Conference Day Two

Thursday, May 23, 2024

8:00 am Check-In & Light Breakfast

8:55 am Chair’s Opening Remarks

9:00 am Navigating the Complexities of Disease Indication Selection for Treg Therapies


  • Acknowledging the unique challenges faced by smaller biotech companies in making strategic decisions when selecting particular disease indications
  • Discussing the implications of disease indication selection on the success of Treg therapies and how this may impact development decisions
  • Offering strategic considerations and decision-making frameworks for smaller biotech’s to capitalize on market opportunity

9:30 am Revolutionizing TNFR2 Agonism for Autoimmunity & CNS Diseases: Creation of High Potency Antibodies with Tiny Concentrations

  • Denise Faustman Director of Immunobiology, Associate Professor of Medicine, Massachusetts General Hospital, Harvard Medical School


  • The design of agonistic antibodies to surface receptors of the TNF family have been challenging to make and dose correctly in human clinical trials
  • Our group has focused on single cell surface hexagonal networks to form the tight clustering for potent intracellular signaling, such as for TNFR2 agonism instead of bridging ADCC mechanisms
  • TNFR2 agonist antibodies with high potency and validity across broad dose responses demonstrate the validity of single cell hexagonal clustering

10:00 am Morning Refreshments & Speed Networking

Cell-Based Approaches

Exploring the Next Steps for Cell-Based Therapies to Address Regulatory Concerns

10:30 am Roundtable Discussion: Unlocking the Full Potential of Treg Cells: Beyond Therapeutic Frontiers


  • As Treg therapies continue to demonstrate their efficacy in autoimmune and inflammatory conditions, this session explores the broader landscape of opportunities that Treg cells can provide for diverse therapeutic applications
  • Delve into the untapped potential of Tregs and discuss how their success can be leveraged to shape the future of immunotherapy
  • Explore avenues for expanding the scope of Treg applications, addressing challenges, and envisioning new possibilities that Tregs can yield across various therapeutic domains

11:00 am A look at the Current Treg Landscape for Autoimmune Diseases & Recent Deals and Emerging Companies


  • Reviewing the current landscape of Treg cell therapies for autoimmune disease
  • Discussing the future of Treg cell therapies in autoimmune disease
  • A review of the recent deals and emerging companies in the Treg space

11:30 am Harnessing the Power of Regulatory T cells for Orca-T: a High-Precision Cell Immunotherapy for AML, MDS & ALL


  • Orca Bio is an allogeneic cell therapy company developing high-precision cell immunotherapies for hematological malignancies and autoimmune disorders
  • Orca-T, Orca Bio’s lead investigational product, is a cell therapy that leverages highly purified regulatory T cells to control immune reconstitution. It’s currently being tested in a pivotal Phase 3 study, Precision-T
  • Orca-T is intended to reduce complications and provide better outcomes for patients
  • Given its clinical profile, Orca-T has the potential to deliver curative treatment to an even wider net of AML and MDS patients

Spearheading the Development of Novel Strategies for Targeted Delivery to Meet the Need for Continued Innovation

12:00 pm Development of CAR-Treg Therapies to Modulate Immune Responses in Transplantation & Autoimmunity


  • Engineering approaches to improve Treg function and stability
  • Characterization of CAR-Tregs persistence in vivo

Non-Cell Based Approaches

Unravelling Clinical Insights into Non-Cell Based Approaches Utilizing Novel Modalities for Enhanced Tolerance in Treg Function

10:30 am Applying a Novel CD8 Treg Directed Therapeutic Approach to the Clinic


  • Discussing the significance of regulatory CD8 T cells in autoimmune disease
  • Discussing the therapeutic potential of modulating CD8 Treg for the treatment of autoimmune disease
  • Advancing the CD8 Treg modulator MTX-101 targeting inhibitory KIR as autoimmune checkpoint into the clinic for the treatment of autoimmune disease

11:00 am Inhibitory Receptor Targeting & Modulation of Treg Biology


  • Introducing inhibitor receptors (IRs) and their role in health and diseases
  • Outlining the potential of inhibitory receptors in modulating Treg biology
  • Discussing mechanisms by which targeting IRs can be used to restore tolerance

11:30 am Analyzing Methylation Status of Treg Signature Genes using Targeted Bisulfite NGS & MS-HRM


  • FOXP3 demethylation is a biomarker for Treg identification and stability
  • Targeted-bisulfite NGS is a useful tool for large scale panels for methylation analysis, such as FOXP3 regulators
  • Methylation-Specific High-Resolution Melting as a tool for smaller scale methylation analysis of single amplicons, without reducing the sensitivity achieved by NGS

Exploring Combination Approaches to Treg Therapies to Elevate Therapeutic Efficacy

12:00 pm Rezpegaldesleukin, a Regulatory T-Cell Selective IL-2 Conjugate: Lessons Learned From Preclinical Design to Clinical Evaluation in Inflammatory Skin Diseases Including Atopic Dermatitis


  • Use of the appropriate design elements and experimental models for generation and preclinical evaluation
  • Using clinical development approaches to address target engagement and assess PK/PD principles
  • Using clinical studies to identify the correlation between the MOA and the indication

12:30 pm Lunch Break & Networking

Cell-Based Approaches

1:30 pm Emerging Approaches to Monitoring CAR Tregs in Patients for the Age of Clinical Translation

  • Zinaida Good Cancer Immunotherapy Instructor, Stanford University


  • Approaches to monitoring CAR Tregs in the clinic through correlative studies, with CD19-CAR therapy for cancer as an example
  • Monitoring phenotype and TSDR methylation to assess stability of CAR Tregs in patients
  • Lineage tracing to link the source of ‘unstable’ CAR Tregs to pre-existing impurities or loss of Treg identity

2:00 pm The Future of Small Molecule In Vivo Activated Cell Therapy: Modulation of Tregs in the Body

  • John Martin Professor of Cardiovascular Medicine, UCL & Adjunct Professor of Internal Medicine, Yale University


  • AZD1656 is a specific activator of glucokinase; but not hexokinase (the latter is used by T effector lymphocytes for migration)
  • A randomised blinded placebo-controlled trial of this drug in diabetic patients with Covid 19 significantly decreased death and time in hospital in the treated group. This trial was undertaken by the speaker and his team. New immunology data from this human study will be presented
  • This approach will be tested in several autoimmune diseases

Non-Cell Based Approaches

1:30 pm CSL as a Target for Inducing Immune Tolerance by Modulating Treg Cells & Application in Autoimmune Diseases

  • Raj Lehal Chief Executive Officer , Cellestia Biotech


  • CSL as a target for inducing immune tolerance by modulating Treg cells and application in autoimmune diseases
  • Small molecule (CB-401/CB-433) mediated inhibition of CSL causes an expansion of Treg cells • CB-401/CB-433 induced Tregs maintain their suppressive characteristics
  • Orally active CB-401/CB-433 alleviate autoimmune disease by re-establishing immune tolerance

2:00 pm Targeted Approaches to Balance Treg Function in Inflammation & Cancer


  • Pharmacological intervention to precisely and selectively modulate regulatory T (Treg) cell function is required to restore immune system homeostasis in autoimmune disease and cancer
  • Egle Therapeutics’ platform harnesses the selectivity of modified cytokines (muteins) combined with antibodymediated Treg targeting
  • Preclinical studies highlight how Egle’s multifunctional immunocytokines display novel and unanticipated mechanisms of action, resulting in high in vivo selectivity for disease-specific Tregsn

2:30 pm Afternoon Refreshments

Guaranteeing Success for Strategic Milestones from Start to End: IND Preparation to Clinical Insights in Treg Therapies

3:00 pm Failure of Singular Approaches to Induce Tolerance: Practical Applications to Boost Treg Function

  • Amy Rosenberg Senior Director - Immunology & Consultant, Epivax


  • The need for a multi-faceted approach to enhance Treg therapeutic induction or restoration of immune tolerance
  • Analysing limitations and failures of approaches in achieving immune tolerance in autoimmunity and allergy
  • The potential for synergies between different therapeutic modalities in enhancing Treg efficacy

3:30 pm Roundtable Discussion: Building a Strategic Roadmap to Prepare for IND & Advance to Clinical Phases


  • Preparing for Investigational New Drug (IND) applications and transitioning to clinical phases
  • Emphasizing the importance of strategic planning to ensure successful progression through clinical development
  • Detailing the essential components required for a successful IND application
  • Highlighting regulatory expectations and common pitfalls in the IND submission process

4:00 pm IL-2 Containing Nanoparticles Induce Tregs & TGF-β-producing NK Cell

  • David Horwitz Co-founder, USC Keck School of Medicine, Nanotherapeutics


  • The tolerogenic effect of IL-2 on Tregs is TGF-β dependent
  • TGF-β producing NK cells are essential for Treg maintenance
  • The NPs induce TGF-β locally so that encapsulation of TGF-β is not needed

4:30 pm Chair’s Closing Remarks

4:40 pm Close of the 6th Treg Directed Therapies Summit