Subcutaneous administration opens the possibility of patient self-administration that could enable mRNA to be used in chronic treatments for protein replacement or regenerative therapies. In this work, we demonstrate that subcutaneous administration of mRNA formulated within LNPs results in measurable plasma exposure of a secreted protein, albeit with dose-limiting local inflammatory responses. Inclusion of steroid prodrugs in the LNPs resulted in increased protein expression and improved tolerability following subcutaneous administration.

Speaker:

Nigel Davies, Ph.D.
Principal Scientist, Advanced Drug Delivery, Pharmaceutical Sciences, R&D
AstraZeneca, Sweden

The clinical promise of mRNA therapeutics and vaccines is clear, but their novelty also brings new challenges to the manufacturing process. Compared to monoclonal antibodies or viral vectors, cell-free mRNA production is faster, but in vitro transcription, purification efficiency, and lipid nanoparticle (LNP)-mediated delivery each add process challenges. In this presentation, we’ll discuss strategies and technologies for addressing these challenges to develop a robust, scalable, and integrated mRNA manufacturing process. We’ll also address the LNP step, insourcing vs outsourcing, and batch management.

Speaker:

Katarina Stenklo
Enterprise Solutions Commercial Activation Leader
Cytiva, Sweden

To support rapid early discovery research efforts for mRNA therapeutics, Codex DNA has developed a first-of-its-kind BioXp mRNA automation solution capable of synthesizing up to 16 highly functional mRNAs from digital sequence in less than 24hrs. This talk will help you understand the values of leveraging the BioXp system workflow for your research. We’ll present the step-by-step workflow from digital sequence input followed by DNA fragment assembly to mRNA synthesis including using the CleanCap technology for mRNA capping.

Dr. Krishna Kannan is Director of R&D at Codex DNA. He received his PhD from the University of Illinois at Chicago. He is a long-time associate of Dr.Dan Gibson– the creator of the Gibson assembly® method and has worked closely with other Synthetic Biology pioneers at the J Craig Venture Institute and Synthetic Genomics Inc., contributing to the development of the first synthetic cell with a minimal genome and the digital-to-biological converter. Dr. Kannan is widely published in leading peer-reviewed publications such as Cell, Science and Nature Biotechnology. Most recently he has been instrumental in synthetic biology workflow innovations at Codex DNA including the first de novo full-length construction of the Covid-19 genome and the novel sequence-to-mRNA synthesis automation workflow.

Speaker:

Dr. Krishna Kannan
Director of R&D
Codex DNA

With the development of mRNA Vaccines and the role they have played in combatting the global COVID-19 pandemic, regulators are developing guidance to assist manufacturers to navigate the emerging regulatory landscape. This session will provide an in-depth analysis of the recently released draft WHO Guidance for CMC Regulatory Strategies for mRNA Vaccines

Speakers:

Paul Dawidczyk
VP, Regulatory Affairs CMC
Moderna Therapeutics

The recently demonstrated efficacy of mRNA-based Covid-19 vaccines has shown promise of this therapeutic format, but also highlighted the need for higher efficiency of mRNA production to meet enormous needs for global vaccine supply. Typical mRNA production process involves three key steps: 1) plasmid DNA (pDNA) production in supercoiled (sc) isoform, linearization and purification, 2) in-vitro transcription (IVT) reaction and 3) mRNA purification. Here we present a chromatographic toolbox and mRNA IVT synthesis for integrated mRNA production from pDNA to mRNA purification, including in-process analytics. This high yield process reduces the overall number of purification steps required, improves recoveries, results in extra low protein impurity and allows for very efficient dsRNA removal.

Speaker:

Aleš Štrancar
Managing Director
BIA Separations, A Sartorius Company

In vitro transcribed (IVT) mRNA has become an effective and scalable therapeutic modality for a wide range of disease indications. However, therapeutic efficacy may still be improved by making modifications to the wild type nucleobase uridine. To aid in the synthesis of therapeutic mRNA, TriLink offers an extensive platform for the manufacturing of modified uridine and mRNA at GMP quality and scale.

Speaker:

Cory Smith
Application Scientist (mRNA)
TriLink BioTechnologies

The next generation of mRNA therapeutics will utilize synthetic biology to enable programmable drugs capable of delivering precise, multi-functional, and curative treatments. In this presentation, I will talk about our efforts in programming mRNA to improve response rates to checkpoint inhibitor therapy for solid tumors as well as other early work that has the potential to revolutionize cancer immunotherapy.

Speaker:

Tasuku Kitada, Ph.D.
President and Head of R&D
Strand Therapeutics

Attending this session will allow attendees to better understand the criteria that should be used for choosing raw materials for mRNA vaccines and therapeutics. They will also learn about several actual cases where proper or improper choices in material selection were made and the impact of those decisions on the drug approval process.

Speaker:

Dr. Darwin Asa
Market Development Manager
Thermo Fisher Scientific

Since the development of Sanger Sequencing in 1986, sequencing technologies have been revolutionizing biomedical research. Sequencing, mapping, and comparing the genomes of cells in healthy and diseased states can alter the way clinicians think about how to treat patients shifting from traditional medicine to a genome-based era of preventive and therapeutic decisions. There is also growing demand for using sequencing to evaluate the quality, safety, and efficacy of nucleic acid-based pharmaceutical products.

In this presentation, Dr. Steven Parker, NGS Expert from Intertek Pharmaceutical Services, discusses sequencing technology, sequencing workflow, the process of developing it for use in a regulatory research environment, and its potential customer applications.

Speaker:

Dr. Steven Parker
NGS Scientist
Intertek Pharmaceutical Services