8:00 am Check-In & Coffee
Preclinical & Translational
Workshop A
9:00 am Deploying Assays to Demonstrate Effective Cell Depletion & Immunosuppression
Synopsis
Assays are critical for demonstrating early efficacy prior to clinical entry. With uncertainty of requirements across cell therapies and how best to measure cell depletion and immune suppression, this workshop will explore the use of cytokine release profiles and targeted biomarker assays, offering insights into selecting the right approach and outlining requirements for approval.
- Discuss contrasting evidentiary hurdles for immunosuppression and cell depletion to streamline preclinical data packages ahead of approval
- Highlight strategies to demonstrate Treg functionality, considering optimized release assays or assay combinations
- Explore suppression assays and cytokine release profiles to evaluate their role as translational efficacy markers
- Utilize targeted biomarker assays to effectively detect cell depletion and immunosuppression signatures
Clinical
Workshop B
9:00 am Designing Clinical Trials in Alignment with Efficient BLA Approval to Accelerate Therapeutic Development & Therapeutic Access
Synopsis
It is key that autoimmune patients get their therapies as fast as possible, especially those with rare autoimmune conditions that have poor prognosis. This workshop will discuss strategies to design clinical trials with BLA regulatory approval in mind, ensuring you can create an effective therapy and accelerate regulatory approval.
- Evaluate IND timeline strategies to build towards a successful BLA approval
- Consider patient population, number and long-term followup requirements to meet FDA standards, to demonstrate durability
- Address key safety considerations and expectations for BLA approval
- Highlight strategies to validate treatment mechanism of action using cytokines and T-cells
- Discuss strategies for evidencing deep tissue depletion, considering biopsy sites in the tissues, bone marrow, lymph nodes and the impact on patient engagement
12:00 pm Lunch & Networking Break
Workshop C
1:00 pm Setting Up for IND Success to Streamline Preclinical & Translational Progression
Synopsis
As the regulatory bar for autoimmune cell therapies continues to rise, it is essential to align your preclinical strategies with evolving FDA expectations. This workshop will explore how to effectively set up your laboratory and generate the right data to support IND submission.
- Outline the essential preclinical data packages and study types needed to support a successful IND submission
- Address challenges in data collection, imaging, and assay development to ensure effective translation from lab to clinic
- Evaluate the opportunity to shift away from mouse models and its implications for therapeutic development and regulatory confidence
- Identify current gaps in IND-enabling studies and strategies to support efficient regulatory progression
Workshop D
1:00 pm Navigating IIT & EMA Regulatory Pathways to Accelerate Cell Therapy Global Development
Synopsis
With mounting FDA regulatory hurdles and a move towards clinical trials outside the US, this workshop explores IIT and EMA development and clinical trial pathways to evaluate the optimal location for therapeutic development and clinical trial execution.
- Compare regulatory and development challenges for the FDA and EMA, to effectively evaluate the optimal agency to initiate clinical trials
- Explore donor and leukapheresis material regulations across the FDA and EMA to ensure compliant material sourcing
- Decode the EMA and IIT pathways to master cross-border compliance
- Leverage the IIT pathway to accelerate development timelines, maintain capital, and generate reliable clinical data
- Investigate cross-border collaboration strategies to streamline clinical developmentÂ