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Showing of Streams
09:45 - 10:15

Networking Refreshment Break

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12:00 - 12:05
Close of Workshop

Close of Workshop

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Showing of Streams
14:45 - 15:15

Networking Refreshment Break

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Showing of Streams
17:00 - 17:05
Close of Workshops

Close of Workshops

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08:00 - 08:15 15 mins
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Workshop Moderator’s Opening Remarks
  • Vivian Lindo - Associate Director, Analytical Sciences, AstraZeneca
08:15 - 08:45 30 mins
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Analytical Characterization Tools for Peptide Therapeutics Physical Stability
  • Ana Santos - Principal Scientist, Formulations, Principal Scientist, Formulations
08:30 - 09:45 75 mins
Info
Workshop 1: Managing CMC Activities to Accelerate Oligonucleotide Development and Manufacturing
Managing CMC Activities to Accelerate Oligonucleotide Development and Manufacturing
  • Workshop Moderator: Thomas Rupp - Owner & Principal, Thomas Rupp Consulting, Germany

Workshop Description:

This half-day pore-conference workshop will address early to mid-phase drug development and related CMC for oligonucleotide therapeutics. A detailed discussion on oligonucleotide therapeutics moving from discovery to clinical trials will be presented in form of case studies. This includes strategies for early clinical CMC development; early phase CMO work; GMP manufacturing; and the regulatory framework around these activities. Workshop attendees will be allowed 15 minutes open discussion after each presentation to deepen or clarify the presentations.


Who should attend?

Anyone interested in early to mid-stage development of oligonucleotide therapeutics; Anyone interested in outsourcing the manufacturing of oligonucleotide therapeutics to a CMO / CRO. This includes R&D Researchers, Manufacturing Personnel, Quality Assurance, Project Management, Business Development and Scientific Management.

08:45 - 09:15 30 mins
Info
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Development and Validation of a Peptide Bioassay
  • Michael Postlethwaithe, Ph.D - Business Development Manager, Bachem AG

Developed and validated biological assays provide a robust identity or meaningful functional potency measure, and are increasingly requested for synthetic peptides. Biological assays must be fit for a specific target, although many such assays have some common ground, such as statistical rigor to mitigate the inherent variability of biological systems. This presentation discusses our experience with biological assays as GMP release tests, using a GLP-1 peptide agonist as an example.

09:15 - 09:45 30 mins
Info
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Understanding the 3-D structures of a Peptide to Determine the Control Strategy for Biological Activity
  • Mark Drew - Business Programme Lead, AstraZeneca

A variety of analytical techniques were employed to gain insight to the higher order structures of a synthetic peptide. Based on the understanding gained, regulatory insight and bioassay stability data, no biological activity test was deemed necessary on the drug product specification. This position has been approved by regulatory authorities.

09:45 - 10:15 30 mins
Networking Refreshment Break
10:15 - 12:00 105 mins
Info
Workshop 1: Managing CMC Activities to Accelerate Oligonucleotide Development and Manufacturing
Managing CMC Activities to Accelerate Oligonucleotide Development and Manufacturing
  • Thomas Rupp - Owner & Principal, Thomas Rupp Consulting, Germany

Workshop Description:

This half-day pore-conference workshop will address early to mid-phase drug development and related CMC for oligonucleotide therapeutics. A detailed discussion on oligonucleotide therapeutics moving from discovery to clinical trials will be presented in form of case studies. This includes strategies for early clinical CMC development; early phase CMO work; GMP manufacturing; and the regulatory framework around these activities. Workshop attendees will be allowed 15 minutes open discussion after each presentation to deepen or clarify the presentations.

Who should attend?

Anyone interested in early to mid-stage development of oligonucleotide therapeutics; Anyone interested in outsourcing the manufacturing of oligonucleotide therapeutics to a CMO / CRO. This includes R&D Researchers, Manufacturing Personnel, Quality Assurance, Project Management, Business Development and Scientific Management.

10:15 - 10:45 30 mins
Info
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Peptide Oligomers - Friends or Enemies?
  • Lise Giehm, Ph.D - Principal Scientist, Zealand Pharma A/S

Peptides offer enormous growth potential as future therapeutics and are recognized as being highly selective and efficacious. Due to their size, they generally have flexible structures and many have a preference for self-assembly. We will present how to investigate peptide structures in liquid formulations, with a focus on the ability to self-assemble as both stable structures and undesired higher order aggregates.

10:45 - 11:15 30 mins
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Late Breaking Presentation
11:15 - 12:00 45 mins
Workshop 2: Analytical Strategies and Technologies for Peptide Therapeutics
Panel Discussion with Workshop Speakers
12:00 - 12:05 5 mins
Close of Workshop
13:00 - 13:15 15 mins
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Workshop Moderator’s Opening Remarks
  • Stefan Vonhoff - Vice President CMC, NOXXON Pharma AG
13:00 - 13:15 15 mins
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Workshop Moderator’s Opening Remarks
  • Mimoun Ayoub, PhD - Director and Head of North American and Emerging Markets, CordenPharma International
13:15 - 13:45 30 mins
Info
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Optimization of Novel Polymeric Delivery Vehicles by Chemical Evolution
  • Ernst Wagner, Ph.D. - Professor and Chair, Pharmaceutical Biotechnology,, Ludwig Maximilans University

Chemical evolution for optimizing synthetic drug delivery carriers includes identification of delivery motifs (e.g. artificial amino acids), their assembly into defined sequences by solid phase synthesis, screening and selection for a defined cargo (nucleic acid, protein, Cas9/sgRNA) followed by carrier sequence variation and next selection round.

13:15 - 13:45 30 mins
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Stage Appropriate CMC Overview and Requirements for a Robust Dossier
  • Mimoun Ayoub, PhD - Director and Head of North American and Emerging Markets, CordenPharma International
13:45 - 14:15 30 mins
Info
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Investigations into Disruptive Delivery Approaches for LNA Antisense Oligonucleotides (ASO LNA)
  • Dr. Michael Keller, Ph.D. - Senior Principal Scientist, pRED, pCMC, Roche

Parenteral or intrathecal administration of antisense oligonucleotides (ASO) have enabled treatment of liver and CNS based diseases, respectively, thanks to the inherent high exposure of the ASO in these tissues. To extend the possible scope of indications to treat with ASO, we looked at feasibility concepts to deliver ASO LNA into tissues where exposure with unformulated ASO LNA generally is insufficient for PD effect.


13:45 - 14:15 30 mins
Info
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
CMC Technical and Regulatory Strategies for Development of Peptides and Oligonucleotides
  • Gary Musso, PhD - President, Musso and Associates LLC

Many factors guide the development pathways taken to meet drug demands and the regulatory requirements of a peptide or oligonucleotide.  Key factors include:  Finance, Support, Clinical Phase, Geography.  I will overview the factors that influence CMC development of “tides” and strategies to keep the program successful when faced with obstacles.

14:15 - 14:45 30 mins
Info
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Oligonucleotide Drug Product (Development) for (Ultra) Orphan Ophthalmic Diseases
  • Vera Brinks - Director, Pharmaceutics, ProQR Therapeutics

Oligonucleotide drug product (DP) development for (ultra) orphan ophthalmic diseases can be challenging from a formulation, primary packaging and manufacturing point of view. This presentation will elaborate on some of the challenges related to intravitreal (IVT) administered products, such as endotoxin and sub-visible particle specifications of DP. Also, considerations for dose accuracy, and the use of (prefilled) syringes will be discussed. Additionally, this presentation will include the possibility of terminal sterilization for oligonucleotide-based products.

14:15 - 14:45 30 mins
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Scale-up Peptide Manufacturing Case Study: Transition from Solid-phase to Liquid Phase Synthesis
  • Bruce Morimoto, PhD - Vice President, Drug Development Operations, Alkahest
14:45 - 15:15 30 mins
Networking Refreshment Break
15:15 - 15:45 30 mins
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Challenges for Peptides Drug Products at the Interface of Formulation, Primary Packaging and Application
  • Stephanie Lemoult, PhD - Senior Principal Scientist-Team Leader, Formulation, Lonza AG
15:15 - 15:45 30 mins
Info
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Drug Product Development and Industrialization for Peptides and Oligos: A CDMO Perspective
  • Umberto Romeo - R&D Manager, Corden Pharma

This presentation will provide a CDMO perspective on the challenges, the requirements and the technologies needed for the successful  formulation, process development and industrialization of oligonucleotide and peptide-based drug products.

15:45 - 16:15 30 mins
Info
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Formulation Development and Device Options for the Subcutaneous Injection of Spiegelmer Drug Product Solution
  • Stefan Vonhoff - Vice President CMC, NOXXON Pharma AG

For pegylated oligonucleotides, concentration and viscosity of the drug product solution are key parameters influencing the choice of devices suitable for subcutaneous self-administration. Data from a formulation development study aiming to reduce the viscosity of the pegylated Spiegelmer solution will be presented. Stability, compatibility and feasibility of the optimized drug product solution were evaluated in a range of devices. Conclusions for the further development of the drug product/device combination will be discussed.

15:45 - 16:15 30 mins
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Late Breaking Presentation
16:15 - 16:45 30 mins
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Late Breaking Presentation
16:15 - 17:00 45 mins
Workshop 4: Accelerating Oligonucleotide and Peptide Drug Development
Panel Discussion with Workshop Speakers
16:45 - 17:00 15 mins
Workshop 3: Drug Product Development Strategies for Oligonucleotides and Peptides
Concluding Remarks and Discussion
17:00 - 17:05 5 mins
Close of Workshops

* 活动内容有可能不事先告知作更动及调整。