SMART AAV Analytical Method Toolbox - Live Online Training

Thursday, 6 February 2025 9 .00 - 15.30 h

Course No. 21882

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Speakers

Dr Sabine Hauck

Dr Sabine Hauck

dequra pharma consult hauck, Chair of ECA ATMP Interest Group

Dr. Ulrike Herbrand

Dr. Ulrike Herbrand

Charles River Laboratories Germany

Dr. Rudolf Zirwes

Dr. Rudolf Zirwes

Charles River Laboratories

Dr Amrita Pai

Dr Amrita Pai

Charles River Laboratories

Anna Liznar

Anna Liznar

PathoQuest

All times mentioned are CET.

Objectives

Following a brief introduction to ATMPs and its regulations, this course will explore optimization strategies for the formulation, production, and analysis of adeno-associated viruses (AAVs). Participants will understand challenges and learn practical solutions for efficiency and compliance. The course highlights common obstacles and offers practical solutions demonstrated through case studies, providing insights into applying GMP principles in different scenarios.

In addition, expert speakers from manufacturing, research, and consultancy will share their experiences and lessons from GMP-compliant environments. Through their presentations, participants will gain a well-rounded understanding of the challenges and best practices in maintaining GMP compliance.

Background

Gene therapies are rapidly gaining popularity as a cutting-edge approach to treating a wide range of diseases, offering enormous potential to address conditions from degenerative diseases to rare congenital neurological disorders. These therapies have the potential to revolutionize medicine by targeting the underlying genetic causes of illnesses, providing long-lasting or even permanent therapeutic effects. Central to many DNA-based gene therapies is the use of viral vectors to deliver genetic material directly into the patient’s cells, and among the various viral vectors available, the most commonly used and highly regarded is the adeno-associated virus (AAV).

The importance of AAV in the field of gene therapy cannot be overstated. Discovered in 1965, AAV has become the preferred vector due to its ability to efficiently transfer genetic material while exhibiting a relatively low immunogenic profile, meaning it is less likely to provoke an unwanted immune response. Additionally, AAV is known for its ability to transduce both dividing and non-dividing cells, making it a versatile tool for a variety of gene therapy applications. Its proven safety record, combined with its effectiveness in delivering therapeutic genes, has led to its widespread adoption in clinical trials and commercial gene therapies.

AAV-based gene therapies are already showing promise in treating conditions such as spinal muscular atrophy, hemophilia, and certain inherited forms of blindness, highlighting their growing role in modern medicine. However, as with any biopharmaceutical product, there are significant challenges associated with the development and production of AAV therapies, particularly in defining the appropriate Critical Quality Attributes (CQAs) that ensure the safety, efficacy, and consistency of the final product. Establishing these CQAs is a complex and highly regulated process, and failure to do so correctly can lead to inefficiencies, wasted resources, and even setbacks in the development process. Determining the best way to define and measure these CQAs—without wasting valuable time and financial resources—is a critical task for any organization working in AAV manufacturing.
 
This course will address these challenges head-on, providing valuable insights into how to effectively define CQAs for AAV products. It will explore strategies to optimize both the analytical methods and the manufacturing processes involved in the production of AAV-based gene therapies and their Quality Control. Participants will learn how to navigate these complexities efficiently and gain practical tools for improving the quality and scalability of AAV production, ultimately helping to bring these life-changing therapies to patients more quickly and reliably.

Target Group

This course is addressed to all people involved in the day-to-day work of AAV with manufacturing, method development and optimization and analysis.

Date & Technical Requirements

Thursday, 06 February 2025, 09.00 h – 15.30 h
All times mentioned are CET

We use WebEx for our live online training courses and webinars. At https://www.gmp-compliance.org/training/online-training-technical-information you will find all the information you need to participate in our trainings and you can check if your system meets the necessary requirements to participate. If the installation of browser extensions is not possible due to your rights in the IT system, please contact your IT department. WebEx is a standard nowadays and the necessary installation is fast and easy.

The presentations will be made available to you prior to the Live Online Training as PDF files. After the event, you will automatically receive your certificate of participation.

Programme

SMART AAV Analytical Method Toolbox - Live Online Training

Seminar Programme as PDF

Introduction in ATMP and their Regulations
(Dr Sabine Hauck)
  • EMA and ICH guidelines for ATMPs, focusing on Gene Therapy products
  • GMP for ATMPs
  • Pharmacopoeial monographs for AAV (Pharm. Eur. 3186, 5.34 and 5.12, USP viral vectors)
Chemistry, Manufacturing and Control (CMC)
Testing for AAV Therapeutics

(Dr Rudolf Zirwes)
  • Identity testing
  • Purity testing
  • Safety testing
From AAV Plasmids to Drug Product – Quality Testing with iDTECT NGS on GMP Grade
(Anna Liznar)
  • Virus detection for adventitious virus testing
  • Genome Identity for sequence verification
  • An outlook into genome integrity & residual DNA characterization
AAV Analytical Toolbox for Stability Assessment
(Dr Sabine Hauck)
  • Selection of suitable Methods
  • Analysis of the stability indicating power
  • Learnings for formulation development
Bioactivity for AAV Therapeutics
(Dr Ulrike Herbrand)
  • Matrix Approach
  • MoA reflection
  • Challenges related to references and control items
Building and Breaking AAV Processes
(Dr Amrita Pai)
  • How is development representative of future manufacturing scale? What data can we collect/analyze? How do we scale up?
  • What tools do we have for making quick decisions? Are they worth the potential tradeoffs?
  • What happens when something goes wrong? How can we be good stewards of time/resources?

ECA-Member*: € 790,-
Regular Fee*: € 890,-
EU/GMP Inspectorates*: € 445,-
APIC Member Discount*: € 840,-

(All prices excl. VAT). Important notes on sales tax.

* also payable by credit card
American Express Visa Mastercard

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Further dates on-site
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Recording
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Do you have any questions?

Please contact us:
Tel.: +49 6221 8444-0
E-Mail: info@gmp-compliance.org

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