Event preview of TIDES USA 2026

From nanotechnology to nucleic acid therapeutics: tackling a global health challenge

Robert Langer (David H. Koch Institute Professor, Massachusetts Institute of Technology) will discuss the remarkable evolution of nucleic acid delivery from its earliest conceptual stages to the present day. In his talk, “From Nanotechnology to Nucleic Acid Therapeutics: How Overcoming Skepticism and Barriers Led to New Cancer Treatments and Ways to Tackle a Global Health Challenge”, Langer will reflect on five decades of progress, beginning with the widespread scientific skepticism of the 1970s surrounding macromolecule delivery, and charting the field's growth into a robust therapeutic platform with an expanding portfolio of approved treatments and a promising pipeline of candidates in development.

Advancements in oligonucleotide therapeutics

James Powell (VP, RNAi CMC and Boulder Site Head, Novo Nordisk) will discuss how oligonucleotide manufacturing has evolved from small-scale synthesis for research tools to high-throughput platforms powering genomics, screening, and functional studies, and now to industrial-scale production for therapeutic applications. In his presentation “The History and Future of Oligonucleotide Manufacturing”, Powell will highlight key advances in chemistry, automation, and quality systems, and will look ahead to emerging directions, including enzymatic synthesis, sustainability initiatives, and digital optimization as drivers of next-generation manufacturing capability.

Anastasia Khvorova (Professor, RNA Therapeutics Institute, UMass Chan Medical School) will examine how advances in chemical engineering have broadened the reach of RNA interference (RNAi) beyond liver-targeted applications. In her presentation “Chemical Innovation in RNAi: From Chemistry to Neurotherapeutic Applications”, Khvorova will address how small interfering RNA (siRNA) modifications can modulate CNS distribution and cerebrospinal fluid clearance, how multivalent architectures support broad brain exposure, and how backbone modifications, such as extended nucleic acid chemistry, improve therapeutic durability.

Vadim Dudkin (Founding Chief Technology Officer, Soufflé Therapeutics) will discuss a novel platform for discovering cell- and tissue-specific ligands and its application in targeted genetic medicines. In his presentation "Novel Ligands for Cell Selective Gene Silencing," Dudkin will describe how ligands targeting receptors identified through a proprietary discovery platform, when conjugated to siRNA, achieved selective gene silencing in skeletal muscle and cardiac myocytes while sparing off-target tissues.

Innovations in gene editing

Kiran Musunuru (Barry J. Gertz Professor for Translational Research, Perelman School of Medicine at the University of Pennsylvania) will explore the translational journey of personalized gene editing therapies and their broader implications for clinical practice. In his presentation "Lessons Learned from a Personalized CRISPR Gene Editing Therapy", Musunuru will discuss the path from the first individual to receive a personalized gene editing therapy to a future of "interventional genetics", in which such approaches become the standard of care for patients with genetic disease.

Laura Sepp-Lorenzino (Biotech Executive, Board Member, and Industry Advisor, GNMmeds LLC) will examine how programmable therapeutics are reshaping the treatment paradigm for genetic and acquired diseases alike. In her talk "From Sequence to Therapy: Innovations in Oligonucleotide and Genome Editing Medicines," Sepp-Lorenzino will survey recent advances in antisense oligonucleotides, siRNAs, and genome editing platforms, with a focus on delivery strategies that enable tissue-specific targeting and effective intracellular trafficking. The presentation will also explore the use of CRISPR technologies in both in vivo applications and cell-based therapies, drawing on case studies and pipeline developments to illustrate how oligonucleotide and genome editing modalities are converging to advance precision medicine and enable durable, disease-modifying treatments across a broad range of indications.

Peptide drug discovery

Ashok Bhandari (Executive Vice President and Chief Discovery Officer, Protagonist Therapeutics) will present on the company's multi-component peptide discovery platform and its clinical validation. In his talk "Precision Peptides, Powerful Impact: Protagonist Story of Peptide Innovation from Platform to Patients," Bhandari will discuss the development of rusfertide, a hepcidin mimetic for polycythemia vera, and icotrokinra, a first-in-class IL-23 receptor antagonist for psoriasis and IBD, alongside a growing pipeline spanning immunology, metabolic diseases, and hematology.

Michael Kopach (Associate Vice President, Synthetic Molecule Design and Development, Eli Lilly and Company) will examine the molecular design and manufacturing innovation underpinning retatrutide, a first-in-class triagonist with potential applications across obesity, type 2 diabetes, obstructive sleep apnea, and osteoarthritic knee pain. In his presentation "Retatrutide: A Breakthrough Triagonist Uniting Molecular Design and Sustainable Manufacturing," Kopach will address how a hybrid solid-phase/liquid-phase peptide synthesis platform enables scalable, sustainable commercial manufacture with exceptionally low process mass intensity.

Beyond the oligonucleotide, gene editing, and peptide sessions, the forum will also include presentations on topics such as mRNA therapeutics, drug delivery, and oligonucleotide CMC. Notable presentations include Kristine McKinney (Vice President and Head, Cancer Vaccines Research, Moderna) on mRNA cancer vaccines, Eric Kool (Professor of Chemistry, Stanford University) on RNA modification chemistry for stabilization and control, and Andreas Kuhn (Senior Vice President, RNA Biochemistry and CMC Development, BioNTech SE) on analytical platform technologies for mRNA-based therapeutics and vaccines.

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