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Thanks to its unique characteristics, mRNA may be employed for the potent treatment of cancer. The potential of mRNA-based cancer therapeutics will be discussed and preliminary clinical data from BioNTech´s mRNA immunotherapy programs will be presented.
ART BioScience is a drug development company, initially focusing on mRNA-based treatment of Duchene muscular dystrophy-the most common
fatal genetic disorder, affecting about 300,000 people worldwide; with approximately 18,000 new cases registered each year.
Delivered to the muscle cells, synthetic mRNA will drive the production of functional protein, providing universal treatment, regardless of the
patient’s genetic background (mutation’s nature and location), opening the whole new area of therapeutics.
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 ex vivo and in vivo skin model systems varying formulation and delivery.
Messenger RNA based therapeutics offer the unique potential for product specific optimization to enable a tailored therapy for different diseases. The optimization does not only comprise the sequence and structure of mRNA but also the delivery system, that has to be adapted for each tissue in order to enable mRNA expression.
Genevant’s Lipid Nanoparticle (LNP) platform is enabling several early and late stage clinical trials. They are designed to deliver their nucleic acid payloads to sites of disease and have been used to target both viral and endogenous gene targets. Here we describe their application to mRNA payloads and draw comparisons to their performance with siRNA.
Messenger (m)RNA is increasingly investigated as a platform technology: The delivery of the genetic information offers potentially broad therapeutic applications. The presentation will deal with mRNA design, manufacturing and analytics as well as approaches to overcome general challenges bringing mRNA therapeutics into market-ready products.
The recent discovery of CRISPR has provided a highly efficient tool for site-directed genome engineering in eukaryotic cells, opening new perspective for innovative gene therapy. However, in vivo administration of the CRISPR-CAS9 system remains a limitation for its therapeutic application. We have designed a new delivery platform that can potently deliver active CAS9 mRNA/gRNA or CAS9/gRNA ribonucleoprotein complexes in cultured cells as well as in vivo. The ADGN-technology is based on amphipathic peptides that form stable neutral nanoparticles with CRISPR-CAS9 complexes through non-covalent electrostatic and hydrophobic interactions. ADGN-Nanoparticles mediate functional delivery of CAS9 mRNA and CAS9:gRNA complexes into human primary cells and T cells, allowing simultaneous robust multiple-gene editing via either Non-Homologous End Joining (NHEJ) or Homology Directed Repair (HDR). ADGN-Nanoparticle promotes in vivo delivery of CAS9 mRNA/gRNA, leading to the expression of active CAS9/gRNA in mice and a robust editing of a selected target gene in specific organs or in tumors. ADGN technology was evaluated in clinically relevant system targeting PCSK9. A single administration of ADGN nanoparticle containing CAS9 mRNA/gRNA resulted in a permanent alteration of PCSK9 and a marked reduction of cholesterol level, without inducing any toxicity or histological alteration. Therefore, given the robustness of the biological response achieved through this approach and the absence of associated toxicity, we are further exploring the ADGN-technology for therapeutic application of CRISPR-CAS9 based genome editing.