Day One

Accelerate Clinical Impact with Smarter Trial Design, Early Apheresis & Patient-Centric Strategies

This track is tailored for leaders in: Clinical, Translation, Medical

• Showcasing autoimmune clinical data achieved without lymphodepletion to demonstrate durable responses while improving patient safety and tolerability
• Sharing evolving PK profiles to illustrate sustained persistence and strengthen confidence in non-conditioning approaches
• Evaluating safety outcomes and patient engagement benefits to positioning nonconditioning approaches as an accessible and scalable therapeutic strategy

• Identify emerging biological targets that unlock new treatment possibilities for complex autoimmune diseases
• Explore multi-target therapeutic approaches that address disease complexity and improve response rates
• Evaluate how to match the right therapeutic modality to the right disease to optimize efficacy, safety, and long-term patient benefit

• Comparing PK profiles in autoimmune diseases and hematological malignancies to uncover differences in exposure, persistence, and dosing strategies
• Highlighting how endpoints, patient selection, and lymphodepletion approaches differ in lupus and MS
• Exploring clinical safety data to enhance autologous CAR-T expansions beyond oncology

Private Lunch Hosted by PBS Biotech

Enjoy lunch as you spark new discussions, build relationships that can drive impact throughout the summit.

• Summarizing trends in recent CGT FDA decisions
• Sharing new tools and platform technologies of interest at the FDA
• Diving into FDA trends and future direction

• Detailing the transition process between FDA, EMA and IIT clinical programs to streamline clinical development across borders
• Discussing strategies for incorporating early clinical readouts from international studies to enable faster decision making before US trial initiation
• Evaluating evolving US regulatory comfort with globally generated data to showcase how aligned global development strategies can accelerate innovation while maintaining regulatory rigor

• Expand clinical momentum by leveraging the IIT pathway to generate early human data across BCMA in vivo CAR and CD19 CAR-T
• Utilizing clinical insights to inform development strategy
• Sharing practical learnings from IND and IIT discussions to navigate regulatory engagement effectively

Build Phase 1 Processes with Speed, Flexibility & a Clear Path to Commercialization

This track is tailored for innovators in: Analytical Development, Process Development, MSAT

• Providing strategies to effectively isolate key starting materials to enhance product yield
• Assessing cell phenotype and fitness to understand how starting material quality impacts manufacturing success
• Applying engineering and process innovations to improve low quality autologous starting material and enable reliable production for clinical supply

• Addressing the in vivo CAR-T tradeoff between durable CAR expression and safety
• Using data from primary human T cells and in vivo models to explore mRNA expression
• Exploring how cap structures, UTR design, and poly(A) tail engineering can deliver what in vivo CAR-T demands: not peak height, but duration

• Moving starting material characterization upstream in the CGT workflow – Immunophenotyping of leukapheresis starting material at the point of collection provides earlier insights into leukopak composition and immune subset variability, helping developers better understand manufacturing inputs, establish acceptance criteria, and support more consistent and predictable cell therapy manufacturing outcomes
• Standardization across decentralized collection networks – Perspectives from leading blood centers participating in a multicenter immunophenotyping study focused on reproducibility, harmonization, and scalable collection and streamlined characterization workflows
• Delivering more actionable starting material data to developers – How characterized leukapheresis products and standardized QC approaches can support manufacturing decisions, reduce variability, and strengthen developer–collector partnerships

Private Lunch Hosted by PBS Biotech

Enjoy lunch as you spark new discussions, build relationships that can drive impact throughout the summit.

• Incorporating complex allogeneic edits as cell line development to reduce additional editing cycles
• Refining scale up readiness to accelerate timelines while protecting product quality and regulatory compliance
• Aligning with regulators on GMP editing component guidelines

• Introducing scientists to strategies for scalable pluripotent and/or mesenchymal stem cell culture, with a focus on how GMP-grade reagents that align with USP<1043> can streamline translation into bioprocessing and clinical manufacturing
• Examining key considerations, in addition to potency and performance, in selecting and qualifying ancillary materials to minimize redevelopment timelines and accelerate regulatory readiness
• Understanding how coupling GMP-ready platforms with compliant reagents drives robust innovation and assures a smooth path to recognize its clinical and therefore commercial values

• Exploring how to match functional and safety assays with regulatory requirements to ensure robust testing strategies
• Evaluating the use of quality testing for virus replication in cell therapy products despite minimal risk to balance regulatory compliance and assay costs
• Sharing insights on balancing regulatory compliance with scientific rationale to reduce unnecessary testing while maintaining patient safety

• Leveraging real-time monitoring to enable tumor rechallenge experiments and deeper insight into CAR-T cytotoxic durability and functional exhaustion
• Identifying clinical correlates for preclinical assay results to ensure streamlined Phase I clinical testing
• Applying dynamic assay data to better characterize the cytotoxic profile of final CAR-T products and support confident preclinical-to–Phase I translation

Scale Up, Reduce Costs & Ensure Quality in Phase 2 & Beyond

This track is tailored for specialists in: Manufacturing, CMC, QC, MSAT 

• Working with development, technical leaders to identify scale-up limitations to anticipate risk and guide late-stage process development
• Optimizing engineering, and process integration strategies to reliably build and reproduce complex products at scale
• Defining metrics and success criteria to measure scale-up readiness, ensure product consistency, and support commercial confidence

• Commercializing cell and gene therapies (CGTs) requires overcoming CMC bottlenecks, scaling complex autologous processes, and ensuring consistent batch performance from late-stage development through commercial supply.
• Minaris supports this transition with proven end‑to‑end manufacturing, regulatory readiness, and global tech transfer expertise—demonstrated through approved therapies like AMTAGVI® and commercial partnerships such as LYFGENIA®, achieving >95% success rates with a “Right First Time” philosophy.
• Outlining practical strategies for process validation, comparability, supply reliability, and scalable cell therapy manufacturing to enable sustainable commercialization and broader global patient access

• Leveraging bioreactors to achieve commercial scale
• Comparing with alternative scale-up methods to identify trade-offs in efficiency, control and product quality
• Analyzing cell phenotype post-scale-up to understand the impact of use on product quality

• Case study: successful IND amendment clearance based on comparability performance from the Cellares Cell Shuttle
• How to apply phase-appropriate comparability approaches based on clinical experience and product knowledge
• Leverage automation to reduce variability, strengthen process understanding, and enable scalable, lifecycle-ready manufacturing

Private Lunch Hosted by PBS Biotech

Enjoy lunch as you spark new discussions, build relationships that can drive impact throughout the summit.

• Building robust, well-understood processes early to enable reliable and efficient scale-up
• Anticipating technical, cost, and operational constraints that may hinder translation from clinical manufacturing to commercial manufacturing
• Maintaining consistent, adaptable manufacturing and analytical systems from early trials to commercial production

• Proven tech transfer model built for scale – applying a structured, repeatable approach refined across multiple programs to reduce rework and accelerate transition into manufacturing
• Partner-in-Plant program with an integrated training model—accelerating adoption, standardizing best practices, reducing variability and downtime, and building durable in-house capability
• Operationalizing 10x scale without compromising consistency – integrating facility design, process standardization, and team training to increase throughput while maintaining reproducibility and quality

• Debating whether commercial manufacturing should prioritize commercial scalability from day one or focus on the fastest path into the clinic, and how each choice shapes regulatory strategy
• Examining how early process design and analytical development decisions impact potential CMC approval for commercial studies

• Mitigating risks through strategic approaches in scale-up process tech transfer and manufacturing
• Building and managing partnerships with CDMOs for manufacturing success
• Applying scientific methodologies and phase-appropriate governance across development stages.