Please Note: Times Are Listed As ET

8:00 am Pre-Conference Virtual Networking

8:50 am Chair’s Opening Remarks

  • Jeffry Lawrence Interim Vice President, Clinical Development, Solid Biosciences

Showcasing the Diversity of Novel Drug Modalities with New Mechanisms to Regulate the Complement System

9:00 am Next-Gen Complement Therapeutics: Opportunities for Indication Expansion and Tissue Targeted Immunomodulation

  • Michael Holers Professor of Medicine & Immunology, University of Colorado School of Medicine

Synopsis

  • Complement can play unique context- and stage-specific roles in the natural history of autoimmune diseases such as rheumatoid arthritis
  • First generation systemic complement inhibitors retain certain limitations with regards to PK/PD relationships and potential adverse events
  • Developing methods to more precisely target inhibition of complement to sites of pathway activation has substantial potential benefit

9:30 am The Potential for Tissue Targeted Complement Inhibition: Generation of a Novel Therapeutic Platform

  • Shelia M. Violette Co-Founder, Chief Scientific Officer & President of Research, Q32 Bio

Synopsis

  • Targeted delivery of negative regulatory proteins potently inhibits complement activation and injury in diseased tissue without systemic blockade
  • Q32 Bio tissue targeted complement therapeutics biodistribute to diseased tissues and provide long-term durable tissue PK/PD and efficacy
  • Q32 Bio tissue targeted complement therapeutics have the potential to be used for a wide range of disease indications in which complement activation is implicated and systemic targeting is not feasible or desirable

10:00 am Recombinant Truncated Human CR1 as a Novel Complement Inhibitor

  • Sandra Wymann Senior Manager, Translational Research, CSL Behring AG

Synopsis

  • Truncated recombinant CR1 shows improved potency compared to fulllength soluble CR1 (in vitro and in vivo)
  • In vitro sialylation of truncated recombinant CR1 shows improved PK and PD properties
  • Truncated recombinant CR1 shows efficacy in disease animal models

10:30 am Virtual Speed Networking & Break

11:30 am Engineering Complement Factor I as a Protease Medicine: Replacement Therapy and Tuning Potency and Specificity for Complement-Mediated Disorders

  • Grant Blouse Senior Vice President, Translational Research, Catalyst Biosciences

Synopsis

  • Overcoming limitations of small molecule and antibody approaches
  • Describing mechanisms by which therapeutic proteases regulate the complement system
  • Discussing the impact of extended half-life on infrequent drug administration

12:00 pm Cascade Biotechnology: Novel Therapeutic Opportunities Using Complement Depletion

Synopsis

  • Cascade’s patented protein, sC3, completely removes active C3 from the circulation
  • sC3 can be delivered subcutaneously, intravenously, topically or in a viral vector
  • sC3 is in development for a variety of indications involving overactivity of the complement system, in the circulatory and retinal compartments

12:30 pm Upstream Complement Inhibition in Peripheral Auto- Immune Neuromuscular Neuropathies

Synopsis

  • Inhibition of lectin and classical pathway shows promise in preclinical models
  • Pathophysiology not driven by terminal pathway
  • ARGX-117 is a potent and long-acting inhibitor of C2

1:00 pm Lunch Networking Break

2:00 pm siRNA Targeting Complement Protein 3 (C3) for Treatment of Patients with C3 Glomerulopathy (C3G)

Synopsis

  • Complement proteins are often exclusively produced by hepatocytes and therefore ideal targets for GalNAc siRNA
  • Blocking the complement cascade and its detrimental downstream effects at C3 level is a promising therapeutic strategy
  • Potent GalNAc-conjugated siRNAs targeting C3 have been identified and tested in vitro and in vivo with proof of mechanism achieved pre-clinically

Understanding Complement Dysregulation in COVID-19 & Key Learnings for Managing Complement Inhibition in Long-COVID

2:30 pm Complement Inhibition in the Setting of COVID-19

Synopsis

  • Complement dysregulation in COVID-19
  • Clinical data on complement inhibition in COVID-19
  • Key scientific questions for R&D

3:30 pm Clinical Impacts of Controlling Dysregulation of the Terminal Complement Pathway and LTB4

Synopsis

  • Role of complement dysregulation in a wide range of diseases
  • Learnings from COVID-19
  • The future role of complement and LTB4 inhibition in chronic diseases

4:40 pm End of Complement-based Drug Development Summit

5:00 pm Chair’s Closing Remarks

  • Jeffry Lawrence Interim Vice President, Clinical Development, Solid Biosciences