主要会議 - 3日目（5月22日）
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Streams
6:45am - 7:45am60 mins
Sunrise Yoga: Wellness Event - Reservation Required
Breakfast Spotlight Presentations
7:45am - 8:15am30 mins
Breakfast Spotlight Presentations 1
Transforming Therapeutics: The Role of Advanced Cap Analogs in mRNA Synthesis
- Sigita Cinciute, PhD - Product Manager, Thermo Fisher Scientific
7:45am - 8:15am30 mins
Breakfast Spotlight Presentations 2
Assessing Critical Quality Attributes for sgRNA and mRNA Characterization LC-MS
- Mihir Thakar - Global Product Manager, Phenomenex
7:45am - 8:15am30 mins
Breakfast Spotlight Presentations 3
Innovation in Assays and Enzymes of RNA Therapeutics Development
- Frank Xu - Vice President and Co-founder, Vazyme Biotech
Case Studies of Preclinical and Clinical Oligonucleotides in Development
8:25am - 8:30am5 mins
Oligonucleotide Discovery, Preclinical and Clinical
Chairwoman’s Remarks: Oligonucleotides for CNS Diseases
- Aimee Jackson, PhD - Chief Scientific Officer, Atalanta Therapeutics
8:25am - 8:30am5 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Chairman’s Remarks: Oligonucleotide Analytics & Regulatory Perspectives
- Claus Rentel, PhD - Vice President, Analytical Development/QC, Ionis Pharmaceuticals, Inc.
8:25am - 8:30am5 mins
Peptide Discovery to CMC
Co-Chairs' Remarks: Peptide CMC Strategies
- Marc Jacob, PhD - Head of Strategic Development, Chromatography Products, YMC America
- Matteo Villain, PhD - Vice President and Global Peptide Technical Lead, Piramal Pharma Solutions
8:25am - 8:30am5 mins
Genome Editing Technology and Applications
Co-Chairs': Genome Editing Preclinical and Clinical Updates: Part 1
- Rubina Parmar, PhD, Atlas Ventures
- Cecilia Fernández, Ph.D. - Entrepreneur in Residence, Mass General Brigham Ventures
8:25am - 8:30am5 mins
Delivery of Macromolecules
Chairman’s Remarks: Oligonucleotide Delivery Strategies
- Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Innovation and Distinguished Research Scientist, Alnylam Pharmaceuticals
8:30am - 9:00am30 mins
Oligonucleotide Discovery, Preclinical and Clinical
TRiM™ Platform for Delivery of RNAi Therapeutics to the Central Nervous System via Subcutaneous Administration
- Tao Pei, PhD - Senior Vice President, Chemistry, Arrowhead Pharmaceuticals
8:30am - 9:00am30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Differentiation of n-1 Positional Isomers in Antisense Oligonucleotides with Orthogonal Mass Spectrometry Methods
- Zifan Li, PhD - Senior Scientist, Biogen
8:30am - 9:00am30 mins
Peptide Discovery to CMC
Industry Perspective on the Selection of Regulatory Starting Materials for Synthetic Peptides
- Subha Mukherjee, PhD - Scientific Director, Synthetic Molecule Design and Development, Eli Lilly and Company
8:30am - 9:00am30 mins
Genome Editing Technology and Applications
Writing DNA with RNA: Genome Engineering by Target-Primed Reverse Transcription
- Cecilia Cotta-Ramusino, PhD - Senior Vice President - Head of Platform, Tessera Therapeutics
8:30am - 9:00am30 mins
Delivery of Macromolecules
Delivery of RNAs with Hydrophilic Nanoparticles and Immune Modulation
- Kunwoo (Ryan) Lee, PhD - Chief Executive Officer, GenEdit
9:00am - 9:30am30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Progress Towards Oligonucleotide Therapeutics for Prion Disease
- Sonia Vallabh, PhD - Director, Prion Therapeutic Science, Broad Institute of MIT & Harvard
9:00am - 9:30am30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
sgRNA Single Nucleotide Resolution by Ion-pairing Reversed Phase Chromatography
- Joshua Jones, PhD - Advisor - Analytical Chemistry - SMDD, Eli Lilly and Company
9:00am - 9:30am30 mins
Peptide Discovery to CMC
Synthetic Peptide Enantiomeric Purity Analysis by Chiral HPLC-MS
- Laura Muehlbauer, PhD - Advisor, Eli Lilly and Company
9:00am - 9:30am30 mins
Genome Editing Technology and Applications
Expanding RNA Editing Applications to CNS Disorders Through AIMer Chemistry Design
- Chikdu Shivalila, PhD - Senior Director of Platform Biology, Wave Life Sciences
9:00am - 9:30am30 mins
Delivery of Macromolecules
The Endosomal Escape Vehicle Platform Safely and Effectively Delivers Oligonucleotide Therapeutics to Skeletal and Cardiac Muscle Tissue for the potential treatment of Duchenne Muscular Dystrophy
- Leo Qian, PhD - Co-Founder and Vice President, Discovery Research, Entrada Therapeutics
9:30am - 10:00am30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Antisense Oligonucleotides for the Treatment of Neurological Disease
- Holly Kordasiewicz, PhD - SVP Research, Franchise Leader Neurology R&D, Ionis Pharmaceuticals
9:30am - 10:00am30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Comparing Analytical Strategies for Diastereomer Profiling of a Fully Thiolated ASO
- Matthew Quinn - Manager, Analytical Development, Global Nucleic Acid Therapies, Novo Nordisk
9:30am - 10:00am30 mins
Peptide Discovery to CMC
Challenges and Strategies in Managing Peptide Aggregation
- El Djouhar Rekaï, PhD - Head of Peptide Process Development & Manufacturing, PolyPeptide Group
9:30am - 10:00am30 mins
Genome Editing Technology and Applications
Prime Editors to Treat Human Liver and Metabolic Diseases
- Jeremy Duffield, MD, PhD, FRCP - Chief Scientific Officer, Prime Medicine
9:30am - 10:00am30 mins
Delivery of Macromolecules
Advancing Therapeutics with Antibody-Oligonucleotide Conjugates: Clinical Progress in Rare Muscle Diseases and Emerging Applications in Precision Cardiology
- Mike Flanagan, PhD - Chief Scientific Officer, Avidity Biosciences
10:00am - 10:45am45 mins
Networking Refreshment Break in Poster and Exhibit Halls (Grand Ballroom and Harbor Ballroom)
10:45am - 11:15am30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Divalent siRNAs as Novel Therapeutics for CNS Indications
- Aimee Jackson, PhD - Chief Scientific Officer, Atalanta Therapeutics
10:45am - 11:15am30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
QC Method Development & Duplex Stability Analysis of siRNAs
- Yen-Chih Wang, PhD - Assistant Director, Analytical Development and Quality Control, Ionis Pharmaceuticals
10:45am - 11:15am30 mins
Peptide Discovery to CMC
Bioproduction Platform to Generate Functionalized Disulfide-constrained Peptide Analogues
- Sunhee Hwang, PhD - Scientist 4, Genentech
10:45am - 11:15am30 mins
Genome Editing Technology and Applications
In Vivo Editing of HBG1/2 Promoters Leads to Fetal Hemoglobin Reactivation in Humanized Mouse Model
- John Zuris, PhD - Director of Delivery and Editing Technologies, Editas Medicine
10:45am - 11:15am30 mins
Delivery of Macromolecules
VECTrans® Platform Achieves Efficient Functional Delivery of Oligonucleotides to the deep brain in NHP at Minimal Systemic Dose
- Guillaume Jacquot, PhD - Program Director, Brain and Tissue Delivery of Oligonucleotides, Vect-Horus
11:15am - 11:45am30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Intrathecal Delivery of Antisense Oligonucleotides for Treatment of a Genetic Form of Epilepsy Oligonucleotides for CNS Diseases
- Barry Ticho - Chief Medical Officer, Stoke Therapeutics
11:15am - 11:45am30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Regulatory Feedback on Late-stage Programs - A CDMO Perspective
- Susann Rosmus, PhD - Head of Quality Assurance, BioSpring GmbH
- Ruven Jilly, PhD - Senior Head of Quality Control Lab, BioSpring GmbH
11:15am - 11:45am30 mins
Peptide Discovery to CMC
Peptide Synthesis by Molecular Hiving
- Laurin Melzig, PhD - Director R&D Process Development, Bachem AG
11:15am - 11:45am30 mins
Genome Editing Technology and Applications
Harnessing Endogenous ADAR for Oligo-Directed RNA Editing
- Tyson Moyer, PhD - Principal Scientist, Korro Bio
11:15am - 11:45am30 mins
Delivery of Macromolecules
Safety, Tolerability and Activity of ABX1100, A CD71 Centyrin siRNA Conjugate Targeting GYS1 in a Phase 1 Study in Normal Healthy Volunteers
- Karyn O'Neil, PhD - Chief Scientific Officer, Ar-o Biotherapeutics
11:45am - 12:15pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Mivelsiran, The First Investigational RNAi Therapeutic in Development for Alzheimer’s Disease, Bench to Bedside
- Jayaprakash Nair, PhD - Vice President, Research, Chemistry and Delivery Science, Alnylam Pharmaceuticals
11:45am - 12:15pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
A Unified Platform for the Online Determination of Multiple mRNA Quality Attributes
- Alexandre Goyon, Ph.D - Principal Scientist, Genentech
11:45am - 12:15pm30 mins
Peptide Discovery to CMC
Peptide CMC Strategies for a PCSK9 Inhibitor
- Cindy Hong, PhD - Associate Principal Scientist in Biocatalysis, Process Chemistry, Merck
11:45am - 12:15pm30 mins
Genome Editing Technology and Applications
Building a Best-in-class RNA Editing Platform for the Treatment of Genetic Diseases
- Sriram Sathy, Ph.D. - Chief Scientific Officer, AIRNA
11:45am - 12:15pm30 mins
Delivery of Macromolecules
Late Breaking Presentation
12:15pm - 12:20pm5 mins
Oligonucleotide Discovery, Preclinical and Clinical
Transition to Spotlight Presentation Rooms
12:15pm - 12:20pm5 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Transition to Spotlight Presentation Rooms
12:15pm - 12:20pm5 mins
Peptide Discovery to CMC
Transition to Spotlight Presentation Rooms
12:15pm - 12:20pm5 mins
Genome Editing Technology and Applications
Transition to Spotlight Presentation Rooms
12:15pm - 12:20pm5 mins
Delivery of Macromolecules
Transition to Spotlight Presentation Rooms
12:20pm - 12:50pm30 mins
Spotlight Presentation 1
Ionizable Lipid Development for Targeted-lipid Nanoparticles
- Sho Toyonaga - Director, Drug Delivery Research, FUJIFILM Pharmaceuticals U.S.A., Inc
12:20pm - 12:50pm30 mins
Spotlight Presentation 2
Scalable Lipid Nanoparticle Formulation Using Impingement Jets Mixing Systems - From R&D to Production
- Nadia Elghobashi-Meinhardt, PhD - Project Manager Customized Solutions, KNAUER
12:20pm - 12:50pm30 mins
Spotlight Presentation 3
GenScript Spotlight Presentation
12:20pm - 12:50pm30 mins
Spotlight Presentation 4
Sciex Spotlight Presentation
12:20pm - 12:50pm30 mins
Spotlight Presentation 5
Chemoenzymatic Ligation for siRNA and sgRNA Manufacturing: Recent Advances and Development Strategies
- David Butler, PhD - Chief Technology Officer, Hongene Biotech Corporation
12:50pm - 1:55pm65 mins
Networking Luncheon in Poster and Exhibit Halls (Grand Ballroom and Harbor Ballroom)
Networking Luncheon in Poster and Exhibit Halls (Grand Ballroom and Harbor Ballroom)
1:10pm - 1:20pm10 mins
TIDES Talks in Poster and Exhibit Halls (Grand Ballroom and Harbor Ballroom)
Accelerating the GLP-1 Revolution: Process Technologies Enabling the Future of Peptide Therapeutics
- Chloe Mitchell, PhD - Field Application Scientist, Gyros Protein Technologies
1:20pm - 1:30pm10 mins
TIDES Talks in Poster and Exhibit Halls (Grand Ballroom and Harbor Ballroom)
Innovative Peptide Manufacturing: Piramal Pharma Solutions and the Power of Fragment Condensation to Overcome Resin Aggregation Challenges
- Matteo Villain, PhD - Vice President and Global Peptide Technical Lead, Piramal Pharma Solutions
1:55pm - 2:00pm5 mins
Oligonucleotide Discovery, Preclinical and Clinical
Chairman’s Remarks: Oligonucleotide Discovery to Clinic Case Studies
- Punit Seth, PhD - SVP, Research, Alnylam Pharmaceuticals
1:55pm - 2:00pm5 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Chairman’s Remarks: Innovations in Oligonucleotide Process Development and Manufacturing
- Firoz Antia, PhD - Vice President, Oligonucleotide & Small Molecule CMC, Denali Therapeutics
1:55pm - 2:00pm5 mins
Peptide Discovery to CMC
Co-Chairs' Remarks: GLP-1 and Beyond: Peptide Therapeutics for Diabetes and Obesity/Oral Peptide and Novel Peptide Delivery Technologies
- Trishul Shah, M.S. - Global Sales and Marketing (interim), Head of Sales, Americas, PolyPeptide Laboratories Inc.
- Gary Musso, PhD - President, Musso and Associates LLC
1:55pm - 2:00pm5 mins
Genome Editing Technology and Applications
Chairman’s Remarks: Genome Editing Preclinical and Clinical Updates: Part 2
- John Zuris, PhD - Director of Delivery and Editing Technologies, Editas Medicine
2:00pm - 2:30pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Delivering the Promise of RNAi to Extra-hepatic Tissues
- Punit Seth, PhD - SVP, Research, Alnylam Pharmaceuticals
2:00pm - 2:30pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Step Changes Towards Continuous Oligonucleotide Manufacturing
- Roman Hillebrand, Ph.D. - Associate Director of Manufacturing Innovation, Arrowhead Pharmaceuticals
2:00pm - 2:30pm30 mins
Peptide Discovery to CMC
Retatrutide: A Novel Triagonist for Metabolic Disorders and CMC Advancements
- Michael Kopach, PhD - Associate Vice President, Eli Lilly and Company
2:00pm - 2:30pm30 mins
Genome Editing Technology and Applications
Advancing Axiomer ADAR RNA Editing Platform: Therapeutic Applications and Progress Towards Clinical Development
- Peter Beal, PhD - Professor, Department of Chemistry, UC Davis
2:30pm - 3:00pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Correcting Aberrant Splicing of UNC13A As a Treatment for People with ALS
- Shila Mekhoubad, PhD - Senior Director, Biology, Trace Neuroscience
2:30pm - 3:00pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Preparation of Anti-Sense Oligonucleotide (ASO) via Hybrid Synthesis
- Xuan Zhou - Associate Director, ASO Process Chemistry Lead, Biogen
2:30pm - 3:00pm30 mins
Peptide Discovery to CMC
Advancing Next-Generation Therapies for Obesity and Related Conditions
- Daniel DeOliveira, PhD - Vice President, Drug Substance Development and Manufacturing, Kailera Therapeutics
2:30pm - 3:00pm30 mins
Genome Editing Technology and Applications
Tune-401: a First-in-class Epigenetic Silencer Developed for the Treatment of Chronic Hepatitis B
- Tyler Goodwin, PhD - Head of Delivery Technologies, Tune Therapeutics
3:00pm - 3:30pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Extending Oligonucleotide Delivery and Gene Knockdown to Specific Cell Types in Kidney
- Alfica Sehgal, PhD - Chief Scientific Officer, Judo Bio
3:00pm - 3:30pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Next Generation Manufacturing Process for siRNA
- Joshua Brooks, PhD - Director, Process Sciences, Alnylam Pharmaceuticals
3:00pm - 3:30pm30 mins
Peptide Discovery to CMC
Fragment-Based Approaches for the Production of Peptide-based Therapeutics for Diabetes and Obesity
- Leendert van den Bos - Chief Executive Officer, EnzyTag BV
3:00pm - 3:30pm30 mins
Genome Editing Technology and Applications
LNP Delivery of AsCas12a Enables Liver Editing and Disease-Associated Protein/Biomarker Reduction for Multiple Targets in Mouse and Non-human Primate Preclinical Models
- Judith Newmark, PhD - Associate Director of Pharmacology and Toxicology, Editas Medicine
3:30pm - 4:00pm30 mins
Networking Refreshment Break
4:00pm - 4:30pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
FORCE(TM) Platform Enables TfR1-mediated Delivery of Therapeutics for Rare Neuromuscular Diseases
- Stefano Zanotti, PhD - Head of Neuromuscular Research, Dyne Therapeutics
4:00pm - 4:30pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Solvent and Reagent in-situ Reuse in Solid Phase Oligonucleotide Synthesis
- Jessica Zelinski - Senior Engineer, Process Development, Eli Lilly and Company
4:00pm - 4:30pm30 mins
Peptide Discovery to CMC
Universal Formulation for Oral Therapeutics As a Cornerstone of Improved Patient Care
- Miriam Kidron - Chief Scientific Officer and Director, Oramed Pharmaceuticals Inc.
4:00pm - 4:30pm30 mins
Genome Editing Technology and Applications
The Therapeutic Application of In Vivo CRISPR Gene Editing
- David Maag, PhD - Executive Director, Clinical Development, Intellia Therapeutics
4:30pm - 5:00pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
INHBE Silencing in Mice Confirms Human Genetic Data and Supports a New Approach for Obesity
- Michael Byrne, PhD - VP, Research Pharmacology, Wave Life Sciences
4:30pm - 5:00pm30 mins
Oligonucleotide Chemistry, Manufacturing and Controls
Multicolumn, Continuous Chromatography for Oligonucleotides
- Robert Gronke, PhD - Senior Principal Scientist, Biogen, Inc
4:30pm - 5:00pm30 mins
Peptide Discovery to CMC
BIONDD™ [BEYOND] - Oral Delivery of Biologics with Bioavailability Similar to Subcutaneous Administration
- Karsten Lindhardt, MSc, PhD, DBE - CEO, Biograil
4:30pm - 5:00pm30 mins
Genome Editing Technology and Applications
Single-course Gene Editing to Durably Lower Harmful Cholesterol
- Troy Lister - Chief Scientific Officer, Verve Therapeutics
5:00pm - 5:30pm30 mins
Oligonucleotide Discovery, Preclinical and Clinical
Using the iNeuroRx® Technology Platform to Develop Disease-Modifying Treatments for ALS and FTD
- Wen-Hsuan Chang, PhD - Associate VP, Discovery & Early Development, AcuraStem
5:00pm - 5:05pm5 mins
Oligonucleotide Chemistry, Manufacturing and Controls
ISPE Baseline Guide for Oligonucleotide Facility Design
- Francis Ring - Director of Manufacturing & Operations, Ionis Pharmaceuticals
- Jim Love - Life Sciences Market Director, Oligo/Peptide, CRB
5:00pm - 5:30pm30 mins
Peptide Discovery to CMC
Ultra Long-acting Anti-obesity Peptides
- Daniel Santi, MD, PhD - President and Co-founder, ProLynx LLC
5:00pm - 5:30pm30 mins
Genome Editing Technology and Applications
Genome Editing Discovery
- Christopher Brown, PhD - Head of Discovery, Metagenomi
5:30pm - 5:35pm5 mins
Close of TIDES 2025
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