Day Two

• Sharing innovations in cmCAR and CellectLNP technology enable a differentiable and highly extensible in vivo cell engineering platform
• Revealing how BR101, a bi-specific in vivo CAR-T pipeline based on cmCAR and CellectLNP, exhibits robust and systemic B-cell depletion and immunereset in NHP
• Exploring how an open-label, single-arm clinical trial has been initiated to evaluate safety and efficacy of BR101 in cancer and autoimmune diseases

• Rational Design of Ionizable Lipid Libraries: Application of Structure-Activity Relationship (SAR) modeling and AI guided molecular design to develop a proprietary lipid library with an optimized therapeutic index and enhanced clinical tolerability
• Optimization for Hepatic Genome Editing: Identification and selection of LNP formulations tailored for high efficiency hepatic delivery, facilitating the technical transition from transient protein replacement to permanent genetic intervention with minimal off-target toxicity
• Strategic Engineering for in vivo CAR-T Programming: Development of LNP formulations specifically optimized to maximize T-cell delivery efficiency, enabling precise in vivo CAR-T cell programming through systemic mRNA delivery

• Advancing non-viral CAR-NK platforms: Development of mRNA and circRNA-based CAR-NK systems enabling rapid, scalable, and safer manufacturing compared to viral approaches
• Overcoming key barriers in solid tumors: Strategies to address antigen shedding (e.g., MSLN), CAR downregulation, and TME-mediated suppression (including TGF-Beta resistance and metabolic reprogramming)
• Expanding therapeutic scope beyond oncology: Application of engineered NK cells in non-malignant diseases, highlighting next-generation targets and translational opportunities

This networking session is your opportunity to get face-to-face with many of the brightest minds working in the cell, gene and mRNA fields and establish meaningful business relationships.

• Introducing Quantum Engine for non-viral gene therapy
• Describing the benefits of dual-targeting CD19/CD20 T(SCM) cells to overcome antigen escape and improve response durability
• Presenting preliminary findings from a Phase I open-label trial of GF-CART01 for relapsed or refractory B-cell hematologic cancers

• Developing a dual-target CAR-T therapy for AML aimed at overcoming antigen heterogeneity and antigen escape–driven relapse
• Sharing insights about GPC3-targeted, multi-modal CAR-T therapy for hepatocellular carcinoma (HCC) to overcome the immunosuppressive tumor microenvironment, enhance T-cell persistence and infiltration, recruit endogenous bystander T cells

• Overview of RNA editing based on trans-splicing ribozyme
• Presenting cancer gene therapy
• Showcasing gene therapy for genetic disorders

• Presenting the NNP Trinity platform, a structure-prediction-driven approach that combines an NSM membrane engineering scaffold, AI-guided design of protein assembly, and a validated 3D bioreactor process for scalable, clinical-grade production of cell-derived natural nanoparticles
• Exploring the B-NNP brain shuttle, a dock-and-transcytose dual-targeting design in which Transferrin Receptor 1 drives receptor-mediated transcytosis across the blood-brain barrier while a second motif anchors the particle to a diseased-brain glycoprotein, enabling deep parenchymal delivery rather than perivascular accumulation
• Outlining applied oligonucleotide delivery with in vivo data on ASO and siRNA cargo loaded into B-NNP, including amyloid-beta reduction in a 5XFAD Alzheimer’s model after systemic IV dosing, with extension to additional CNS targets