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Key Sessions

Hari Pujar

mRNA Vaccines and Therapeutics: On the Progress from Promise to Reality

Moderna Therapeutics

Nov 09
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08:50 - 09:00 10 mins
Chairperson’s Opening Remarks
09:00 - 09:30 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
mRNA Vaccines and Therapeutics: On the Progress from Promise to Reality
  • Hari Pujar - Vice President, Technical Development and Manufacturing, Moderna Therapeutics

Messenger RNA offers unparalleled breadth and depth for the discovery and development of novel drugs and vaccines. The potential to direct specific tissue translation of both wild type and engineered intracellular, membrane-bound, and secreted proteins (and combinations thereof), coupled with rapid transition from preclinical to clinical development, has enabled Moderna and its partners to progress several development candidates into the clinic. This potential is being realized by the parallel development of different modalities (e.g. prophylactic and therapeutic vaccines, paracrine and systemic drugs).  This talk will review the scientific and engineering accomplishments enabling pharmaceutical development.

09:30 - 10:00 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
mRNA-Based Cancer Immunotherapeutics
  • Robert Jabulowsky, PhD - Deputy Head of Project Management, BioNTech AG

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.

10:00 - 10:30 30 mins
mRNA Therapeutics and CRISPR Therapeutics
VEGF mRNA in Cardiovascular Disease
  • Anna Collén, PhD - Project Leader VEGF Project, AstraZeneca
10:30 - 11:00 30 mins
Morning Coffee Break
11:00 - 11:30 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
mRNA Therapy for the Rare Genetic Disease
  • Aleks Schein - CSO, Head of RNA Therapeutics, ART BioScience

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.

11:30 - 12:00 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Towards mRNA therapeutics for skin diseases
  • Dr Markus Mandler, Ph.D. - Chief Science Officer, Accanis Biotech

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.

12:00 - 12:30 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Optimizing RNA therapeutics
  • Gemma Navarro - Senior Scientist, Formulation and Delivery Department, CureVac AG, Germany

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.

12:30 - 13:30 60 mins
Lunch
13:30 - 14:00 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Development of mRNA Therapeutics with LNP
  • James Heyes - SVP Technology Development, Genevant Sciences Corp.

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.

14:00 - 14:30 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
mRNA manufacturing
  • Dr Christoph Kröner - Head of IVAC Mutanome Lead Structure, BioNTech RNA Pharmaceuticals GmbH, Germany

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.

14:30 - 15:00 30 mins
Afternoon Coffee Break
15:00 - 15:30 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
In Vivo Gene Editing with CRISPR Using Peptide based Nanoparticle
  • Gilles Divita - CEO, Divincell, France

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.

15:30 - 16:00 30 mins
Info
mRNA Therapeutics and CRISPR Therapeutics
Addressing the Scalability and Manufacturing Challenges of CRISPR Therapeutics

If you are interested in giving this presentation please contact catherine.marshall@knect365.com

16:00 - 16:05 5 mins
End of Conference Day 4 and End of EuroTides 2018