Conference Day Two | Thursday, June 5
7:30 am Check-In & Coffee
8:20 am Chairβs Opening Remarks
8:30 am Envisioning the Future Evolution of In Vivo Hematopoietic Stem Cell Engineering & Gene Editing Treatments
Synopsis
- Establishing in vivo HSC engineering approaches by leveraging validated biology from ex vivo therapeutics to eliminate patient and manufacturing burdens and expand treatable patient populations
- Combining large gene insertions and lineage-specific control in vivo to enable multicellular interventions to diversify treatment opportunities
- Evaluating in vivo HSC engineering opportunities in gene editing methods, including Cas9, base editing, prime editing, and targeted insertions, alone and in combination with insertions
9:00 am Roundtable Discussion: Harnessing Investor & Pharma Insights to Facilitate Successful, Lasting, Progressive Collaborations & Investments
Synopsis
- Revealing the considerations pharma companies face when deciding to enter the market
- Delving into the investorsβ mindset to understand what drives their expectations, concerns and evaluation of biotech investments
- Discussing the factors that determine the commercial viability of treatmentsΒ
9:45 am Morning Break & Networking
10:15 am Unlocking Novel Strategies & Technologies to Produce, Targeted, Stable Vehicle Delivery Options & Stimulate Clinical Progression
Synopsis
- Navigating complex design processes to produce delivery vehicles that protect the therapeutic payload, enhance stability and facilitate targeted delivery
- Spotlighting novel technologies to transform vehicle delivery manufacturing and produce safe, stable delivery vehicles
10:45 am Leveraging Delivery Model Insights to Engineer Enhanced Delivery Vehicles to Refine Target Specificity & Reduce Immunogenicity
Synopsis
- Identifying and discussing strategies for vehicle optimization to ensure efficient treatment delivery for a variety of payloads
- Exploring the mechanisms that drive immunogenicity and how we can overcome them
- Utilizing enhanced delivery vehicles to effectively balance treatment potency and toxicity prevention
11:15 am Roundtable Discussion: The Transgene Insertion or Transient Expression Debate: Exploring the Superior Method for Payload Delivery
Synopsis
- Exploring which process is best utilized for curative or symptom regulation improvements in oncology and autoimmune-based diseases
- Discussing the efficacy and safety impacts of each payload delivery method to identify the premier delivery method
- Evaluating preclinical data highlighting the persistence and treatment response outcomes to provide insight into possible clinical outcomesΒ
12:00 pm Lunch Break & Networking
1:00 pm Reimagining CAR & Protein Manufacturing Processes to Revolutionize Treatment Payload Manufacturing
Synopsis
- Modifying the CAR manufacturing process to overcome manufacturing challenges caused by the hydrophobic domain of CARs
- Understanding the complexities of protein multidomain to enhance and simplify the protein purification process during manufacturing
- Exploring cargo expression suppression in lentiviral manufacturing to maximize efficient production and prevent interference in particle productionΒ
1:30 pm Navigating Protein Delivery Mechanisms to Overcome Tolerance & Immunogenicity
Synopsis
- Navigating protein delivery mechanisms of action in the muscle or brain to explore delivery beyond the liver
- Highlighting factors that mediate tolerance to foreign viral capsids produced by AAV delivery
- Understanding the extent to which protein-induced immunogenicity impacts therapy delivery, potency and efficiency to improve treatment efficacy
2:00 pm Panel Discussion: βEvaluating LVV & LNP Delivery Method Clinical Potential in In Vivo Cell & Gene Therapies
Synopsis
- Discussing the long-term efficacy and durability of LVV and LNP-based therapies to understand the impact of delivery on disease regression and treatment
- Exploring how LVV, LNP and RNP technologies can evolve to produce enhanced therapeutic outcomes
- Evaluating the potential of the delivery vehicles to deliver complex payloads
2:45 pm Afternoon Break & Networking
3:15 pm Exploring the Possibilities of In Vivo Gene Editing to Diversify Treatment Opportunities
Synopsis
- Exploring how in vivo genetic editing could unlock treatments for previously untreatable genetic disorders and complex diseases
- Understanding how advances in genetic editing will enable more tailored, patient-specific therapies for improved outcomes
- Discuss the hurdles of ensuring sustained effects and safety of in vivo genetic editing therapies over time
3:45 pm Shaping the Next Generation of In Vivo Gene Editing
Synopsis
- Explore cutting-edge technologies improving the accuracy and efficiency of in vivo genetic editing techniques, such as CRISPR and base editing
- Address the evolving methods to enhance delivery systems, ensuring targeted and efficient gene editing in diverse tissues and organs
- Delving into metabolism changes as a result of in vivo gene editing
- Sharing preclinical successes
4:15 pm Reimagining Gene Editing Capabilities: Exploring In Situ Gene Editing of the Microbiome to Expand Addressable Targets
Synopsis
- Exploring bacterial expression from the core microbiome treat immune and chronic diseases
- Highlighting bacterial viral vectors as effective delivery vehicles for gene editing payloads