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7:30am - 8:20am

Registration and Coffee

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8:20am - 8:30am

Chairperson's Remarks

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8:30am - 9:30am
Info

Antisense Technology: Today and Tomorrow

Today antisense technology is delivering its promise. At Ionis, antisense is delivering transformational medicines that fundamentally alter the course of diseases that range from the rare to the very common. We, at Ionis, expect to have at least ten new medicines enter pivotal trials in 2019 and 2020. The medicines focus on treating a wide range of diseases with targets in various organs and are administered by several routes of administration attest to the breadth of value antisense technology may bring in the coming years. Equally importantly, we continue to make fundamental advances in understanding the molecular mechanisms via which antisense agents of various chemistries produce the observed effects. As our mechanistic understanding increases, we rapidly convert those advances into greater therapeutic index, tolerability and patient convenience by coupling advances in ASO medicinal chemistry to these biological insights and creating advanced ASO designs that immediately enter our pipeline. 

In the presentation, I will provide an update on the current status of the technology and the benefits to patients it is delivering, then focus on the work that my group and I are doing to create an even brighter future.

  • Stanley Crooke, M.D, PhD - Chief Executive Officer, Ionis Pharmaceuticals, Inc.
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9:30am - 10:15am
Info

RNAi Therapeutics: Onpattro and Beyond

In 2006, the Nobel prize in Physiology or Medicine was awarded for the discovery of RNA interference (RNAi). In 2018, the first RNAi therapeutic became a reality for patients with hereditary TTR amyloidosis. The approval of Onpattro paves the way for a whole new class of medicines. Beyond Onpattro, I will discuss how our RNAi therapeutic platform has enabled the current liver-targeted pipeline via a reproducible and modular approach to drug discovery and development. Finally, I will share recent advances in extra-hepatic-delivery (central nervous system, eye) at Alnylam and how they offer promise for major neurogenerative and ocular disorders.

  • Kevin Fitzgerald, Ph.D - Senior Vice President and Head of Research, Alnylam Pharmaceuticals
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10:15am - 10:45am

Networking Refreshment Break

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10:45am - 11:15am
Info

A Platform for In-solution Enrichment from Large Libraries to Identify Peptide Inhibitors of Protein-Protein Interactions

Here we report a platform for improving the affinity of peptide-based inhibitors of protein-protein interactions using non-canonical amino acids. With this platform—which is inherently selective for high-affinity binders—we realized up to ~100 or ~30-fold gains in affinity for binders to the oncogenic ubiquitin ligase MDM2 or the HIV capsid protein C-terminal domain (C-CA). We demonstrated the utility of the identified compounds as functional PPI inhibitors by rendering them cell permeable via macrocyclization to target MDM2 in cancer cells

  • Bradley Pentelute, PhD - Professor, Chemistry, Massachusetts Institute of Technology
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11:15am - 11:45am

Rare Diseases and RNA-Targeted Medicines

  • Sarah Boyce - President, Akcea Therapeutics
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11:45am - 12:15pm

Late Breaking Keynote Presentation

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Showing of Streams
1:25pm - 1:30pm
Chairperson's Remarks

Chairperson's Remarks

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1:30pm - 2:00pm
Info

Using Artificial Intelligence to Accelerate Drug Development and Precision Medicine

Artificial Intelligence (AI) can drive cars, beat humans at their own games and transcribe speech. Biology and medicine present much greater challenges, but the recent exponential growth in biomedical data has made AI assisted drug development a reality. Brendan Frey, a pioneer in this field, will discuss how Deep Genomics is using AI for target discovery and drug development, including its potential application to personalized therapies. He will give his perspective on the opportunities and challenges that lie ahead, such as guaranteeing prediction accuracy and establishing trust with clinicians and regulators.

  • Brendan Frey, PhD - Founder and CEO, Deep Genomics
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2:00pm - 2:30pm
Info

Parameters for Accelerated Development of Antisense Oligonucleotides as Personalized Medicine

We will discuss our experience with the accelerated development of the patient-specific drug milasen, as rescue therapy for a young girl with rapidly progressive neurodegenerative disease, which raises questions about opportunities and challenges in the deployment of antisense oligonucleotides for personalized medicine.

  • Timothy Yu, M.D., Ph.D. - Attending Physician, Division of Genetics and Genomics, Boston Children's Hospital
More
2:30pm - 3:00pm

Development of Neoantigen-Based Personalized Cancer Vaccine, NEO-PV-01

  • Jesse Dong, PhD - Vice President, Peptide Chemistry, Neon Therapeutics
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3:00pm - 3:45pm

Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break

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3:45pm - 4:15pm
Info

mRNA as a Platform Technology Ideally Suited for Individualized Therapeutics

Neo-epitopes derived from tumor mutations are ideal targets for immunotherapy against cancer. As the vast majority of cancer mutations are unique to the individual patient, personalized approaches are needed. By combining sequencing of tumor samples and bioinformatic algorithm with our mRNA platform, which allows efficient manufacturing of one batch for each patient, we could implement and verify this approach in a first-in-concept clinical trial in melanoma patients. This has demonstrated that mRNA is a versatile platform technology especially applicable for manufacturing of individualized therapeutics.

  • Andreas Kuhn, PhD - Vice President, RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH
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4:15pm - 4:45pm
Info

Manufacturing NeoAntigen Peptides: Requirements and Pragmatism

Traditional approaches to manufacturing therapeutic peptides cannot meet the throughput needed for NeoAntigen Peptides. The need for NeoAntigen Peptides challenges the industry to think “out-of-the-box”. This presentation discusses pragmatic manufacturing approaches to consider that balance the need for Quality, GMP, and throughput.

  • Trishul Shah, MS - Director Business Development, PolyPeptide Group
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4:45pm - 5:15pm

Panel Discussion with Session Speakers: Personalized Medicine and Individualized Therapies

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5:15pm - 6:45pm
Info

Networking Reception in Poster and Exhibit Hall

Join fellow attendees and speakers for the TIDES opening night networking reception. Enjoy drinks and appetizers with colleagues while viewing the exhibits and scientific poster sessions.

More
7:30am - 8:20am 50 mins
Registration and Coffee
8:20am - 8:30am 10 mins
Chairperson's Remarks
8:30am - 9:30am 60 mins
Info
Antisense Technology: Today and Tomorrow
  • Stanley Crooke, M.D, PhD - Chief Executive Officer, Ionis Pharmaceuticals, Inc.

Today antisense technology is delivering its promise. At Ionis, antisense is delivering transformational medicines that fundamentally alter the course of diseases that range from the rare to the very common. We, at Ionis, expect to have at least ten new medicines enter pivotal trials in 2019 and 2020. The medicines focus on treating a wide range of diseases with targets in various organs and are administered by several routes of administration attest to the breadth of value antisense technology may bring in the coming years. Equally importantly, we continue to make fundamental advances in understanding the molecular mechanisms via which antisense agents of various chemistries produce the observed effects. As our mechanistic understanding increases, we rapidly convert those advances into greater therapeutic index, tolerability and patient convenience by coupling advances in ASO medicinal chemistry to these biological insights and creating advanced ASO designs that immediately enter our pipeline. 

In the presentation, I will provide an update on the current status of the technology and the benefits to patients it is delivering, then focus on the work that my group and I are doing to create an even brighter future.

9:30am - 10:15am 45 mins
Info
RNAi Therapeutics: Onpattro and Beyond
  • Kevin Fitzgerald, Ph.D - Senior Vice President and Head of Research, Alnylam Pharmaceuticals

In 2006, the Nobel prize in Physiology or Medicine was awarded for the discovery of RNA interference (RNAi). In 2018, the first RNAi therapeutic became a reality for patients with hereditary TTR amyloidosis. The approval of Onpattro paves the way for a whole new class of medicines. Beyond Onpattro, I will discuss how our RNAi therapeutic platform has enabled the current liver-targeted pipeline via a reproducible and modular approach to drug discovery and development. Finally, I will share recent advances in extra-hepatic-delivery (central nervous system, eye) at Alnylam and how they offer promise for major neurogenerative and ocular disorders.

10:15am - 10:45am 30 mins
Networking Refreshment Break
10:45am - 11:15am 30 mins
Info
A Platform for In-solution Enrichment from Large Libraries to Identify Peptide Inhibitors of Protein-Protein Interactions
  • Bradley Pentelute, PhD - Professor, Chemistry, Massachusetts Institute of Technology

Here we report a platform for improving the affinity of peptide-based inhibitors of protein-protein interactions using non-canonical amino acids. With this platform—which is inherently selective for high-affinity binders—we realized up to ~100 or ~30-fold gains in affinity for binders to the oncogenic ubiquitin ligase MDM2 or the HIV capsid protein C-terminal domain (C-CA). We demonstrated the utility of the identified compounds as functional PPI inhibitors by rendering them cell permeable via macrocyclization to target MDM2 in cancer cells

11:15am - 11:45am 30 mins
Rare Diseases and RNA-Targeted Medicines
  • Sarah Boyce - President, Akcea Therapeutics
11:45am - 12:15pm 30 mins
Late Breaking Keynote Presentation
12:15pm - 1:25pm 70 mins
Info
Spotlight Presentations 1
Track Record of Oligonucleotide and Peptide Manufacturing in Large Scale by Solution Phase Approach AJIPHASE®.
  • Daisuke Takahashi, PhD - Senior Principal Researcher, Bio-functional, Research Institute for Bioscience Products and Fine Chemicals, Ajinomoto Co. Inc.

Peptide and oligonucleotide drug development are growing and a practical manufacturing method has been strongly required. Most of peptide and oligonucleotides are synthesized by solid-phase method, however, some solution phase-based technologies are developing in the world for future demand. We will describe solution-phase methods which has not been so clarified, such as usability, scalability and reliability in detail by using AJIPHASE® track records and will discuss both methods by comparison of yield, side reaction and purity of obtaining API.

12:15pm - 1:25pm 70 mins
Info
Spotlight Presentations 2
Lipid Nanoparticles – From Benchtop to Clinic: A CDMO Perspective
  • Di Bush, PhD - Formulations Director, Avanti Polar Lipids Inc

With the recent growth in lipid-based delivery platforms for oligonucleotide therapies, novel lipids, in particular cationic lipids, have become a great point of interest. As a one-stop CDMO for lipid synthesis, cGMP manufacturing, formulations development, and lipid analytical services, Avanti provides a unique perspective on bringing a novel cationic lipid from the benchtop to the clinic.

12:15pm - 1:25pm 70 mins
Info
Spotlight Presentations 3
Development of Synthetic Self-replicating RNA Platforms for Oncology Vaccines and Therapeutics
  • Anton McCaffrey, PhD - Senior Director of Research and Development, Biology, TriLink BioTechnologies, Inc.
  • Nathaniel Wang, PhD - Head of R&D, RNA Medicines, Synthetic Genomics Inc.

RNA as a vaccine and therapeutic modality has become a focus of significant interest. SGI has developed a self-amplifying RNA replicon showing improved protein expression, immunogenicity, and resistance to immune shutdown. This improved RNA replicon lowers the effective dose required for immunization and improves immunological memory enabling novel oncology therapeutics.


12:15pm - 1:25pm 70 mins
Info
Spotlight Presentations 4
Selecting an Asian CMO Partner for Therapeutic Oligonucleotide Manufacturing
  • J.O Adams - Head of Research, Ribo Biologics Co. Ltd.

There has been significant growth in the number of clinical manufacturing and research organizations in China. In the past, western companies were reluctant to outsource manufacturing to Asian companies, but, with the harmonization of regulations between China and the rest of the world, as well as stricter IP protection, the Asian CMOs and CROs now adhere to the same strict standards as their American and European counterparts; thus, are now ideal partners for you manufacturing and research projects.

12:15pm - 12:45pm 30 mins
Info
Spotlight Presentations 5
Biospring Spotlight Presentation
  • Mario Jauker, PhD - Head of Process Characterization, BioSpring
1:25pm - 1:30pm 5 mins
Chairperson's Remarks
1:30pm - 2:00pm 30 mins
Info
Using Artificial Intelligence to Accelerate Drug Development and Precision Medicine
  • Brendan Frey, PhD - Founder and CEO, Deep Genomics

Artificial Intelligence (AI) can drive cars, beat humans at their own games and transcribe speech. Biology and medicine present much greater challenges, but the recent exponential growth in biomedical data has made AI assisted drug development a reality. Brendan Frey, a pioneer in this field, will discuss how Deep Genomics is using AI for target discovery and drug development, including its potential application to personalized therapies. He will give his perspective on the opportunities and challenges that lie ahead, such as guaranteeing prediction accuracy and establishing trust with clinicians and regulators.

2:00pm - 2:30pm 30 mins
Info
Parameters for Accelerated Development of Antisense Oligonucleotides as Personalized Medicine
  • Timothy Yu, M.D., Ph.D. - Attending Physician, Division of Genetics and Genomics, Boston Children's Hospital

We will discuss our experience with the accelerated development of the patient-specific drug milasen, as rescue therapy for a young girl with rapidly progressive neurodegenerative disease, which raises questions about opportunities and challenges in the deployment of antisense oligonucleotides for personalized medicine.

2:30pm - 3:00pm 30 mins
Development of Neoantigen-Based Personalized Cancer Vaccine, NEO-PV-01
  • Jesse Dong, PhD - Vice President, Peptide Chemistry, Neon Therapeutics
3:00pm - 3:45pm 45 mins
Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break
3:45pm - 4:15pm 30 mins
Info
mRNA as a Platform Technology Ideally Suited for Individualized Therapeutics
  • Andreas Kuhn, PhD - Vice President, RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH

Neo-epitopes derived from tumor mutations are ideal targets for immunotherapy against cancer. As the vast majority of cancer mutations are unique to the individual patient, personalized approaches are needed. By combining sequencing of tumor samples and bioinformatic algorithm with our mRNA platform, which allows efficient manufacturing of one batch for each patient, we could implement and verify this approach in a first-in-concept clinical trial in melanoma patients. This has demonstrated that mRNA is a versatile platform technology especially applicable for manufacturing of individualized therapeutics.

4:15pm - 4:45pm 30 mins
Info
Manufacturing NeoAntigen Peptides: Requirements and Pragmatism
  • Trishul Shah, MS - Director Business Development, PolyPeptide Group

Traditional approaches to manufacturing therapeutic peptides cannot meet the throughput needed for NeoAntigen Peptides. The need for NeoAntigen Peptides challenges the industry to think “out-of-the-box”. This presentation discusses pragmatic manufacturing approaches to consider that balance the need for Quality, GMP, and throughput.

4:45pm - 5:15pm 30 mins
Panel Discussion with Session Speakers: Personalized Medicine and Individualized Therapies
5:15pm - 6:45pm 90 mins
Info
Networking Reception in Poster and Exhibit Hall

Join fellow attendees and speakers for the TIDES opening night networking reception. Enjoy drinks and appetizers with colleagues while viewing the exhibits and scientific poster sessions.