RFPT01--Nucleotide-Based Therapeutics: Delivery & Gene Editing

Thursday, September 26, 2024

9:00 AM – 9:50 AM ET

Location:Theater B (Exhibit & Poster Hall)

Chair(s)

PH

Patrick Harrison

Department of Physiology, BioSciences Institute
University College Cork
SS

Shingo Suzuki, PhD (he/him/his)

University of Alabama at Birmingham

This rapid fire session on Nucleotide-Based Therapeutics: Delivery & Gene Editing showcases eight research projects on lipid nanoparticle (LNP) and viral vector delivery, codon optimization and improvements in mRNA delivery, advances in prime editing and selection of editing sites in the context of ancestral diversity. The approaches described in this session show how strong basic sciences underpins the therapeutic pipeline for everyone with CF.
Presenters will be given the opportunity to succinctly present the essence of their poster data one after another. After the session, questions can be addressed with the presenter by visiting their poster during the open Poster Hall hours.

Learning Objectives:

Participants should be able to compare and contrast the different challenges of developing LNP and viral vectors for in vitro and in vivo gene delivery and to summarize the role of codon optimization of coding sequences and mRNA circularization in the context of gene delivery.
Participants should be able to better explain and discuss the challenges of design and selection of gene editing sites in the context of our diverse ancestry, as well as describe the advantages and challenges in the journey to optimize prime editing to restore CFTR function in cells with a range of different CF-causing variants.
Overall, participants at this symposium should then be able apply their combined understanding of delivery and editing strategies to engage in a constructive debate on how to move forward in the development of nucleotide-based therapeutics.