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DIA/FDA Oligonucleotide-Based Therapeutics Conference

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Session 5 Track 1: Liver Targeted Therapeutics, Clinical Experience

Session Chair(s)

Louis O'Dea O'Dea, MD

Louis O'Dea O'Dea, MD

  • Chief Medical Officer and President
  • BIORCHESTRA (USA) Inc, United States
Xuan Chi, MD, PhD

Xuan Chi, MD, PhD

  • Supervisory Pharmacologist
  • CDER, FDA, United States
The speakers will present data on 3 clinical programs using liver-directed RNA therapeutic modalities. The programs will cover late phase development of a GalNAc-amended SiRNA (Inclisiran), a mid-phase GalNAc-amended ASO (Pelacarsen) and on early phase development of an LNP-formulated Crispr molecule. In addition to providing an update on the progress of each program, the session will allow the audience to compare and contrast the various programs, approaches, and challenges, and how each sponsor has found solutions that can be applied more broadly across the field of oligo therapeutics. Finally, the session is planned to allow time for fruitful exchanges between attendees and speakers.
Learning Objective :
  • Understand why oligo-based therapeutics and CRISPR-based gene editing may be uniquely suited to address certain genetic diseases
  • Understand the specific challenges of developing RNA-based therapeutics
  • Understand the challenges inherent in each of 2 different oligo-based and 1 CRISPR-based gene editing therapeutic modalities
  • Gain insight into the impact of tissue- and cell-specific targeting of these therapeutics on safety and efficacy outcomes

Speaker(s)

Tom Thuren, MD, PhD

Inclisiran (Leqvio), a GalNAc-siRNA Anti-PCSK9 Late-phase Clinical Development and Experience From Early Post-Marketing Data

Tom Thuren, MD, PhD

  • Vice President
  • Novartis, United States
Sotirios Tsimikas, MD

Pelacarsen, a GalNAc-ASO against Apo(a) in CVD: Phase 2 Results and Phase 3 Plan

Sotirios Tsimikas, MD

  • Senior Vice President, Global Cardiovascular Development
  • Ionis Pharmaceuticals, United States
David E Lebwohl, MD

In Vivo CRISPR/Cas9 Editing of the TTR Gene with NTLA-2001 in Patients with Transthyretin Amyloidosis: Phase 1 Results

David E Lebwohl, MD

  • Chief Medical Officer
  • Intellia Therapeutics, United States

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