Pre-Conference Focus Day

24th January, 2023

Next Generation Nucleic Acid Therapeutics (Beyond mRNA)

The understanding of disease and the accuracy of nucleic acid medicine to resolve complicated health problems is crucial. A comprehensive and profound knowledge of biology will allow the finest variety of therapeutic targets or a combination therapy for the industry. Vast proficiency in several nucleic therapeutic methods and delivery approaches let the field to produce effective nucleic acid drug formats and the most efficient delivery methods to treat many different diseases.

The Recent Outlook of Nucleic Acid-Based Therapeutics

9:00 am Discussing the Current & Future Landscape of Nucleic Acid-based Therapeutics

Synopsis

  • Examining platform nanotechnologies, both viral and non-viral
  • Exploring ex-vivo and in vivo approaches, along with targeting extrahepatic tissues
  • Overcoming common challenges within the field

9:30 am The Evolution of the Adult Vaccine Market: A Holistic Perspective

  • Charles Jones Senior Director, mRNA Commercial Strategy & Innovation, Pfizer

Synopsis

  • Showcasing a unique perspective of the adult vaccines across many disease categories with >20 launches expected in the next five years
  • Understanding how driven by the rapid development of innovative scientific platforms such as RNA, could bring 100+ new products across 30+ disease areas
  • Debating how the expansion of products will create increased complexity across all stakeholders, which will be explored in more detail using the US as a case study

10:00 am Employing Digital Tools for RNA Vaccine & Therapeutic Production

Synopsis

  • Understanding RNA platform technology and its transformative potential for the flexible and rapid production of high-quality and low-cost vaccines
  • Realising how analytical technologies and computer modeling techniques are applied to accelerate the development and mass production of RNA vaccines and therapeutics
  • Levergaing digital twins and soft sensors for real-time monitoring and process control
  • Presenting the “pre-qualification” concept for expediting development and regulatory approval, based on the platform nature of both the RNA vaccine manufacturing process and of the Quality by Digital Design (QbDD) framework
  • An exclusive look at a dashboard for big data analysis and visualisation

10:30 am
Morning Networking Break

The Next Generation of RNA Vaccines: Understanding Self-Amplifying RNA & Small Interfering RNA

11:00 am Assessing Next Generation Self-Replicating RNA Vectors to Enable New Approaches to Immunotherapy

Synopsis

  • Understanding how next generation synthetic srRNA vectors can lower dose by orders of magnitude compared to current srRNA designs
  • Developing new srRNA products requires independent optimisation of vectors, gene inserts, and delivery
  • Improving srRNA bioactivity to enable new approaches for immunotherapies in oncology
  • Following these platform improvements, Replicate Biosciences have designed a srRNA vaccine targeting resistance mutations in endocrine receptor positive breast cancer

11:30 am Leveraging Small Activating RNA: From Concept to Phase II Clinical Trials

  • Nagy Habib Head of Surgery Hammersmith Campus , Imperial College London

Synopsis

  • Exploring the development of RNA activation to target tumour associated macrophages
  • Harnessing the upregulation of transcription factors genes to the bone marrow
  • Outlining the administration of RNA activation drug in over 130 patients with advanced cancer
  • Showcasing safety profile and clinical outcomes

12:00 pm Utilising a Non-Lipid Non-Viral Self-Amplifying RNA Delivery Technology for Therapeutics & Vaccines

Synopsis

  • Introducing the ChaESAR technology, a next-generation approach for RNA delivery
  • Leveraging ChaESAR for superior drug design
  • Understanding how an easy production system is geared for rapid scale up and is an easy fit with existing supply chain paradigms for distribution globally

12:30 pm
Networking Lunch Break

Evaluating the Delivery of Next-Generation Nucleic Acid Vaccines & Therapeutics

1:30 pm Leveraging Polymeric Nanoparticles for the Delivery of Next-Generation mRNA Vaccines & Therapeutics

Synopsis

  • Examining the polymeric nanoparticle delivery platform
  • Reviewing an innovative concept in vaccine development, thermostable vaccines
  • Evaluating the delivery of next-generation nucleic acid vaccines and therapeutics

2:00 pm Discovering Oligonucleotide Senotheraputics for the Diseases of Ageing

  • Lorna Harries Chief Scientific Officer - Research & Development Lead, Senisca

Synopsis

  • Taking a deeper look at how RNA processing, a major component of molecular stress resilience which is a major driver of cellular senescence in multiple human tissue types
  • Identifying the points of intervention that can be targeted for restoration of correct mRNA processing
  • Examining a designed portfolio of oligonucleotide interventions exploiting these auto-regulatory loops to restore regulated splicing factor expression in senescent primary human cells and demonstrated that these can ameliorate multiple aspects of the senescent cell phenotype and attenuate markers of disease phenotypes
  • Addressing dysregulated mRNA processing in senescence provides an opportunity to target the causes of age-related disease at their roots, rather than their consequences

2:30 pm Exploring Novel Tools for Intracellular Delivery of RNA Therapeutics: From Small Molecules to Proteins

Synopsis

  • Repurposing of cationic amphiphilic drugs (CADs) to promote cytosolic delivery of small nucleic acid therapeutics (siRNAs, ASOs)
  • Exploiting CADs as structural and functional components of lipid nanoparticles (LNPs) for cytosolic delivery of mRNA
  • Applying lung surfactant proteins as endogenous and biocompatible enhancers of intracellular RNA delivery

3:00 pm
Afternoon Networking Break

Navigating Advances in Oligonucleotide Drug Delivery

3:30 pm Leveraging Bioengineering Approaches for Non-viral Delivery of Oligonucleotides

Synopsis

  • Examining nanoparticles for oligonucleotide delivery
  • Discussing hydrogel and scaffolds for oligonucleotide delivery
  • Debating the advantages and challenges of bioengineering methods for oligonucleotide release

4:00 pm Harnessing Next Generation LNA Antisense Oligonucleotide (ASOs) Platform, LNAplus™

Synopsis

  • Exploring rapid generation of high-quality development candidates – six months from idea to candidate
  • Parallel evaluation of efficacy and safety – starting at the in-silico stage
  • Overview of successful partnerships across therapeutic areas
  • Presenting examples from the diversified proprietary pre-clinical pipeline for indications of oncology and inflammatory and fibrotic diseases of the kidney and liver

4:30 pm
End of Pre-Conference Focus Day