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

Welcome and Introduction

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

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

Oligonucleotide Synthesis: Reminders of the Classics and Exploring the New
Masad J. Damha, PhD, FCIC, McGill University

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

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

Discovery And Characterization Of Spike N-terminal Domain-binding Aptamers For Rapid SARS-CoV-2 Detection
Lucy Yang, University of Washington

Scalable Synthesis Of DNA And RNA Oligomers Using Mechanochemistry
James Thorpe, McGill University

Differential Uptake Of Antisense Oligonucleotides In Mouse Hepatocytes And Macrophages Revealed By Simultaneous Two-photon Excited Fluorescence And Coherent Raman Imaging
Prabuddha Mukerjee, University of Illinois at Urbana Champaign

Simultaneous, Single-particle Measurements Of Size And Loading Give New Insights Into The Structure Of Drug-delivery Nanoparticles
Albert Kamanzi, PhD, University of British Columbia

Investigating The Impact Of Epitranscriptomic Modifications On RNA Ensembles
Hala Abou Assi, PhD, Duke University

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

Catalytic Asymmetric Synthesis of P-Containing Scaffolds with P(III) and P(V) TransfersScott Miller, PhD, Yale University

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


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

On Demand:
5' Phosphofluoridate Modifications Impact Sirna Activity Profiles
Andrew Varley, BSc, MSc, Ontario Tech University

Backbone Hydrocarbon-constrained Nucleic Acids Modulate Hybridization Kinetics For RNA
Graeme Freestone, PhD, Ionis Pharmaceuticals

Aminooxy Click Chemistry (aocc): A Versatile Bioconjugation Approach
Dhrubajyoti Datta, PhD, Alnylam Pharmaceuticals Inc.

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

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

Antisense Oligonucleotide Therapies for ALS
Robert H. Brown MD, DPhil, University of Massachusetts Medical School

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

Development of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1
Pushkal Garg, MD, Alnylam Pharmaceuticals

N=1 Collaborative
Timothy Yu, MD, PhD, Harvard/Boston Children's Hospital

Panel Discussion with Maarika Kimbrell, JD, USFDA

On Demand:
Exon-skipping Antisense Oligonucleotide Therapy For A Patent With Nieman Pick Type C
Haiyan Zhou, MD, PhD, University College London

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


Intronless genes trigger Argonaute surveillance and a novel mode of co-translational silencing
Craig Mello, PhD, UMass Medical School

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

Harnessing RNAi for a New Class of Medicine
John Maraganore, PhD, Alnylam Pharmaceuticals
On Demand: A Genome-wide Screen Of Sirna Molecules Against Sars-cov-2
Omer Weissbrod, Eleven TX Identification Of Small Interfering RNAs For Allele-selective Silencing Of Murine Von Willebrand Factor
Yvonne Jongejan, MSc, Leiden University Medical Center Imaging Huntingtin Messenger RNA For Diagnosis And Therapy
Eric Wickstrom, PhD, Thomas Jefferson University

Tuesday, September 28

9.00-9.50 Networking – Virtual Coffee Meetup

10.00-11.15 Session IV: Delivery

Chair: Annabelle Biscans, PhD, AstraZeneca

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

Extra-hepatic delivery of Centyrin-targeted siRNA conjugates
Karyn T. O'Neil, PhD, Aro Biotherapeutics

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

On Demand:
Unlocking the Potential of Oligonucleotide Therapeutics for Duchenne Muscular Systrophy Through Enhanced Delivery
James McArthur, PhD, PepGen

Endosomal Escape Vehicle Conjugation Enhances Functional Delivery Of Oligonucleotides
Ziqing "Leo" Qian, PhD, Entrada Therapeutics

Improved Delivery And Therapeutic Efficacy Of Oligonucleotide Therapeutics With The Use Of Optimer Delivery Vehicles - Edward Barnes, PhD, Aptamer Group

11.30-12.35 Session V: Awards Session

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

Welcome & Announcemnts:
Best Talk Award – NextGen Session
Paper of the Year Awards Presentation


Dr. Alan M. Gewirtz Memorial Scholarship Award for Graduate Students
Sequence Controlled DNA-Polymer Conjugates for The Delivery of Nucleic Acid Therapeutics
Hassan Fakih, McGill University


Dr. Alan M. Gewirtz Memorial Scholarship Award for Postdocs
Heteroduplex-structure Reduces CNS-toxicity Of ASO With Intrathecal Administration And
Enhances Therapeutic Index In Rodents And Non-human Primates
Kotaro Yoshioka, MD, PhD, Tokyo Medical and Dental University


Mary Ann Liebert publishers, Inc. Young Investigator Award
Antisense Oligonucleotides to Treat Inherited Retinal Diseases
Alex Garanto, PhD, Radboudumc

On Demand:
Paper of the Year Awards

Basic Research Category:
Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates
Vasant Jadhav, PhD, Alnylam Pharmaceuticals

12.35-12.45 Break / Networking

12.45-14.00 Session VI: Genome and RNA Editing

Chair: Keith Gagnon, PhD, University of Southern Illinois

Programmable RNA Editing: New Approaches, New Challenges
Prashant Mali, PhD, University of California, San Diego

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

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

On Demand:
CLUSTER Guide RNAs Enable Precise And Efficient RNA Editing In Cell Culture And In Vivo
Philipp Reautschnig, PhD, University of Tübingen

Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing Of SERPINA1 Transcripts, Yielding Functional Alpha-1 Antitrypsin Protein In A Mouse Model For Alpha-1 Antitrypsin Deficiency
Prashant Monian, PhD, Wave Life Sciences

Iterative Optimization of a Universal Chemically Modified Anti-CRISPR-Cas9 Oligonucleotide: Toward In Vivo Proof-of-Concept
Christopher L. Barkau, Southern Illinois University

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

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

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

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

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

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

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

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

Meetings with Bob
Martín Egli, PhD, Vanderbilt University

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

Family Tribute
Reed Letsinger & Sue Letsinger

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

Poster Award Winners - Announced

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

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

RNAi-based ApoE Targeting in Alzheimer's Disease: Innate Immune Response Activation for Plaque Clearance Modulation
Anastasia Khvorova, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School

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

On Demand:
Guanidine-bridged Nucleic Acid Enhances Potency Of Antisense Oligonucleotides In Heart
Tadashi Umemoto, PhD, Luxna Biotech Co. Ltd.

Efficient Exon 53 Skipping Of The Human Dystrophin Transcript In A Mouse Model For Duchenne Muscular Dystrophy
Sarah Engelbeen, Leiden University Medical Center

Structure-based Optimization Of Lipophilic Sirna Conjugates For Albumin Binding Yields Improved Pharmacokinetics And Carrier-free Tumor Gene Silencing
Ella Hoogenboezem, Vanderbilt University

DUET-01: Bispecific Oligonucleotide Targeting TLR9 and STAT3 Signaling for B Cell Lymphoma Immunotherapy
Alan Horsager, PhD, Duet Therapeutics

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


SSRI-conjugated oligonucleotides for the treatment of major depressive disorder and Parkinson´s disease
Analía Bortolozzi Biassoni, PhD, Institute of Biomedical Research of Barcelona

High-resolution visualization of nucleic acid-based therapeutics in cells and tissues using NanoSIMS
Haibo Jiang, PhD, The University of Hong Kong/The University of Western Australia

Using Nuclear Imaging to Understand Delivery and Distribution of Intrathecal ASOs in Humans
Jenna Sullivan, PhD, Biogen

On Demand:
Stealth Fluorescence Labeling For Live Microscopy Imaging Of Nucleic Acid Therapeutics
Tom Baladi, PhD, AstraZeneca

Targeting 3’ And 5’ Untranslated Regions With Antisense Oligonucleotides To Stabilize Frataxin MRNA And Increase Protein Expression
Marek Napierala, UAB

Systemically Administered Dna/rna Heteroduplex Oligonucleotides Achieve Blood To Brain Delivery And Efficient Gene Knockdown In The CNS
Takanori Yokota, MD, PhD, Tokyo Medical and Dental University

13.15-13.30 Break / Networking

13.30-15.10  Session X: Oligonucleotide Clinical Studies

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

Development of the Moderna COVID-19 Vaccine
Randy Hyer, MD, PhD, MPH, Moderna

Overview on Pulmonary Program IONIS-ENAC-2.5Rx
Ken Newman, MD, Ionis Pharmaceuticals

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

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:
Evaluation Of Dmd Transcripts Imbalance Pre And Post Golodirsen Treatment In Myod-converted Fibroblasts From 4053-101 Clinical Trial Patients
Rachele Rossi, PhD, UCL

15.10-15.20 Annual Meeting Closing Remarks

15.30-17.00 Annual Meeting Wrap Party

Sponsored by: Grab a refreshment and snacks and join us for a virtual party you will LOVE. We’ll reveal the winners of the Talent Show, laugh together at comedian Don McMillian (back in 2021 by popular demand), followed by BarNone Trivia a live team trivia game! Prizes will be awarded to the winning teams. Don’t miss out!
A big shout out to ChemGenes, our party sponsor - they continue to champion the OTS Annual Meeting and the Wrap Party for 2021.