7:45 am Registration & Morning Coffee
Advancing Targeted Extra-Hepatic Delivery to Specific Tissues & Organs to Widen the Therapeutic Index of Gene Editing
8:45 am Chair’s Opening Remarks
9:00 am Panel Discussion: Assessing the State of Play & Visualizing the Future: Challenges & Opportunities for Extra-Hepatic In Vivo Gene Editing
Synopsis
- Examining the current landscape of in vivo gene editing delivery
- Delving into the critical bottlenecks preventing extra-hepatic delivery and identifying the side effects of overdosing to the liver
- Setting a roadmap to accelerate drugs to patients
10:00 am Harnessing Mobile Genetic Elements to Write DNA Sequences with RNA Beyond the Liver
Synopsis
- Exploring LNPs as a versatile delivery system for RNA cargo, well-suited for the all-RNA system of Gene Writers
- Uncovering LNPs’ systemic administration and biology towards organs other than the liver
- Optimizing and quantifying delivery to establish therapeutically relevant extra-hepatic delivery
10:30 am Morning Break & Speed Networking
Synopsis
Our speed networking is the ideal opportunity to get face-to-face time with the brightest minds working in gene editing delivery and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the conference and beyond.
11:30 am Identifying Delivery System Modifications to Avoid the Liver & Target the Desired Tissue for Maximum Efficacy
Synopsis
- Uncovering how to identify proteins that target the tissue of choice by rational and random design
- Understanding whether the protein can be fragmented to reduce the size of the therapeutic package
- Assessing whether this method can eliminate extra-hepatic delivery
12:00 pm Roundtable Discussion: Exploring the Viability of Engineering Delivery Systems That Hit Only One Cell/Tissue Type
Synopsis
- Assessing the current advances in engineering tissue specificity
- Debating whether precise in vivo gene delivery is a pipe dream or viable
- Sharing experiences with new advances to maximize the therapeutic window of in vivo gene editors
12:45 pm Networking Lunch
1:45 pm Exploring Pulmonary Delivery for Gene Editing for Therapeutic Efficacy
Synopsis
- Introducing novel peptide-based delivery strategies to target pulmonary tissues
- Assessing delivery to airway cells using different animal models
- Discussing therapeutic potential of gene editor delivery to the lungs
2:15 pm Crossing the Blood Brain Barrier with Targeting, Safety & Efficacy
Synopsis
- Delivery targeted only to central nervous system
- Avoiding off-target effects, including hepatic accumulation
- Effectively targeting and treating specific brain diseases
2:45 pm Optimizing Ocular Delivery to Accelerate In Vivo Therapy into the Clinic for Rare Retinal Disorders
Synopsis
- Assessing strategies to advance ocular gene editing delivery
- Understanding how the engineering differentiates from systemic delivery
- Addressing safety concerns
3:15 pm Afternoon Networking Break
Discussing Next Generation & Novel Gene Editors for Increased Editing Targeting
4:00 pm Inserting Transposable Elements into Genomes to Take Advantage of Natural Systems
Synopsis
- Discussing the benefits of transposable elements relative to CRISPR/Cas9 in the context of size and tissue accessibility
- Overcoming the continuous diversification of transposable elements to optimize their therapeutic use
- Targeting transposable elements to the desired DNA sequence and cell type
4:30 pm Exploring Novel Cas Nucleases to Widen the Targeting Index of CRISPR/Cas Systems
Synopsis
- Discovering new Cas nucleases with more DNA sequence targets to widen the targeting ability of gene editors
- Understanding why different Cas nucleases target different sequences to aid rational design
- Minimizing off-target editing with novel nucleasesÂ