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7:30 am 8:25 am (55 mins)

Main agenda

Registration and Coffee

7:45 am 8:15 am (30 mins)

Main agenda

Breakfast Presentation: Large-Scale Production of Liposomal Oligonucleotides: Lessons Learned from Successful Product and Process Developments

Polymun Scientific GmbH is a pioneer in the formulation and large-scale production of liposomal dosage forms. Various oligonucleotides, such as siRNA, saRNA, mRNA and DNA, various small molecular weight compounds and various liposomal vaccine products have been formulated using Polymun’s proprietary solvent injection technology. Most of the oligonucleotide formulations are based either in the interaction between a positively charged phospholipid with the negatively charged oligonucleotide, or in the action of bivalent cations which crosslink phosphate groups of polynucleotides with the ones of phospholipids. Optimal oligonucleotide encapsulation can be achieved by very well control over the ratio of the components of the liposomal formulation at the point of liposome formation. Along years, several techniques have been proposed for the large-scale production of liposomes, but their robustness and efficiency in the entrapment of hydrophilic compounds is debated. Our proprietary technology is an upscale of the ethanol injection method and is based on the specially designed injection module that allows perfect control over the process parameters, like concentration and ratio of API and lipid at the point of the injection. Manufacturing of liposomal formulations with Polymun’s proprietary crossflow solvent injection technology in a cGMP compliant environment, allows full-scalable, batch to batch consistent and robust liposome production. The unique advantages of our technology is the high production output in short process times and the mild manufacturing conditions, which decrease markedly the risk of API, lipid and product instability. Additionally, optimization of process parameters such as flow rates, choice of the optimal solvent(s), choice of the appropriate buffer(s) and optimization of the process temperature, allow the production of a tailored liposomal product according to the requirements of our clients. Next to the optimization of the production parameters, the analytic methods related to the characterization of the liposomal product with regards to composition and physicochemical stability over time are set up and optimized by Polymun’s QC department. Over years, plenty of different liposomal formulations and over 30 different oligonucleotides have been developed and amongst those, promising candidates have been transferred from laboratory scale preparation into large scale cGMP manufacturing. By now, Polymun Scientific guided 15+ projects into Phase I clinical trials. Among those projects are injectable liposomal formulations, liposomal vaccines and topical formulations. With our current equipment and qualified processes and personnel, we are capable of producing volumes ranging from several milliliters up to few hundred liters. Furthermore, Polymun is equipped with an automated filling line with capacity of 10000 vials and has the capacity to lyophilize liposomal products. The cGMP lyophilizer is capable of lyophilizing up to 5000 vials per run.

  • Charalampos Koutsoulas, Ph.D. - Scientific Project Leader, Polymun Scientific, Austria

7:45 am 8:15 am (30 mins)

Main agenda

Liquid-Phase Oligonucleotide Synthesis by Segment Condensation

Nagase introduces unique technologies for peptide and nucleotide area. One is Liquid-Phase Nucleic Acid Segment Synthesis and another is Maruoka Catalyst® which can create UNAA (Unnatural amino acid).

  • Masanori Kataoka, Ph.D. - Chief Technical Officer, Shikoku Nucleic Acid Chemistry (S-NAC)

7:55 am 8 am (5 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Chairman’s Remarks

  • Chairman Lubomir Nechev, Ph.D. - Vice President, Process Sciences, Alnylam Pharmaceuticals

8 am 8:30 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Driving Large Scale Oligonucleotides to Be Commercially Viable

Oligo manufacture is a triumph of platform design in that any reasonable size oligo can be prepared with only limited modifications to a standard process. As demands increase however, inherent inefficiencies in the platform will have a disproportionate impact that could impact application in more mainstream therapeutic indications. I will share the view that developing a large scale oligo provides sufficient motivation to challenge and redevelop the process.

  • Presenter Louis Diorazio, Ph.D. - Principal Scientist, Chemical Development, AstraZeneca

8:30 am 9 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

GalNAc Solid Support as Starting Material for the Oligonucleotide Conjugate Manufacturing Process

The GalNAc starting material for the oligonucleotide manufacturing process will be described. General principles for starting material selection and appropriate control strategies will be discussed.

  • Presenter Roumen Radinov, Ph.D. - Associate Director, Process Chemistry, Alnylam Pharmaceuticals

9 am 9:30 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Conjugation of Oligonucleotides Under Non-Aqueous Conditions

Recently there has been considerable focus in the conjugation of oligonucleotides with non-nucleotide moieties, for example, enabling targeting of therapeutic oligonucleotides to specific organs and tissues in vivo. Traditionally, conjugations are performed in aqueous solvents and require several equivalents of conjugate group, thus increasing the cost of manufacture.

The conjugation reaction under non-aqueous conditions, allows for drastic reduction of the amount of conjugate needed or the use of non-aqueous compatible conjugates.

  • Presenter Christoph Rosenbohm, Ph.D. - Senior Director, Research Operations, Roche Innovation Center Copenhagen

9:30 am 10 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Solid vs. Solution Phase GalNAc/Oligonucleotide Conjugation

  • Presenter Andrew Rodriguez, Ph.D. - Assistant Director, Process Organic Chemistry, Ionis Pharmaceuticals

8:25 am 8:30 am (5 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Chairman’s Remarks

  • Chairman Dmitry Samarsky, Ph.D. - Chief Scientific Officer, Silence Therapeutics, Germany

8:30 am 9 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

This Is Your Brain on Antisense Oligonucleotides: Distribution, Activity and Application to the Treatment of Severe Neurodegenerative Disease

Multiple antisense oligonucleotides (ASOs) are currently in various stages of clinical development for severe neurological diseases. Because ASOs do not cross the blood brain barrier, ASOs must be administered directly into the cerebrospinal fluid (CSF) of the central nervous system (CNS).    ASOs then distribute broadly within the CNS, modulate their RNA targets, and display the expected pharmacology. This presentation will provide an update on our work that supports the development of ASOs for the treatment of Parkinson’s disease, as well as other severe neurological diseases, particularly those stemming from increased or aberrant expression of a toxic protein or RNA.   

  • Presenter Eric Swayze, Ph.D. - Vice President, Chemistry, Ionis Pharmaceuticals, Inc.

9 am 9:30 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Modified Aptamers As a New Generation of Nucleic Acid Therapeutics

We have recently developed a new generation of aptamers modified at the 5-position of pyrimidine nucleotides with enhanced affinity, slow dissociation rates, and intrinsically high nuclease resistance.    For therapeutics, we have focused on extracellular targets in indications where unique properties of these molecules have distinct advantages as drug candidates.

  • Presenter Dan Drolet - Senior Director, Pharmacology, SomaLogic, Inc.

9:30 am 10 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Reduced in vivo Tumor Grown Using CAR T-cells Treated ex vivo with PD-1 Targeting sd-rxRNA®

Silencing of immunosuppressive genes, such as PD-1, by ex vivo treatment with a self-deliverable siRNA (sd-rxRNA) may improve the efficacy of CAR-T cells for the treatment of solid tumors. Meso-CAR-T cells (expressing antibody fragments that target mesothelin) were treated ex vivo with an anti-PD-1 sd-rxRNA. Direct tumor injection of these modified CAR-T cells in a mouse model of human ovarian cancer (expressing mesothelin and PD-L1) results in a statistically significant reduction of tumor growth.

  • Presenter Karen Bulock, Ph.D. - Vice President Research, RXi Pharmaceuticals Corp.

8:25 am 8:30 am (5 mins)

Peptide Chemistry Manufacturing and Controls

Chairman’s Remarks

  • Chairman Alex Fässler, Ph.D. - Chief Operating Officer, Europe, Bachem AG

8:30 am 9 am (30 mins)

Peptide Chemistry Manufacturing and Controls

High Quality Starting Materials: One Important Key to Success

The focus on starting materials for SPPS increased significantly. A holistic approach for high starting material quality to ensure high API quality will be presented. The methodology comprises analytical methods based on the manufacturing processes of amino acid derivatives. Furthermore, a deep inside knowledge of starting material impurities is important to understand the origin of API impurities. Tight specifications and reliable sources will guarantee that the established control strategy remains effective.

  • Presenter Dirk Bächle, Ph.D. - Director Quality Control II, Bachem

9 am 9:30 am (30 mins)

Peptide Chemistry Manufacturing and Controls

Characterization of Starting Resins and Resin Properties

  • Presenter Brad DeHoff, Ph.D. - Director, R&D Strategy, Corden Pharma Colorado Inc

9:30 am 10 am (30 mins)

Peptide Chemistry Manufacturing and Controls

Regulatory Change Control for Peptide Starting Materials: A Case Study

Amino acid derivatives (AAD) are used as starting materials for the synthesis of peptide APIs. Therefore, AAD quality is critical and should be thoroughly established and controlled. Besides incoming material testing a key element for consistently ensuring AAD quality is the qualification of the supply chain including change control for the AAD manufacturers and their manufacturing processes. In addition to these quality and GMP expectations, the AAD manufacturers must be registered with the regulatory authorities. In most regions post-approval changes of manufacturer will require pre-approval by the authority. A case study will be presented demonstrating how authority requirements for changes of AAD manufacturers can be met. The associated implications and consequences will be discussed. 

  • Presenter Gerhard Haas, Ph.D. - VP Quality Assurance and Regulatory Affairs, Bachem AG

8:25 am 8:30 am (5 mins)

Peptide Discovery, Preclinical and Clinical

Chairman’s Remarks

  • Chairman John Nuss, Ph.D. - Vice President, Drug Discovery, Ferring Research Institute

8:30 am 9 am (30 mins)

Peptide Discovery, Preclinical and Clinical

Proteomics Methods to Discover Bioactive Peptides and Small Proteins

Proteomics and genomics methods have been combined to identify many novel protein-coding genes in the human genome. These genes encode peptides and small proteins, which we refer to as microproteins, and contain members with fundamental cellular activities. This lecture will focus on methods to discover and characterize these novel genes.

  • Presenter Alan Saghatelian, Ph.D. - Professor, Salk Institute for Biological Studies

9 am 9:30 am (30 mins)

Peptide Discovery, Preclinical and Clinical

Recombinant Expression of Protein and Peptide Therapeutics with Multiple Non-Natural Amino Acids

Synthorx is a drug discovery company that has commercialized a semi-synthetic DNA alphabet to develop a recombinant discovery and manufacturing system for proteins with multiple non-natural amino acids.  The ability to produce recombinant protein therapeutics of any size with multiple, distinct non-natural amino acids allows for the development of a pipeline of new therapeutics that have not been accessible using existing technologies. We will discuss the features and performance of our system for novel therapeutic protein production. 

  • Presenter Jerod Ptacin, Ph.D. - Principal Scientist, SynthoRx

9:30 am 10 am (30 mins)

Peptide Discovery, Preclinical and Clinical

The Application of Assembly Line Methodologies to the Development and Manufacture of Complex Synthetic Oligomers

Swedish Biomimetics has developed and proven µLOT, the world’s first continuous multi-stage peptide synthesis technology. It offers scope for faster development, lower cost of goods and a simplified regulatory approval process. This will be the first public presentation of this technology, whose potential has already generated significant commercial interest.

Speaker for this Presentation: 

Kimon Roussopoulos, Ph.D.Head of Research and Development, Swedish Biomimetics

Additional Author of the Work That Will be Discussed in This Presentation:

Andrew Prewer, Chief Scientist, Swedish Biomimetics 

  • Presenter Kimon Roussopoulos, Ph.D. - Head of Research and Development, Swedish Biomimetics 3000 Ltd.

10 am 10:45 am (45 mins)

Main agenda

Networking Refreshment Break in Poster and Exhibit Hall

10:44 am 10:45 am (1 mins)

mRNA Therapeutics

Chairperson’s Remarks

  • Örn Almarsson - Head of Formulations, Moderna Therapeutics

10:45 am 11:15 am (30 mins)

mRNA Therapeutics

Preclinical and Clinical Development of mRNA Therapeutics

  • Presenter Örn Almarsson - Head of Formulations, Moderna Therapeutics

11:15 am 11:45 am (30 mins)

mRNA Therapeutics

Cancer Immunotherapy: Individualized mRNA-based Vaccines for the Treatment of Cancer

Cancer, a primary cause of mortality with high unmet medical need, arises from the accumulation of genomic mutations and epigenetic changes that constitute a hallmark of cancer. The talk will focus on the current approaches to use these hallmarks for the generation of mRNA-based cancer vaccines modulating the immune response to specifically target the cancer of an individual patient.

  • Presenter Matthias Miller, Ph.D. - Project Manager, BioNTech AG

11:45 am 12:15 pm (30 mins)

mRNA Therapeutics

Overview of RaNA's mRNA Up-regulation Platform and mRNA Technology

  • Thomas McCauley, Ph.D. - Chief Scientific Officer, RaNA Therapeutics

12:15 pm 12:45 pm (30 mins)

mRNA Therapeutics

Ex vivo and in vivo Modification of Dendritic Cells with mRNA

Modification of dendritic cells (DC) with mRNA allows their loading with tumor antigens and their functional programming. To reprogram immature DC towards potent antigen (Ag) presenting cells, we provide 3 molecular adjuvants: mRNA coding for: caTLR4, mimicking TLR-4 activation; CD40L, mimicking ‘licensing’ of DCs and CD70 to provide an extra stimulus for the priming of CD8+ T cells. The mixture of these three mRNA’s is referred to as ‘TriMix’. mRNA encoding the full-length tumor specific or associated antigens is used to direct the immune system to the desired targets. Dendritic cells modified ex vivo have been used in numerous clinical trials. But mRNA can also be used directly, i.e. by injection of naked mRNA in vivo to reprogram and load the professional antigen presenting cells. Preclinical and clinical mRNA based immunization studies will be discussed.

  • Presenter Kris Thielemans, M.D., Ph.D. - Chief Scientific Officer, eTheRNA immunotherapies NV

10:45 am 11:15 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

GalXC cGMP Manufacturing Experience

  • Presenter Emma Wright, D.Phil. - Senior Director, Manufacturing, Dicerna Pharmaceuticals, Inc.

11:15 am 11:45 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Strategies for Scaling, Optimizing and Understanding the Manufacture of GalNAc-Conjugated Oligonucleotides

  • Presenter Chris Shaffer - Associate Director, Pharmaceutical Development, Regulus Therapeutics

11:45 am 12:15 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

PANEL DISCUSSION: Qualification of Impurities for Oligonucleotides

  • Moderator Lubomir Nechev, Ph.D. - Vice President, Process Sciences, Alnylam Pharmaceuticals
  • Panelist Claus Rentel, Ph.D. - Executive Director Analytical Development QC, Ionis Pharmaceuticals, Inc.
  • Panelist René Thürmer, Ph.D. - Deputy Head, Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices
  • Panelist Mike Webb, Ph.D. - Vice President, Manufacturing, Atlantic Healthcare
  • Panelist Louis Diorazio, Ph.D. - Principal Scientist, Chemical Development, AstraZeneca

12:15 pm 12:45 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Sequence Fidelity of Chemical Oligonucleotide Synthesis

  • Presenter Hüseyin Aygün, Ph.D. - Chief Scientific Officer, BioSpring GmbH

10:45 am 11:15 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Locked Nucleic Acid: Enabling RNA Therapeutics

RNA therapeutics has over the past decade developed from concept, clinic to market. However, the development has been hampered by a too simplistic approach to the underlying drug discovery principles resulting in unnecessary attrition rates during development. The lessons learned have resulted in new concepts to conduct drug discovery leading to increases in the productivity of RNA therapeutics. Locked Nucleic Acids are recognized as a preferred chemistry for RNA therapeutics and this will be presented in the context of lessons learned and LNA drug discovery case stories.

  • Presenter Maj Hedtjærn - Head of Drug Discovery, RNA Therapeutics Research, Roche Innovation Center Copenhagen

11:15 am 11:45 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

RNAi Structures in Animals: An RNAi Platform and Preclinical Update

  • Presenter Bob Brown, Ph.D. - Chief Scientific Officer, Senior Vice President, Research, Dicerna Pharmaceuticals

11:45 am 12:15 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

RNAi Based Human Therapeutics: From Discovery to Applications

This presentation will describe our progress in chemical modifications and delivery strategies (Lipid Nanoparticles and GalNAc Conjugates). A number of different applications will be discussed.

  • Presenter Muthiah (Mano) Manoharan, Ph.D. - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.

12:15 pm 12:45 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Oligonucleotides Targeting Periostin Ameliorates Pulmonary Fibrosis

In this study, we demonstrated that antisense oligonucleotides against periostin significantly decrease the levels of transforming growth factor-β1 and collagen and fibrotic score in the lungs from mice with lung fibrosis compared to control animals, suggesting that administration of periostin antisense may be an effective therapeutic approach for pulmonary fibrosis.

  • Presenter Esteban Gabazza, M.D., Ph.D. - Professor in Medicine and Chairman, Mie University Graduate School Of Medici

10:45 am 11:15 am (30 mins)

Peptide Chemistry Manufacturing and Controls

Downstream Process Controls and Aspects of the API Solid State

  • Presenter Jon Holbech Rasmussen, Ph.D. - Director Global Development, PolyPeptide Group, Belgium

11:15 am 11:45 am (30 mins)

Peptide Chemistry Manufacturing and Controls

Critical Quality Attributes for Activated PEGs

To successfully develop PEGylated biopharmaceuticals a holistic approach has to be employed involving the selection of the most appropriate PEG reagent available in different chemistries, sizes and shapes as well as an appropriate quality control strategy. The later preferably includes the pharmaceutically active protein or peptide, the PEG reagent and the final PEG-protein/peptide API. As a reliable supplier for the global life science industry, MilliporeSigma developed powerful analytical methods that are able to characterize the PEG reagents as well as the PEGylated proteins or peptides. Crucial quality parameters characterizing activated PEGs are the molecular mass, the polydispersity and the presence of non-activated and activated impurities. At least as important as those parameters is the additional implementation of a PEGylation use test verifying the applicability of the corresponding PEG reagents.

  • Raymond Behrendt, Ph.D. - Project Manager R&D, Merck

11:45 am 12:15 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Implementation of Appropriate cGMPs for Pre-Clinical Through Commercial Drug Substance

  • Presenter Gary Erickson, Ph.D. - CEO, CBL Biopharma LLC

12:15 pm 12:45 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Etelcalcetide: Overcoming Challenges in the Commercialization of a Late Development In-licensed Peptide Program

  • Presenter Asher Lower, Ph.D. - Senior Scientist, Pivotal Drug Substance Process Development, Amgen

10:45 am 11:15 am (30 mins)

Peptide Discovery, Preclinical and Clinical

Protein Engineering to Improve the Expression of Protein-Intein Fusions for Development of Protein-Peptide Conjugates

Expressed protein ligation uses self-splicing inteins to enable the production of site-specific protein conjugates. While widely used in research settings, less effort has been invested in developing an intein platform suitable for manufacturing of bioconjugate pharmaceuticals. We will present progress in engineering intein constructs to improve the expression and stability of the resulting proteins, with the goal of making commercial use of this technology feasible.

  • Presenter Alex Jacobitz - Postdoctoral Fellow, Amgen

11:15 am 11:45 am (30 mins)

Peptide Discovery, Preclinical and Clinical

Development of Cytotoxic Bicyclic Peptide Drug Conjugates and Applications in Molecular Targeted Cancer Therapy

The Bicycle ® platform allows hugely diverse libraries of constrained, bicyclic peptides (Bicycles®), generated with homo-trifunctional organochemical scaffolds, to be displayed on the surface of viable bacteriophage. These proprietary Bicycle libraries have been repeatedly screened against a range of targets, including receptors, interleukins, enzymes and serine proteases, using the "Bicycle process"; which involves sequential rounds of evolution-driven iterative phage display selections. The output from these selections are high affinity peptides, which typically deliver exquisite target specificity unachievable with conventional small molecule approaches, while retaining cross species activity. The presentation will describe and exemplify the Bicycle® platform and highlight the power of the technology by describing its application to identify drugs for the future treatment of oncology. Specifically by describing a case study, namely the identification and development of the Bicyclic Peptide Drug Conjugate BT1718.

  • Presenter Dan Teufel, Ph.D. - Head of Chemistry, Bicycle Therapeutics Limited

11:45 am 12:15 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Development of Macrocyclic Peptides for the Treatment of Complement-Mediated Disorders

  • Presenter Alonso Ricardo - Senior Vice President, Research, Ra Pharmaceuticals

12:15 pm 12:45 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Invention of Grazoprevir: A Hepatitis C Virus NS3/4a Protease Inhibitor

  • Presenter John McCauley - Vice President, Transportation and Logistics, Cargill Asia Pacific Holdings

12:45 pm 1:55 pm (70 mins)

Main agenda

Networking Luncheon in Poster and Exhibit Hall

1:45 pm 1:50 pm (5 mins)

Peptide Discovery, Preclinical and Clinical

Chairman's Remarks

  • Waleed Danho, Ph.D. - Distinguished Research Leader and Consultant for Peptides, Member of the Scientific Advisory Board

1:50 pm 2:15 pm (25 mins)

Peptide Discovery, Preclinical and Clinical

Half-Life Extended NaV1.7 Inhibitory Peptide-Antibody Conjugate Biodistributes to Nerve

We report on NaV1.7 inhibitory peptide-antibody conjugates as a novel modality for channel blockade. Venom-derived peptides inhibit NaV1.7 but suffer from a short half-life in vivo. We describe the site-specific conjugation of potent, selective GpTx-1 peptide analogs to an engineered cysteine residue in a carrier monoclonal antibody. The conjugates blocked NaV1.7 currents, extended in vivo half-life by 130-fold, and biodistributed to nerves in mice.

  • Justin Murray, Ph.D. - Senior Scientist, Hybrid Modality Engineering, Amgen, Inc.

2:15 pm 2:40 pm (25 mins)

Peptide Discovery, Preclinical and Clinical

Discovery of Selepressin and Merotocin, Two Clinical Candidates from Neurohypophyseal Family of Hormones

The peptidic hormones oxytocin and vasopressin display numerous biological actions mediated by closely related receptors, OTR, V1aR, V1bR, and V2R. We have recently nominated two oxytocin/vasopressin-related clinical candidates: selepressin, a selective V1aR agonist for treatment of vasodilatory hypotension and merotocin, a selective OTR agonist for lactation support. These programs exemplify the challenges associated with developing peptide therapeutics. Highlights of SAR studies and selected pharmacodynamic and pharmacokinetic properties will be presented.

  • Presenter Kazimierz Wisniewski, Ph.D. - Senior Scientist II, Ferring Research Institute

2:40 pm 3:05 pm (25 mins)

Peptide Discovery, Preclinical and Clinical

Setmelanotide for the Treatment of Rare Monogenic Forms of Obesity: The MC4-Pathway

  • Presenter Lex Van der Ploeg, Ph.D. - Chief Scientific Officer, Rhythm Pharmaceuticals

3:05 pm 3:30 pm (25 mins)

Peptide Discovery, Preclinical and Clinical

Elastin-Like Polypeptide Biopolymers Enhance the Pharmacology of Therapeutic Peptides

ELP fusion proteins undergo a fully reversible, temperature-dependent phase transition. ELP fusions with a transition temperature slightly below body temperature can form a coacervate at the subcutaneous injection site, from which the protein is slowly released. PhaseBio’s clinical pipeline includes PB1046, a long-acting VIP-ELP fusion for treatment of cardiopulmonary diseases.

  • Presenter Jim Ballance, Ph.D. - VP Research & Scientific Affairs, PhaseBio Pharmaceuticals, Inc.

1:55 pm 2 pm (5 mins)

Peptide Chemistry Manufacturing and Controls

Chairman’s Remarks

  • René Thürmer, Ph.D. - Deputy Head, Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices

2 pm 2:30 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Ferring QbD Approach to Peptide APIs in Development

Quality by Design (QbD) is a Systematic Approach to Development. Ferring’s approach to QbD embraces the principles outlined in ICH Q8(R2), Q9, Q10 and Q11. This talk will provide practical examples to illustrate how QbD principles and tools are applied by Ferring for peptide API development.

  • Presenter Mette Bryder, M.D. - QbD Faciliatator, Ferring Pharmaceuticals A/S

2:30 pm 3 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Regulatory Perspectives on Characterization of Quality Attributes of Therapeutic Peptides

From an analytical and regulatory perspective peptides are interesting since they present a link between products derived from biotechnology and the small molecular chemical compounds.  What to control is one of the key questions in connection with regulatory submissions during clinical development and marketing authorization applications and identifying critical quality attributes (CQAs) is vital during pharmaceutical development. The presentation will address the control strategy for synthetic peptides. Requirements for starting materials, manufacturing, characterization, specifications and analytical testing will be discussed.

  • Presenter René Thürmer, Ph.D. - Deputy Head, Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices

3 pm 3:30 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Peptide Synthesis Using Our Coupling Agent AllessanCap® with Case Study

  • Presenter Mimoun Ayoub, Ph.D. - Director, Global Peptides, Oligonucleotides, Lipids, Carbohydrates & Injectables Platforms, CordenPharma International, Switzerland

1:55 pm 2 pm (5 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Chairman’s Remarks

  • Chairman Emma Wright, D.Phil. - Senior Director, Manufacturing, Dicerna Pharmaceuticals, Inc.

2 pm 2:30 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Liquid Phase Oligonucleotide Synthesis with Membrane Separation for Efficient Large Scale Manufacturing

We introduce a liquid phase oligonucleotide synthesis (LPOS) strategy that rivals classical Merrifield solid phase synthesis (SPOS) for both yield and purity, but unlike SPOS is readily scaled. After each chain extension membrane separation is used to purify the growing oligo. To enhance the separation, three oligos are linked to a central tri-star hub. This enables LPOS in standard chemical process plant; in-process reaction monitoring is facile; and building block usage is minimized. The technical and economic potential of this approach will also be described.

  • Presenter Piers Gaffney, Ph.D. - Engineering Chemist, Imperial College London

2:30 pm 3 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

New Purification Process for ASOs

  • Presenter Robert Gronke, Ph.D. - Senior Principal Scientist, Technical Development, Biogen, Inc

3 pm 3:30 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

AJIPHASE®: Novel Solution Phase Approach for Oligonucleotides

A new technology of large scale synthesis for oligonucleotides has been strongly demanded to establish a steady procurement strategy of drug substances, and AJIPHASE® technology has been proven as a practical method to solve the issue. We will update our track record for oligonucleotides.

  • Presenter Hideaki Sato - General Manager, Technical Support and Marketing, GeneDesign, Inc., Japan

1:55 pm 2 pm (5 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Chairman’s Remarks

  • Chairman Arthur Levin, Ph.D. - Executive Vice President R&D, Avidity Biosciences LLC

2 pm 2:30 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Strategies to Optimize Oligonucleotide Safety in Drug Discovery and Candidate Selection

It is well established that some oligonucleotide therapeutics cause toxicities which may be limiting to their clinical development. At Roche pRED, we developed a set of predictive, human relevant in vitro tests to optimize the systemic safety of new oligonucleotide therapeutics. Application of these tests during drug discovery allows for an efficient, parallel optimization of safety and efficacy and may lead to clinical candidates with an overall improved safety profile. The presentation will provide an overview of these test systems, their validation and application in oligonucleotide discovery and candidate selection.

  • Presenter Franz Schuler, Ph.D. - Head of Drug Disposition & Safety, Pharm Sciences, F. Hoffmann-La Roche Ltd

2:30 pm 3 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Cobitolimod, a Novel Immunomodulatory Oligonucleotide Entering Clinical Phase IIb for Local Treatment of Active Ulcerative Colitis

  • Christine Dieterich Johansson, Ph.D. - Senior Project Manager CMC and Pre-clinical, InDex Pharmaceuticals

3 pm 3:30 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Assessment of the Effects of 2′-Methoxyethyl Antisense Oligonucleotides on Platelet Count in Cynomolgus NHPs

  • Presenter Scott Henry, Ph.D. - Vice President of Nonclinical Development, Ionis Pharmaceuticals, Inc.

1:55 pm 2 pm (5 mins)

mRNA Therapeutics

Chairman’s Remarks

  • Nigel Horscroft - Vice President Development RNArt, CureVac AG

2 pm 2:30 pm (30 mins)

mRNA Therapeutics

Targeted mRNA Delivery to the Liver for Intracellular Enzyme Replacement Therapy (i-ERT)

  • Presenter Michael Houston, Ph.D. - Chief Scientific Officer, PhaseRx

2:30 pm 3 pm (30 mins)

mRNA Therapeutics

Therapeutic mRNA delivery: Into Hepatocytes and Beyond

Functional delivery of RNA payloads to target cells present a major hurdle in the development of nucleic acid therapeutics. We set up an unbiased screening process for LNPs by applying mRNA aiming for evaluation of biodistribution and efficiency. Subsequent characterization of a selected liver targeting LNP with siRNA identifies various cell types to be targeted. Additionally, we demonstrate long-lasting reduction of therapeutically relevant proteins, in vivo by co-delivering Cas9-mRNA and synthetic guide-RNA with this LNP.

  • Presenter Adrien Weingärtner, Ph.D. - Group Leader mRNA, Silence Therapeutics

3 pm 3:30 pm (30 mins)

mRNA Therapeutics

RNAntibody® - A Potent mRNA Technology for Therapeutic Antibodies

An important field for protein therapies is the production of recombinant antibodies. We demonstrate that mRNA technology can be used to produce various antibodies in vivo. In animals, either formulated or non-formulated mRNA leads to antibody titers sufficient to provide protection in various challenge models. Thus RNAntibody® technology provides a potent means for passive immunization as well as for the delivery of other therapeutic antibodies.

  • Presenter Nigel Horscroft - Vice President Development RNArt, CureVac AG

3:30 pm 4:15 pm (45 mins)

Main agenda

Networking Refreshment Break in Poster and Exhibit Hall

4:15 pm 4:45 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Therapeutic mRNA: Manufacturing and Regulatory Considerations

Messenger (m)RNA is increasingly investigated as a platform technology for multiple therapeutic applications. My presentation will cover GMP manufacturing of therapeutic mRNA as well as applicable regulatory guidelines. As an exemplary application, the focus will be on delivering the genetic information of antigens into professional antigen-presenting dendritic cells for immunotherapies against cancer.

  • Presenter Andreas Kuhn - Vice President RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH, Germany

4:45 pm 5:15 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Case Study of Bioanalytical Approaches for Assessing Biodistribution of Modified mRNA Therapeutics

Oligonucleotide therapeutics offer a novel way for the Pharmaceutical Industry to ameliorate disease. By targeting the intracellular machinery, the use of these new drug entities can manipulate protein production to the benefit of a patient. AstraZeneca (AZ) is currently collaborating with Moderna Plc to develop chemically modified messenger RNA (mRNA) drug entities as potential therapeutics for several disease areas. These projects are extremely diverse and have different bioanalytical requirements & challenges. Both PK and PD measurements (of the modified mRNA and the encoded protein, respectively) need to be suitably assessed. Moreover, mRNA can be susceptible to circulating nuclease enzymes in blood so may need to be encapsulated (e.g. within lipid nanoparticles, LNPs) for increased protection. As such, the bioanalytical requirements for a single modified mRNA (preclinical) study may include the mRNA, the protein product and the LNP plus its’ breakdown products (e.g. cationic lipids) in numerous tissues & matrices. Understanding the holistic distribution and effects of these therapeutics & their constitutive parts is a key step in the path to creating safe & tolerable medicines. This presentation will highlight a recent case study assessing the biodistribution of an administered modified mRNA therapeutic, including the analytical strategies & techniques used.

  • Presenter Neil Henderson, Ph.D. - Associate Principal Scientist, AstraZeneca

5:15 pm 5:45 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Biochemical, Biophysical and Cellbased Characterization of mRNA, A Novel Drug Modality

There are numerous CMC challenges associated with the development of any novel therapeutic. One thing that differentiates mRNA from non-coding small RNAs are the challenges associated with characterizing long coding mRNAs. This presentation will discuss some of the challenges associated with biochemical, biophysical and cell based characterization of mRNAs. The biochemical and biophysical properties of mRNAs will be compared and contrasted against two other modalities, small non-coding RNAs and proteins.

  • Presenter Kristian Link, Ph.D. - Senior Manager, Analytical Development, Moderna Therapeutics

4:15 pm 4:45 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Selection of Well-Tolerated GalNAC-siRNAs by Screening for RNAi-mediated Off-Target Effects in Rodent Toxicity Studies

  • Presenter Maja Janas, Ph.D. - Senior Scientist, Investigative Toxicology, Alnylam Pharmaceuticals

4:45 pm 5:15 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Recent Advances in the Design and Delivery of microRNA-based Therapeutics

The role of microRNAs in regulating the expression of genes has made them attractive targets for therapeutic intervention. Regulus is developing a portfolio of oligonucleotide therapeutics that can modulate the activity of microRNAs, including both “anti-miRs” to block microRNA function and “miR-mimics” that can supplement the activity of endogenous microRNAs. We have established a robust platform for the design and discovery of anti-miR therapeutics, and have made significant progress in improving the design and functional delivery of miR-mimics. Regulus has identified several strategies to enhance delivery to specific tissues or cell types, whether through the appropriate placement of nucleotide and backbone modifications or by way of conjugation to targeting ligands. Recent advances in microRNA therapeutic technology, including an overview of compounds in development, will be discussed.

  • Presenter Charles R. Allerson, Ph.D. - Director, Chemistry, Regulus Therapeutics

5:15 pm 5:45 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Enhancing ASO Activity in Extra- Hepatic Tissues

  • Presenter Punit P. Seth, Ph.D. - Executive Director, Medicinal Chemistry, Ionis Pharmaceuticals

4:15 pm 4:45 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Single to Multiple Disulfide Bridgerich Peptides: A Perpetually Evolving Approach in Process Development and Scale-Up

The large abundance of bioactive single and multiple cystine-rich scaffold peptides in nature has fostered the pharma and biotech industry to move from exploration to exploitation of those complex molecules. Over the last decade, the number of disulfide bridges in peptides evaluated in clinical studies has continuously been growing, leading the manufacturers to deeply reconsider their approach of chemical route / process selection and scale-up. We propose an overview of our strategy for setting an optimized, safe and cost effective process of single / multiple disulfide bridge peptides.

  • Presenter El Djouhar Rekaï, Ph.D. - Head of Operation Products, PolyPeptide Group

4:45 pm 5:15 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Insights from PolyPeptide Group: Impurities in Peptide Manufacturing

For any API the understanding and control of the impurity profile is of paramount importance. The impurity profile of an API is the rather complex result of the API itself, including storage, stability and salt selection, the processing decisions, and the selection and the quality of the starting materials. With examples, this presentation will show that following a systematic approach it is possible to predict and eventually control the impurity profile effectively. Ultimately, the use of such approaches in the development of an API are extremely valuable for making the best informed decisions at the right time during the development.

  • Presenter Jon Holbech Rasmussen, Ph.D. - Director Global Development, PolyPeptide Group, Belgium

5:15 pm 5:45 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

LC-MS, A Key Technique to Understanding the Fate of Impurities in Ferring’s Synthetic Therapeutic Peptides

LC-Ms is the key technique used to support the understanding of the fate of impurities. Impurities can originate from the starting materials or can be formed during the synthesis and in the final purification steps. Several examples from Ferring’s synthetic therapeutic peptide portfolio will be discussed to show the complexity when working with synthetic therapeutic peptides and fate of impurities.

  • Presenter Jörgen Kjellgrenn Sjögren, Ph.D. - Senior Research Scientist, Ferring Pharmaceuticals A/S

4:15 pm 4:45 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Discovery and Development of Novel Macrocycle Drugs: Outer Membrane Protein Targeting Antibiotics (OMPTA), A New Class with a Novel Mode of Action

Polyphor has established two complementary, fully synthetic and proprietary macrocycle technologies: The presentation will cover the most recent developments and data from our break-through antibiotic research on a new series of macrocyclic peptides that have outstanding broad-spectrum activity against the most serious and relevant Gram-negative ESKAPE pathogens involved in hospital and community acquired infections. We have recently shown exceptional in vitro activity on the most recently emerging antibiotic-resistant strains, and in animal model studies we have shown excellent in vivo efficacy against relevant bacterial strains, including resistant strains. We will also give an update on our lead antibiotic Murepavadin (POL7080), which is a narrow-spectrum, targeted therapy for Pseudomonas infections in critically ill patients in the ICU. POL7080 has successfully completed Phase II and we are preparing the path forward for a Phase III registration trial.

  • Presenter Daniel Obrecht, Ph.D. - Chief Scientific Officer, Co-Founder, Polyphor Ltd.

4:45 pm 5:15 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Dasiglucagon (ZP4207) Single-Dose Rescue Treatment for Acute, Severe Hypoglycemia Events, Phase II Update

Dasiglucagon is a novel analogue of human glucagon in clinical development for use in a ready-to-use rescue pen for the treatment of hypoglycaemia and as an essential component in an insulin-glucagon dual hormone pump (the ‘artificial pancreas’) an important advancement in the treatment of Type 1 diabetes.

  • Presenter Andrew Parker, Ph.D. - CSO & SVP, Head of Research & External Innovation, Zealand Pharma A/S

5:15 pm 5:45 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Clinical Development of Elamipretide for Rare and Common Diseases

Age-related decline in mitochondrial function and bioenergetics failure underlie many chronic diseases. Certain mitochondria-targeted tetrapeptides have been found to protect mitochondrial structure and promote cellular bioenergetics. Elamipretide is currently in clinical development for rare mitochondrial genetic diseases (myopathy, LHON, Barth Syndrome) and chronic diseases (heart failure, macular degeneration, muscle weakness).

  • Presenter Hazel Szeto, M.D., Ph.D. - Scientific Founder, Stealth Biotherapeutics

4:15 pm 4:45 pm (30 mins)

mRNA Therapeutics

Therapeutic mRNA: Manufacturing and Regulatory Considerations

  • Presenter Andreas Kuhn - Vice President RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH, Germany

4:45 pm 5:15 pm (30 mins)

mRNA Therapeutics

Case Study of Bioanalytical Approaches for Assessing Biodistribution of Modified mRNA Therapeutics

Oligonucleotide therapeutics offer a novel way for the Pharmaceutical Industry to ameliorate disease. By targeting the intracellular machinery, the use of these new drug entities can manipulate protein production to the benefit of a patient. AstraZeneca (AZ) is currently collaborating with Moderna Plc to develop chemically modified messenger RNA (mRNA) drug entities as potential therapeutics for several disease areas. These projects are extremely diverse and have different bioanalytical requirements & challenges. Both PK and PD measurements (of the modified mRNA and the encoded protein, respectively) need to be suitably assessed. Moreover, mRNA can be susceptible to circulating nuclease enzymes in blood so may need to be encapsulated (e.g. within lipid nanoparticles, LNPs) for increased protection. As such, the bioanalytical requirements for a single modified mRNA (preclinical) study may include the mRNA, the protein product and the LNP plus its’ breakdown products (e.g. cationic lipids) in numerous tissues & matrices. Understanding the holistic distribution and effects of these therapeutics & their constitutive parts is a key step in the path to creating safe & tolerable medicines. This presentation will highlight a recent case study assessing the biodistribution of an administered modified mRNA therapeutic, including the analytical strategies & techniques used.

  • Presenter Neil Henderson, Ph.D. - Associate Principal Scientist, AstraZeneca

5:15 pm 5:45 pm (30 mins)

mRNA Therapeutics

Biochemical, Biophysical and Cell-based Characterization of mRNA, A Novel Drug Modality

There are numerous CMC challenges associated with the development of any novel therapeutic. One thing that differentiates mRNA from non-coding small RNAs are the challenges associated with characterizing long coding mRNAs. This presentation will discuss some of the challenges associated with biochemical, biophysical and cell based characterization of mRNAs. The biochemical and biophysical properties of mRNAs will be compared and contrasted against two other modalities, small non-coding RNAs and proteins.

  • Presenter Kristian Link, Ph.D. - Senior Manager, Analytical Development, Moderna Therapeutics

5:45 pm 6:45 pm (60 mins)

Main agenda

Networking Reception in Poster and Exhibit Hall