Cellular immunotherapies: targeting new frontiers

• As momentum builds for in vivo CAR-T and TCR therapies, particularly using mRNA-LNP delivery platforms, how can the field overcome delivery and technological hurdles to accelerate this paradigm shift?

• How to address persistent manufacturing and logistical challenges limiting the scalability of autologous CAR-T therapies (e.g., through point-of-care manufacture)?

• Tackling key scientific and technical hurdles preventing the successful translation of CAR-T therapies into solid tumor indications.

• How can developers clearly differentiate their cellular immunotherapy products and platforms in terms of efficacy, safety, and scalability?

• How can the field navigate the challenges of moving beyond CD19 and BCMA to secure approvals for novel targets and indications (e.g., autoimmune diseases)?

• How is the field evolving to mitigate the most pressing safety concerns
  (e.g., insertional mutagenesis, cytokine
  release syndrome) and predict patient
  response?

• What are the barriers to advancing NK and macrophage-based therapies, and how is renewed interest in MSCs shaping
  development strategies?

Gene therapy analytics/CMC

• Improving vector characterization and QC readiness to ensure scalability and minimize risk of product release delays. 

• How can in vivo functional assays be used to demonstrate that a therapy can deliver the intended gene and produce a therapeutic effect? 

• Assessing the integration of process analytical technologies (PAT) (e.g., mass spectrometry, MALS) to provide real-time quantitative data, ensuring vector quality and consistency.  

• How can novel digital tools such as multiomics, NGS, and real-time release testing be successfully incorporated into a validated, regulatory-compliant framework? 

• What are the risks in deferring analytical strategy until late in development, and how are experienced teams embedding CMC thinking from the earliest stages? 

• Identifying assay lifecycle readiness—how can we know how robust, transferable, and automation-compatible our methods are? 
  
  
  
  
  
  
  
  
  
  
  
  
  

Induced pluripotent stem cells (iPSCs)

• How to design effective pivotal trials and demonstrate efficacy for iPSC-derived therapies targeting complex diseases like Type 1 diabetes and Parkinson’s? 

• What are the major technical and operational barriers to scaling iPSC manufacturing to commercial scale, and how can automation and process standardization help close the gap between clinical promise and commercial feasibility? 

• How are safety characterization technologies for iPSCs (e.g., karyotyping, tumorigenicity testing, whole genome sequencing, pluripotency assays, flow cytometry, targeted mutational analysis, imaging techniques) being integrated into QA/QC workflows? 

• How are iPSCs advancing drug discovery and deepening our understanding of disease mechanisms through predictive in vitro models? 

• How can the field ensure the long-term genetic stability of allogeneic iPSC lines? 

• How are developers interpreting evolving regulatory expectations for PSC starting material characterization? 
  
  
  
  
  
  

Cell therapy manufacturing
and supply chain  

• Interrogating selection and acquisition of cellular starting materials and critical raw/ancillary materials–how can consistency, quality, and supply be ensured across autologous and allogeneic supply chains? 

• Streamlining autologous patient pathways: reducing logistical and clinical complexity to improve patient access, including safer conditioning regimens and minimizing off-target effects. 

• Enabling decentralized and point-of-care manufacturing: exploring the infrastructure, regulatory harmonization, and workforce training needed to scale CAR-T and other autologous therapies closer to the patient. 

• Enhancing upstream processing with intelligent bioreactors: how are developers leveraging smart bioreactor technologies to improve process control, scalability, and reproducibility in cell expansion? 

• Addressing the growing demand to adopt closed, automated, platform-based manufacturing systems to improve scalability, consistency, and cost-efficiency. 

• How can we overcome the challenges of isolating high-purity cell populations in downstream processing while maintaining viability and functionality? 

Non-viral delivery:
making a real-world impact 

• Overcoming plateauing momentum as non-viral delivery technologies transition from hype to the hard realities of clinical translation.

• Addressing key bottlenecks in non-viral delivery—transfection efficiency, stability, cellular uptake, immunogenicity, targeting, and long-term integration. 

• Getting beyond the liver: how can the field overcome the persistent challenge of achieving cell-type-specific tropism in extrahepatic tissues with non-viral vectors?  

• Improving LNP stability, targeting, and safety to help enable delivery of complex payloads. 

• Expanding the synthetic toolkit: what is the translational potential of emerging platforms (e.g., mechanoporation, electroporation, synthetic DNA vectors, gene circuits, carbon fiber particles, hybrid micelles, EVs/exosomes)? 

• What in vivo delivery innovations are needed to expand the therapeutic reach of gene editing and CAR-T cell therapy? 

• How can developers, regulators, and solution providers collaborate to build fit-for-purpose evaluation frameworks for novel delivery systems? 
  
  
  
  

AI & digital transformation in CGT 

• How can AI be effectively integrated into CGT translational R&D, manufacturing, and analytics, particularly considering the challenges in updating legacy systems? 

• How to build high-quality datasets and data infrastructure for AI/ML applications in CGT? 

• How and where specifically should AI be applied in nonclinical and clinical development? 

• How can AI integration in CGT manufacturing overcome challenges in real-time process control, digital batch record generation, and predictive maintenance to enable adaptive, intelligent production systems? 

• How can blockchain and digital QA/QC tools, including multiomics analysis, improve traceability and compliance (e.g., in real-time release testing)? 

• What are the regulatory considerations for employing AI-driven tools in CGT workflows? 
  
  
  
  
  
  
  
  
  
  
  
  
  

Gene editing: making the cut   

• How can the personalized CRISPR-based medicine field build on early clinical promise? 

• Assessing key delivery challenges for in vivo gene editing: how are technologies like LNPs and electroporation evolving to meet them? 

• Optimizing process and analytical workflows for ex vivo gene-edited therapies 

• As in vivo applications accelerate, how can developers safeguard against unintended edits, chromosomal rearrangements, and long-term genomic instability while improving precision and minimizing immunogenicity? 

• Building a robust evidence base to support clinical translation of editing tools beyond CRISPR (e.g., base editing, prime editing, epigenome editing)? 

• How are AI-driven design and multiomics helping to refine CRISPR variants and evaluate the functional impact of emerging editing platforms? 

• How should the field respond to growing ethical, societal, and regulatory concerns around human heritable genome editing (HHGE)? 
  
  
  
  
  
  
  
  

Manufacturing scale-up/
scale-out and automation

• Exploring scalable, automated solutions to make cell and gene therapies more accessible and affordable without compromising quality or safety. 

• Clarifying what constitutes a ‘platform’ and how standardized tools (e.g., modular CAR-T production frameworks, AAV vector backbones) can streamline development and reduce complexity. 

• Tackling the cost, infrastructure, and donor variability challenges in scaling out autologous workflows and scaling up allogeneic platforms. 

• Supporting commercial readiness: enabling seamless end-to-end automation by integrating upstream and downstream systems with real-time analytics and controls. 

• Developing robust, flexible supply networks to support international commercialization. 

• Innovating sustainable models for low-volume, high-cost therapies where traditional manufacturing approaches are economically unviable. 

• Building tomorrow’s CGT workforce: addressing the talent gap in CGT manufacturing through training, cross-sector collaboration, and technology transfer from academia to industry.  

Cell therapy analytics/CMC 

• How can in-line analytical monitoring systems and PAT (e.g., Raman spectroscopy, microfluidics-based QC), enable real-time release testing and efficient batch management? 

• Investigating the development of robust, phase-appropriate assays to address the inherent heterogeneity of autologous products and ensure comparability across lots and sites, including multiplexing assays. 

• Leveraging NGS to supplement potency and identity assays (e.g., where orthogonal methods are limited or inconclusive). 

• Streamlining analytical workflows at scale: overcoming bottlenecks in sterility testing and cell monitoring to support high-throughput, GMP-compliant manufacturing (e.g., through biocalorimetry and cartridge-based flow cytometry). 

• How can we realize the potential for AI-powered multiomics analytics to integrate genomic/ transcriptomic/ proteomic/epigenomic data, and link product attributes directly to clinical outcomes? 

• Assessing in vivo versus in vitro assays: how should developers go about selecting the right assay for their use case? 

• Measuring real-world therapeutic durability: harnessing long-term patient outcomes data to help meet evolving safety, efficacy, and economic demands. 

Viral vector manufacturing & platform evolution    

• How are developers working to reduce variability and establish scalable, reproducible manufacturing platforms for both AAV and lentivirus? 

• Assessing ongoing safety challenges in AAV (e.g., immunogenicity, genome integration, long-term persistence).  

• Interrogating why upstream and downstream manufacturing struggles (yield, impurity removal, empty capsid clearance) continue to limit AAV production scalability. 

• Breaking through pre-existing immunity and targeting limitations: how is the field addressing the challenge of pre-existing antibodies and the need for reliable extrahepatic targeting to expand therapeutic reach? 

• How will lentiviral vectors compete with emerging non-viral in vivo delivery platforms in the face of high manufacturing costs, limited transduction efficiency, and the risk of random integration?  

• How can next-generation engineered capsids enable targeted delivery to tissues like muscle and the CNS?

• What bioreactors and other technologies are needed to enable efficient, high-volume vector production?