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Key Sessions

Chad Mirkin, PhD

FEATURED PRESENTATION: Rational Vaccinology: Structure-Function Relationships in the Development of Immunotherapeutic Agents

Northwestern University

7:00am - 7:45am

Registration and Coffee

Showing of Streams
10:00am - 10:45am

Networking Refreshment Break in Poster and Exhibit Hall

Showing of Streams
12:15pm - 12:20pm
Transition to Spotlight Presentation Rooms

Transition to Spotlight Presentation Rooms

Showing of Streams
12:50pm - 1:55pm

Networking Luncheon in Poster and Exhibit Hall

Showing of Streams
5:45pm - 6:45pm

Networking Reception in Poster and Exhibit Hall

7:00am - 7:45am 45 mins
Registration and Coffee
7:45am - 8:15am 30 mins
Breakfast Spotlight Presentation 1
Readiness to One-stop Manufacturing Service for Oligonucleotides and Phosphoramidites for Commercial Scale
  • Kyeong Eun Jung, PhD - Senior Vice President, Head of Oligo and R&D Division, ST Pharm
more
7:45am - 8:15am 30 mins
Breakfast Spotlight Presentation 2
GMP Liposome Production from Microfluidics to Large-scale
  • Charalampos Koutsoulas, PhD - Head of Liposome R&D, Polymun Scientific GmbH
more

Polymun Scientific GmbH is a pioneer in the formulation and large-scale production of liposomal dosage forms. Various oligonucleotides such as siRNA, saRNA, DNA and numerous mRNAs have been formulated using Polymun's proprietary solvent injection technology. Additional to nucleic acids, various small molecular weight compounds and various liposomal vaccine products have been successfully produced by Polymun's fully scalable solvent injection technology. Polymun is also specialized in manufacturing of pre-clinical and clinical material of cGMP quality with its robust, innovative solvent injection technology.

8:00am - 8:05am 5 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Chairperson's Welcome and Opening Remarks
  • Chairperson Shuling Guo, PhD - Executive Director, Ionis Pharmaceuticals, Inc.
more
8:25am - 8:30am 5 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Chairperson's Welcome and Opening Remarks
  • Chairperson Doug Brooks, PhD - Consultant, Matapalo Pharma Consulting Services
more
8:25am - 8:30am 5 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Chairperson's Welcome and Opening Remarks
  • Chairperson Alex Fässler, PhD - Chief Operations Officer, Bachem Holding AG
more
8:25am - 8:30am 5 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Chairperson's Welcome and Opening Remarks
  • Chairperson Rami Hannoush, PhD - Principal Scientist and Group Leader, Genentech
more
8:30am - 9:00am 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Oligo Market Expansion and Responding to the Capacity and Technological Challenges
  • Joe Guiles, PhD - Head of Development, Agilent Technologies, Inc.
more
8:30am - 9:00am 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Higher Molecular Weight (HMW) Peptide Impurities - Control Strategies and Acceptance Criteria
  • Ralph Schönleber, PhD - Vice President for Research & Development, Bachem AG
more

Focusing on typical process stages, BACHEM's standard approach for optimization of process parameters regarding aggregation is presented within different case studies. This involves consideration of suitable aggregation screening techniques based on light scattering, turbidity and fluorescence spectroscopy. In addition, analytical methods suitable for control of HMW (such as size-exclusion chromatography, gel electrophoresis, flow microscopy) as well as suggested acceptance criteria for release and IPCs are discussed.

8:30am - 9:00am 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Plants as Sources for Peptide Lead Discovery
  • David Craik, PhD - Professor of Biomolecular Structure, Institute for Molecular Bioscience, University of Queensland
more

Previously, plants have been extensively mined to discover small molecule drug leads. With recent advances in peptide characterization, plants have now become valuable sources of novel bioactive peptides. This presentation will focus on plant-derived cyclic peptides as ultra-stable scaffolds for drug design.

8:35am - 9:00am 25 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Develop Antisense Therapies for Genetic Diseases
  • Shuling Guo, PhD - Executive Director, Ionis Pharmaceuticals, Inc.
more

Antisense technology provides a direct route from genetic sequence to therapy. Antisense oligonucleotides (ASOs) can be delivered via systemic administration and local delivery into CNS, eye or lung. In this talk, I will discuss our efforts at Ionis in developing novel therapies for genetic diseases using antisense technology.

9:00am - 9:30am 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
From Discovery to the Clinic: Translational Strategies for Oligonucleotides
  • Patrick Haslett, MD - Executive Director of Clinical Research, Alnylam Pharmaceuticals
more
9:00am - 9:30am 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
White Paper on Oligonucleotide Impurities
  • Daniel Capaldi, PhD - Vice President Analytical and Process Development, Ionis Pharmaceuticals, Inc.
more
9:00am - 9:30am 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Producing the Highly Potent Peptide
  • Rick Dauer, PhD - Distinguished Engineer, Program Management Organization, Corden Pharma Colorado
more

Production of highly potent compounds presents a host of challenges. When combined with peptide production, a multidisciplinary team approach is required to deliver superior GMP quality product while maintaining the health and well-being of researchers and operations staff. This talk presents the strategy, approach and solutions employed at Corden Pharma Colorado when producing highly potent peptide drug substances.

9:00am - 9:30am 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Massively Parallel Synthesis and Screening of Linear Peptides and Macrocycles using Peptide Microarrays
  • Lauren Goodrich, PhD - Scientist, Technology Innovation, Roche
more

We have developed a peptide microarray synthesis and screening platform. Using a chemical catalog of over 300 amino acid building blocks, we synthesize 18 million unique linear or cyclic peptides in a 24-48 hour run on a microarray surface. 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.

9:30am - 10:00am 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
mRNA Therapies for Acute Regenerative and Chronic Approaches
  • Nils Bergenhem, PhD - Director, Strategy and External Innovation, AstraZeneca Pharmaceuticals
more

We are taking VEGF-A delivered via mRNA into clinical phase 2A development for cardiovascular disease and phase 1 for wound healing. Development of these acute treatments will be discussed as well as approaches for chronic delivery of mRNA therapeutics.

9:30am - 10:00am 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Carbon-Carbon Bond Formation on Peptides
  • Janos Kodra, PhD - Principal Scientist, Protein & Peptide Chemistry, Novo Nordisk A/S
more

Carbon-carbon bond construction is the basis for all of organic synthetic chemistry. The multifunctional nature of peptides has limited the toolbox of carbon-carbon bound forming reactions that currently are being used routinely in combination with peptide chemistry. The talk will focus on our work using N-heterocyclic carbenes to catalyse Carbon-Carbon bond formation on peptides. This represents a new approach to post-modify bioactive peptides as well as performing peptide ligation through Carbon-Carbon bond formation.

10:00am - 10:45am 45 mins
Networking Refreshment Break in Poster and Exhibit Hall
10:45am - 11:15am 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Development of RNAi-based Modulation of sFLT1 As a Novel Approach for Treatment of Preeclampsia
  • Anastasia Khvorova, PhD - Professor, RNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School
more

Preeclampsia (PE) is a placentally-induced hypertensive disorder that causes pregnancy-related morbidity and mortality. Clinical manifestations of PE result from excess circulating soluble vascular endothelial growth factor receptor FLT1 (sFLT1) of placental origin. Here we identify short interfering RNAs (siRNAs) that selectively silence three sFLT1 mRNA isoforms responsible for placental overexpression of sFLT1. When fully chemical stabilized and hydrophobically modified, these siRNAs accumulate in placenta and reduce circulating sFLT1 in pregnant mice. In a baboon model of PE, one dose of siRNAs suppressed sFLT1 overexpression and clinical symptoms of PE. Our results establish a path toward a new treatment paradigm for PE.

10:45am - 11:15am 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Manufacturing Process Development for PMO
  • Bao Cai, PhD - Director, Process Development, Sarepta Therapeutics
more

Manufacturing Process Development for PMO: From Discovery Research to Commercial ProductionEteplirsen (EXONDYS 51®) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved in 2016 under accelerated approval. Eteplirsen drug substance manufacturing process was developed applying quality by design approaches, focusing on risk analysis, impact assessment, and control strategy. To produce drug substance of suitable quality, the drug product quality target product profile (QTPP) was determined, and a list of the attributes of drug substance, critical to the intended quality of the drug product, was created. These critical quality attributes (CQAs) of the drug substance were then used to guide the development of a robust and controlled drug substance manufacturing process to meet the commercial demands. The early phase process was evaluated to identify critical process parameters in each unit operation and areas of improvement to reduce risk to CQAs while improving process performance and scalability. These improvements were developed and implemented to provide the commercial process. The drug substance quality was confirmed by a comparability study to demonstrate the CQAs were maintained.

10:45am - 11:15am 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Highly Pure Octreotide - Development and Scale-Up of a State-of-the-art SPPS Process
  • Stefan Eissler, PhD - Director, API Manufacturing, Bachem AG
more

In this case study the CMC development, scale-up and large scale manufacturing of Octreotide Acetate will be described. The talk will also consider the control strategy (starting material quality, in-process controls, API specifications). Process robustness is demonstrated comparing process parameters and drug substance quality attributes from process development and scale-up batches.

10:45am - 11:15am 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Engineering Potent NaV1.7 Inhibitory Peptide−Antibody Conjugates
  • Justin Murray, PhD - Principal Scientist, Hybrid Modality Engineering, Amgen, Inc.
more

The voltage-gated sodium channel NaV1.7 is a genetically validated pain target under investigation for the development of analgesics. We describe NaV1.7 inhibitory peptide−antibody conjugates as an alternate construct for potential prolonged channel blockade through chemical derivatization of engineered antibodies. Further improvements in potency have been achieved through the discovery of JzTx-V from the Chinese earth tiger tarantula Chilobrachys jingzhao and incorporation of selective analogs into peptide-antibody conjugates.

11:15am - 11:45am 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Stereochemical Control of Antisense Oligonucleotides Enhances Target Efficacy
  • Zhong Zhong, PhD - Vice President and Head of Biology, Wave Life Sciences
more


Oligonucleotide therapeutics need to reach tissues of interest in sufficient quantity to modulate their specific gene targets. With Wave’s proprietary chemistry platform, which allows for the precise design and control of the stereochemistry of oligonucleotides, we have evaluated how chirality influences the spectrum of antisense oligonucleotide (ASO) properties ranging from RNaseH activity, stability, immune activation, and tissue distribution. Using phenotypic assays in relevant human cell models mediated by gymnosis (ie, free uptake by cells without transfection agents), we screened for stereopure ASO compounds with target modulation. With chemistry and stereochemical control optimized, we were able to improve the potency and efficacy of ASOs in vitro. These lead molecules were then evaluated in appropriate animal models where human gene targets were expressed. This presentation will review data from three discovery programs targeting APOC3 in hepatocytes, MALAT1 in eye, and C9Orf72 in brain and spinal cord. Emerging evidence suggests that stereopure ASOs selected on the basis of gymnotic assay conditions also have better target efficacy in vivo, and this approach may offer a more stringent discovery filter for clinical candidates.


11:15am - 11:45am 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Antisense Oligonucleotide Purification Process: Successes and Challenges During Scale-up
  • Robert Gronke, PhD - Senior Principal Scientist, Technical Development, Biogen, Inc
more

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.

11:15am - 11:45am 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Purification of Peptides by Twin-column Countercurrent Chromatography
  • Thomas Müller-Späth, PhD - Senior Scientist, Institute for Chemical and Bioengineering, ETH Zurich
more

Continuous countercurrent chromatography is a valuable tool for the purification of peptides produced by chemical synthesis. Using a process comprising two reverse-phase columns and internal recycling (MCSGP), peptides are purified on a preparative scale with high yield and purity simultaneously while the generation of side-fractions and re-chromatography is avoided. The talk introduces the process concept and recent results of preparative peptide purification by countercurrent chromatography, such as a UV-based dynamic process control concept.

11:15am - 11:45am 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Emerging Approaches for Peptide Drug Discovery
  • Rami Hannoush, PhD - Principal Scientist and Group Leader, Genentech
more

This talk will describe our group's efforts on utilizing peptide-based scaffolds to enable the development of novel technologies for peptide drug discovery. The presentation will highlight some of the challenges and emerging technologies for peptide lead identification and development.

11:45am - 12:15pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Sustained Suppression of Huntingtin mRNA and Protein throughout the Central Nervous System after Intrathecal Administration of Antisense Oligonucleotides
  • Holly Kordasiewicz, PhD - Executive Director, Ionis Pharmaceuticals
more

Huntington's disease is a fatal dominantly inherited neurodegenerative disease, for which there is currently no cure. Since ASOs do not cross the blood brain barrier, CNS targets in patients can be accessed via direct delivery to the cerebral spinal fluid. I will present here a set of experiments determining the pharmacodynamics and pharmacokinetics of huntingtin mRNA-targeting ASOs in larger brains using the clinical route of delivery, intrathecal bolus injection.

11:45am - 12:15pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Scale Matters - The Importance of Designing Scalable Processes and Equipment
  • John Batal - Director of Engineering, Nitto Avecia
more

As the oligonucleotide market continues to mature it becomes increasingly important that sponsors and their partners work together to develop processes and equipment that can make an effective transition from small clinical scales to larger commercial demand. Experience, innovation, flexibility, and capacity are all factors that play significant roles in successfully building a robust commercial supply. Finding the right partners for growth is imperative for this growing market.

11:45am - 12:15pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Transitioning Manufacturing Development from Clinical Supply to Registration and Pre-Commercial Process from the Small Company Perspective
  • Dave Garman, PhD - Chief Technology Officer, NoNO, Inc.
more

There are significant differences in the manufacturing and testing requirements between supplying drug for clinical trials and registering a drug for market access. For small to mid size companies, these development expenses are often pushed to late stages when the risk of failure in clinical studies is lower. We examine the transition to commercial development of API manufacturing processes to mitigate risks of a FDA refusal to file in the context of our Phase 3 peptide therapeutic NA-1.

11:45am - 12:15pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Small Scaffold Proteins for Drug Discovery
  • John Dwyer - Director, Protein Engineering, Ferring Research Institute
more
12:15pm - 12:20pm 5 mins
Transition to Spotlight Presentation Rooms
12:20pm - 12:50pm 30 mins
New Insights into the Sequence Fidelity of Chemically Synthesized Oligonucleotides
  • Hüseyin Aygün, PhD - Managing Director / Chief Scientific Officer, BioSpring GmbH
more
12:20pm - 12:50pm 30 mins
Comparison between AJIPHASE® and Solid-phase Synthesis for Large Scale Oligonucleotide Manufacturing
  • Daisuke Takahashi, PhD - Senior Principal Researcher, Bio-functional, Research Institute for Bioscience Products and Fine Chemicals, Ajinomoto Co. Inc.
more

We have developed a practical synthetic method AJIPHASE® which retains scale up advantage for oligonucleotide synthesis using solution-phase approach. The efficacy of AJIPHASE® has been proven with the successful synthesis of various antisense oligonucleotides with the same impurity profiles as it of solid-phase synthesis. This presentation will cover the comparison in detail of AJIPHASE® and solid-phase synthesis for oligonucleotide procurement in large volume.

12:20pm - 12:50pm 30 mins
Update on USP Standards for Therapeutic Peptides and Impurity Measurements
  • Michael Huang, PhD - Senior Science & Standards Liaison, U.S. Pharmacopeia
more
12:50pm - 1:55pm 65 mins
Networking Luncheon in Poster and Exhibit Hall
1:55pm - 2:00pm 5 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Chairperson's Remarks
  • Chairperson Cindy Berman, PhD - Toxicology Consultant, Consultant
more
1:55pm - 2:00pm 5 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Chairperson's Remarks
  • Chairperson Cindy Berman, PhD - Toxicology Consultant, Consultant
more
1:55pm - 2:00pm 5 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Chairperson's Remarks
  • Chairperson Ved Srivastava, PhD - Vice President, Chemistry, Intarcia Therapeutics, Inc.
more
1:55pm - 2:00pm 5 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Chairperson's Remarks
  • Chairperson Ved Srivastava, PhD - Vice President, Chemistry, Intarcia Therapeutics, Inc.
more
1:55pm - 2:00pm 5 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
Chairperson's Remarks
  • Chairperson Andreas Kuhn, PhD - Vice President, RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH
more
1:55pm - 2:00pm 5 mins
Track 6: Delivery of Macromolecular Therapeutics
Chairperson's Remarks
  • Marian Gindy, PhD - Executive Director, Pharmaceutical Sciences, Merck Research Laboratories
more
2:00pm - 2:20pm 20 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Introduction of the Oligonucleotide Safety Working Group (OSWG)
  • Jeffrey Foy, PhD - Director, Nonclinical Writing & Documentation, Celgene
more
2:00pm - 2:20pm 20 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Introduction of the Oligonucleotide Safety Working Group (OSWG)
  • Jeffrey Foy, PhD - Director, Nonclinical Writing & Documentation, Celgene
more
2:00pm - 2:30pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Engineering "Smartness" into Insulin: Glycosylated Insulins with Responsivity to Physiological Levels of Glucose
  • Songnian Lin, PhD - Director, Chemistry Modalities, Merck Research Laboratories
more

"Smart" insulin with responsivity to changing glucose levels has long been the holy grail of insulin development. Novel glycosylated insulins were created which interact with both insulin receptor and an endogenous lectin. Proof of biology was successfully demonstrated at physiological glucose levels with these dual action compounds in vitro and in higher species.

2:00pm - 2:30pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Engineering "Smartness" into Insulin: Glycosylated Insulins with Responsivity to Physiological Levels of Glucose
  • Songnian Lin, PhD - Director, Chemistry Modalities, Merck Research Laboratories
more

"Smart" insulin with responsivity to changing glucose levels has long been the holy grail of insulin development. Novel glycosylated insulins were created which interact with both insulin receptor and an endogenous lectin. Proof of biology was successfully demonstrated at physiological glucose levels with these dual action compounds in vitro and in higher species.

2:00pm - 2:30pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
mRNA Therapeutics: Ex-vivo and In-vivo Modification of Antigen Presenting Cells
  • Kris Thielemans, MD, PhD - Chief Scientific Officer / Professor, eTheRNA immunotherapies NV / Vrije Universiteit Brussel
more

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 designed a set of molecules that mimic closely activation of these cells during the initiation of an adaptive immune response. We provide 3 molecular adjuvants: mRNA coding for a constitutive active variant of TLR4, mimicking TLR-4 activation; CD40L mRNA, mimicking the 'licensing' of DCs when Th cells and DCs interact and CD70 to provide an extra stimulus for the priming of CD8+ T cells and inducing their proliferation and survival. 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. To enhance a broad immune response and provide help to the CTLs, we ensure HLA-class-I and class-II presentation by modifying the antigen sequence by adding a DC-LAMP-derived lysosomal targeting sequence. Dendritic cells modified ex vivo have been used in numerous clinical trials. The results of the investigator initiated studies performed in Brussels for the treatment of melanoma will be presented. 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 in lymph nodes or at the tumor site. The latter approach is now being implemented in clinical studies.

2:00pm - 2:30pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Overcoming Endosomal Entrapment in Drug Delivery with Cyclic Cell-Penetrating Peptides
  • Dehua Pei, PhD - Professor, Department of Chemistry and Biochemistry, Ohio State University
more

Most biological modalities (e.g., peptides, proteins, and nucleic acids) are internalized by mammalian cells through endocytic mechanisms, leading to their entrapment inside the endosomes and lysosomes. We discovered a family of cyclic cell-penetrating peptides that effectively exit the early endosome and have >100% cytosolic delivery efficiency (compared to 2% for Tat). I will discuss the mechanism by which these cyclic CPPs escape from the endosomes and their applications in the cytosolic delivery of peptide, protein, and nucleic acid cargoes in vitro and in vivo.

2:20pm - 2:30pm 10 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Short Updates from OSWG Subcommittees
2:20pm - 2:30pm 10 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Short Updates from OSWG Subcommittees
2:30pm - 3:00pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
From Excitement to Despair to Exhilaration in 13 months - The Story of Arrowhead's Move from DPCs to the TRiM™ Platform
  • Thomas Schluep - Vice President, Program Management, Arrowhead Pharmaceuticals
more

DPC-related animal toxicity led to discontinuation of Arrowhead's DPC-related clinical programs in December, 2016. Fortunately, our new direct conjugate TRiM™ platform had proven itself in the Amgen licensed Lp(a) program. With modular design, we moved from crisis to new submissions for first-in-human testing of 2 drugs in just under 13 months.

2:30pm - 3:00pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
From Excitement to Despair to Exhilaration in 13 months - The Story of Arrowhead's Move from DPCs to the TRiM™ Platform
  • Thomas Schluep - Vice President, Program Management, Arrowhead Pharmaceuticals
more

DPC-related animal toxicity led to discontinuation of Arrowhead's DPC-related clinical programs in December, 2016. Fortunately, our new direct conjugate TRiM™ platform had proven itself in the Amgen licensed Lp(a) program. With modular design, we moved from crisis to new submissions for first-in-human testing of 2 drugs in just under 13 months.

2:30pm - 3:00pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Clinical Development of Oral Calcitonin for Osteoporosis
  • Nozer Mehta, PhD - Principal, Peptide Technologies LLC
more

Salmon calcitonin (sCT) is a 32 amino acid peptide hormone that inhibits osteoclasts and regulates serum calcium. It is currently marketed in injectable and nasal spray formulations. Studies on the oral delivery of calcitonin have been carried out with different technologies. Using the PeptelligenceTM technology, which enhances paracellular intestinal transport of the peptide, several early phase studies demonstrated a significantly increased absorption of sCT and a reduction in CTX-1, a marker for bone resorption. A global Phase III double-blind, placebo controlled trial in postmenopausal women with osteoporosis demonstrated that women randomized to oral sCT had a mean percent increase from baseline in lumbar spine bone mineral density (LS BMD) that was greater than those randomized to sCT nasal spray or placebo. A subsequent Phase II study of oral sCT for the prevention of postmenopausal osteoporosis was also successfully completed, and resulted in an increase in LS BMD, a reduction in total proximal femur BMD loss, and reduced serum CTx-1 in postmenopausal women with low bone mass and increased risk of fracture. The data from the clinical development program will be presented.

2:30pm - 3:00pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Clinical Development of Oral Calcitonin for Osteoporosis
  • Nozer Mehta, PhD - Principal, Peptide Technologies LLC
more

Salmon calcitonin (sCT) is a 32 amino acid peptide hormone that inhibits osteoclasts and regulates serum calcium. It is currently marketed in injectable and nasal spray formulations. Studies on the oral delivery of calcitonin have been carried out with different technologies. Using the PeptelligenceTM technology, which enhances paracellular intestinal transport of the peptide, several early phase studies demonstrated a significantly increased absorption of sCT and a reduction in CTX-1, a marker for bone resorption. A global Phase III double-blind, placebo controlled trial in postmenopausal women with osteoporosis demonstrated that women randomized to oral sCT had a mean percent increase from baseline in lumbar spine bone mineral density (LS BMD) that was greater than those randomized to sCT nasal spray or placebo. A subsequent Phase II study of oral sCT for the prevention of postmenopausal osteoporosis was also successfully completed, and resulted in an increase in LS BMD, a reduction in total proximal femur BMD loss, and reduced serum CTx-1 in postmenopausal women with low bone mass and increased risk of fracture. The data from the clinical development program will be presented.

2:30pm - 3:00pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
mRNA-based Cancer Immunotherapeutics
  • Robert Jabulowsky, PhD - Deputy Head of Project Management, BioNTech AG
more

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 the worldwide first systemic mRNA cancer vaccination trial will be presented.

2:30pm - 3:00pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Targeted Delivery of Antisense Oligonucleotides to Pancreatic β-cells
  • Shalini Andersson, PhD - Senior Director, Drug Metabolism & Pharmacokinetics, AstraZeneca
more

Conjugation of antisense oligonucleotides to GLP1R ligands represents a novel concept to target delivery of therapeutic oligonucleotides to GLP1R expressing cells. This opens the possibility to develop treatments of disease caused by or associated with aberrant gene expression in the pancreatic ß-cells whilst reducing the risk for off-target effects.

3:00pm - 3:30pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Improved Specificity of Conjugate siRNAs through Chemical Modifications
  • Mark Schlegel, PhD - Senior Scientist, RNAi Discovery, Alnylam Pharmaceuticals
more

We will present our design efforts towards to use of novel chemical modifications for the mitigation of off-target effects of our GalNAc conjugate siRNAs, including an assessment of the impact on potency, stability, and specificity in vivo.

3:00pm - 3:30pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Improved Specificity of Conjugate siRNAs through Chemical Modifications
  • Mark Schlegel, PhD - Senior Scientist, RNAi Discovery, Alnylam Pharmaceuticals
more

We will present our design efforts towards to use of novel chemical modifications for the mitigation of off-target effects of our GalNAc conjugate siRNAs, including an assessment of the impact on potency, stability, and specificity in vivo.

3:00pm - 3:30pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Designing Peptides for the Medici Drug Delivery SystemTM
  • Andrew Young, MD, PhD - Chief Scientific Officer, Intarcia Therapeutics, Inc.
more

The Medici Drug Delivery SystemTM, a miniature (44 x 4 mm) osmotic pump that is placed subcutaneously in an office procedure, delivers formulated peptides continuously for at least 6 months. The convenience of this system is anticipated to promote compliance. However, the necessarily limited drug payload imposes a high barrier on in vivo potency of the encapsulated drug. Peptides of such potency rarely exist in nature, and must be designed. Approaches to achieving such potency will be discussed.

3:00pm - 3:30pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Designing Peptides for the Medici Drug Delivery SystemTM
  • Andrew Young, MD, PhD - Chief Scientific Officer, Intarcia Therapeutics, Inc.
more

The Medici Drug Delivery SystemTM, a miniature (44 x 4 mm) osmotic pump that is placed subcutaneously in an office procedure, delivers formulated peptides continuously for at least 6 months. The convenience of this system is anticipated to promote compliance. However, the necessarily limited drug payload imposes a high barrier on in vivo potency of the encapsulated drug. Peptides of such potency rarely exist in nature, and must be designed. Approaches to achieving such potency will be discussed.

3:00pm - 3:30pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
VEGF mRNA in Cardiovascular Disease
  • Anna Collén, PhD - Project Leader VEGF Project, AstraZeneca
more
3:00pm - 3:30pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Design and Development of Lipid Nanoparticles for mRNA Vaccines
  • Marian Gindy, PhD - Executive Director, Pharmaceutical Sciences, Merck Research Laboratories
more
3:30pm - 4:15pm 45 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Networking Refreshment Break in Poster and Exhibit Hall
3:30pm - 4:15pm 45 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Networking Refreshment Break in Poster and Exhibit Hall
3:30pm - 4:15pm 45 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Networking Refreshment Break in Poster and Exhibit Hall
3:30pm - 4:15pm 45 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Networking Refreshment Break in Poster and Exhibit Hall
3:30pm - 4:15pm 45 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
Networking Refreshment Break in Poster and Exhibit Hall
3:30pm - 4:15pm 45 mins
Track 6: Delivery of Macromolecular Therapeutics
Networking Refreshment Break in Poster and Exhibit Hall
4:15pm - 4:45pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Translational Insights of Oligonucleotide ADME and Toxicology: From Bench to Bedside with a GalNAc-conjugated Anti-miR
  • Steven Neben, PhD - Senior Director, Research and Development, Regulus Therapeutics
more

A case study will be presented for nonclinical and early clinical development of a novel oligonucleotide inhibitor of a hepatocyte microRNA, targeted to liver by conjugation to GalNAc. Novel findings from pharmacokinetics, pharmacodynamics, and toxicology data provide important learnings that can be applied to development of other drugs in the class.

4:15pm - 4:45pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Translational Insights of Oligonucleotide ADME and Toxicology: From Bench to Bedside with a GalNAc-conjugated Anti-miR
  • Steven Neben, PhD - Senior Director, Research and Development, Regulus Therapeutics
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A case study will be presented for nonclinical and early clinical development of a novel oligonucleotide inhibitor of a hepatocyte microRNA, targeted to liver by conjugation to GalNAc. Novel findings from pharmacokinetics, pharmacodynamics, and toxicology data provide important learnings that can be applied to development of other drugs in the class.

4:15pm - 4:45pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Oral Delivery of Constrained Peptides to Treat Gastrointestinal Disorders
  • Mark Smythe, PhD - Founder and Vice President, Protagonist Therapeutics
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Protagonist Therapeutics is a clinical-stage company focused on developing peptide-based drug candidates. Our focus is on developing oral peptide drugs that target biological pathways of marketed injectable antibody drugs. This presentation will outline strategies for development of potent antagonists that are stable to the gastrointestinal tract and suitable for oral delivery.

4:15pm - 4:45pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Oral Delivery of Constrained Peptides to Treat Gastrointestinal Disorders
  • Mark Smythe, PhD - Founder and Vice President, Protagonist Therapeutics
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Protagonist Therapeutics is a clinical-stage company focused on developing peptide-based drug candidates. Our focus is on developing oral peptide drugs that target biological pathways of marketed injectable antibody drugs. This presentation will outline strategies for development of potent antagonists that are stable to the gastrointestinal tract and suitable for oral delivery.

4:15pm - 4:45pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
Essential Quality Attributes of mRNA-containing Lipid Nanoparticles
  • Peter Lutwyche, PhD - CTO & Vancouver Site Head, Genevant Sciences Corporation
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This presentation will review quality attributes required for optimum biological performance of mRNA-containing LNP, along with appropriate methods for testing those attributes.

4:15pm - 4:45pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Robust In Vivo Gene Editing with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 RNA Components
  • Amy Rhoden Smith, PhD - Principal Scientist, Intellia Therapeutics
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We present development of a novel LNP-mediated delivery system for Cas9 mRNA and chemically synthesized sgRNA capable of producing significant liver editing in the transthyretin (Ttr) gene in vivo. This work highlights the potential of this method for effective therapeutic liver delivery of CRISPR/Cas9 gene editing components.

4:45pm - 5:15pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
DCR-PHXC vs. DCR-PH1: A Comparison of Nonclinical Development Programs for siRNA Delivered Using GalNAc Conjugates or Lipid Nanoparticles
  • Jennifer Lockridge, PhD - Senior Vice President, Program Development, Dicerna Pharmaceuticals, Inc.
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4:45pm - 5:15pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
DCR-PHXC vs. DCR-PH1: A Comparison of Nonclinical Development Programs for siRNA Delivered Using GalNAc Conjugates or Lipid Nanoparticles
  • Jennifer Lockridge, PhD - Senior Vice President, Program Development, Dicerna Pharmaceuticals, Inc.
more
4:45pm - 5:15pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Development of Neoantigen-Based Personalized Cancer Vaccine NEO-PV-01
  • Jesse Dong, PhD - Vice President, Peptide Chemistry, Neon Therapeutics
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Neon Therapeutics is pursuing an exciting clinical development program of a personalized cancer vaccine, NEO-PV-01, which targets patient-specific tumor neoantigens to engage the immune system to precisely and selectively attack tumors. Our objective is to create and deepen anti-tumor immune responses and broaden the range of cancers treatable via immuno-oncology approaches.

4:45pm - 5:15pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Development of Neoantigen-Based Personalized Cancer Vaccine NEO-PV-01
  • Jesse Dong, PhD - Vice President, Peptide Chemistry, Neon Therapeutics
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Neon Therapeutics is pursuing an exciting clinical development program of a personalized cancer vaccine, NEO-PV-01, which targets patient-specific tumor neoantigens to engage the immune system to precisely and selectively attack tumors. Our objective is to create and deepen anti-tumor immune responses and broaden the range of cancers treatable via immuno-oncology approaches.

4:45pm - 5:15pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
Quality Control of mRNA for Preclinical and Clinical Studies
  • Andreas Kuhn, PhD - Vice President, RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH
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Messenger (m)RNA is increasingly investigated as a platform technology for multiple therapeutic applications. My presentation will cover quality control aspects of mRNA manufactured for preclinical and clinical studies. This will include the description of approaches to identify critical quality attributes as well as an overview about assays to test the different parameters.

4:45pm - 5:15pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Centyrin Designer Proteins for Targeted Delivery
  • Karyn O'Neil, PhD - Chief Scientific Officer, Ar­ō Biotherapeutics
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We are developing Centyrins for targeted delivery where it is desirable to focus the activity of a therapeutic cargo to a specific cell type. Centyrins are novel FN3 domains based on the sequence of human tenascin C that retain the affinity properties of antibodies with the stability, solubility and tissue penetration properties of small molecules. The biophysical properties of Centyrins provide unique advantages for therapeutic payload delivery for even challenging moieties such as oligonucleotides and nanoparticles.

5:15pm - 5:45pm 30 mins
Track 1: Oligonucleotide Discovery, Preclinical and Clinical
Panel Discussion
5:15pm - 5:45pm 30 mins
Track 2: Oligonucleotide Chemistry, Manufacturing and Controls
Panel Discussion
5:15pm - 5:45pm 30 mins
Track 3: Peptide Chemistry, Manufacturing and Controls
Heat Shock Protein Chaperoned Long Synthetic Peptide Cancer Vaccines
  • Mark Findeis, PhD - Senior Director, Research Biochemistry, Agenus, Inc.
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Vaccination with tumor-derived neoepitopes has emerged as an increasingly active area of effort to prime the immune system of cancer patients with the goal of stimulating a therapeutic immune response against their disease. Challenges in the execution of this strategy range from identification of neoepitopes by genomic or HLA ligandome analyses, to prioritization of neoepitopes using bioinformatic techniques, manufacturing of a vaccine, conducting clinical studies and evaluating immune response and clinical efficacy. The Agenus ASV™ platform uses a variety of approaches to address these challenges and provide individualized and personalized vaccines within a clinically relevant timeframe. This presentation will discuss these strategies with an emphasis on efficiency in manufacturing long synthetic peptide antigens and complexing them with a protein chaperone to facilitate effective transport to antigen presenting cells.

5:15pm - 5:45pm 30 mins
Track 4: Peptide Discovery, Preclinical and Clinical
Heat Shock Protein Chaperoned Long Synthetic Peptide Cancer Vaccines
  • Mark Findeis, PhD - Senior Director, Research Biochemistry, Agenus, Inc.
more

Vaccination with tumor-derived neoepitopes has emerged as an increasingly active area of effort to prime the immune system of cancer patients with the goal of stimulating a therapeutic immune response against their disease. Challenges in the execution of this strategy range from identification of neoepitopes by genomic or HLA ligandome analyses, to prioritization of neoepitopes using bioinformatic techniques, manufacturing of a vaccine, conducting clinical studies and evaluating immune response and clinical efficacy. The Agenus ASV™ platform uses a variety of approaches to address these challenges and provide individualized and personalized vaccines within a clinically relevant timeframe. This presentation will discuss these strategies with an emphasis on efficiency in manufacturing long synthetic peptide antigens and complexing them with a protein chaperone to facilitate effective transport to antigen presenting cells.

5:15pm - 5:45pm 30 mins
Track 5: mRNA, CRISPR and Hot Topics in Oligonucleotides
Characterization of Critical Quality Attributes of mRNA, A Novel Therapeutic Modality
  • Kristian Link, PhD - Associate Director, Analytical Development, Moderna Therapeutics
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Using mRNA to create new therapeutics is complex and requires overcoming novel scientific and technical challenges. mRNA production process heavily relies on in vitro enzymatic synthesis instead of chemical synthesis due to its length. Recent advances towards mRNA production and delivery prompt a need for robust analytical methods capable of characterizing this new class of drugs. mRNA product-related impurities include short mRNAs resulting from either premature termination of transcription or in-process degradation, uncapped mRNAs, and point mutations, insertions/deletions. Multiple case studies utilizing a combination of biochemical and biophysical methods will be discussed on characterization of mRNA product- related impurities and variants for successful development of mRNA therapeutics.

5:15pm - 5:45pm 30 mins
Track 6: Delivery of Macromolecular Therapeutics
Development of mRNA Nanomedicines for Delivery
  • Robert Jabulowsky, PhD - Deputy Head of Project Management, BioNTech AG
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Messenger RNA (mRNA)-based nanomedicines constitute a new class of pharmaceutical products with potential applications in tumor therapy, vaccination, or protein substitution therapy. A key prerequisite for translation of such novel therapeutic concepts into clinical practice are pharmaceutical formulations for parenteral application of the RNA to the patient and to deliver the RNA to the target site. Further technological challenges refer to GMP-compliant manufacturing and quality control of such systems. Here approaches for assembly and control of injectable RNA nanomedicines are presented. They dispose high biopharmaceutical availability as well as efficient and organ-selective expression of the RNA. Case studies of novel RNA pharmaceuticals for tumor immunotherapy are given, where dedicated manufacturing processes offer a fully GMP-compliant, cost-effective and highly scalable production.

5:45pm - 6:45pm 60 mins
Networking Reception in Poster and Exhibit Hall