OPT Congress 2024 Plenary Session Program
WEDNESDAY, MARCH 13
3:45 pm Plenary Chairperson's Remarks
Jim Weterings, PhD, Vice President Research, RNA Therapeutics & Delivery, Sirnaomics
3:50 pm PLENARY PRESENTATION: Biomimetic Chemistry of RNA Therapeutics
Mano 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.
4:20pm PLENARY PRESENTATION: Applications for mRNA Therapeutics: Immunological Issues and Considerations
Arthur Krieg, MD, Adjunct Professor, University of Massachusetts, Chan School of Medicine
From an immunological perspective, there are 3 distinct categories of mRNA therapeutics, including: 1. Protein expression mRNAs (including e.g., enzyme replacement, antibody expression, gene editing with encoded programmable nuclease), wherein any immune activation is highly undesirable; 2. Infectious disease mRNA vaccines such as COVID (immune activation desirable to induce neutralizing antibodies); and 3. Cancer mRNA vaccines (immune activation desirable to induce CD8+ T cells able to kill tumors). Achieving these distinct immune effects requires intentional design of the mRNA and delivery system, which will be reviewed.
THURSDAY, MARCH 14
8:35 am Plenary Chairperson's Remarks
Paloma Giangrande, PhD, Chief Technology Officer, Eleven Therapeutics
8:40 am PLENARY PRESENTATION: Realizing the Promise of in vivo CRISPR Therapeutics
Laura 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. Coller, PhD, Bloomberg Distinguished Professor of RNA Biology and Therapeutics, Johns Hopkins University
We have created a therapeutic technique that enhances mRNA translation. This technology has numerous clinical applications and works by binding to mRNA and improving translation. The approach offers key benefits: it is disease-modifying, restoring normal protein levels; it is mutation agnostic; it can be tailored to precisely control expression, reducing the risk of overexpression; and lastly, it is applicable across indications and highly versatile.