Gene Therapy

Robust commercial scale bioprocessing workflows must be established along with strategies for sterility assurance during the production of allogeneic products.
Bioprocessing considerations include:
• Establishing a robust process
• Ensuring process compatibility
• Creating the sampling plan
• Planning for media production

Sterility assurance considerations include:
• Using GMP materials
• Qualifying raw materials
• Adopting closed processing
• Performing aseptic process simulations
• Ensuring proper sterile filtration
• Understanding extractables, leachables, and particulates

The AAV vector manufacturing process involves cell lysis and nuclease treatment. Advanced techniques, such as using high salt concentration and a salt-tolerant endonuclease, can enhance vector titer and infectivity. Additionally, the development of a non-animal origin, salt-tolerant Benzonase® endonuclease has shown significant impact on AAV5 capsid titers and AAV2 vector infectivity.

In this webinar, you will learn:
• Key principles of Business Continuity Management
• Best practices for a robust supply chain
• How it translates into success stories, like with the Benzonase® family.

• The gene therapy field is poised to deliver an increasingly broad and impactful set of treatments across many different diseases.
• A linchpin for success is a robust, cost-efficient, and scalable viral vector manufacturing workflow.
• Standardized production methods and fit-for-purpose workflow technologies are now available to address the unique development and manufacturing needs of viral vectors.
• Access to the necessary capacity and expertise along with strategies for a simplified pathway to GMP manufacturing, a shorter process development timeline, and a reduction in the exorbitant fixed costs associated with facility development are also available to help realize the true potential of gene therapy manufacturing space.

With a process monitoring tunnel you can monitor 100s of CPPs in a single graphical interface that captures the overall health – both past and present – of your bioprocess and reliably predicts its future health.

• While gene therapies can offer remarkable outcomes for patients, realizing their full potential requires an efficient workflow for viral vector production.
• Faced with limited process development and manufacturing expertise, gene therapy innovators can partner with integrated solution providers to fill these gaps and ensure a robust, optimized, and scalable viral vector production process.
• An integrated solution provider offers significant advantages in contrast to an assemblage of multiple suppliers focused on individual workflow steps which may lack a holistic view of operations.

Take a few minutes and navigate through our mAb, ADC, mRNA, Vaccine, and Cell and Gene Therapy process templates. Go ahead and dive into each unit operation to uncover the best option for your process.

• Viral vectors are the most reliable and sought-after method for gene delivery.
• Use of adherent cell culture for manufacturing these vectors, however, can lead to limited cell growth, lower productivity, and necessitate use of animal-derived media supplements which increase the risk of introducing adventitious agents.
• The VirusExpress® 293 AAV production platform reduces risk and accelerates timelines with use of chemically defined animal component-free cell culture media that has been optimized for viral vector manufacturing.

21 CFR Part 11 and Annex 11’s purpose is to ensure electronic records and signatures are equivalent to paper-based records and handwritten signatures. Beyond proving equivalency, software used for electronic records and signatures offer benefits like access control, audit trails, data integrity, and easy search and retrieval of information.

• Cell and gene therapies have delivered remarkable outcomes for patients with a wide variety of diseases and conditions.
• These therapies rely on adeno-associated viral (AAV) vectors and lentiviral vectors and are essential for delivery of genetic material into target cells.
• Use of adherent cell cultures for vector production are limited in terms of scalability and have historically relied on media formations that were not optimized and supplemented with fetal bovine serum.
• In contrast, suspension culture systems deliver significant benefits for upstream viral vector production processes including higher AAV and lentivirus vector titers and robust scalability.