Day Two - mRNA - Hanson Wade Template 6.0

Explore the Agenda

Focus Day

Monday, July 29

Day One

Tuesday, July 30

Day Two

Wednesday, July 31

8:00 am Morning Check In & Coffee

8:20 am Chair’s Opening Remarks

De-Risking Your Pipelines with Guidance from Regulatory Experts to Realize the Promise of mRNA

8:30 am Discussing the Regulatory Expectations for mRNA-Based Therapeutics & Vaccines Against Infectious Diseases, Cancer & Rare Diseases

What are the regulatory differences between mRNA vaccines and therapeutics targeted to infectious, oncological, or rare indications?
What can the next generation of mRNA vaccines and therapeutics expect as regulatory requirements?
Are there any transferable regulatory learnings from mRNA vaccines and therapeutics for gene editing applications that use mRNA technology,
including CRISPR?

9:00 am TFF in RNA Workflow: Process Optimization for mRNA, saRNA, & LNP Manufacturing

Delta regenerated cellulose flat sheet membranes with 100 kDa molecular weight cutoff for TFF in RNA workflows
TFF optimization for mRNA, saRNA, and LNPs where a single filter can work across modalities
How process conditions can impact LNP processing time
Scalability data from bench (93 cm2 ) to manufacturing-scale (2.5 m2 ) using LNPs, discussing proper scaling factors

9:30 am Panel Discussion: Uniting Regulatory Experts from Industry & Agencies to Navigate the Complex Landscape for mRNA-Based Medicines

How do the FDA, EMA and MHRA define a “platform” and what are the paths available to mRNA drug developers to utilize “platforms” as a way to expedite approvals
Is it the right time to start developing mRNA specific regulatory guidance and should there be separate guidelines for each therapeutic area and/or application?
What tools and expectations apply to companies exploring novel mRNA applications without an established platform or previous experience with large-scale batch production?

Panel Moderator: Emily English

Virtual Speaker: Marco Cavaleri

10:15 am Empowering the mRNA Industry with Nucleic Acid Therapeutics: Innovating for Success

Thermo Fisher Scientific’s extensive capabilities and tailored solutions in nucleic acid therapeutics, specifically for the mRNA industry
Detailed overview of Thermo Fisher Scientific’s ability to deliver highly impactful and effective solutions in this rapidly evolving field
An inspiring real-life example showcasing Thermo Fisher Scientific’s successful support for businesses in the mRNA industry, highlighting their reliability and commitment to progress

10:45 am Morning Networking Break

Discovery Translation Clinical Development CMC

Discovery

Accelerating the Development of mRNA-Based Vaccines & Therapeutics to Fight Infectious & Oncological Diseases

11:15 am Advancing Immunoinformatic Tools for Epitope Mapping to Supercharge Development of mRNA-Based Personalized Cancer Vaccines

Streamlined and automated cancer vaccine design platforms are key to develop on-demand therapeutics
Enhanced antigen selection enables the removal of inhibitory sequences that curtail vaccinemediated immune responses
Computational vaccinology toolki

11:45 am Radar Therapeutics: Pioneering Precision-Expressed mRNA Therapeutics

Overview of Radar’s proprietary RNA sense-and-respond platform for precise expression of mRNA/gene therapeutics
RADAR enables cell-type specific RNA expression by sensing intracellular RNA markers that define the cell type, disease state or cell state of interest to activate translation of a synthetic transcript
RADAR enables precise, autonomous cell-selectivity that can bring specificity to broadly-targeting delivery vectors for DNA and mRNA
Discussing the platform’s applicability to complex diseases

12:15 pm Innovac’s End-To-End Approach to Capitalize on the mRNA Revolution in the Vaccine Industry

Describing Innovac’s platform technologies and pipeline to drive the next wave of therapeutic and prophylactic mRNA vaccines
Selecting and stratifying disease targets and patient populations for mRNA vaccines
Revealing new pre-clinical data from multiple vaccine projects targeting infectious diseases and cancer

Translation

Unlocking in vivo Cell Engineering Through mRNA Therapeutics Aiding the Fight Against Autoimmune & Oncological Diseases

11:15 am Harnessing mRNA Technology to Unlock Cell Engineering Applications Against Solid Tumors

Introducing Factor’s proprietary platform applying mRNA to in vivo cell engineering therapeutics
Transforming CAR-T cells in vivo to target solid tumors
Expressing gene-editing proteins in vivo to turn “cold” tumors into “hot” tumors

11:45 am Implementation of Phase Appropriate mRNA Analytics in a Contract Testing Environment: Balancing the Development & Qualification Approach in a mRNA Program

The analytical requirements for characterization of mRNA Drug Substance and Drug Products have been defined by the USP as well as sponsor companies participating in early phase clinical trials
Balancing the effort spent on method development and qualification/validation is critical to keeping with timelines and controlling analytical costs
How to utilize a suite of mRNA Analytical platform methods to streamline your process development team’s ability to optimize the scale up and manufacturing process
Effective implementation of full cGMP versus process development service analytical offerings in the same lab space

12:15 pm In Vivo Induction of CD19 CAR-T Cells using mRNA as a Therapeutic for Autoimmune Indications

Describing Capstan’s LNP platform capable of extra-hepatic delivery
Targeting the hematopoietic system with tolerable doses of mRNA-LNP
Discussing the pre-clinical CAR-T data in mouse and NHPs using Capstan’s technology

Clinical Development

Key Clinical Case Studies on mRNA-Based Vaccines & Therapeutics Against Rare Diseases

11:15 am Unveiling Moderna’s Latest Efforts to Target Rare Diseases with Protein Replacement Using mRNA Therapeutics

Introducing Moderna’s latest expansions into rare diseases with mRNA protein therapy
Revealing proof of concept from early stage trials against propionic and methylmalonic acidemia, and type 1 glycogen storage disease
Discussing safety data from prolonged exposure to mRNA therapeutics targeted at the above indications

11:45 am Overcoming dsRNA Challenge: Innovation in Assays & Enzymes

Optimization of IVT-PP for dsRNA is challenging
Next-Generation T7: Overall performance enhanced ds-SensorTM: State-of-the-art dsRNA assay

12:15 pm Leveraging the LUNAR® Platform to Develop RNA Medicines for Diseases with Significant Unmet Medical Needs & Accelerated Clinical Paths

Overview of the ARCT-032 program – an mRNA replacement therapy to deliver a new copy of the CFTR mRNA into CF patients’ airways
Overview of the ARCT-810 Program – an mRNA medicine that enables OTC patients to make healthy functional OTC enzymes in their liver cells
ARCT-154 demonstrates non-inferiority to original strain and superior immunogenicity to Omicron BA.4/5 variant compared to a first-generation mRNA vaccine booster

CMC

Deciphering Critical Quality Attributes & Embracing Next Generation Analytical Techniques for Enhanced mRNA Characterization

11:15 am Understanding Critical Quality Attributes for mRNA in a Cell Therapy Setting

Review of the recent general quality guidelines for mRNA
Applying the guidelines to mRNA quality in the context of a cell therapy program
Strategies for analytical development, qualification and validation for mRNA in a cell therapy context

11:45 am Comprehensive Evaluation of T7 Polymerase for Enhanced Yield & Quality in mRNA Production

A high throughput screening (HTS) workflow was established to enable comprehensive evaluation of T7 polymerase as a critical raw material in mRNA production
Four commercially available wild type T7 polymerases were systematically evaluated via DOE based optimizations, subsequently lot-to-lot variations, and scalability studies
A deep profile of each T7 polymerases including their performance in mRNA production and cost analysis was established to support ReciBiopharm’s advanced mRNA technology platform

12:15 pm Process & Analytical Development Advances for eRNA Therapeutics

Introduction to Sail Biomedicines’ bioplatform for programmable medicines
Overview of the differences between the techniques used for eRNA compared to mRNA
How to align RNA analytical methods to streamline manufacturing

12:45 pm Lunch & Networking Break

Discovery Translation Clinical Development CMC

Discovery

Elucidating the Fate of the mRNA Payload Inside the Cell for Increased Delivery Efficiency & Expression

1:45 pm Decoding the Dance: Unravelling the Moves of LNP-mRNA-based Vaccines

Understanding the Mechanism of Action of vaccines based on LNP-mRNA technology
Investigating the internalization and trafficking of cellular LNP, as well as the release of RNA cargo
Unravelling the Mechanism of Endosomal Escape

Translation

Enabling Improved Potency Assays for Faster & Robust Efficacy Characterization

1:45 pm Critical Quality Attributes: Development of a Potency Platform Assay for mRNA-Based Therapeutics & Vaccines

Current guidelines and ongoing discussions with health authorities regarding potency assay strategies
Overview of existing technologies
Introduction of a potency assay platform approach

Clinical Development

Unlocking Novel Disease Areas with Disruptive Companies Using Next Generation mRNA Therapeutics

1:45 pm Updates & Interim Data of mRNA-Based Cancer Vaccines to Treat EBV-Related Cancers

Presenting the latest clinical data for breakthroughs in the treatment of EBV-related cancers, including NPC and NKT lymphoma
Highlighting safety and efficacy based on ongoing enrolment and interim data from clinical trials
Demonstrating the transformative potential of mRNA therapeutics in the treatment of viralrelated malignancies

2:15 pm Clinical Updates on Gene Editing Therapeutics Targeting Rare Diseases in the Liver

Introducing YolTech’s high throughput discovery platform for novel CRISPR-CAS proteins
Revealing clinical trial data from the lead program targeting ATTR with a gene editing approach
Revealing clinical trial data from a second program targeting PCSK9 with a gene editing approach

CMC

Upgrading Platforms Through the Latest Digital & Technological Advancements for Faster mRNA Manufacturing

1:45 pm Harnessing Digital Twin for mRNA Manufacturing

Mechanistic models are being constructed for all unit operations for the manufacturing of mRNA biotherapeutics
The models are integrated with models for constraints, uncertainties, and disturbances to form a digital twin for integrated manufacturing
The digital twin is used to analyze the comparative performance for batch and continuous manufacturing

2:15 pm Evolving Self-Amplifying RNA Development by Pinpointing Influences & Tailoring Manufacturing Scale

Identifying the differences in materials and piloting the process development to optimize the results
Navigating the landscape of new technologies, and how to incorporate this into your program
Discussing the future of self-amplifying RNA and customized therapies, and scaling processes to meet patient needs

2:15 pm Afternoon Networking Break

Gearing up for 2025: Licensing, Partnership & Financial Considerations in the Dynamic mRNA Landscape

3:15 pm Wellcome Leap’s R3 Program: RNA Readiness & Response

R3: “RNA Readiness + Response” program goals and demonstrations at scale, to enable equitable access to RNA products and economically sustainable pandemic preparedness
Living RNA Biofoundries based on multi-product, multi-scale, standardised manufacturing processes
Demonstration on diverse RNA products and delivery systems stretch the edges of the product space
Digital tools for product design and manufacturability verification enable democratised access to manufacturing runs, for a vibrant, self-sustaining RNA ecosystem

3:45 pm Robust mRNA Production with End-to-End In-Process Analytics

Design of an efficient multi-step chromatographic process using CIMmultus re-usable columns to achieve scalable production of safe mRNA drug substance with low dsRNA, low fragments and residual DNA
Deployment of fast analytical methods based on PATfix to reduce cost of drug substance production, and to optimize production and purification processes in shorter development timelines
Overcoming challenges of in-process LNP drug product characterization by automated analysis of LNP quality attributes with minimal material handling

4:15 pm Planning for the Second Half of 2024: Investments, Partnerships & De-Risking Your mRNA Pipeline for 2025

Seizing opportunities for investments and partnerships in a thriving mRNA company
Overcoming challenges within a turbulent funding climate for mRNA companies to gain a scientific advantage
Key recommendations to avoid risks and ensure success

4:45 pm Chair’s Closing Remarks

5:00 pm End of 4th mRNA-Based Therapeutics Summit 2024

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Focus Day

Day One

Day Two – mRNA