Day Two

• Analysis of the limitations in existing lentiviral systems identified engineering constraints that shaped the starting architecture for a new lentiviral platform, enabling clearer mechanistic differentiation 
• Preclinical proof of concept readouts and benchmarking data will be presented for the first time

• CRISPR directed gene editing of master regulator genes controlling oncogenesis in cutaneous squamous cell carcinoma
• Tumor cell specific disruption of NRF2 and EGFR genes as a platform approach to the treatment of squamous cell carcinoma of the lung
• Direct delivery strategies for enhancing the effectiveness of gene editing in cancer treatment

• Defining the unmet medical need that can be fulfilled with an in vivo CAR-T approach
• Ideal features necessary for a safe and effective mast cell target
• Design and function of novel mRNA-LNP CAR constructs targeting human mast cells

• Developing lentivector systems using novel viral pseudotypes for improved in vivogene delivery and possibly enabling patient redosing
• Demonstrating in vivo efficacy of CD19 CAR delivery in liquid tumor models using a novel pseudotype
• Applying this novel lentivector system to the delivery of TCRs in melanoma models, expanding therapeutic reach into solid tumor indications

Aligning editing durability with disease specific biological drivers, ensuring long-lasting therapeutic benefit-specific biological drivers

Balancing targeted delivery capabilities with cargo design requirements, maximizing safety and functional persistence

Designing persistence, monitoring, and retreatment frameworks suited to long-term in vivo modulation, enabling reliable patient management over time-term 

• Sharing early clinical data from ongoing patients dosing, accelerating confidence in therapeutic potential
• Implementing adaptive cohort designs and monitoring frameworks, improving trial flexibility and patient safety
• Aligning eligibility and dosing strategies with evolving biomarker insights, ensuring efficient global progression 

Linking immune cell kinetics and clinical scores to dose and retreat timing in autoimmunity, sharpening endpoint selection

Mapping early oncology kinetics to response and event management, improving cohort design and monitoring

Distilling predictive versus misleading preclinical readouts, streamlining data packages for efficiency