Conference Day One - Wednesday | July 10, 2024
7:50 am Check-In & Coffee
8:50 am Chairβs Opening Remarks
Evaluating Promises & Challenges to Propel In Vivo Approaches as the Definitive Solution
9:00 am Industry Leaderβs Fireside Chat: Supercharging In Vivo as the Frontier of Cell Therapy & Comparing to Ex Vivo
Synopsis
- Accentuating the bottlenecks of ex vivo therapies, such as the need for costly manufacturing processes and limited access, to consider the need to move towards a new engineering technique
- Championing in vivo therapies by highlighting the solutions they could bring to the ex vivo cell therapy and gene editing field
- Reaffirming the shift of interest towards in vivo therapies as the reality of clinical progression comes to the forefront
10:00 am Morning Break
Optimizing Target Specificity to Minimize Off-Target Toxicity & Prioritize Safety, Allowing Progression Towards Clinics
11:00 am Panel Discussion: Improving Target Specificity by Reviewing Methods to Reduce Off-Site Effects & Innovations Needed to Increase Target Precision
Synopsis
- Identifying challenges in target specificity to collaboratively review current techniques
- Assessing the advantages and disadvantages of each method to improve target specificity
- Planning for future improvements and driving new innovations to elevate target specificity
Focusing on Gene Editing in the Liver & Other Tissues, & Cell Engineering Beyond Oncology
11:30 am Developing In Vivo Editing To Target HSCs
Synopsis
- Describing how Sanaβs Retargetable Fusogen platform directly couples receptor recognition to cell entry, in contrast to endosomal escape delivery systems, enabling high on vs. off-target cell specificity in vitro and in vivo
- Showing development of a T cell-specific fusogen to enable in vivo CAR-T therapy
- Highlighting data showing the progression of HSC in vivo gene editing using a virus-like particle (VLP) system
12:00 pm Lunch Break & Networking
Finding the Balance Between Efficacy & Toxicity to Secure Safe & Promising Outcomes
1:00 pm Examining Efficacy of In Vivo Treatments & Sharing Pre-Clinical Data
Synopsis
- Addressing clinical CAR-T indications, efficacy signals, the need for lymphodepletion, effective doses, toxicities, and correlatives (integrations per cell, durability, exhaustion etc.)
- Evaluating the performance of in vivo generated CAR-T cells in well-known pre-clinical models to allow us to predict likely clinical superiority
1:30 pm Exploring How Efficacy & Toxicity Link with Drug Exposure
Synopsis
- Understanding connections between increased drug exposure and increased efficacy in models
- Discussing the optimal drug exposure to balance off-site toxicity with increased efficacy
2:00 pm Afternoon Break & Speed Networking
Synopsis
As this community re-unites for the third time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections.
Discussing Innovations in Platform Development: CAR-M Therapies & Administration Methods
3:00 pm Reprogramming Myeloid Cells for Cancer: CAR-M from Ex Vivo to In Vivo
Synopsis
- Targeting macrophages with CARs for solid tumors
- Investigating pre-clinical and clinical data with ex vivo CAR-M cell therapies
- Describing our in vivo CAR-M platform that is being developed in partnership with Moderna
3:30 pm Lessons Learned from Intravenous Administration of Gene Delivery Vehicles for In Vivo Cell Engineering
Synopsis
- Exploring intravenous administration of gene delivery vehicles to inform the development of in vivo CAR-T
- Scaling the manufacturing of products for intravenous administration
- Examining the role of synthetic biology to achieve the targeting precision required for in vivo cell engineering