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Key Sessions

Chandra Vargeese, PhD

From Stereopurity to Precision Medicine: Optimizing the Properties of Antisense Nucleic Acid Therapeutics

WAVE Life Sciences

Feb 27
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8:00am - 8:55am

Registration and Coffee

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Showing of Streams
10:30am - 11:10am

Networking Refreshment Break with Poster and Exhibit Viewing

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Showing of Streams
12:45pm - 1:55pm

Networking Luncheon & Poster Viewing in Exhibit Hall

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Showing of Streams
5:30pm - 7:00pm

Networking Cocktail Reception in the Exhibit and Poster Hall

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8:00am - 8:55am 55 mins
Registration and Coffee
8:55am - 9:00am 5 mins
Main Conference
Chairperson's Remarks
  • James Thompson, PhD - Head of CMC Project Management, Moderna Therapeutics, Inc.
9:00am - 9:30am 30 mins
Main Conference
An Introduction to mRNA Therapeutics
  • James Thompson, PhD - Head of CMC Project Management, Moderna Therapeutics, Inc.
9:30am - 10:00am 30 mins
Info
Main Conference
Novel mRNA Immunotherapies
  • 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 and other diseases. The potential of mRNA-based therapeutics will be discussed and preliminary clinical data from BioNTech´s mRNA immunotherapy programs will be presented.

10:00am - 10:30am 30 mins
Info
Main Conference
Effective Delivery of Therapeutic Proteins by mRNA
  • Nigel Horscroft, D.Phil - Vice President Development RNArt, CureVac AG

In recent years the field of mRNA therapeutics has expanded significantly. mRNA drugs can fill critical gaps not met by traditional small molecule drugs, available biological treatments, and emerging gene therapies. Our data supports the ability of sequence-engineered chemically unmodified mRNA to achieve ample protein expression.

10:30am - 11:10am 40 mins
Networking Refreshment Break with Poster and Exhibit Viewing
11:10am - 11:15am 5 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Chairperson's Remarks
11:10am - 11:15am 5 mins
Peptide Track: Peptide Discovery and Development
Chairperson's Remarks
11:15am - 11:45am 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
From Stereopurity to Precision Medicine: Optimizing the Properties of Antisense Nucleic Acid Therapeutics
  • Chandra Vargeese, PhD - SVP, Head of Drug Discovery, WAVE Life Sciences

Wave Life Sciences has developed proprietary synthetic chemistry and manufacturing capabilities that we are using to design and produce stereopure antisense oligonucleotides (ASOs) for patients with serious, genetically defined disease. In our evaluation of ASOs that promote RNase H-mediated activity or exon-skipping activity, we have explored the relationships among sequence space, chemical modification and backbone stereochemistry to optimize their pharmacological properties. We have discovered a relationship between the molecular configuration of our ASOs and their activity. We will present data from multiple programs that illustrate how our chemistry platform allows us to optimize ASOs for mechanism (e.g., gene knockdown or splicing), properties in vitro and in vivo, and distribution to an array of tissues.

11:15am - 11:45am 30 mins
Info
Peptide Track: Peptide Discovery and Development
Plants as Biofactories for Producing Peptide-based Pharmaceuticals
  • David Craik, PhD - Professor of Biomolecular Structure, Institute for Molecular Bioscience, University of Queensland

This presentation will provide an update on our progress using plants to discover novel cyclic peptides and then using crop plants to produce 'designer' cyclic peptides with applications in cancer, multiple sclerosis and cardiovascular disease.

11:45am - 12:15pm 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
The Role of Base Modifications in Folding, Binding and Pharmacology of DNA-based Aptamers
  • Nebojsa Janjic, PhD - Chief Science Officer, SomaLogic, Inc.

Base modifications front-loaded into SELEX libraries have expanded the range of proteins for which high-affinity aptamers can be selected.  The composition and physico-chemical properties of modified side chains profoundly influence folding, binding, metabolic stability and pharmacokinetic properties of aptamers, with implications for development of new diagnostics and therapeutics.

11:45am - 12:15pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Genetically Encoded Selection of Novel Cyclic and Bicyclic Architectures
  • Ratmir Derda, PhD - Associate Professor, Department of Chemistry, University of Alberta

The talk will describe genetically-encoded (GE) platform for discovery of macrocyclic and macrobicyclic peptides synthesized by aqueous late-stage functionalization of readily-available libraries of peptides displayed on phage. The structure of these chemical post-translational modifications can be encoded in the genome of phage using silent encoding technology. The resulting phage displayed libraries of "unnatural" macro(bi)cyclic peptides could be used to target either proteins or cells and tissues; the latter targets are difficult to address with DNA/RNA or bead-based libraries. Expanded chemical space offers value-added properties such as stability to aggressive protease environment and incorporation of unnatural chemotypes that are known to increase bioavailability

12:15pm - 12:45pm 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
Next Generation Locked Nucleic Acids
  • Konrad Bleicher, PhD - Expert Scientist, F. Hoffmann-La Roche Ltd.

Locked Nucleic Acids (LNA) have come a long way from research tools to clinically validated drug candidates. As we are constantly developing this platform, potential next generation analogues have been identified, showing very promising drug properties. Here we will report our latest observations of backbone modified LNA with a particular focus on thiophosphate modifications, including stereo-defined internucleoside linkages.

12:15pm - 12:45pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Intracellular Screening for Selective Antagonists of an Oncogenic Transcriptional Regulator
  • Jody Mason, PhD - Senior Lecturer In Biochemistry, University of Bath

Our research focuses on design, intracellular library screening and selection of peptide antagonists with high affinity and selectivity for inhibiting protein-protein interactions implicated in disease. Therapeutically relevant targets that include the oncogenic Activator Protein-1 transcriptional regulator, and β-amyloid / α-synuclein proteins implicated in AD/PD. Target-specificity is further enhanced by expressing off-target proteins during selection with antagonists downsized and refined using structure-inducing constraints and non-natural sequences.

12:45pm - 1:55pm 70 mins
Networking Luncheon & Poster Viewing in Exhibit Hall
1:55pm - 2:00pm 5 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Chairperson's Remarks
1:55pm - 2:00pm 5 mins
Peptide Track: Peptide Discovery and Development
Chairperson's Remarks
2:00pm - 2:30pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
In Vivo Activity of Authentic and Theoretical Impurities in RNAi Therapeutics
  • Bob Brown, PhD - Chief Scientific Officer, Senior Vice President, Research, Dicerna Pharmaceuticals
2:00pm - 2:30pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Inhibition and Degradation of Drug Targets Using bioPROTAC mRNAs – A Novel Approach with Broad Therapeutic Potential
  • Jeff Chang, PhD - Associate Scientist and Post Doc Fellow, MSD International GmbH (Singapore)

To tackle historically intractable targets, we have developed a platform employing targeted degradation. Specifically, we have engineered fusion constructs involving two components I) mini-proteins/peptides with high-affinity against therapeutic targets linked to II) truncated E3 ligase receptors. These ‘bioPROTACs’ have proven broadly successful with many constructs showing robust degradation activity. Currently, we aim to apply this technology as research tools and therapeutically by pursuing delivery strategies of bioPROTAC mRNAs.

2:30pm - 3:00pm 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
Optimizing Delivery of mRNA Therapeutics
  • Peter Lutwyche, PhD - Chief Technology Officer, Genevant Sciences Corporation

Lipid Nanoparticles are the leading technology for delivery of mRNA therapeutics. This presentation will describe the features of LNP critical for maximizing the therapeutic index of mRNA products.

2:30pm - 3:00pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Massively Parallel Synthesis and Lead Optimization of Peptidomimetics
  • Jigar Patel - Director, Roche Madison

We have developed a maskless photolithography peptide synthesis and screening platform that has been applied both for lead discovery and optimization. Our chemical catalog of over 350 amino acid building blocks combined with rapid library design and robust workflows, allows synthesis of over 18 million unique linear or cyclic peptides in less than 48 hrs on a single glass slide. The process is scalable and large libraries of millions of peptides can be synthesized on demand. We have made incremental upgrades and exploring end-point detection, real-time kinetic interaction, and cell-based phenotypic assays. Here, we will describe the core technology and its application to rapidly and systematically evolve high-affinity, high-specificity binding peptides to protein targets in a reproducible and digitally controlled process. We will demonstrate some case-examples, and recent process upgrades that enable real-time kinetic analysis for thousands of peptide-protein interactions in parallel.

3:00pm - 3:30pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Presentation Title TBA
  • Satoshi Inoue, PhD - Scientist, Ajinomoto Co., Inc.
3:00pm - 3:30pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
A New Scenario for Disulfide Peptide Synthesis Based on Npys Chemistry
  • Yoshio Hayashi, PhD - Professor, Department of Medicinal Chemistry, Tokyo University of Pharmacy and Life Science

Chemistry based on 3-nitro-2-pyridinesulfenyl (Npys) group gave us a new scenario on disulfide peptide synthesis. Two topics will be discussed in the lecture, i.e., new solid- or no-solid-supported Npys-sulfenates (Npys-OR) as disulfide bond-forming agents and solid-phase-assisted disulfide ligation method. The latter actualizes intriguing “disulfide-driven cyclic peptides synthesis”.

3:30pm - 4:00pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Networking Refreshment Break with Poster and Exhibit Viewing
3:30pm - 4:00pm 30 mins
Peptide Track: Peptide Discovery and Development
Networking Refreshment Break with Exhibit and Poster Viewing
4:00pm - 4:30pm 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
Antisense Oligonucleotide Purification Process: Successes and Challenges During Scale-up
  • Robert Gronke, PhD - Senior Principal Scientist, Technical Development, Biogen, Inc

Recently, our first full scale GMP batch for an antisense oligonucleotide was manufactured in the newly built synthesis suite at Biogen. A four-step purification process was then carried out in the existing flexible volume manufacturing facility that, up until this point, has been used for manufacturing Biogen's protein-based parenterals. This was our first test case to demonstrate that Biogen can manufacture ASOs safely, at scale, and achieve high purity and yield. Results are presented on the scalability of the ASO process from bench to GMP scale, highlighting successes and challenges faced with scale-up of the downstream ASO process.

4:00pm - 4:30pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Peptide Drug Discovery Case Study: Appropriate Screening for ADME Properties and Pseudo Allergic Reactions at An Early Stage is Key to Success
  • Raffaele Ingenito, PhD - Senior Scientist, IRBM Science Park

The key to success for development of peptide therapeutics is to use an appropriate screening funnel to correctly drive the SAR at the early stage of the project. This presentation will address important issues to consider in peptide development, namely: subcutaneous and plasma metabolism with in vitro/in vivo correlation to optimize bioavailability; potential reactions of peptide induced pseudo-allergy; immunogenicity; oligomerization and aggregation tendencies.

4:30pm - 5:00pm 30 mins
Info
Oligonucleotide Track: Preclinical and Clinical Updates
NittoPhase® HL, High Loading Polymeric Solid Support for Efficient and Cost-Effective Oligonucleotide Synthesis
  • Mohammad Ahmadian, Ph.D. - Senior Director, Kinovate Life Sciences, A Nitto Company

When it comes to “Therapeutic Oligonucleotide manufacturing” performance and quality of the solid support matter. In this presentation, quality and performance of NittoPhase® HL is discussed and advantages of this cGMP manufactured solid support in comparison with other solid supports are discussed.

4:30pm - 5:00pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Beyond Antibodies: Targeting VEGF-A and PD-1 with Synthetic D-proteins
  • Dana Ault-Riche, Ph.D. - CEO, Reflexion Pharmaceuticals

Small proteins have historically been under-utilized as human therapeutics because they are rapidly metabolized, have short half-lives and can be neutralized by anti-drug immune responses. However, proteins composed entirely of D-amino acids (“D-proteins”) are resistant to proteases, have longer half-lives and significantly reduced immunogenicity. These properties also enable D-proteins to better penetrate tissues and solid tumors. Because D-proteins are chemically manufactured they can be engineered in ways not possible using biologic manufacturing. Mirror image phage display technology has been used to create D-proteins that are potent inhibitors of VEGF-A, an important therapeutic target for treating cancer and eye diseases. D-protein inhibitors of PD-1, an important immune-oncology target, have also been developed. Therapies combining VEGF-A and PD-1 inhibition have resulted in improved clinical outcomes for liver, lung and kidney cancers. This presentation will focus on the development of D-protein inhibitors to VEGF-A and PD-1.

5:00pm - 5:30pm 30 mins
Oligonucleotide Track: Preclinical and Clinical Updates
Intertek Presentation Title TBA
5:00pm - 5:30pm 30 mins
Info
Peptide Track: Peptide Discovery and Development
Of Peptides and PANDAS: Innovative Preclinical Assessment Tools for Safety and Efficacy of Protein and Peptide Therapeutics
  • Annie De Groot, MD - Founder, CEO and CSO, Epivax, Inc.

The FDA recently released a new draft guidance defining the equivalence of a rDNA peptide product and a synthetic peptide product. The draft guidance enables generic manufacturers of peptide drugs to file an Abbreviated New Drug Application (ANDA) for synthetic peptide drug products that refer to listed drugs of rDNA origin. Since the processes for manufacturing the generic and reference drug (RLD) are not equivalent, peptide drugs can be associated with impurities. Impurities can result from changes in the sequences due to deletions, insertions, integration of incorrect amino-acids and modifications and also impurities related to the synthetic production. The FDA draft guidance requires manufacturers to prove that the synthetic peptide product does not contain impurities that have an increased affinity for major histocompatibility complexes and potential for engaging immune response, which may drive undesired anti-drug antibody development. We have used both immunoinformatics-driven analysis and in vitro validation assays to perform immunogenicity risk assessment of peptide generics. This combination of in silico and in vitro tools is referred to the PANDA assay which can be used to support generic peptide drug equivalency in an ANDA application. This presentation will provide insight as to the process of performing the PANDA assay, illustrating the process with two case studies (such as Calcitonin and Teriparatide).

5:30pm - 7:00pm 90 mins
Networking Cocktail Reception in the Exhibit and Poster Hall