Conference Day Two - Wednesday | July 11, 2024
8:00 am Check-In & Coffee
8:50 am Chairβs Opening Remarks
Comparing Modes of Delivery: Viral vs Non-Viral, to Gain Insights into Transient & Permanent Delivery Methods
9:00 am Discussing Non-Viral Delivery of DNA to Spotlight this Unique Approach to In Vivo CAR-T
Synopsis
- Highlighting non-viral delivery mechanisms to engineer cells in vivo
- Understanding the advantages and disadvantages of this mode of delivery compared to other forms
- Sharing insights from the NanoCell technology platform
9:30 am Targeted mRNA-LNPs for Tunable Engineering of the Immune System
Synopsis
- Describing the advantages and challenges presented by in vivo reprogramming of the immune system using non-cell based and non-viral based approaches
- Revealing the type and size of payload that can be delivered using LNPs to gain insights into the utilization of this technology
- Discussing the scalability, cost, and manufacturing of non-viral vectors such as LNPs to improve and streamline processes
- Examining the safety and efficacy of LNPs compared to other modes of delivery
10:00 am Morning Break & Networking
11:00 am Examining the Use of Lentivirus Vectors as a Delivery Mechanism to Engineer Cells In Vivo
Synopsis
- Showcasing platforms using viral vectors such as lentiviruses to reflect on how this technology can be used to deliver in vivo cell and gene therapies
- Reaffirming the safety of viral vectors to deliver cell engineering and gene editing treatments
11:30 am Exploring Viral Phylogeny for Engineering Optimized Gene Delivery Vectors
Synopsis
- Characterizing diverse viral envelope proteins for specific and efficient cell targeted gene delivery
- Identifying novel cell targeting moieties for use in our engineered lentiviral delivery systems
- Developing CARs targeting new tumor antigens
12:00 pm Panel Discussion: Addressing Safety of Delivery via Viral or Non-Viral Modes in Response to Concerns in the Ex Vivo Space
Synopsis
- Navigating the safety of delivery mechanisms to collaborate in ensuring vectors are as safe as possible
- Clarifying the latest FDA guidelines in the ex vivo CAR-T space to apply insights to the in vivo space
- Evaluating how this may impact in vivo pipelines using viral vectors to plan for the future
12:45 pm Lunch Break & Networking
Driving In Vivo as the Frontier for Multiple Facets of Cell & Gene Therapy: Focusing on Gene Editing in the Liver & Other Tissues, & Cell Engineering Beyond Oncology
1:45 pm Roundtable Discussion: Exploring In Vivo Gene Editing in the Liver, HSCs, & Beyond
Synopsis
- Unveiling new data highlighting the promising potential of pursing pipelines beyond the liver
- Elucidating the efficacy and durability in vivo gene editing in animal models
- Learning about the new indications that in vivo gene editing beyond the liver could be applied to
2:15 pm Determining the Progression of In Vivo in Indications Beyond Oncology Such As Autoimmune Diseases
Synopsis
- Reflecting on the application of ex vivo cell and gene therapies to consider the in vivo potential of this indication
- Analyzing pre-clinical data suggesting promise for indications outside of oncology to begin mapping out future progression towards the clinics
- Examining how the chronic but not life-threatening nature of autoimmune diseases may impact the safety concerns surrounding CAR-T treatments to evaluate their effect on in vivo
2:45 pm Afternoon Break & Networking
Spotlighting Companies Closest to the Clinic to Understand Priorities & Challenges at this Stage of Development
3:15 pm Panel Discussion: Unveiling the Challenges that Arise in Scaling-Up from Pre-Clinical to Early Clinical Development
Synopsis
- Understanding what models and data may be required to progress towards the clinic
- Hearing about challenges encountered in scaling up to plan for more streamlined processes in the future
- Discovering the bottlenecks that have been faced in scaling up manufacturing to plan for potential challenges ahead