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RNA Leaders USA
RNA breakthroughs, Case Studies and Partnerships
September 23-25, 2026Hynes Convention Center
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Friday, September 25 - EST (Eastern Time, UTC-05:00

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Friday, September 25 - EST (Eastern Time, UTC-05:00
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Showing 1 of 1 Streams
8:00am - 9:00am
Registration
Showing 2 of 2 Streams
9:00am - 9:25am
Presentation: Development and progression to the clinic of antisense oligonucleotide AS-241 to advance treatment of ALS and FTD
  • Wen-Hsuan Chang, PhD - Associate VP, Discovery & Early Development, AcuraStem
9:25am - 9:50am
Presentation: Clinical Overview of Phase 1a TX-MC138 targeting solid tumors and transition to Phase 2a
  • Zdravka Medarova, PhD - Co-Founder and Chief Scientific Officer, TransCode Therapeutics
9:50am - 10:15am
Presentation: Innovating Oligonucleotide Production: Chemoenzymatic Synthesis
  • Wuming Yan - Chief Scientist Oligonucleotides, Asymchem
9:00am - 9:25am
Presentation: Advancing circRNA expression systems applied to in vivo CAR cell Therapy
  • Lubor Gaal - CFO, Circio AB
9:25am - 9:50am
Presentation: In Vivo CAR T with RNA Medicines for Scalable and Safe Immune Cell Reprogramming
9:50am - 10:15am
Presentation: Harnessing circRNA therapeutics for durable and tolerable treatments in oncology and autoimmune diseases – Preclinical Updates
  • Peter Weinstein - CEO, Circurna
Showing 1 of 1 Streams
10:15am - 11:20am
Partnering Hour
Showing 2 of 2 Streams
11:20am - 11:45am
Presentation: Advancing PGN-EDODM1 , an enhanced peptide for clinical studies to treat DMD1
  • Jane Larkindale - VP Clinical Science, PepGen Inc.
11:45am - 12:10pm
Presentation: Phase 2 clinical development of Mivelsiran for Cerebral Amyloid Angiopathy
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Innovation and Distinguished Research Scientist, Alnylam Pharmaceuticals
12:10pm - 12:35pm
Presentation: Advancing siRNA Manufacture From Conventional to Thermostable Enzymatic Ligation
  • Xiaocen (Chris) Li, PhD - Supervisor, LGC Axolabs
11:20am - 11:45am
Presentation: Unlocking tRNA Biology For Stop Codon Disease – First in human trial updates
11:45am - 12:10pm
Presentation: Advancing mRNA Therapeutics with Strand Signal Stack
  • Tasuku Kitada, Ph.D. - President and Head of R&D, Strand Therapeutics
12:10pm - 12:35pm
Presentation: Advancing Therapeutic Angiogenesis: First-in-Human Trial of HM2002 Circular RNA Therapy for Ischemic Heart Failure in CABG Patients
Showing 1 of 1 Streams
12:35pm - 2:00pm
Lunch
Showing 1 of 1 Streams
2:00pm - 2:30pm
Keynote Presentation: Advocacy to Action - The Rare Disease Patient Perspective
2:30pm - 3:15pm
Keynote Panel: RNA Drug Discovery – Fostering the Next Wave of RNA Medicines
  • Laura Sepp-Lorenzino, PhD - Biotech Executive, Board Member and Industry Advisor, GNMmeds LLC
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Innovation and Distinguished Research Scientist, Alnylam Pharmaceuticals
  • Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
3:15pm - 4:00pm
Keynote Panel: Funding and Partnerships Dynamics in Genetic Medicines
  • Daniel Abernathy - Director, McQuade Center for Strategic Research & Development, Otsuka
  • Elena Diez Cecilia - Senior Director External Innovation DPDS, Johnson & Johnson
  • Soyoung Park - General Partner, 1004 Venture Partners
4:00pm - 4:15pm
Closing Remarks
Thursday, September 24
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