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Meeting Agenda

All Times Listed in Eastern Time (ET)

Sunday, September 26

9.00-9.50 Networking – Virtual Coffee Meetup





10.00-12.00 Preconference Session: Oligo Therapeutics – The Basics


Oligonucleotide Therapeutics Education Workshop: Oligo Therapeutics – The Basics Chair: Marc Lemaitre, PhD, M_L Consult

10.00-10.05
Welcome and Introduction

10.05-10.30
Successes and Challenges of Current Technologies for Developing Therapies for Genetic Diseases
Meena, PhD, Stoke Therapeutics

10.30-10.55
Method Validation Approaches for Oligonucleotides and Raw Materials
Heidi G. Heck, Nitto Denko Avecia

10.55-11.20
Recent Developments in Oligonucleotide Synthesis, and Reminders of the Classics
Masad J. Damha, PhD, FCIC, McGill University

11.20-11.45
Antisense Drug Development Guideposts: An Ionis View
Richard Geary, PhD, Ionis Pharmaceuticals

11.45-12.00
Q&A Panel




12.15-14.20 Preconference Session: Next-Gen Early Career Scientists


Co-chairs: Leonora Abdullahu, McGill University Aurélie Lacroix, PhD, Sixfold Biosciences 12.15-12.45

Session Keynote:
Chemical Biology, CRISPR, RNA Therapeutics, and More – Just Love What You Do!
Keith Gagnon, PhD, Southern Illinois University

12.45-13.00
Short Talk 1 selected from submitted abstracts

13.00-13.15
Short Talk 2 selected from submitted abstracts

13.15-13.30
Short Talk 3 selected from submitted abstracts

13.30-13.45
Short Talk 4 selected from submitted abstracts

13.45-14.00
Short Talk 5 selected from submitted abstracts

Voting for Best Talk will be available at the conclusion of the session.
Winner will be announced Tuesday during Awards Session




12.00-12.15 Break / Networking





14.00-15.00 Virtual Social Meetups


General Networking Meetup Social Next-Gen Early Career Scientist Social





Monday, September 27

10.00-10.15 Welcome & Opening Remarks


Annemieke Aartsma-Rus, PhD, Leiden University Medical Center
OTS President

Daniel O’Reilly, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School
2021 Meeting Organizing Co-Chair




10.15-11.30 Session I : Nucleic Acid Chemistry


Chair: Masad J. Damha, PhD, FCIC, McGill University

10.15-10.40
Sequence-based Design of Small Molecules Targeting RNA Structure
Matthew Disney, PhD, The Scripps Research Institute

10.40-11.05
A New Class of Oligonucleotide Building Blocks
Maureen McKeague, PhD, McGill University

11.05-11.30
Catalytic Asymmetric Synthesis of P-Containing Scaffolds with P(III) and P(V) Transfers
Scott Miller, PhD, Yale University

On Demand:
Short Talks selected from submitted abstracts




9.00-9.50 Networking – Virtual Coffee Meetup





11.30-11.45 Break / Networking





11.45-13.45 Session II: Rare Diseases and the Role of Patient Advocates


Chair: Timothy Yu, MD, PhD, Harvard/Boston Children's Hospital

11.45-12.05
n-Lorem, a Charitable Solution for Patients with Ultra-Rare Mutations
Stanley T. Crooke, MD, PhD, n-Lorem Foundation

12.05-12.25
Topic: ALS
Robert H. Brown MD, DPhil, University of Massachusetts Medical School

12.25-12.45
Preliminary Studies on Use of ASOs to Reverse Imprinting and Treat Angelman Syndrome
Elizabeth M. Berry Kravis, MD, PhD, Rush University Medical Center

12.45-13.05
Topic: Pediatric Application of Lumasiran
Pushkal Gard, MD, Alnylam Pharmaceuticals

13.05-13.25
Title TBA
Timothy Yu, MD, PhD, Harvard/Boston Children's Hospital

13.25-13.45
Panel Discussion with Maarika Kimbrell, JD, USFDA




13.45-14.00 Break / Networking





15.15-16.15 Virtual Social Meetups





14.00-15.15 Session III: 20th Anniversary of Mammalian RNAi


Chair: John Maraganore, PhD, Alnylam Pharmaceuticals

14.00-14.25

TBA
Craig Mello, PhD, University of Massachusetts Medical School

14.25-14.50
A Journey with the Argonautes
Leemor Joshua-Tor, PhD, Cold Spring Harbor Laboratory

14.50-15.15
Harnessing RNAi for a New Class of Medicine
John Maraganore, PhD, Alnylam Pharmaceuticals





Tuesday, September 28

9.00-9.50 Networking – Virtual Coffee Meetup





10.00-11.15 Session IV: Delivery


Chair: Annabelle Biscans, PhD, AstraZeneca

10.00-10.25
Combinatorial Development of RNA Delivery Systems
Daniel G. Anderson, PhD, Massachusetts Institute of Technology

10.25-10.50
Centyrin Mediated Delivery of siRNA to Extra Hepatic Tissues
Karyn T. O'Neil, PhD, Aro Biotherapeutics

10.50-11.15
Novel Technologies for Enhancing Oligonucleotide Delivery in Neuromuscular Disease
Matthew Wood, PhD, University of Oxford

On Demand:
Short Talks selected from abstract submissions




11.30-12.35 Session V: Awards Session


Chair: Annemieke Aartsma-Rus, PhD, Leiden University Medical Center

12.45-12.50
Welcome
NextGen Session (26 Sept) - Best Talk Award Announcement
Paper of the Year Award Announcement

12.50-13.10
Dr. Alan M. Gewirtz Memorial Scholarship Award for Graduate Students
To Be Announced

13.10-13.30
Dr. Alan M. Gewirtz Memorial Scholarship Award for Postdocs
To Be Announced

13.30-13.50
Mary Ann Liebert publishers, Inc. Young Investigator Award
To Be Announced

Awards application deadline July 9




12.35-12.45 Break / Networking





12.45-14.00 Session VI: Genome and RNA Editing


Chair: Keith Gagnon, PhD, University of Southern Illinois

15.30-15.55
Programmable RNA Editing: New Approaches, New Challenges
Prashant Mali, PhD, UCSD/Shape Therapeutics

15.55-16.20
In Vivo Epigenome Editing with CRISPR Systems
Charles Gersbach, PhD, Duke University

16.20-16.45
Prime Editing: Precise and Versatile Genome Editing without Double-Strand Breaks or Donor DNA
Andrew Anzalone, MD, PhD, Prime Medicine

On Demand:
Short Talks selected from abstract submission




11.15-11.30 Break / Networking





14.00-14.15 Break / Networking





14.15-16.45 Session VII: Bob Letsinger, PhD – 100 Years of History


Co-chairs: Mano Manoharan, PhD, Alnylam Pharmaceuticals and Masad J. Damha, PhD, FCIC, McGill University

14.15-14.20
Welcome and Introduction
Masad J. Damha, PhD, FCIC, McGill University

14.20-14.35
Robert Letsinger: The Father of Synthetic DNA Chemistry
Marvin H. Caruthers, PhD, University of Colorado Boulder

14.35-14.50
Bob Letsinger: Inspiration, Mentor, Motivating Genius, and Life-long Friend
Hon Kelvin K. Ogilvie C.M.,Ph.D.,D.Sc.,F.C.I.C.,HCol., Senator(r), Acadia University

14.50-15.05
Bob Letsinger: "Well, let's see what you can do."
Paul Miller, PhD, Johns Hopkins Bloomberg School of Public Health

15.05-15.20
Robert Lewis Letsinger: Early Years
Margaret Schott, PhD, Northwestern University

15.20-15.35
Bob Letsinger – True Pioneer of Nucleic Acid Chemistry, Mimetics and Conjugates
Sergei M. Gryaznov, PhD, MAIA Biotechnology, Inc.

15.35-15.40
Business With Bob: Navigating the Start-up World with an Oligonucleotide Giant
Chad Mirkin, PhD, Northwestern University

15.40-15.55
Meetings with Bob
Martín Egli, PhD, Vanderbilt University

15.55-16.10
TBA

16.10-16.25
Hundred Years of Bob Letsinger and Early Years of Oligonucleotide Therapeutics
Muthiah Manoharan, PhD, Alnylam Pharmaceuticals

16.25-16.45
Q&A with Panel and Concluding Remarks




16.45-17.45 Virtual Social Meetups






Wednesday, September 29

9.00-9.50 Networking – Virtual Coffee Meetup





10.00-11.45 Session VIII: Oligonucleotide Preclinical I


Co-chairs: Shalini Andersson, PhD, AstraZeneca and Jimmy Weterings, PhD, AstraZeneca

10.00-10.05
Poster Award Winners - Announced

10.05-10.30
PCSK9 ASO Story including Phase 1 Data (Dyslipidemia/ Liver targeting)
Björn Carlsson, PhD, AstraZeneca

10.30-10.55
SLN360 - A Case Study for Overcoming the Challenge of a Species-restricted mRNA Target
Marie Wikström Lindholm, PhD, Silence Therapeutics

10.55-11.20
CNS RNAi PK/PD in Animal Models of Different Brain Size
Anastasia Khvorova, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School

11.20-11.45
ALN-APP for the Treatment of Autosomal Dominant Alzheimer's Disease
Kirk Brown, PhD, Alnylam Pharmaceuticals

On Demand:
Short Talks selected from abstract submissions




11.45-12.00 Break / Networking





12.00-13.15 Session IX: Oligonucleotide Preclinical II


Co-chairs: Holly Kordasiewicz, PhD, Ionis Pharmaceuticals and Bruno Godinho, PhD, Atalanta Therapeutics

12.00-12.25

Topic: SSRI-conjugated oligos for the treatment of PD and MDD
Analia Bortolozzi, Institute of Biomedical Research of Barcelona

12.25-12.50
Topic: High-resolution visualization and quantification of therapeutic oligos by NanoSIMS
Haibo Jiang, University of Australia/The University of Hong Kong

12.50-13.15
Using Nuclear Imaging to Understand Delivery and Distribution of Intrathecal ASOs in Humans
Nicolas Currier, Biogen

On Demand:
Short Talks selected from abstract submissions




13.15-13.30 Break / Networking





13.30-14.50  Session X: Oligonucleotide Clinical Studies


Co-chairs: Martin Maier, PhD, Alnylam Pharmaceuticals and Richard Geary, PhD, Ionis Pharmaceuticals

13.30-13.50
Development of the Moderna COVID-19 Vaccine
Adam Bloomfield, MD, FAAP, Moderna

13.50-14.10
Topic: Overview on Pulmonary Program IONIS-ENAC-2.5Rx
Ken Newman, MD, Ionis Pharmaceuticals

14.10-14.30
Harnessing the Promise of Liver-Directed CRISPR Gene Editing for Rare Diseases
Laura Sepp-Lorenzino, PhD, Intellia Therapeutics

14.30-14.50
Dose-related and prolonged reductions in blood pressure with a RNAi therapeutic targeting angiotensinogen in hypertensive patients: Interim results from a Phase 1 study with ALN-AGT01
Stephen Huang, MD, Alnylam Pharmaceuticals

On Demand:
Late Breaking Talks




14.50-16.00 Closing Remarks





16.00-17.00 Annual Meeting Wrap Party


Details to follow, but you won't want to miss on the fun!