Resources

Stealth Mission: Novel mRNA Vaccine Delivery System

The recent success of mRNA vaccines during the COVID pandemic has vaulted this technology from its humble research beginnings to international recognition by the scientific and medical community as a groundbreaking measure in combating a host of diseases. The delivery of mRNA molecules has been dominated by the use of lipid nanoparticles (LNPs). However, while LNPs have proven effective, they provide investigators with several challenges—most importantly, the activation of unwanted cell-mediated immune responses. Alternative mRNA delivery systems offer the capability to stealthily deliver nucleic acids to their target and evade the immune system while generating a potent antigen-specific CD8+ T-cell response.

In this webinar cohosted by GEN and The CRISPR Journal, we will delve into the delivery question surrounding mRNA research and therapeutics. Dr. Helen McCarthy will review the key issues around RNA delivery and describe a novel peptide-based drug delivery system that her company has developed. This system is based on a sequence of 30 amino acids and condenses anionic cargo, like mRNA, irrespective of size, into nanoparticles for highly efficient entry into cells.

Chemically Modified mRNA as a Biologic: Developing an Analytical Framework for Success

Developing an analytical framework for success using a chemically modified mRNA can be compared with those for traditional biologics. Though much of the regulatory filing requirements can be compared with those for traditional biologics, others are not yet well defined. As such, it’s especially important that control on quality and consistency is maintained to ensure efficacy, purity, and safety.

Like traditional biologic products and processes, mRNA-based products require controls on:

• Quality of the starting material (including cell banking)
• Consistency of manufacturing processes
• Identification of critical process parameters
• Clearance of impurities
• Establishment of environmental controls to meet safety requirements
• Thorough characterization of structure, purity, and efficacy

Khaled Yamout, Senior Director, Manufacturing and Quality Control at
TriLink BioTechnologies, will discuss some of these challenges and solutions to support the requirements for characterization of structure, purity, and, efficacy of a well characterized mRNA drug substance.

Treating Viral Infections with mRNA-Encoded Cas13

The potential of gene editing as a therapeutic has come to fruition in the past several years, as clinical data from various studies has shown very positive results. Merging that potential with novel mRNA technologies—which have also demonstrated their value and versatility during the COVID-19 pandemic—could provide scientists and clinicians with an unmatched weapon in the arsenal to fight various diseases. In particular, Cas13 has been used to target RNA viruses in cell culture, but only recently has efficacy in animal models been demonstrated.

in this webinar, cohosted by GEN and The CRISPR Journal, that is diving deep into the burgeoning world of mRNA research and therapeutics. Our distinguished guest for this event, Dr. Philip Santangelo, will introduce important aspects of how to use mRNA to express Cas13 and use it as an antiviral agent. Additionally, Dr. Santangelo will discuss guide design, screening, the importance of controls, and how to apply this approach in vivo against multiple pathogens

Challenges and Considerations for mRNA Therapeutic Development

Due to its extreme potential to revolutionize the biopharma industry, the benefits of mRNA as a novel therapeutic are well known. However, the path leading to the development of mRNA medicines can be challenging and is often less discussed. Qualities like nucleic acid stability, immunogenicity, and delivery of the final product are all critical factors that face mRNA medicines and need to be addressed more frequently. Thankfully, novel methodologies and techniques to assist drug developers are on the rise and have the potential to streamline production workflows and increase production and efficiency.

This is the second webinar in a series of four, cohosted by GEN and The CRISPR Journal, that is diving deep into the burgeoning world of mRNA research and therapeutics. Our distinguished guest for this event, Dr. Edward Miracco, will take us through his extensive experience with mRNA chemistry, sequence design, and commercial-scale production. Dr. Miracco has faced many challenges in his work and will tell us about several vital considerations that could significantly aid your mRNA research endeavors.

Influenza mRNA Vaccines: Mechanisms and Methodologies

The power of mRNA vaccines became well established due to the COVID-19 pandemic, but we are just beginning to scratch the surface of their therapeutic versatility. Numerous global projects are underway to employ mRNA technology to combat human diseases such as cancer, HIV, and influenza. In a four-part webinar series over the next several months, we will hear from a diverse group of investigators who are at the vanguard of mRNA research and the novel ways they are utilizing this technology.

In this first webinar, our distinguished speaker, Dr. Norbert Pardi, will be discussing cutting-edge work on the development of broadly protective mRNA-based influenza vaccines. Not only will he discuss the mechanism of action of mRNA vaccines, Dr. Pardi will also tell us how his laboratory utilized a lipid nanoparticle-encapsulated, nucleoside-modified mRNA vaccine platform to deliver a combination of conserved influenza virus antigens.

TIDES mRNA Technologies Digital Week: Analytical Methods for a Well Characterized mRNA Drug Substance

Though much of the regulatory filing requirements for mRNA-based therapeutics can be compared with those for traditional biologics, others are not yet well defined. Like traditional biologic products and processes, mRNA-based products require controls on the quality of the starting material (including cell banking), consistency of manufacturing processes, identification of critical process parameters, clearance of impurities, establishment of environmental controls to meet safety requirements, and the thorough characterization of structure, purity, and efficacy. This has posed some challenges in analytical method development to ensure qualification for intended use. We discuss some of these challenges and solutions to support the requirements for characterization of structure, purity, and efficacy of an mRNA Drug Substance.

mRNA Modifications that Matter: Modified Uridine as a Therapeutic Tool

In vitro transcribed (IVT) mRNA has become an effective and scalable therapeutic modality for a wide range of disease indications. It has been shown that modifications to wild-type uridine may improve the efficacy of these therapeutics. To aid in the synthesis of these compounds, TriLink is developing an extensive platform to manufacture modified uridine and mRNA at both GMP quality and scale.

Fine-Tuning mRNA Fate and Function: Modified Uridine as a Therapeutic Tool

In this presentation Cory Smith, Application Scientist, TriLink BioTechnologies will discuss in vitro transcribed (IVT) mRNA and how it has become an effective and scalable therapeutic modality for a wide range of disease indications. 

Therapeutic Advances Using In Vivo CRISPR Genome Editing

In this webinar, Dr. Laura Sepp-Lorenzino Chief Scientific Officer at Intellia, will share the company's progress in both in vivo and ex vivo genome editing approaches. In particular, she will discuss the recent advances made regarding NTLA-2001—the first-ever investigational CRISPR-Cas9 therapy candidate to be systemically delivered for the treatment of ATTR amyloidosis.

The Future of RNA Therapeutics is Modular

In this webinar from GEN and The CRISPR Journal, Professor Dan Peer will discuss the state of RNA-based therapeutics, particularly developing a self-assembled modular platform that enables the construction of a theoretically unlimited repertoire of RNA-targeted carriers. He will present a novel approach for delivering modified mRNA to specific cell types in vivo utilizing this platform.

mRNA Applications: Development of mRNA and its Applications

In recent years, the development of mRNA and its applications has dramatically expanded across therapeutic areas. The clinical development of mRNA products includes a number of unique features and considerations that are rooted in mRNA’s molecular characteristics. In this webinar, May Guo, MBA, Director of Business Development at TriLink BioTechnologies, will highlight important aspects to consider as you develop your mRNA tools and drugs.

Advantages of In vivo DNA Barcoding in Nanoparticle Selection: Quantifying how thousands of nanoparticles deliver nucleic acids in vivo using DNA barcodes.

In this video James Dahlman, PhD, Assistant Professor, Department of Biomedical Engineering at the Georgia Institute of Technology discusses, RNA therapeutics and the role of  in vivo DNA barcoding in nanoparticle selection.

Vaccine and Trial Efforts Against COVID-19

In this Webinar Dr. Tal Zaks, CMO at Moderna, will discuss vaccine and trial efforts against COVID-19

Self-Amplifying mRNA Vaccines for Pandemic Rapid Response

In this webinar, you will hear from Dr. Andy Geall about data innovations from seven years of research at Novartis Vaccines on self-amplifying mRNA technology and how this approach could enable the concept of vaccines on demand as a rapid response to a real threat.

SARS-CoV-2: Going Viral

In this Webinar Dr. Gregory A. Poland, Director of Mayo Vaccine Research Group at the Mayo Clinic will discuss, SARS-CoV-2: Going Viral.

Coronaviruses in Livestock: Vaccines and International Transport of Virus

In this Webinar Dr. Mark Kehrli, Center Director at the National Animal Disease Center will discuss, Coronaviruses in Livestock: Vaccines and International Transport of Viruses.

Base Editing: Putting Precision into Genomic Medicine

In this presentation, Dr. Nicole Gaudelli will discuss how her group has transformed off-the-shelf base editors from a technology developed in academia to a promising therapeutic tool through engineering, screening, and directed evolution.

COVID-19: A Prototype Pathogen Demonstration Project for Pandemic Preparedness

In this Webinar Dr. Barney S. Graham, Deputy Director, VRC, NIAID NIH will discuss, COVID-19: A prototype pathogen demonstration project for pandemic preparedness.

A CRISPR Vision for the Future of Cell Therapy

In this webinar, T.J. Cradick, PhD, who was until recently head of genome editing at CRISPR Therapeutics, will introduce how genome editing is used to engineer cell therapies.

Why Use mRNA: Video 1 of 8 from the TIDES 2018 Workshop by Dr. Anton McCaffrey.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses the benefits of using mRNA for manufacturing therapeutics.

CRISPR-Based In Vivo Genome Editing and Base Editing

In this webinar, Dr. Wen Xue, Assistant Professor, RNA Therapeutics Institute at the University of Massachusetts Medical School will present, a modular delivery strategy for homology-directed repair by combining lipid nanoparticle delivery of Cas9 mRNA with adeno-associated viruses (AAV) encoding a sgRNA and a repair template.

mRNA Applications: Summarizing mRNA applications that include genome engineering, gene replacement, and immunobiology. Part 2 of 8 of the TIDES 2018 workshop series by Dr. Anton McCaffrey.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses, mRNA applications, including gene engineering, gene replacement and immunobiology.

mRNA Biology: Video 3 of 8 from the TIDES 2018 Workshop featuring Dr. Anton McCaffrey. Includes biology of mRNA location and sensor recognition.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses the biology behind mRNA therapeutics.

Manufacturing Design Considerations: Video 4 of 8 from the TIDES 2018 Workshop featuring Dr. Anton McCaffrey. Includes: planning the optimal mRNA, mRNA synthesis, mRNA manufacturing processes, research phase, plasmid template, and poly A polymerase.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses planning, manufacturing design considerations and processes involved with research and clinical applications.

WVC COVID-19 Webinar: Panel on Vaccine Trial Data, Antibodies and Safety

This panel focuses on the lessons learned from COVID-19 vaccine human trials to date: our current understanding on the evolving trial data, neutralizing antibodies and safety of these COVID-19 vaccine candidates.

mRNA Capping: Video 5 of 8 from the TIDES 2018 Workshop featuring Dr. Anton McCaffrey. m6Am, Cap 0, Cap 1, and Cap 2 explained in this summary of endogenous cap structures.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses the cap structures .m6Am, Cap 0, Cap 1, and Cap 2 for optimal mRNA synthesis.

In Vitro mRNA Capping: Video 6 of 8 TIDES 2018 featuring Dr. Anton McCaffrey discussing mRNA capping methods, capping characteristics, ARCA, CleanCap, and comparison data.

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses the capping characteristics, capping methods and comparison data between legacy capping methods ARCA, and CleanCap^®, TriLink’s, novel, new capping technology.

Chemical Modification of mRNA: Part 7 of 8 of the TIDES 2018 workshop series by Dr. Anton McCaffrey. Includes topics surrounding modified RNA sequence space, sequence engineering, uridine depletion, purification methods, effect of cap structures, and var

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses chemical modification of mRNA including purification methods and sequence engineering.

Design & Manufacturing of Chemically Modified mRNA Therapeutics: Design and Manufacturing of Chemically Modified Messenger RNA Therapeutics - Entire Video TIDES 2018 Workshop by Dr. Anton McCaffrey

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses, the Design & Manufacturing of Chemically Modified mRNA Therapeutics: This complete video highlights mRNA therapeutic manufacturing from research to clinical applications.

The Path to GMP Production: Video 8 of 8 from the TIDES 2018 workshop series by Dr. Anton McCaffrey

In this video Dr. Anton McCaffrey, Senior Director of Emerging Science and Innovation, at TriLink BioTechnologies discusses, the path to GMP production including planning and timelines.