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

• Comparing DNA‑based nanoparticle delivery with LNP and lentiviral systems, enabling stronger, longer yet non‑integrating gene expression to enhance therapeutic optionality
• Revealing emerging biodistribution insights including an unexpected liver‑escape profile, expanding understanding of tissue targeting and informing future mechanism‑of‑action work
• Highlighting manufacturing simplicity, safety signals, and unpublished preclinical data, equipping attendees with actionable perspectives on platform readiness and translational potential

• 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 vivo gene 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 

• Optimizing base editor and payload design for efficient liver‑targeted delivery enabling durable, single‑dose functional gene inactivation in vivo
• Assessing off‑target, bystander and edit‑outcome profiles using discovery‑stage analytics strengthening confidence in precision and long‑term safety for clinical advancement
• Aligning potency and comparability strategies with modality‑specific editing dynamics streamlining CMC readiness and accelerating progress toward lasting therapeutic benefit

• 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