2024年 기조 강연

3월13일(수)

3:45 pm Plenary Chairperson's Remarks
Dmitry Samarsky, PhD, CTO, Sirnaomics

3:50 pm PLENARY PRESENTATION Biomimetic Chemistry of RNA Therapeutics
Mano ManoharanMano Manoharan, PhD, Distinguished Scientist & Senior Vice President, Innovation Chemistry, Alnylam Pharmaceuticals
Achieving success in RNA therapeutics depends on proper understanding of mechanisms of nature. In stages of discovery, delivery, and development of RNA-based therapeutics we follow and mimic many natural processes. We will illustrate this concept by taking several key steps of molecular mechanisms involved and examples of medicines which are either approved or in clinical development.

3월14일(목)

8:35 am Plenary Chairperson's Remarks
Paloma Giangrande, PhD, Independent Consultant

8:40 am PLENARY PRESENTATIONRealizing the Promise of in vivo CRISPR Therapeutics
Laura Sepp-LorenzinoLaura Sepp-Lorenzino, PhD, CSO, Intellia Therapeutics
NTLA-2001 is an investigational CRISPR-based therapy being evaluated in a Phase 1, two-part, open-label study in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or with cardiomyopathy (ATTR-CM). NTLA-2002 is being developed for hereditary angioedema (HAE), designed to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of the disease. NTLA-2002 is being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE. Preclinical and clinical data for both programs will be presented.  

9:10 am PLENARY PRESENTATION Harnessing RNA Metabolism for Precision RNA Therapeutics
Jeffery M. CollerJeffery M. Coller, PhD, Bloomberg Distinguished Professor of RNA Biology and Therapeutics, Johns Hopkins University




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