Pre-Conference Workshops (am) & Main Conference Day 1 Plenary (pm) - CET (Central European Time, GMT+01:00)
The workshop will give an overview of manufacturing processes and CMC strategies for long and complex oligonucleotides, like sgRNA or aptamers. It will emphasize the CMC development from early discovery phase throughout the clinical phases and spotlight on cleaning/carryover, process development, analytical development, raw materials selection and control, impurity characterization, analytical tools and setting of specifications. The workshop will be split into a manufacturing-focused part ranging from early process development to scale-up, and an analytical part addressing process-, and product-related impurity formation and their control. After the presentations the speakers will jointly chair a panel discussion on CMC aspects. Furthermore, bioanalytical tools for oligonucleotides will be addressed to support preclinical and clinical development. Different bioanalytical assays will be presented in terms of their specific characteristics.
Topics to be Discussed:
- CMC activities for long and complex oligonucleotide therapeutics or theranostics
- Raw material selection and control
- Understanding and controlling process and product-related impurities
- Analytical and bioanalytical tools and their application
- Specific and sensitive bioanalytical methods to investigate pharmacokinetics, biodistribution as well as elimination
Who Should Attend?
Anyone interested in CMC, Impurity Control Strategies, and in-house or outsourced manufacturing of oligonucleotide therapeutics during pre-clinical and clinical development of synthetic RNA Therapeutics or Theranostics. This includes R&D Researchers, Manufacturing Personnel, Quality Assurance, Project Management.
- Thomas Rupp - Managing Director, Axolabs Berlin GmbH
- Chris Oswald - Founder, Owner, and Principal Consultant, Coswald Consulting LLC
- Chris Oswald - Founder, Owner, and Principal Consultant, Coswald Consulting LLC
- Thomas Rupp - Managing Director, Axolabs Berlin GmbH
- Christian Heinlein - Group Leader Bioanalytics at Axolabs GmbH, Axolabs GmbH
- Andrea Schuster - Senior Scientist, Axolabs GmbH
- Judy Carmody, Ph.D. - Founder and Principal Consultant, Carmody Quality Solutions, LLC
- Melanie Cerullo - Cheif Quality & Regulatory Officer, ReciBioPharm
This presentation delves into innovative Chemistry, Manufacturing, and Controls (CMC) strategies aimed at expediting development processes without compromising quality. Emphasizing clear communication channels and streamlined workflows, it offers practical approaches for maximizing efficiency in pharmaceutical development, regulatory compliance, and manufacturing. By harnessing the power of CMC, organizations can navigate challenges, enhance collaboration, and achieve accelerated development timelines while upholding stringent quality standards.
- Judy Carmody, Ph.D. - Founder and Principal Consultant, Carmody Quality Solutions, LLC
In the rapidly evolving field of gene editing, development and manufacturing demand a comprehensive understanding of CMC processes. This presentation introduces a case study that delves into the CMC best practices and invaluable lessons learned in the journey from sequence to IND for a Gene Editing Program. This case study reflects on the challenges faced, setbacks encountered, and the adaptation of strategies during the entire development/manufacturing process. Insights gained from real-world experiences offer valuable guidance to sponsors and serve as a vital resource for CMC stakeholders, offering a roadmap for success in bringing gene editing therapies from sequence to IND.
- Melanie Cerullo - Cheif Quality & Regulatory Officer, ReciBioPharm
Workshop Overview:
Over the last 20 years, peptide therapeutics have evolved from simple to complex. Peptide modifications are very common including the incorporation of unnatural amino acids, conjugation, and cyclization. This workshop will consist of a collection of presentations with a focus on various peptide modification chemistries leading to improve metabolic and therapeutic properties with a focus on case studies as examples of these approaches and applications.
- Bruce Morimoto, PhD - Vice President, Drug Development, Alto Neuroscience
- Alastair Hay, Ph.D. - Vice President, Peptides, Almac
- David Craik, PhD - Professor of Biomolecular Structure, University of Queensland
- Anna Koijen, PhD - Principal Scientist, EnzyTag B.V.
- Alastair Hay, Ph.D. - Vice President, Peptides, Almac
- Christian Becker, Ph.D. - Professor and Head of the Institute of Biological, University of Vienna
- William Fang - Vice President of Oligonucleotide and Peptide Development, WuXi TIDES
- Youme Gim - Team Leader, Oligonucleotide CDMO I, ST Pharm
- Yogesh Sanghvi, PhD - President, Rasayan Inc.
Methylated nucleotides are known to shape structures and functions of natural and therapeutic RNAs. We discovered the catalytic potential of RNA as methyltransferase ribozyme and continue to develop novel ribozymes for posttranscriptional installation of site-specific RNA modifications. Combining guide and enzyme in one molecule of functional RNA, RNA-alkylating ribozymes find synthetic applications in vitro and are promising for modifying intracellular targets.
- Takumi Okuda, PhD - Postdoc, Laboratory of Claudia Höbartner, University of Würzburg
As our drug modality landscape is expanding, so are our therapeutic opportunities with the promise of unlocking curative and disease-modifying outcomes. Driving drug modality innovation with both internal discoveries and external partnerships is core to Novo Nordisk. Exemplified by our nucleic acid based portfolio, we are with a strategic blend of internal and external investments building a pipeline and platform technologies with the aim of transforming nucleic acid therapies from the few patients today to the many tomorrow.
- Karina Thorn, PhD - Corporate VP and Head of Research, Global Nucleic Acid Therapies (GNAT), Novo Nordisk
Since the early clinical findings of using glucagon like peptide-1 (GLP-1) to regulate blood glucose levels in the late 80´s there has been an increasing interest to discover and develop GLP-1 receptor agonists for treatment of metabolic disorders. The increased non-clinical and clinical understanding of the mechanism of action of GLP-1 and the parallel technical development have now brought the scientific community to a very high level of understanding with structural insight of the interaction of peptides and small molecules with GPCR class B receptors. The discovery of semaglutide is a fantastic example on how technology and biologic understanding have developed in parallel ending with a superior peptide for treatment of diabetes and obesity.
- Jesper Lau, PhD - Vice President, Digital Science & Innovation, Novo Nordisk A/S
- Patrizio Mattei, PhD - Expert Scientist, Medicinal Chemistry, F. Hoffmann-La Roche Ltd.
Founded in January 2020, n-Lorem is a non-profit discovering, developing ,manufacturing and providing personalized ASO treatment for nano-rare patients for free for life. Patients, with pathological nano-rare mutations (n<30 WW) are by definition the rarest of rare patients. These patients are rarely diagnosed and cannot be addressed today with any commercial model. N-Lorem has industrialized the ASO treatment of these patients. Our approach, experience and learnings will be discussed.
- Stanley Crooke, MD, PhD - Founder and CEO, n-lorem Foundation