Rutgers University, 허은미 Cal. Tech.

2015 (Spring) Mi Jung Kim (Duke University)

2015 (Fall) Minyoung Park (The Rockefeller University)

KASBP-KSEA FELLOWSHIP

2013 Sung In Lim (University of Virginia)

2014 Keun-woo Jin (Temple University)

KASBP-KUSCO FELLOWSHIP

2008 김현호 National Institutes of Health, 온택범 Harvard Medical School, 주원아 Wistar Institute

KASBP-KRICT FELLOWSHIP

2009 신승식 Rutgers University, 정은주 Columbia University, 백규원 University of Pennsylvania

KASBP-KHIDI FELLOWSHIP

2010 배재현 Yale University, 조희연 Boston College

Preliminary Program

For more information, please visit www.kasbp.org

2017 SPRING SYMPOSIUM ATTENDEES

Last Name

First Name Korean Affiliate Area Group Discussion

1. Ahn Kyonghoon 안경훈 Daewoong pharmaceutical company KR Respiratory/metabolic/cardiovascular/Neurogenerative

2. Aoyagi kazuko Celerion NJ Respiratory/metabolic/cardiovascular/Neurogenerative

3. Baek Kyuwon 백규원 AMO Lifescience KR Cell and Gene Therapy/Viral infection/Rare disease

4. Baek Jonathan 백인환 대원제약 주식회사 KR BD/Legal/VC:

5. Bang Hanseong 방한성 CMIC CMO USA NJ BD/Legal/VC:

6. Barker Thomas Foley Hoag LLP DC BD/Legal/VC:

7. Cha Bumjoon 차범준 University of Massachusetts Lowell MA Cell and Gene Therapy/Viral infection/Rare disease

8. Chang Hemmie 장혜미 Foley Hoag MA BD/Legal/VC:

9. Chang Jintae 장진태 CORESTEM KR Cell and Gene Therapy/Viral infection/Rare disease

10. Chang Dong-Eun 장동은 CJ Research Center America MA BD/Legal/VC:

11. Chang Kern 장건희 Janssen R&D PA Respiratory/metabolic/cardiovascular/Neurogenerative

12. Cho Jaemin 조재민 KCRN Research MD PK/PD/pre-clinical/Clinical Science:

13. Cho Min-Kyu 조민규 Novartis MA Chemistry

14. Choe Yun H. 최윤 Lucas and Mercanti, LLP NJ BD/Legal/VC:

15. Choi AHyun 최아현 Univ. of Massachusetts, Medical School MA Immuno-oncology/Autoimmune/Inflammatory

16. Choi Yong Jin 최용진 UC Berkeley CA Cell and Gene Therapy/Viral infection/Rare disease

17. Choi Sungwook 최성욱 U mass medical school MA BD/Legal/VC:

18. Choi Jun Young 최준영 Nitto Avecia MA

19. Choi Younggi 최영기 FORMA Therapeutics CT Chemistry

20. Choi Suktae 최석태 Celgene Inc NJ PK/PD/pre-clinical/Clinical Science:

21. Choo Min-Kyung 추민경 MGH MA

22. Chung Seungwon 정승원 AbbVie IL Chemistry

23. Chung Jaeyoon 정재윤 Boston University MA Respiratory/metabolic/cardiovascular/Neurogenerative

24. Chung WonWoo 정원우 MCPHS Universty MA Pharmacy

25. Chung Kyung Hyun 정경훈 MCPHS MA PK/PD/pre-clinical/Clinical Science:

26. Chung HaeWon 정해원 The University of Texas at Austin TX Cell and Gene Therapy/Viral infection/Rare disease

27. Doh Hyounmie 도현미 Dong-A ST

KOREA

Immuno-oncology/Autoimmune/Inflammatory

28. Eo Jinsu 어진수 Center for engineering in medicine MA

29. Eom Tae Ung 엄태웅 Samyang Biopharmaceuticals Corp. KR

30. HAHM Sean 함성원 The Yakup Shinmoon KR

31. Hahn William Dana-Farber Cancer Institute MA BD/Legal/VC:

32. Hailey Jeong 정혜연 MCPHS University MA BD/Legal/VC:

33. Han Heeoon 한희운 University of Pennsylvania PA

34. Han Celine Garam 한가람 Dana-Farber Cancer Institute MA Immuno-oncology/Autoimmune/Inflammatory

35. Han Sangyeul Cell Signaling Technology MA Immuno-oncology/Autoimmune/Inflammatory

36. Ho Seok-Man 호석만 Icahn School of Medicine at Mount Sinai NY Respiratory/metabolic/cardiovascular/Neurogenerative

37. Hong Peter 홍성원 Regeneron Pharmaceuticals NY PK/PD/pre-clinical/Clinical Science:

38. Huh Jeongho 허정호 Greencross Corp. KR

39. Hwang So-Young 황소영 Genosco MA

40. Hwang Ji Young 황지영 Geisel School of Medicine at Dartmouth NH Immuno-oncology/Autoimmune/Inflammatory

41. Hwang Soo Seok 황수석 Yale University School of Medicine CT Immuno-oncology/Autoimmune/Inflammatory

42. Hwang Seongwoo 황성우 PTC Therapeutics NJ Chemistry

43. Hyun Grace 현은지 MCPHS University MA Pharmacy

44. IM WEON BIN 임원빈 DONGA ST KR Chemistry

Last Name

First Name Korean Affiliate Area Group Discussion

45. Im Eunju 임은주 Nathan S. Kline Institute, NYU Medical center NY Respiratory/metabolic/cardiovascular/Neurogenerative

46. In Hwa Chung 정인화 Sungwun pharmacopia KR Immuno-oncology/Autoimmune/Inflammatory

47. JEONG HEE-JIN 정희진 Dana-Farber Cancer Institute MA

48. JEONG HEYKYEONG 정혜경 Temple University PA Respiratory/metabolic/cardiovascular/Neurogenerative

49. Jeong Jae Uk 정재욱 GSK PA BD/Legal/VC:

50. Jin Joon Yung 진준영 CJ CheilJedang MA BD/Legal/VC:

51. Jo Seunghee 조승희 Agios Pharmaceuticals MA Cell and Gene Therapy/Viral infection/Rare disease

52. Jo Hakryul Agios Pharmaceutical Inc. MA Cell and Gene Therapy/Viral infection/Rare disease

53. Joh Nathan Amgen CA

54. Jung Young Chun 정영춘 Zafgen Inc. MA Chemistry

55. Kang Unbeom 강운범 Dana-Farber Cancer Institute MA PK/PD/pre-clinical/Clinical Science:

56. Kang Young Bok 강영복 Mass General Hospital MA Immuno-oncology/Autoimmune/Inflammatory

57. Kang Pilsoo 강필수 Genzyme MA Cell and Gene Therapy/Viral infection/Rare disease

58. Kang Jungwook 강정욱 Rutgers Univ. School of Pharmacy NJ Pharmacy

59. Kang Jin Young 강진영 Rockefeller University NY Immuno-oncology/Autoimmune/Inflammatory

60. Ki Dong Hyuk 기동혁 Dana-Farber Cancer Ins./Harvard M. S. MA

61. Kim Ah Ram 김아람 Boston Children's Hospital MA Cell and Gene Therapy/Viral infection/Rare disease

62. Kim Alexander 김지혁 Boston College MA Pharmacy

63. Kim Hyungchul 김형철 Novartis MA

64. KIM Paul T. Foley Hoag LLP DC BD/Legal/VC:

65. Kim Younghoon 김영훈 Sanofi-Genzyme MA Cell and Gene Therapy/Viral infection/Rare disease

66. Kim Jungeun 김정은 MCPHS university MA Pharmacy

67. Kim Minji 김민지 Curis, Inc MA BD/Legal/VC:

68. Kim YeonJin 김연진 Harvard School of Dental Medicine MA Immuno-oncology/Autoimmune/Inflammatory

69. Kim Eunkyung 김은경 Genosco MA PK/PD/pre-clinical/Clinical Science:

70. Kim Judith Rubin and Rudman LLP MD BD/Legal/VC:

71. Kim Jong-Gyun 김종균 Yuhan Corporation KR Immuno-oncology/Autoimmune/Inflammatory

72. KIM BUMJUN 김범준 Northeastern University MA Pharmacy

73. Kim Jia 김지아 Stony Brook University NY Cell and Gene Therapy/Viral infection/Rare disease

74. Kim Junil 김준일 University of Pennsylvania PA Cell and Gene Therapy/Viral infection/Rare disease

75. Kim Sung ki 김성기 MCPHS university MA Pharmacy

76. Kim Yongmin 김용민 MCPHS Pharmacy MA Pharmacy

77. Kim Sahee 김사희 RevHealth NJ Pharmacy

78. Kim Sehoon 김세훈 Merck MA Immuno-oncology/Autoimmune/Inflammatory

79. Kim Mi-Sook 김미숙 Takeda MA

80. Kim Sung-Kwon 김성권 Alexion Pharmaceuticals CT Immuno-oncology/Autoimmune/Inflammatory

81. Kim Dae-Shik 김대식 Eisai Inc MA Chemistry

82. KIM KYOUNGTAE 김경태 Nieman Foundation of Journalism HARVARD MA Immuno-oncology/Autoimmune/Inflammatory

83. kim Youngjin 김영진 rockefeller university NY Pharmacy

84. Kim Joonyul 김준열 Proximity Biosciences LLC AL BD/Legal/VC:

85. Kim Han-Joo 김한주 Yuhan Corporation

OTHER

Respiratory/metabolic/cardiovascular/Neurogenerative

86. Kim Youngsun 김영선 Adello Biologics NJ BD/Legal/VC:

87. Kim Hai-Young 김혜영 Merck MA Immuno-oncology/Autoimmune/Inflammatory

88. Kim Leo Incyte Corporation DE Immuno-oncology/Autoimmune/Inflammatory

89. Kim Jisu 김지수 Massachusetts college of pharmacy MA Pharmacy

90. Kim Min-woo 김민우 Zafgen Inc. NJ Chemistry

91. Kim Young Woo 김영우 Access Bio NJ Immuno-oncology/Autoimmune/Inflammatory

Last Name

First Name Korean Affiliate Area Group Discussion

92. Kim Jae-Hun 김재훈 IFF NJ Chemistry

93. Kim Bae-Hoon 김배훈 HHMI at Yale School of Medicine CT Cell and Gene Therapy/Viral infection/Rare disease

94. KOH JONG SUNG 고종성 GENOSCO MA BD/Legal/VC:

95. Koo Jaseok Yale School of Medicine CT

96. Kwon Byungsu 권병수 MIT chemistry MA Chemistry

97. Kwon Jason 권재혁 Indiana University School of Medicine IN Cell and Gene Therapy/Viral infection/Rare disease

98. Kwon Sam 권상열 Vesta Pharmaceuticals, Inc. IN

99. Kwon Hokeun 권호근 Harvard Medical School MA Immuno-oncology/Autoimmune/Inflammatory

100. Kwon Eunjeong 권은정 MGH MA

101. Lee Young Eun 이영은 Monell Chemical Senses Center PA Chemistry

102. Lee Hyun-Hee 이현희 Merck MA Immuno-oncology/Autoimmune/Inflammatory

103. Lee Myung Yeol 이명렬 Amolifescience KR Cell and Gene Therapy/Viral infection/Rare disease

104. Lee Hak-Myung 이학명 Shire MA

105. Lee Dooyoung 이두영 Applied BioMath MA PK/PD/pre-clinical/Clinical Science:

106. Lee Jeongwook 이정욱 Wyss Institute, Harvard University MA Immuno-oncology/Autoimmune/Inflammatory

107. Lee Jongsoon 이종순 Joslin Diabetes Center/Harvard M Medical School MA Respiratory/metabolic/cardiovascular/Neurogenerative

108. Lee Sam 이상엽 CRScube America Inc. MD BD/Legal/VC:

109. Lee Kyungjin 이경진 STP America Research NJ Chemistry

110. Lee HeaYeon 이혜연 Northeastern University NY Respiratory/metabolic/cardiovascular/Neurogenerative

111. LEE JAEKYOO 이재규 GENOSCO MA BD/Legal/VC:

112. Lee Joonsoo 이준수 Shire MA

113. Lee Tae Gyu 이태규 Osong New Drug Development Center KR Immuno-oncology/Autoimmune/Inflammatory

114. Lee James Jungkue 이정규 Bridge Biotherapeutics, Inc KR BD/Legal/VC:

115. Lee Hwaseong 이화성 Samyang Biopharmaceuticals Corp. KR

116. Lee Seung-Mi 이승미 Rutgers University NJ Pharmacy

117. Lee Seungkyu 이승규 Boston children's hospital MA Respiratory/metabolic/cardiovascular/Neurogenerative

118. Lim Sungtaek 임성택 Sanofi Pharmaceuticals MA Chemistry

119. Lim Hyungwook 임형욱 Novartis MA Immuno-oncology/Autoimmune/Inflammatory

120. Lim Hanjo 임한조 Genentech CA PK/PD/pre-clinical/Clinical Science:

121. Ma Sunghoon 마성훈 Exelixis CA Chemistry

122. Moon Ji Yoon 문지윤 MCPHS Worcester MA Pharmacy

123. Moon Young-Choon 문영춘 PTC Therapeutics NJ Chemistry

124. Nam Gyeongsug 남경숙 Daewoong pharmaceutical company KR Immuno-oncology/Autoimmune/Inflammatory

125. Nam Spencer 남성한 SV Investment Corporation MA

126. Oh Chris Chigon 오치곤 ENVIGO KR PK/PD/pre-clinical/Clinical Science:

127. Oh Hyungsuk 오형석 Harvard Medical School MA Cell and Gene Therapy/Viral infection/Rare disease

128. Paik Ik-Hyeon 백익현 WAVE Life Sciences, Inc. MA Cell and Gene Therapy/Viral infection/Rare disease

129. Park Soo-Hee 박수희 Novartis MA Respiratory/metabolic/cardiovascular/Neurogenerative

130. Park Sangho 박상호 Merck & Co MA Immuno-oncology/Autoimmune/Inflammatory

131. Park Angie Inkyung 박인경 OncoMed Pharmaceuticals CA Immuno-oncology/Autoimmune/Inflammatory

132. Park Daniel 박종호 Rutgers University NJ Pharmacy

133. PARK JAHA 박자하 Boston University MA BD/Legal/VC:

134. Park JiYoung 박지영 Rutgers University NJ Pharmacy

135. Park Jinkyu 박진규 Internal Medicine, Yale University CT

136. Park Keun Woo 박근우 burke-cornell medical research institute NY Respiratory/metabolic/cardiovascular/Neurogenerative

137. Park Jeehae 박지혜 Harvard Medical School MA Cell and Gene Therapy/Viral infection/Rare disease

138. PARK SUNGHO 박성호 SVinvestment KR

Last Name

First Name Korean Affiliate Area Group Discussion

139. Park YoungSeoub 박영섭 Green Cross KR Immuno-oncology/Autoimmune/Inflammatory

140. Park Jihoon 박지훈 National Institutes of Health MD Immuno-oncology/Autoimmune/Inflammatory

141. Park Min 강민 STP America Research NJ

142. Park Jeonghan 박정한 STP America Research NJ Chemistry

143. Park Hee Dong 박희동 LG Chem, Life Science BU KR Respiratory/metabolic/cardiovascular/Neurogenerative

144. Park Dong Ho 박동호 Massachusetts Eye and Ear Infirmary MA Immuno-oncology/Autoimmune/Inflammatory

145. Park Seung-Yeol 박승열 BWH MA Immuno-oncology/Autoimmune/Inflammatory

146. Park Jiyoung 박지영 University of Pennsylvania PA Cell and Gene Therapy/Viral infection/Rare disease

147. Park Jonghoon 박종훈 Life Sciences Company, LG Chem KR Immuno-oncology/Autoimmune/Inflammatory

148. Park Jaehong 박재홍 Takeda Oncology MA Immuno-oncology/Autoimmune/Inflammatory

149. Rhu Hong Yeol 류홍열 Yale University CT

150. Shim Jaehoon 심재훈 Boston children's hospital MA Cell and Gene Therapy/Viral infection/Rare disease

151. Shin Jaeyoun 신재윤 Columbia University NY Chemistry

152. Shin Baehyun 신배현 MGH/Harvard Medical School MA Respiratory/metabolic/cardiovascular/Neurogenerative

153. Shin Seung-Wook 신승욱 L&J Biosciences, Inc MD Respiratory/metabolic/cardiovascular/Neurogenerative

154. Shin Hyunjin 신현진 Takeda MA

155. Song HoJuhn 송호준 Genosco MA Immuno-oncology/Autoimmune/Inflammatory

156. Song JK 송정근 L&J Bioscience MD PK/PD/pre-clinical/Clinical Science:

157. Suh Hyunsuk 서현석 Pfizer MA

158. Suh Kathryn MCPHS NJ Pharmacy

159. Suh Byung-Chul 서병철 GENOSCO MA Chemistry

160. Suh K. Stephen Hackensack Meridian Health NJ Cell and Gene Therapy/Viral infection/Rare disease

161. suh Sandy Exeltis NJ Immuno-oncology/Autoimmune/Inflammatory

162. Sung Moo Je 성무제 Novartis MA Chemistry

163. Um Moonkyoung 엄문경 law firm MA BD/Legal/VC:

164. Won Yougun 엄유근 Harvard Medical School MA BD/Legal/VC:

165. Won Kwang-Ai 원광애 LG Chem Life Science R&D CA Immuno-oncology/Autoimmune/Inflammatory

166. Woo Jonghye 우종혜 MGH MA Respiratory/metabolic/cardiovascular/Neurogenerative

167. Yang Eun-Jin 양은진 Decision Resources Group MA BD/Legal/VC:

168. Yang Garp Yeol 양갑열 STP America Research NJ

169. Yang Hanseul 양한슬 The Rockefeller University NY Cell and Gene Therapy/Viral infection/Rare disease

170. Yi B. Alexander 이병두 Novartis MA Respiratory/metabolic/cardiovascular/Neurogenerative

171. Yi Michael 이용민 Celerion Inc. KR PK/PD/pre-clinical/Clinical Science:

172. yim yeong shin 임영신 MIT MA Respiratory/metabolic/cardiovascular/Neurogenerative

173. Yoon Seongkyu 윤성규 University of Massachusetts Lowell MA Cell and Gene Therapy/Viral infection/Rare disease

174. Yoon Chan 윤석찬 Rutgers University NJ Pharmacy

175. Yoon Taeyoung 윤태영 Dong-A ST KR

176. Yoon Derek 윤동민 Aju IB Investment MA BD/Legal/VC:

177. You Kwontae 유권태 Broad Institute MA Immuno-oncology/Autoimmune/Inflammatory

178. You Dahea 유다혜 Rutgers University NJ PK/PD/pre-clinical/Clinical Science:

179. Yu Mikyung 유미경

Brigham and Women's Hospital/ Havard Medical School

MA

180. Yun Esther 윤예진 University of Calgary CA Cell and Gene Therapy/Viral infection/Rare disease

181. Yun Jeong-Ho 윤정호 YPSO-FACTO MA Chemistry

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