Conference Day One
Wednesday, March 8, 2023

7:40 am Morning Registration & Welcome Coffee

8:30 am Program Director’s Opening Address

Synopsis

We’re excited to be welcoming you to the inaugural Targeted Intracellular Delivery Summit!

We look forward to using the next two days to move the needle when it comes to validating complex drug delivery via novel vehicles which overcome complex biological barriers and triumph over efficacy demonstrations.

8:40 am Chair’s Opening Remarks: The Breakthrough in mRNA/DNA LNP Innovations – Low N/P ratio, Nontoxic, and High Transfection Efficiency

Synopsis

Overview of Me-Too LNPs with the conclusion that the Me-Too LNPs do not meet with the needs of rapidly developed genetic vaccines and therapeutics

Share Cureport’s novel strategies of LNP innovations

Compare the key differences between the novel LNP and Me-Too LNPs

Share the in vitro and in vivo data of the novel LNPs

Setting Strategies for Target Tissue Delivery

9:00 am Targeting Specific Cells with Recombinant Vesicles

Synopsis

  • Methods of cell targeting by using affibodies and antibodies
  • Avoiding endosomal degradation for Golgi membrane and cell nucleus localization
  • Opportunities for oral and transdermal delivery

9:30 am INspire – A Novel Intracellular Delivery Platform for Protein-Based Therapeutics

Synopsis

  • The INspire intracellular delivery platform is based on conjugation of proteinbased therapeutic payloads to a chemically modified carrier protein which enables cell internalization and efficient endosomal escape
  • The INspire carrier was shown in vitro to deliver various payloads with differen functionalities and to inflict biological effects in various cancer models
  • The platform exhibited clinically relevant pharmacokinetics and biodistribution profiles as well as in vivo functionality in several oncology models

10:00 am Morning Break & Speed Networking

Synopsis

Kick start your morning by making valuable connections in our speed networkingsession with industry peers working in novel delivery platform design.

10:45 am Rational Design of Dendriplexes Towards Improved Intracellular Delivery of mRNA

Synopsis

  • Rational design of a dendriplex series for intracellular delivery of mRNA
  • Functionalisation of side chains and effects on cellular trafficking characteristics
  • The utility of endosomal escape assays in directing design choice principles

11:15 am Physiologically Targeted Intracellular Delivery using Phosphatidylserine

Synopsis

  • Understand the critical importance phosphatidylserine in efferocytosis and cell membrane fusion
  • Describe how this normal physiologic response to cell injury allows important, context dependent extrahepatic targeting
  • Share recent positive clinical experience and potential future applications of this technology

11:45 am Enabling Extrahepatic Delivery of siRNA with Novel Targeting Ligands

  • Donna Klein Scientific Director, RNA & Targeted Therapies, Janssen Biologics BV

Synopsis

  • Novel targeting ligands for enhanced siRNA delivery
  • CNS delivery of siRNA in neurological diseases

12:15 pm Networking Lunch

Synopsis

Your opportunity to schedule 1-2-1 meetings with your fellow colleagues and our event partners to find out their scientific innovations, technology capabilities, and assay development opportunities!

New to this space? Get in touch here and we can facilitate a conversation for you!

1:30 pm RNA Cell Therapy for Autoimmune Disease

Synopsis

  • RNA Cell Therapy is a new modality with the potential for disease modifying impacts in autoimmune disease
  • The combinatorial power of our RNA Armory platform has the potential to enable precision targeting of cellular therapies
  • The quality control strategy for RNA Cell Therapy leverages best practices for both mRNA therapeutics and conventional cell therapies

The Route Towards Engineering Technologies Mediating Intracellular Delivery

2:00 pm Maximizing Intracellular Delivery by Fusogenic Intrinsic Nanovesicles

Synopsis

  • Fusosome platform can specifically fuse to the surface of target cells
  • Fusosome is a platform technology that facilitates membrane-editing and direct delivery of therapeutic cargos into the target cell cytoplasm
  • We reappraised the idea that extracellular vesicles function as garbage cans and repurposed them as a therapeutic agent

2:30 pm Sapreme’s SPT001 Endosomal Escape Enhancers Enable Targeted, Intracellular Delivery – In Vitro and In Vivo Case Studies

  • Guy Hermans Chief Executive Officer, Sapreme Technologies

Synopsis

  • SPT001 endosomal escape enhancer conjugates efficiently escape the endosome
  • Entry into the target cell endosome is targeting ligand dependent, and SPT001 is selective for the endosomal compartment. Hence, delivery is target cell uptake specific
  • SPT001 is targeting ligand agnostic, and has been shown to deliver both oligonucleotides and proteins. This versatile system therefore allows many different targeted, intracellular delivery applications

3:00 pm Afternoon Break

Synopsis

Explore the exhibition hall to find out more on the opportunities available to leverage delivery platforms for seeing your pipeline achieve its full potential.

3:30 pm Development of Endosomal Escape Vehicles (EEV) to Enhance the Functional Delivery of Oligonucleotides

  • Leo Qian Co-Founder & Vice President, Discovery Research, Entrada Therapeutics

Synopsis

  • The EEV platform consists of a library of proprietary cyclic peptides with unique chemistry that enable improved uptake and endosomal escape
  • EEV-therapeutics can be designed to enhance functional delivery to target tissues
  • EEV-oligonucleotide corrected splicing deficits in preclinical models of neuromuscular diseases

4:00 pm Panel Discussion: Innovating Targeted Intracellular Delivery Vehicles: At the Intersection of Academia & Industry

  • Allen Horhota Senior Director & Head of Delivery, Orna Therapeutics
  • Rui Zhang Associate Director, LNP Formulation, Tessera Therapeutics

Synopsis

  • With resources limited, what is the key to academic and industry collaboration to facilitate innovation at pace?
  • How can better bridges be forged so opportunities aren’t missed to design the next winning vehicle?
  • What forums, consortia and touch points can be leveraged to enable cross-learnings and meaningful collaborations to progress targeted intracellular delivery?
  • Which fundamental questions on trafficking would industry to get answered from academic research?
  • How can results from basic research enter efficiently into pharmaceutical development?

4:30 pm Leveraging LNP’s: Overcoming Limitations Leveraging Industry’s Winning Vehicle

  • Qi-Ying Hu Vice President, Head of Formulation & Delivery, Omega Therapeutics

Synopsis

  • Leveraging LNPs for the delivery of oRNATM molecules to target cells
  • Addressing & overcoming liver accumulation for cell-population targeting
  • Route towards the identification of targeting approaches which enable the design of LNP vehicles with the chemical properties which will then interact with these unique markers / targets

5:00 pm Chair’s Closing Remarks & End of Day One