Informa
基調セッション

Muthiah (Mano) Manoharan, PhD

RNAi治療薬のデリバリー:siRNAの薬理学的特性の改善

Alnylam Pharmaceuticals, Inc.

Jesper Lau, PhD

セマグルチドの創薬−AlaスキャンからGLP-1類似体の構造設計へと至る道筋

Novo Nordisk A/S

Thazha P. Prakash, Ph.D.

オリゴヌクレオチド治療薬開発の新たな戦略と技術

Ionis Pharmaceuticals

Hiroaki Suga, Ph.D.

擬天然ペプチドのリボソーム型生体外発現に関する最近の研究成果

University of Tokyo, Japan

2月26日
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12:45pm - 1:55pm

昼食

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3:30pm - 4:00pm

ネットワーキングブレイク

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6:00pm - 7:00pm

ネットワーキングディナー

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9:00am - 12:45pm 225 mins
Info
ワークショップ
  • Co-Moderator Marc Lemaitre, PhD - Chief Operating Officer / Principal, Sirnaomics, Inc. / ML Consult
  • Co-Moderator Thomas Rupp - Owner & Principal, Thomas Rupp Consulting, Germany

This workshop will address early drug development and CMC of oligonucleotide therapeutics. A detailed discussion of moving oligonucleotide therapeutics from discovery to clinical trials will include a description of strategies for early clinical development, including GMP synthesis, analytical controls and specifications; nonclinical, CMC, manufacturing and scale-up and the regulatory framework for preparation of IND-IMPD dossiers. Participants will also gain a basic understanding of the considerations and requirements for taking an oligonucleotide therapeutic into first-in-human clinical trials.

Speakers:

CMC Oligos - Real World Case Studies

Marc M. Lemaitre, Ph.D., Principal, ML Consult, USA

Oligo Synthesis Scale-up and Process Related Issues and Troubleshooting

Thomas Rupp, Owner & Principal, Thomas Rupp Consulting, Germany

Additional Workshop Speakers to Be Confirmed

9:00am - 9:15am 15 mins
Info
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
ワークショップ
  • Co-Moderator Bruce Morimoto, PhD - Vice President, Scientific Affairs, Alkahest
  • Co-Moderator Christopher Rhodes, PhD - President and CEO, Drug Delivery Experts

This practical, introductory workshop will address early drug development of peptide therapeutics. A detailed discussion of moving peptide therapeutics from discovery to clinical trials will include a description of strategies for early clinical development, including GMP synthesis, analytical controls and specifications; formulation strategies; pharmacokinetics and toxicology study designs and requirements; and the regulatory framework for preparation of IND-IMPD dossiers. Participants will gain a basic understanding of the considerations and requirements for taking a peptide therapeutic into first-in-human clinical trials.

Who should attend?

Anyone interested in preclinical/clinical development of peptide therapeutics including scientists in discovery research, manufacturing, project management, drug development, business development and regulatory affairs.

9:15am - 10:00am 45 mins
Info
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
ペプチド原薬を合成するうえでの課題
  • Robert Hagopian - Director Business Development, PolyPeptide Group

An overview of solid versus liquid phase peptide manufacturing approaches will be presented. This will be followed by potential challenges including aggregation, scale-up, stability, changes in manufacturing early versus late phase, and other related notes.

10:00am - 10:45am 45 mins
Info
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
開発早期段階に対応する分析法、予備処方、処方
  • Christopher Rhodes, PhD - President and CEO, Drug Delivery Experts

The discussion will focus on all aspects of drug product development. We will start with analytical methods required for drug product, including in process controls, QC release methods, and stability, and how they differ from those for drug substance. In addition, we will discuss preformulation and formulation development of peptides to support non-clinical research studies, toxicity studies, and clinical programs. A third topic area to cover will be the selection, design, and development of delivery systems for peptides.

10:45am - 11:15am 30 mins
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
ネットワーキングブレイク
11:15am - 12:00pm 45 mins
Info
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
ペプチドのIND申請、非臨床安全性試験:薬物動態、生物学的分析、毒性学
  • Bruce Morimoto, PhD - Vice President, Scientific Affairs, Alkahest

Peptides bridge small molecules and biologics, not only in their size, but also in specificity and selectivity. These unique properties of peptides require specialized consideration when designing and executing the safety studies to support first-in-human clinical trials. This talk will outline and discuss the pharmacokinetics and bioanalysis of peptides and the design of toxicology studies for peptides.


12:00pm - 12:45pm 45 mins
Info
ワークショップ#2:ペプチド治療薬開発入門:臨床開発、CMCおよびそれ以降の段階へと進めていくための戦略
米国におけるペプチド薬とペプチド生物製剤の製品開発の類似点と相違点
  • Duu-Gong Wu, PhD - Senior Director, Regulatory Consulting, PPD

With the advancement of immunotherapies, the development of peptide therapeutic cancer vaccines and preventive vaccines as biologics has entered new therapeutic areas.  The regulation and the process in FDA governing the review and approval these products as biologics under PHS Act are quite different relative to other therapeutic peptides regulated as drugs. The presentation will discuss the similarity and differences in the review process and data requirements between a peptide drug and a peptide biologic.

12:45pm - 1:55pm 70 mins
昼食
1:55pm - 2:00pm 5 mins
基調講演
議長の挨拶
2:00pm - 2:30pm 30 mins
Info
基調講演
RNAi治療薬のデリバリー:siRNAの薬理学的特性の改善
  • Muthiah (Mano) Manoharan, PhD - Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.

This presentation will focus on key advances in these areas: 1) LNP mediated delivery and the approval of the first RNAi therapeutic; 2) Advances in GAlNAc conjugates and 3) Chemical modifications for improving the pharmacology of siRNAs.

2:30pm - 3:00pm 30 mins
基調講演
セマグルチドの創薬−AlaスキャンからGLP-1類似体の構造設計へと至る道筋
  • Jesper Lau, PhD - Vice President, Protein and Peptide Chemistry, Novo Nordisk A/S
3:00pm - 3:30pm 30 mins
Info
基調講演
オリゴヌクレオチド治療薬開発の新たな戦略と技術
  • Thazha P. Prakash, Ph.D. - Director, Medicinal Chemistry, Ionis Pharmaceuticals

Inability to selectively deliver antisense oligonucleotide (ASO) therapies to ß-cells is a substantial barrier to the development of treatments for ß-cell specific diseases. We show that the GLP1 receptor can be used as a targeting approach for efficient and selective delivery of ASO to pancreatic ß-cells in cells and in animals.

3:30pm - 4:00pm 30 mins
ネットワーキングブレイク
4:00pm - 4:30pm 30 mins
Info
基調講演
擬天然ペプチドのリボソーム型生体外発現に関する最近の研究成果
  • Hiroaki Suga, Ph.D. - Professor of Chemistry, School of Science, University of Tokyo, Japan

This lecture discusses recent progress in ribosomal in-vitro expression of pseudo-natural peptides containing D-amino acid, beta-amino acids, and other exotic amino acids. This method opens a new opportunity to explore novel sequence space of pseudo-natural peptides.

6:00pm - 7:00pm 60 mins
ネットワーキングディナー

* 不測の事態により、事前の予告なしにプログラムが変更される場合があります。