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 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

  • Colin Exline Head of Gene Editing, Primera Therapeutics
  • Dharini Shah Senior Director - Research & Immunology, Independent Expert
  • Nicholas Boyle Founder, President, Chief Executive Officer & Director, Abintus Bio
  • Cécile Bauche Founder & Chief Scientific Officer, Alaya.bio

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 Fireside Chat: Unveiling the Challenges that Arise in Scaling-Up from Pre-Clinical to Early Clinical Development

  • Adrian Bot CSO, Capstan Therapeutics
  • Dharini Shah Senior Director - Research & Immunology, Independent Expert

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

4:00 pm Chair’s Closing Remarks

4:10 pm End of Conference Day Two