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Session 5 Track 1: Liver Targeted Therapeutics, Clinical Experience
Session Chair(s)
Louis O'Dea, MD
Chief Medical Officer and President
BIORCHESTRA (USA) Inc, United States
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)
Inclisiran (Leqvio), a GalNAc-siRNA Anti-PCSK9 Late-phase Clinical Development and Experience From Early Post-Marketing Data
Tom Thuren, MD, PhD
Novartis, United States
Vice President
Pelacarsen, a GalNAc-ASO against Apo(a) in CVD: Phase 2 Results and Phase 3 Plan
Sotirios Tsimikas, MD
Ionis Pharmaceuticals, United States
Senior Vice President, Global Cardiovascular Development
In Vivo CRISPR/Cas9 Editing of the TTR Gene with NTLA-2001 in Patients with Transthyretin Amyloidosis: Phase 1 Results
David E Lebwohl, MD
Intellia Therapeutics, United States
Chief Medical Officer
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