Advanced Training in Nucleic Acid Formulation and Manufacturing

Introduction:

Nucleic acid therapeutics, including DNA, RNA, and PNA, hold tremendous promise for revolutionizing healthcare through their ability to target and modulate disease mechanisms at the molecular level. However, the successful development and commercialization of nucleic acid-based drugs require overcoming numerous formulation challenges, including stability, delivery, and regulatory compliance. This advanced training program offers a comprehensive overview of formulation strategies and manufacturing considerations for nucleic acid therapeutics, encompassing preformulation studies, process development, regulatory requirements, and emerging technologies. Through a combination of theoretical learning and practical application, participants will gain the knowledge and skills necessary to drive innovation and excellence in nucleic acid formulation and manufacturing.

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Trainer Profile:

Dr. Len Pattenden, Managing Director, CMC Biopharma Ltd

Len has over 30 years of experience in both industry and academia. Len has been a company founder, COO and GMP site head/license holder, overseeing biopharma development and manufacture as well as outsourced CDMO activities. Len has undertaken development and manufacturing of 18 new biological entities, including first-in-man/first-in-class cell and gene therapies, viral vectors, biologics, and monoclonal antibodies. Len’s regulatory and licensing experience covers the MHRA, EMA and FDA. Len works as a CMC consultant for small and large pharmaceutical companies. He is a member of a Regulatory working group, the ISPE and BIA

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Methodology

The training program will utilize a combination of lectures, case studies, workshops, and interactive discussions to facilitate active learning and engagement. Participants will have opportunities to apply theoretical knowledge to practical challenges through problem-solving exercises and collaborative activities. The course will be delivered by an industry expert with extensive experience in nucleic acid formulation and manufacturing.

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Key Outcomes

Upon completion of the course, participants will:

• Enhance interdisciplinary collaboration and communication skills to facilitate successful implementation of mammalian cell culture processes
• Gain insights into formulation principles and strategies for nucleic acids, including RNA, DNA, and PNA
• Acquire practical insights into the formulation of viral vectors for gene therapy applications
• Develop the skills necessary to optimize formulations, scale up processes, and navigate regulatory requirements
• Learn about emerging trends and novel technologies in nucleic acid formulation and delivery
• Enhance interdisciplinary collaboration and communication skills to facilitate successful development and commercialization of nucleic acid therapeutics

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Who Should Attend?

This training program is essential for professionals involved in nucleic acid formulation and manufacturing, including:

• Formulation Scientists
• Process Development Engineers
• Quality Assurance/Quality Control Specialists
• Regulatory Affairs Professionals
• Project Managers
• Researchers and Scientists in R&D
• Bioprocess Engineers
• Biopharmaceutical Manufacturing Professionals

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Schedule:
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DAY 1:
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Session 1: Introduction to Nucleic Acid Formulation

14:00 - 15:00 Title: Unravelling the Complexity: Fundamentals of Nucleic Acid Formulation

Summary:

• Overview of nucleic acids: DNA, RNA, and PNA
• Introduction to formulation principles for nucleic acids
• Preformulation considerations and challenges

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Session 2: RNA Formulation: Lipid Nanoparticles (LNPs)

15:00 - 16:00 Title: Harnessing the Power of LNPs: Formulation Strategies for RNA Therapeutics

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Summary:

• Understanding the role of lipid nanoparticles (LNPs) in RNA delivery
• Key considerations for formulating RNA therapeutics using LNPs
• Case studies and practical insights into LNP-based RNA formulations

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16:00 - 16:15 Coffee Break

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