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Feedback from Moderna: Discovery and Development of mRNA Therapeutics
Unmodified mRNA, a Versatile Drug Substance
CureVac AG, Germany
Messenger (m)RNA is increasingly investigated as a platform technology for multiple therapeutic applications. The employment of mRNA to deliver the genetic information of antigens into antigen-presenting dendritic cells offers new options for immunotherapies against cancer, especially in the context of individualized treatment. The presentation will deal with the GMP manufacturing of mRNA as well as approaches to overcome challenges of conducting an individualized clinical trial with an appropriate infrastructure setup. Part of the presentation will also be about first experiences from our clinical program.
The delivery of genetic information has emerged as a valid therapeutic approach. Several studies using chemically modified molecules have demonstrated that mRNA is able to act as potent vaccine as well as to promote expression of therapeutic proteins without inducing an adverse immune response. Here we provide various examples to demonstrate that our technology based on chemically unmodified mRNA serves the needs of a powerful therapeutic platform.
To be effective, mRNA pharmaceuticals need to be delivered into the cytoplasm of cells. This presentation will review approaches to achieving this, with emphasis on Arbutus’ industry-leading lipid nanoparticle (LNP) nucleic acid delivery system. siRNA-containing LNP products are currently in clinical development in several indications, and critical information on efficacy and tolerability obtained from these studies has been used to develop optimized LNP for the delivery of mRNA. This presentation will provide an overview of key findings from clinical development of siRNA-containing LNP and describe the progress made by Arbutus in design of mRNA-containing LNP.
Skin offers various opportunities with regard to development of mRNA-based therapeutics: diseases with validated molecular targets/attractive markets and direct access facilitating quantification of mRNA expression/clinical activity. mRNA is a new drug format capable of exceeding existing protein-based therapeutics. ACCANIS develops proprietary IVT-mRNAs addressing validated targets for specific skin conditions. We systematically modified specific IVT-mRNAs and tested the most interesting ones in skin explant systems varying formulation and delivery.