Day One

• Distilling the past year’s clinical and translational progress into portfolio-relevant proof points, sharpening investment, and resource allocation
• Prioritizing indications and modalities where in vivo approaches add differentiated value alongside existing assets, focusing pipeline sequencing
• Aligning global development routes, data portability, and CMC readiness with near-term regulatory and commercial realities, accelerating time to value

• Engineering myeloid, NK, and CAR-T programs with cell specific activity, enabling  precise immune cell programming and broader therapeutic reach across indications
• Implementing repeatable RNA-LNP delivery to achieve deep lymph node depletion and immune reset, supporting translation of autoimmune concepts into first-in-human readiness
• Combining CAR-T and myeloid programming with emerging targeted LNPs and stable genomic integration systems, strengthening future strategies for solid tumor control and long-term clinical durability

• NHP data demonstrating the potential of targeted mRNA LNPs to enable in vivo immunotherapy for autoimmune disease models
• Robust B-cell depletion across peripheral blood, spleen, and lymphoid tissues using a proprietary targeted LNP delivery platform
• Evidence of immune reset biology with emerging safety and tolerability supporting continued preclinical development

Highlighting the development of a new in vivo immune cell engineering modality for cancer immunotherapy, delivering differentiated therapeutic potential

Designing first-in-human clinical trial strategies for this novel approach, accelerating proof-of-concept while ensuring patient safety

Outlining future pipeline ambitions and long-term goals, driving sustained innovation and clinical impact 

• T cell-specific delivery and genomic site-specific genome editing enables potent and safe in vivo CAR-T cell generation 
• Precise genomic integration of the CAR behind the endogenous TRAC promoter enables homogenous CAR-T cell generation with enhanced potency from native regulation
• In vivo CAR-T cell efficacy and tolerability across numerous clinically relevant models support progression toward first in human readiness

• CD3scFv-engineered AAV capsids enable T-cell-specific targeting and activation of resting T cells in vivo, delivering CAR constructs via a single IV injection — bypassing leukapheresis, ex vivo manufacturing, and lymphodepletion
• Preclinical data in Raji lymphoma NSG mouse models demonstrate rapid, potent CAR-T expansion and complete tumor eradication, with superior safety from nonintegrating, episomal AAV persistence
• Platform expansion into autoimmune diseases, powered by proprietary Safety Shield (miRNA-based mRNA control) and BAC-to-AAV manufacturing technologies, positions AAVivo for broader, more accessible cell therapies

• How venture, government-backed and alternative funders are assessing the opportunity in in vivo cell and gene therapy, and where they see the strongest long-term value
• What is shaping funder conviction today, from early translational signals to platform potential and the pathway to clinical and commercial validation
• How funder perspectives on risk, timelines and capital deployment are evolving as in vivo approaches move closer to broader clinical and commercial reality

• Reviewing Capstan’s clinical milestones and acquisition to uncover lessons shaping next-generation strategies
• Highlighting pivotal breakthroughs and remaining challenges to accelerate progress in in vivo gene editing
• Exploring future opportunities and emerging technologies to drive innovation and maximize patient impact

• Assessing how investors and pharma teams are integrating in vivo approaches into evolving pipeline and portfolio strategies
• Identifying the evidence and value inflection points shaping investment decisions for in vivo cell and gene therapy programs
• Aligning milestones, data packages and deal structures with shifting search, evaluation and investment priorities across the sector

Unwind after the day and meet fellow in vivo developers who are actively exploring collaborations. Whether you’re pairing novel gene editing tools with new delivery approaches or seeking new partnerships, this relaxed mixer creates space for conversations and connections that drive real in vivo progress.