Conference Day Two - Wednesday | July 11, 2024
8:00 am Check-In & Coffee
8:50 am Chair’s 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 Novel Humanized Particles for In Vivo Delivery
Synopsis
- Engineering novel delivery strategies to overcome existing limitations in the field
- Delivering an array of cargoes, including large cargo like prime editors
- Targeting delivery to specific cell types to enhance safety and efficacy
11:30 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
12:00 pm Lunch Break & Networking
1:00 pm Panel Discussion: Addressing the Safety of Delivery in the In Vivo CAR-T 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
1:45 pm 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
2:15 pm In Vivo Cell Engineering using oRNA®
Synopsis
- Orna is leveraging its groundbreaking circular RNA technology paired with its extensive LNP delivery platform to enable the next generation of in vivo cell therapies
- Featuring robust delivery of oRNA to immune cells enabling in vivo CAR mediated depletion of target B cells in vivo
- Demonstrating key progress showing in vivo editing of hematopoietic stem cells to enable development of novel therapies for the hemoglobinopathies and beyond
Spotlighting Companies Closest to the Clinic to Understand Priorities & Challenges at this Stage of Development
2:45 pm Fireside Chat: 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