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16th Annual Meeting of the Oligonucleotide Therapeutics Society
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Agenda

 

Click here to download a PDF of the agenda.

All times listed are in Eastern Time (ET)
We recognize that our attendees reside in many different time zones around the world; unfortunately we are simply unable to accommodate every attendee with an ideal schedule in their local timezone. We have done our best to arrange the schedule in a way that meets the needs of as many of our constituents as possible. 


Note that all live sessions will also be available on demand after the conclusion of the session so you may watch the presentations at your convenience.


Agenda subject to change

  [+] Show Description
    • Sunday, September 27, 2020
    •  
      09:00  -  09:50
      Networking Meetup - Virtual Coffee Talk
      10:00  -  12:00
      Preconference Session: Oligonucleotide Therapeutics Education Workshop
      Nucleic Acid Therapeutics Development: How To...
      Chair:
      		 Marc Lemaitre, PhD, M_L Consult
      10.00-10.05 Welcome & Introduction
      10.05-10.30 The ADME of siRNA GalNAc Conjugates
      Chris MacLauchlin, PhD, Alnylam 
      10.30-10.55 CMC of Oligonucleotides: Categorization of Impurities
      Thomas Rupp, Thomas Rupp Consulting UG
      10.55-11.20 Nonclinical Safety Evaluation Strategies for Antisense Oligonucleotide Therapeutics
      Cathaline den Besten, PhD, ProQR Therapeutics
      11.20-11.45 Control of Impurities in Double Stranded SiRNA Therapeutics
      Adel Far, PhD, Novartis Pharmaceuticals Canada
      11.45-12.00 Q&A Panel
      Invited Speakers:
      Session Chair:
      12:00  -  12:15
      Break - Networking
      12:15  -  12:50
      Preconference Session: Next-Gen Early Career Scientist Session
      Chairs: Erin M. McConnell, PhD, McMaster University
      Joseph Ochaba, PhD, Ionis Pharmaceuticals
      12.15-12.20Welcome and Introduction
      12.20-12.50 Session Keynote:
      Peptides and Engineered EVs for Delivery of Biotherapeutics
      Samir EL Andaloussi, PhD, Karolinska Institut
      On Demand Short Talks:
       Therapeutic potential of antagomiR-23b for treating Myotonic Dystrophy
      Beatriz Llamusi, PhD, Arthex Biotech SL
       Antisense oligonucleotide-mediated knockdown of mitochondrial fusion and fission factors modulates mitochondrial morphology and rescues mitochondrial dysfunction
      Daniel Garcia, PhD, Ionis Pharmaceuticals
       Design and Enhanced Gene Silencing Activity of Spherical 2′-Fluoroarabinose Nucleic Acids (FANA-SNAs)
      Hassan Fakih, PhD Student, McGill University
       Next Generation Small Nucleic Acid-Based Inhibitors of CRISPR-Cas9
      Chris Barkau, BSc, Southern Illinois University School of Medicine
       Splice-Switching Antisense Oligonucleotides for the Treatment of Cystic Fibrosis
      Wren E. Michaels, Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science
       Antisense oligonucleotide-mediated exon skipping for therapeutic reading frame correction in CLN3 Batten disease
      Jessica Centa, Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science

      		Voting for Best Talk
      A $500 Award will be given for the "Best Talk" of the NextGen session based on audience votes
      Invited Speakers:
      Session Chairs:
      13:00  -  14:30
      Virtual Social Meetups

      #1 - Early Career Scientist Social
      Sponsored by ChemGenes Logo PNG

      #2 - General Networking
    • Monday, September 28, 2020
    •  
      09:00  -  09:50
      Networking Meetup - Virtual Coffee Talk
      10:00  -  10:10
      Meeting Opening & Welcome
      Annemieke Aartsma-Rus, PhD, Leiden University Medical Center, OTS President
      Jonathan K. Watts, PhD, University of Massachusetts Medical School
      Session Chair:
      10:10  -  11:00
      Keynote Presentation:
      Knocking Down Disease – RNAi Therapeutics
      What We Have Accomplished and Where We Need To Go
      Judy Lieberman, MD, PhD, Harvard University & Boston Children’s Hospital
      Keynote Speaker:
      11:00  -  11:15
      Break - Networking
      11:15  -  12:30
      Session I: Nucleic Acid Chemistry
      Chair: Masad J. Damha, PhD, FCIC, McGill University
      11.15-11.40 New Artificial Biomolecules For Oligonucleotide Therapeutics
      Takeshi Wada, PhD, Tokyo University of Science
      11.40-12.05 Generation of Nucleobase-modified Functional Nucleic Acids for Biosensing and Therapeutic Applications 
      Marcel Hollenstein, PhD, Institut Pasteur
      12.05-12.30 Controlled Template-Independent Enzymatic Synthesis of RNA Oligonucleotides
      Jonathan Rittichier, PhD, Harvard University
      On Demand
      		Short Talks: 
       Small molecule-like oligonucleotides as novel antifungal reagents against tRNA 2´-phosphotransferase (Tpt1)
      Leonora Abdullahu, McGill University
       Fluorinated therapeutic agents to combat ALS
      Miguel Garavís, PhD, Instituto de Química-Física Rocasolano, IQFR-CSIC
       2′-O-methylation redistributes the RNA secondary structural ensemble
      Hala Abou Assi, PhD, Duke University
      Invited Speakers:
      Session Chair:
      12:30  -  12:45
      Break - Networking
      12:45  -  14:00
      Session II: Delivery: New Tissues and Technologies
      Chair: Chair: Laura Sepp-Lorenzino, PhD, Intellia Therapeutics
      12.45-13.10 Recent Progress with Antibody Oligonucleotide Conjugates
      Art Levin, PhD, Avidity BioSciences
      13.10-13.35 Selective Organ Targeting (SORT): A Strategy for Effective Tissue Specific mRNA Delivery and CRISPR/Cas Gene Editing
      Daniel Siegwart, PhD, UT Southwestern
      13.35-14.00Vaccinating Against Neoantigens Induced in Tumors and Pathogen Infected Cells
      Eli Gilboa, PhD, University of Miami, Miller School of Medicine
      On Demand
      		Short Talks: 
       Bioengineering bacterial outer membrane vesicles as delivery systems for RNA therapeutics targeted to lung epithelial cytosols
      Elena Eftychiou, Northumbria University
       The morphology and formation of lipid nanoparticles entrapping nucleic acids
      Jayesh Kulkarni, PhD, University of British Columbia
      Invited Speakers:
      Session Chair:
      14:00  -  14:15
      Break - Networking
      14:15  -  15:05
      Session III: Cellular Delivery and Endosomal Escape
      Chair: Steve Dowdy, PhD, UCSD School of Medicine
      14.15-14.40 An Intracellular Depot Supports the Extended Duration of GalNAc-siRNA Conjugates
      Christopher Brown, PhD, Alnylam Pharmaceuticals
      14.40-15.05 Design of Lipid Nanoparticle (LNP) Systems to Enable Gene Therapies
      Pieter Cullis, PhD, The University of British Columbia
      On Demand
      		 Short Talks:
       Efficient, targeted cytoplasmic delivery of oligonucleotides via Sapreme’s endosomal escape enhancers
      Guy Hermans, PhD, Sapreme Technologies BV
       Overhanging duplex oligonucleotides for enhanced systemic delivery
      Kotaro Yoshioka PhD, MD, Department of Neurology and Neurological Science, Tokyo Medical and Dental University
       Improved Functional Delivery of RNA therapeutics in the Eye Using Fatty-Acid Conjugation
      Charles Allerson, PhD, DTx Pharma, Inc.
       Visualizing endosomal escape of ligand-conjugated siRNA
      Hampus Du Rietz, Department of Clinical Sciences, Oncology, Faculty of Medicine, Lund University
      Invited Speakers:
      Session Chair:
      15:15  -  16:45
      Virtual Social Meetup
    • Tuesday, September 29, 2020
    •  
      09:00  -  09:50
      Networking Meetup - Virtual Coffee Talk
      10:00  -  10:50
      Session IV: Genome Editing
      Chair: Ross Wilson, PhD, University of California, Berkeley
      10.00-10.25 A Modular CRISPR/Cas9 Genome Editing Platform for Durable Therapeutic Knockout and Targeted Gene Insertion Application
      Anthony Forget, PhD, Intellia Therapeutics
      10.25-10.50 Systemic Delivery of PNA Nanoparticles to Correct F508del CFTR In Vivo
      Alexandra Piotrowski-Daspit, PhD, Yale University
      On Demand
      		Short Talks:
       Development of receptor-targeted nanocomplexes for in vivo delivery of CRISPR/Cas9 as a potential therapy for Cystic Fibrosis
      Amy Walker, PhD, UCL GOS Institute of Child Health
       5′ Modifications Improve Efficacy of DNA Donors for Precision Genome Editing
      Zexiang Chen, RNA Therapeutics Institute, University of Massachusetts Medical School
       Gene Editing and Cis-Trans Activity Effects with Heavily Modified Cas12a 5' Pseudoknots
      Ramadevi Chilamkurthy, Department of Biochemistry and Molecular Biology, School of Medicine, Southern Illinois University
      Invited Speakers:
      Session Chair:
      10:50  -  11:00
      Break - Networking
      11:00  -  12:15
      Session V: Big Data, AI and Machine Learning in Oligonucleotide Therapeutics
      Chair: Peter Hagedorn, PhD, Roche
      11.00-11.25 Fireside Chat with Brendan Frey, CEO & Founder of Deep Genomics
      Brendan Frey, PhD, FRSC, Deep Genomics
      11.25-11.50High-throughput Analysis Reveals Rules for Target Binding by AGO1–4 RISCs
      Karina Jouravleva, PhD, University of Massachusetts Medical School
      11.50-12.15 Acute Neurotoxicity of Antisense Oligonucleotides After Intracerebroventricular Injection into Mouse Brain Can Be Predicted from Sequence Features
      Peter Hagedorn, PhD, Roche
      Invited Speakers:
      Session Chair:
      12:15  -  12:30
      Break - Networking
      12:30  -  13:40
      Session VI: Awards Presentations and Talks
      Chair: Annemieke Aartsma-Rus, PhD, OTS President
       12.30-12.40Welcome & Award Announcements
      Next-Gen Early Career Scientist Session - Best Talk Award 
      Paper of the Year Awards
      Lifetime Achievement Award Announcement
      12.40-13.00 Dr. Alan M. Gewirtz Memorial Scholarship Award for Graduate Students
      RNAi modulation of APOE as a potential therapeutic for Alzheimer Disease
      Chantal M. Ferguson, RNA Therapeutics Institute, University of Massachusetts Medical School
      13.00-13.20 Dr. Alan M. Gewirtz Memorial Scholarship Award for Postdoctoral Fellows and Junior Industrial Professionals
      Designing New Chemically Engineered siRNAs for Extrahepatic Delivery
      Annabelle Biscans, PhD, AstraZeneca
      13.20-13.40Mary Ann Liebert publishers, Inc. Young Investigator Award
      Advancing the Safety and Reach of RNAi Therapeutics
      Maja M. Janas De Angelis, PhD, Alnylam Pharmaceuticals, Inc.
       On DemandShort Talks:
       Paper of the Year Award - Basic
      Antisense-mediated transcript knockdown triggers premature transcription termination 
      Jong-Sun Lee, PhD, UT Southwestern
       Paper of the Year Award – Late Discovery
      Lowering PCSK9 through siRNA based therapeutics: A revolution in the management of a common disease
      Frederick J.  Raal, PhD, University Of The Witwatersrand, Johannesburg and
      Kausik K Ray, MD, MPhil, Dept of Primary Care & Public Health Imperial Clinical Trials Unit, UK
      Invited Speakers:
      Session Chair:
      13:40  -  14:00
      Break - Networking
      14:00  -  15:30
      Session VII: Early Stage Technology & Novel Mechanisms
      Chair: Isabel Aznarez, PhD, Stoke Therapeutics
      14.00-14.25 Understanding the function of RNA-binding proteins by integration of in vitro and vivo data
      Christopher B. Burge, PhD, Massachusetts Institute of Technology
      14.25-14.50 Counteracting the Chromatin Effects of a Splicing-correcting ASO Enhances Its Therapeutic Efficacy in SMA Mice
      Adrian R. Krainer, PhD, Cold Spring Harbor Laboratory
      14.50-15.15 Antisense Approaches for the Treatment of Cystic Fibrosis
      Shuling Guo, PhD, Ionis Pharmaceuticals
      On Demand
      		 Short Talks:
       Rational Design of Aptamer Switches with Tunable pH Response
      Ian Thompson, BASc, Stanford University
       Looping in phase separation: Controlling RNA:DNA hybrids during transcription
      Jacob Schwartz, PhD, University of Arizona
      Invited Speakers:
      Session Chair:
      15:15  -  16:45
      Virtual Social Meetup
    • Wednesday, September 30, 2020
    •  
      09:00  -  09:50
      Networking Meetup - Virtual Coffee Talk
      10:00  -  11:00
      Session VIII: Turning RNA Against an RNA Virus: Oligo Therapeutics in the Fight Against COVID-19
      Chair:
      		Jonathan Watts, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School
      10.00-10.20 ALN-COV: an Investigational RNAi Therapeutic for COVID-19
      Akin Akinc, PhD, Alnylam Pharmaceuticals
      10.20-10.40 Antisense oligonucleotides as therapeutic agents for SARS-CoV-2
      Lisa Henderson, PhD, NINDS
      10.40-11.00Fighting SARS-CoV-2 with systemically and locally delivered oligonucleotide cocktails
      Jonathan Watts, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School
       

      Invited Speakers:
      Session Chair:
      11:00  -  11:15
      Break - Networking
      11:15  -  12:30
      Session IX: Preclinical Results
      Chair: Martin Maier, PhD, Alnylam Pharmaceuticals
      11.15-11.40Preclinical Development of Antisense Oligonucleotides for Inherited Retinal Diseases
      Jim Swildens, PhD, ProQR 
      11.40-12.05mRNA Chemistry and Manufacturing Process Modulates Innate Immune Activation
      John Joyal, PhD, Moderna
      12.05-12.30Preclinical Efficacy of a Precision Medicine Approach Using a TANGO ASO in the Dravet Mouse Model
      Anne Christiansen, PhD, Stoke Therapeutics
      On DemandShort Talks:
       ASO-mediated selective inhibition of DNA damage response activation at telomeres improves aging phenotypes and extends lifespan in different animal models and species
      Fabrizio d’Adda di Fagagna, IFOM – the FIRC Institute for Molecular Oncology Foundation; IGM-CNR – Istituto di Genetica Molecolare-Consiglio Nazionale delle Ricerche
       Next Generation miR-29 Mimic as a Therapy for Pulmonary Fibrosis
      Rusty Montgomery, PhD, miRagen Therapeutics, Inc.
       Stereopure oligonucleotides support efficient ADAR-mediated RNA editing in non-human primates
      Chikdu Shivalila, PhD, Wave Life Sciences
      Invited Speakers:
      Session Chair:
      12:30  -  12:45
      Break - Networking
      12:45  -  14:15
      Session X: N=1 and Rare Diseases / Patients as Investigators
      Chair:  Tim Yu, MD, PhD, Harvard/Boston Children's Hospital
      12.45-12.55 Facilitating The Development Of Oligonucleotide Therapeutics For N-Of-1+ Diseases
      Annemieke Aartsma-Rus, PhD, Leiden University Medical Center
      12.55-13.20 ASOs For Primary Prevention of Prion Disease: A Patient-led Initiative
      Sonia Vallabh, PhD, The Broad Institute
      13.20-13.45 Models and Mechanisms of FUS-ALS:  In Vivo Studies in Mouse and Man
      Neil Shneider, MD, PhD, Columbia University
      13.45-14.15 Early Forays into Individualized Therapeutics: Ethical Considerations
      Alison Bateman-House, PhD, MPH, MA, Grossman School of Medicine, NYU Langone Health 
       On DemandShort Talk:
       Patient-Specific Antisense Oligonucleotide Therapeutic Development for a Girl with Mucolipidosis
      Margot Cousin, PhD, Mayo Clinic
      Invited Speakers:
      Session Chair:
      14:15  -  14:30
      Break - Networking
      14:30  -  16:10
      Session XI: Oligonucleotide Clinical Studies
      Co-Chairs:   James Hamilton, MD, MBA, BS, Arrowhead Pharmaceuticals
        Arthur M. Krieg, MD, Checkmate Pharmaceuticals
       14.30-14.50Antisense Therapies for Lipid Disorders
      Sam Tsimikas, MD, FACC, FAHA, FSCAI, Ionis Pharmaceuticals
      14.50-15.10
      		 Human Translation of GalNAc-siRNA Conjugates with Improved Specificity
      Vasant Jadhav, PhD, Alnylam Pharmaceuticals
      15.10-15.30
      		 VIR-2218, an X-targeted siRNA for the Functional Cure of HBV
      Phillip S Pang, MD, PhD, Vir Biotechnology, Inc.
      15.30-15.50
      		 Chronic Liver-Specific LDHA Suppression in the Treatment of Primary Hyperoxaluria: Evaluating the Safety and Efficacy of the Investigational Therapy, Nedosiran
      Kerry S. Russell, MD, PhD, Dicerna and Bob Brown, PhD, Dicerna
       15:50-16.10TRiM Platform Derived siRNA Therapeutics: Targeting Hepatocytes and Beyond
      James Hamilton, MD, MBA, BS, Arrowhead Pharmaceuticals
      Invited Speakers:
      Session Chairs:
      16:10  -  16:20
      Closing Remarks & Meeting Adjournment
      16:30  -  18:00
      Annual Meeting Wrap Party

      Sponsored byChemGenes Logo PNG

      The Wrap Party – Virtual Edition         
      A big shout out to ChemGenes, our party sponsor! They continue to champion the OTS Annual Meeting and the Wrap Party. So be sure to grab a refreshment and snacks (on you this year!) and join us on Zoom for a virtual party you will LOVE. We’ll be laughing together, puzzling together and……well, we can’t share every detail. Don’t miss out!!
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