Explore the Agenda
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
Track Name
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
Track Name
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