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7 am 8:25 am (85 mins)

Main agenda

Early Morning Exhibit Viewing and Breakfast in the Exhibit Hall

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

Main agenda

Chairman’s Remarks

  • Firoz Antia, Ph.D. - Director, Chemical Process R&D, Biogen

8:30 am 9 am (30 mins)

Main agenda

in vivo Gene Delivery and Genome Editing: How Advances in siRNA and mRNA Delivery Enable in vivo CRISPR/Cas9 Delivery

  • Presenter Daniel Anderson, Ph.D. - Associate Professor Chemical Engineering, Massachusetts Institute of Technology

9 am 9:30 am (30 mins)

Main agenda

Taking Advantage of Modified RNAs to Improve the CRISPR System Considering Off-target Effects and in vivo Strategies

  • Presenter Thazha P. Prakash, Ph.D. - Director, Medicinal Chemistry, Ionis Pharmaceuticals

9:30 am 10 am (30 mins)

Main agenda

High-resolution Interrogation of the Human Noncoding Genome Using Pooled CRISPR Screens

Although the noncoding genome plays a major role in gene regulation and harbors many common disease genetic variants, we lack tools for rapid identification and manipulation of noncoding functional elements. In addition to genome-scale screens targeting protein-coding genes, we have recently developed techniques for adapting CRISPR screens into noncoding regions of the genome to find functional elements where mutations trigger resistance to targeted chemotherapies for melanoma.

  • Presenter Neville Sanjana, Ph.D. - Core Faculty Member, New York Genome Center, New York University

10 am 10:55 am (55 mins)

Main agenda

Networking Refreshment Break in Poster and Exhibit Hall

10:55 am 11 am (5 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Chairman’s Remarks

  • Paul Metz - Principal Consultant, Metz Biotechnology Consulting, LLC

11 am 11:30 am (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Evolution of the Reference Standard with Product Development Cycle

  • Presenter Kyunghee (Connie) Cho - Associate Director, Regulus Therapeutics

11:30 am 12 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Application of ESI-MS/MS for Oligonucleotide Sequencing Methods

ESI-MS/MS sequencing is used to confirm if a synthesized oligonucleotide is consistent with the proposed molecular sequence.  Using targeted charge states and varying collision energies, data from multiple experiments is combined to sequence oligonucleotides up to 35-mers.  Fragmentation differences observed between oligonucleotide types will be presented including data from method validations using isobaric sequences to demonstrate specificity.

  • Presenter Edward Huber, Ph.D. - Director, Analytical Development/QC, Nitto Denko Avecia

12 pm 12:30 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Validation of Analytical Methods for Oligonucleotide Therapeutics

  • Presenter Claus Rentel, Ph.D. - Executive Director Analytical Development QC, Ionis Pharmaceuticals, Inc.

10:55 am 11 am (5 mins)

Peptide Discovery, Preclinical and Clinical

Chairman’s Remarks

  • Chairman Christopher Rhodes, Ph.D. - President and CEO, Drug Delivery Experts

11 am 11:30 am (30 mins)

Peptide Discovery, Preclinical and Clinical

Tailoring Peptides for Less Frequent Dosing and Non-invasive Delivery

The development within biotechnology has given access to various technologies that are applicable to optimize the properties of endogenous proteins and peptides to stable and efficacious drugs with improved pharmaceutical profiles. The in vivo parameters that often are being optimized includes renal clearance (by adding mass and charge to the protein), liver elimination (by removal or hiding of specific receptor signatures), and plasma elimination (by elimination of proteolytic sites with amino acid substitutions). In addition the biophysical properties addressing stability of drug product is often being optimized to obtain a successful drug candidate. At Novo Nordisk the attachment of fatty acids to proteins and peptides has been an important focus area to change not only the in vivo properties, but also the biophysical properties of the drug candidates. These matters will be discussed using examples from GLP-1 and insulin to meet patients’ needs for less frequent dosing and for non-invasive delivery

  • Presenter Jesper Lau - Vice President, Diabetes Protein & Peptide Chemistry, , Novo Nordisk, Denmark

11:30 am 12 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Sublingual Delivery of Peptides and Small Proteins: Exenatide, Insulin and IL-2 As Examples

  • Presenter Yves Decadt - Chief Executive Officer, BioLingus

12 pm 12:30 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Formulation Approaches to Overcome Barriers to Oral Delivery of Macromolecules

A majority of biological therapies are delivered by injection. In addition to poor patient adherence, the injectable route may not be optimal for chronic treatments. Despite significant efforts, there has been limited success to develop oral formulations of macromolecules. This talk will provide insights on challenges in oral macromolecule delivery, and present strategies to rapidly screen biomolecules potential for oral delivery and assess formulation strategies in parallel.

  • Presenter Ronak Savla, Ph.D. - Scientific Affairs Manager, Catalent Pharma Solutions

10:55 am 11 am (5 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Chairman’s Remarks

  • Elena Feinstein, M.D., Ph.D. - Chief Scientific Officer, Quark Pharmaceuticals

11 am 11:30 am (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Advancing CRISPR Technologies for Therapeutic Application

Genome editing technologies, including the CRISPR/Cas9 system, allow for precise and corrective molecular modifications to treat the underlying cause of genetic diseases.  Editas Medicine is developing CRISPR based therapeutics across a broad range of indications.  There are certain technical challenges that exist for translating genome editing medicines based on CRISPR to the clinic.  These challenges lie in both the creation of appropriately high quality drug substance and development of relevant measurement technologies for the drug and its activities.

This presentation will focus on the progress we have made in the following areas:

  • high quality Cas9 protein, gRNA and RNPs using proprietary technologies coupled to appropriate analytics;
  • pharmacokinetic assay systems for quantitating Cas9 complexed to the gRNA, apoCas9 and the gRNAs to measure cellular and animal exposure and bio-distribution; and
  • pharmacodynamic measurement technologies that allow for size insensitive measurement of editing including small and large insertions and deletions, inversions and chromosomal translocation in a scalable, multiplex compatible format.

Investment in the fundamental technologies required for drug development will ensure we advance to the clinic with a clear understanding of the CRISPR drug, pharmacokinetics and pharmacodynamics. 

  • Presenter Christopher Wilson, Ph.D. - Director of Lead Finding, Editas Medicine

11:30 am 12 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Chemical Modification of Guide RNAs for SpyCas9 and AsCpf1 CRISPR Nucleases

Unmodified RNA oligonucleotides are rapidly degraded by serum or cellular nucleases, limiting their activity, and further can trigger innate immune responses in mammalian cells. Chemical modification was optimized for both the SpyCas9 crRNA/tracrRNA and the AsCpf1 crRNA. Highly functional modified variants were developed that are compatible for use as an RNP complex, directly introduced into mammalian cells using to achieve high editing efficiency with minimal side effects.   

  • Presenter Mark Behlke, M.D., Ph.D. - Chief Scientific Officer, Integrated DNA Technologies

12 pm 12:30 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

A New Dimension in LNA Therapeutics

In recent years, LNA has become a leading modality in RNA, enabling new breakthroughs in the area of mRNA and microRNA targeting therapeutics. Recent improvements in LNA technology will be shown and illustrated by the latest preclinical data. Traditionally, LNA has been synthesized with stereo random phosphorothioate internucleoside linkages (PS). We have now seen that stereo defined LNA PS are strong determinants for activity influencing key drug properties. The importance of PS in therapeutic oligonucleotides is a “hot topic” and the use of “one compound” stereo defined LNA will be discussed and placed within the bigger picture of oligonucleotide drug discovery.   

  • Presenter Troels Koch, Ph.D. - VP & Head of Research, RNA Therapeutics, Roche pRED, Roche Innovation Center Copenhagen, Denmark

10:55 am 11 am (5 mins)

Peptide Chemistry Manufacturing and Controls

Chairman’s Remarks

  • Chairman Fanyu Meng, Ph.D. - Principal Scientist, Merck Research Labs

11 am 11:30 am (30 mins)

Peptide Chemistry Manufacturing and Controls

Analytical Strategy for Continuous Manufacture Process Development for Peptides

There is increasing interest in therapeutic peptides for design of novel therapy in pharmaceutical and bio-tech industry, due to their general high selectivity, efficiency, excellent safety and tolerability profile in human. Once a peptide is selected for further drug development, it needs to be manufactured in large scale with consistent quality, according to GMP rules. In this presentation, analytical control strategy on continuous manufacture development of synthetic peptides will be demonstrated.

  • Presenter Alicia Zeng, Ph.D. - Scientist, Process Development, Amgen, Inc.

11:30 am 12 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

2D-LC as an On-line Desalting Tool Allowing Peptide Identification Directly from MS Unfriendly HPLC Methods

Peptide impurities and positional isomers separated under MS unfriendly conditions are successfully identified by Agilent 2D-LC/MS system using the second dimension LC as an on-line desalting tool. Labor intensive, time consuming and error-prone off-line fraction collection and translation of chromatographic methods are circumvented.

  • Presenter Hao Luo, Ph.D. - Associate Principal Scientist, Merck & Co.

12 pm 12:30 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Application of High Resolution MS for the Analysis and Characterization of Peptides

The presentation will describe the use of high resolution mass spectrometry applications for both development and commercial peptides. Case studies will be presented including peptides that involve ligation technologies, the utility of MS in confirming the removal of solid phase coupling reagents and impurity identification in life cycle management activities.

  • Presenter Marion King, Ph.D. - Analytical Development Manager, Ipsen Manufacturing Ireland Ltd

12:30 pm 1:40 pm (70 mins)

Main agenda

Lunch on Your Own

1:40 pm 1:45 pm (5 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

Chairman’s Remarks

  • Paul Metz - Principal Consultant, Metz Biotechnology Consulting, LLC

1:45 pm 2:15 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

QbD Approaches to Oligonucleotide Process Development

  • Presenter Gillian Turner, Ph.D. - QbD Lead, GlaxoSmithKline

2:15 pm 2:45 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

The Application of DoE to Oligonucleotide Synthesis – Generating Process Understanding

  • Presenter Ben Andrews, Ph.D. - Scientific Investigator, GlaxoSmithKline

2:45 pm 3:15 pm (30 mins)

Oligonucleotide Chemistry, Manufacturing and Controls

CMC Strategies for Managing Late Stage Development of Global Oligonucleotides

  • Presenter Vidhya Gopalakrishnan, Ph.D. - Senior Vice President, Quark Pharmaceuticals

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

Oligonucleotide Chemistry, Manufacturing and Controls

Scaling up for Commercial Supply of Alicaforsen and Treating Patients

Atlantic Healthcare is an emerging trans-Atlantic pharmaceutical company with a core focus on gastrointestinal disorders including Inflammatory Bowel Disease (IBD). Atlantic’s lead product Alicaforsen enema is progressing through pivotal phase 3 clinical trial for the treatment of IBD, Pouchitis, due to read out 2H 2017. Alicaforsen is an antisense oligonucleotide, licensed from Ionis Pharmaceuticals, Inc., which provides the potential for the treatment of inflammation via multiple delivery formulations. Alicaforsen has been granted Orphan Drug designation by the FDA and EMA and a letter of Fast Track for the treatment of Pouchitis in recognition of the unmet medical need. In February 2017 Atlantic received FDA agreement to initiate filing the NDA on a rolling basis. Atlantic selected Nitto Avecia Inc. for commercial supply of alicaforsen API. The original manufacturing process was developed 12 years ago. Incorporating new approaches, Atlantic and Nitto Avecia are collaborating and developing the manufacturing methods and processes. This presentation will describe the experience with patients and the steps taken to prepare the alicaforsen API process for commercial supply.

  • Presenter Janette Thomas, Ph.D. - Director of International Operations, Atlantic Healthcare plc
  • Presenter Mike Webb, Ph.D. - Vice President, Manufacturing, Atlantic Healthcare

1:40 pm 1:45 pm (5 mins)

Peptide Discovery, Preclinical and Clinical

Chairman’s Remarks

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

1:45 pm 2:15 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

The Galinpepimut-S (GPS) Clinical Development Program in Immuno-oncology (IO): Novel Approach Using a Mixture of Heteroclitic Peptides for Epitope Immunization against the Wilms Tumor-1 (WT1) Protein across Several Tumor Types

GPS is a unique active immunotherapy against the premier target onco-antigen, WT1, possessing innovative properties, optimal for the current/ future IO landscape. Ph2 studies showed elicitation of immune responses and a definite clinical activity ‘signal’ in AML, mesothelioma and myeloma. Future studies, incl. Ph3 trials, are being planned.

  • Nicholas Sarlis, M.D., Ph.D. - Chief Medical Officer, Senior Vice President, Sellas Life Sciences Group

2:15 pm 2:45 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

Translating B Cell Epitope Peptide-Based Cancer Vaccines to the Clinic and Future Combination Immunotherapies

We have created and established a portfolio of validated B-cell peptide epitopes against multiple receptor tyrosine kinases to expedite the development of new paradigm shifting cancer immunotherapies. We have identified the most biologically effective combinations of EGFR (HER-1), HER-2, HER-3, VEGF and IGF-1R peptide vaccines/mimics to selectively inhibit multiple receptors and signaling pathways. We have translated two HER-2 combination peptide vaccines to the clinic in a Phase 1/2b trial to safely deliver curative and transformative cancer immunotherapies to advanced cancer patients. This presentation will detail our clinical trial and basic studies based on the development of combinatorial immunotherapeutic strategies that act synergistically to enhance immune-mediated tumor killing aimed at addressing mechanisms of tumor resistance for several tumor types.

  • Pravin Kaumaya, Ph.D. - Professor, Vaccine Development/Peptide & Protein Engineering Laboratory, The Ohio State University Medical Center

2:45 pm 3:15 pm (30 mins)

Peptide Discovery, Preclinical and Clinical

CMC strategy for a clinically approved “Nanoparticle-in-oil based vaccine delivery system (water free) with an effective, long lasting and durable immune responses against “Multi peptide Vaccine” encoding T- Cell for treatment of Ovarian Cancer immunotherapy (IO) and B-cell Epitope for RSV (Infectious disease (ID)

DepoVax™ technology is a novel Nanoparticle-in-oil based vaccine platform in which tumor-associated Antigens (TAA) and infectious B-cell epitopes are encapsulated in nanoparticle and suspended in oil. The oil acts as an adjuvant that greatly enhances the potency of peptide based Cancer Vaccines and elicits a strong cytotoxic T Cell and B Cell responses (unique pharmacokinetics of stable short peptide antigens over several weeks). This approach helps to achieve 1) the right therapeutic target - validated, disease-specific multi peptide antigens capable of targeting tumors and not healthy cells 2) the right enhancement technology - DepoVax optimizes the presentation of peptide or Oligonucleotide or protein based antigens and generates enhanced immunogenicity and 3) the right clinical strategy - Support the immune system by combining DepoVax™ with effective immunomodulatory agents (Polytherapy)

  • Leeladhar Sammatur - Vice President, Product Development and Manufacturing, Immunovaccine, Inc.

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

Peptide Discovery, Preclinical and Clinical

Development of Peptide Vaccines for Cancer: Overcoming the Challenges for Innovation

  • Yasuhide Uejima, Ph.D. - General Manager, Quality Assurance & Pharmaceutical Development, GreenPeptide Co Ltd

1:40 pm 1:45 pm (5 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Chairman’s Remarks

1:45 pm 2:15 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Update on the Development of Oligonucleotides at Quark

  • Presenter Elena Feinstein, M.D., Ph.D. - Chief Scientific Officer, Quark Pharmaceuticals

2:15 pm 2:45 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Clinical Development of AST-005, A Topicallyapplied Antisense Spherical Nucleic Acid for the Treatment of Psoriasis

Exicure, Inc. is developing a topical antisense oligonucleotide, called AST-005, which is targeted to tumor necrosis factor alpha mRNA for the treatment of chronic plaque psoriasis. Drug discovery and development efforts by Exicure revolve around the use of spherical nucleic acid (SNA) constructs, which are 3-dimensional arrangements of oligonucleotides where the nucleic acids are densely packed and radially oriented. When arranging oligonucleotides in this way, properties arise that are distinct from the “linear” nucleic acids (i.e., nucleic acids not arranged in the SNA format). Most importantly, oligonucleotides arranged in the SNA geometry exhibit skin penetration properties and increased cellular uptake when compared to linear nucleic acids. A Phase 1 clinical trial of AST-005 executed in 2016 indicated that AST-005 was safe, well tolerated and produced a dose dependent knockdown of the TNF mRNA transcript in the psoriatic lesional skin. This presentation will describe the development of AST-005, the non-clinical toxicology program and results, as well as the Phase 1 clinical trial and its results.

  • Presenter Weston Daniel, Ph.D. - Director of Program Management, Exicure, Inc.

2:45 pm 3:15 pm (30 mins)

Oligonucleotide Discovery, Preclinical and Clinical

Therapeutic microRNA Modulation: From Concept to the Clinic

  • Presenter Christina Dalby, Ph.D. - Director, Chemistry & Biochemistry, miRagen Therapeutics

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

Oligonucleotide Discovery, Preclinical and Clinical

Emerging Therapeutic Profile of RNA Interference against Hepatic Targets

  • Presenter Patrick Haslett, M.D. - Executive Director of Clinical Research, Alnylam Pharmaceuticals

1:40 pm 1:45 pm (5 mins)

Peptide Chemistry Manufacturing and Controls

Chairman’s Remarks

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

1:45 pm 2:15 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

The Galinpepimut-S (GPS) Clinical Development Program in Immuno-oncology (IO): Novel Approach Using a Mixture of Heteroclitic Peptides for Epitope Immunization against the Wilms Tumor-1 (WT1) Protein across Several Tumor Types

GPS is a unique active immunotherapy against the premier target onco-antigen, WT1, possessing innovative properties, optimal for the current/ future IO landscape. Ph2 studies showed elicitation of immune responses and a definite clinical activity ‘signal’ in AML, mesothelioma and myeloma. Future studies, incl. Ph3 trials, are being planned.

  • Nicholas Sarlis, M.D., Ph.D. - Chief Medical Officer, Senior Vice President, Sellas Life Sciences Group

2:15 pm 2:45 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Translating B Cell Epitope Peptide-Based Cancer Vaccines to the Clinic and Future Combination Immunotherapies

We have created and established a portfolio of validated B-cell peptide epitopes against multiple receptor tyrosine kinases to expedite the development of new paradigm shifting cancer immunotherapies. We have identified the most biologically effective combinations of EGFR (HER-1), HER-2, HER-3, VEGF and IGF-1R peptide vaccines/mimics to selectively inhibit multiple receptors and signaling pathways. We have translated two HER-2 combination peptide vaccines to the clinic in a Phase 1/2b trial to safely deliver curative and transformative cancer immunotherapies to advanced cancer patients. This presentation will detail our clinical trial and basic studies based on the development of combinatorial immunotherapeutic strategies that act synergistically to enhance immune-mediated tumor killing aimed at addressing mechanisms of tumor resistance for several tumor types.

  • Pravin Kaumaya, Ph.D. - Professor, Vaccine Development/Peptide & Protein Engineering Laboratory, The Ohio State University Medical Center

2:45 pm 3:15 pm (30 mins)

Peptide Chemistry Manufacturing and Controls

Design & Clinically Approved “Nanoparticle-in-oil Based Vaccine Delivery system (Water Free) for Effective, Long Lasting and Durable Immune Responses against “Multi peptide Vaccine” Encoding T- Cell for Treatment of Ovarian Cancer Immunotherapy (IO) and & B-cell Epitopes for RSV (Infectious Disease ID)

DepoVax™ technology is a novel Nanoparticle-in-oil based vaccine platform in which tumor-associated Antigens (TAA) and infectious B-cell epitopes are encapsulated in nanoparticle and suspended in oil. The oil acts as an adjuvant that greatly enhances the potency of peptide based Cancer Vaccines and elicits a strong cytotoxic T Cell and B Cell responses (unique pharmacokinetics of stable short peptide antigens over several weeks). This approach helps to achieve 1) the right therapeutic target - validated, disease-specific multi peptide antigens capable of targeting tumors and not healthy cells 2) the right enhancement technology - DepoVax optimizes the presentation of peptide or Oligonucleotide or protein based antigens and generates enhanced immunogenicity and 3) the right clinical strategy - Support the immune system by combining DepoVax™ with effective immunomodulatory agents (Polytherapy)

  • Leeladhar Sammatur - Vice President, Product Development and Manufacturing, Immunovaccine, Inc.

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

Peptide Chemistry Manufacturing and Controls

Development of Peptide Vaccines for Cancer: Overcoming the Challenges for Innovation

  • Yasuhide Uejima, Ph.D. - General Manager, Quality Assurance & Pharmaceutical Development, GreenPeptide Co Ltd

3:45 pm 3:46 pm (1 mins)

Main agenda

Close of Conference