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Process development for successful gene therapies

FUJIFILM Diosynth Biotechnologies’ critical strengths include our experienced personnel who are adept at developing processes that are cGMP-ready by design and our state-of-the-art facilities that provide flexible biomanufacturing solutions to common scale-up challenges. Our development to manufacture design retains flexibility to incorporate process optimizations to improve your product’s safety, stability, and efficacy attributes.

To successfully manufacture gene therapy and viral vaccine products – your process needs to be cGMP-ready by design. From the very beginning, our main focus is to achieve a robust and reliable large-scale manufacturing process.

Process development involves several challenges, including optimizing the upstream/downstream processes, cost-efficient scale-up, and monitoring and achieving the required product quality attributes.
We strive to address these challenges though concerted efforts, utilizing our experience and foresight to identify areas of greatest risk well ahead of cGMP manufacture. In the event that any challenges arise, our team are always able to work together, with our partners, to achieve the best possible outcome.

It all starts with choosing the right combination of a high quality host/virus system. A choice that will mainly depend on the system’s ability to effectively produce the target product, but safety and toxicity needs to be taken into account as well.

Viral vectors

Our team of experts have broad experience in working with BSL-2 viruses and routinely work a variety of viral vectors including:

  • Several serotypes of Adenovirus and recombinant adenoviral vectors
  • Several serotypes of recombinant Adeno-associated virus (AAV)
  • Recombinant Lentiviral vectors
  • Poxvirus
  • Baculovirus
  • Influenza virus
  • Herpes simplex virus

Host cell platforms for upstream development

Selecting the right host cell is critical, because different host cells have different capabilities and different growth properties, including their suitability for growth in adherent vs suspension cultures.

  • HK-293 can serve as an excellent production cell line to make AAV and lentivirus. Typically in suspension processes.
  • C6 human cell line are often used for propagation on Influenza A and B.
  • Sf9/Sf21 are well-established hosts with regulatory traction.
  • CHO are well-established, characterized and proven hosts with great versatility.
  • EB66 are mainly used for antibody production.
  • Vero have regulatory traction with an approved product, but are not compatible with suspension processes which may present some manufacturing challenges.
  • MDCK have good virus growth advantage and adapt well to serum free media.

Viral Gene Therapy expression systems

Vaccine expression systems

FUJIFILM Diosynth Biotechnologies is a global leader in gene therapy development and production. We use a wide variety of cell-based expression systems to produce early and late-phase viral vectors and advanced therapies manufacture.

Gene therapies

Downstream Process Development

One of the major steps in development of an optimal, scalable and cost effective production process is identifying and establishing an appropriate downstream process. Delays in optimization and establishment of ideal purification strategies not only add to the product development costs but can also significantly delay commercialization of the products.

Normally, downstream purification involves at least a clarification step, an ultra-filtration step, chromatography steps to capture and refine the product, and virus inactivation steps for safety purposes.

But there is really no generic solution to purification, because different vectors present different purification challenges. A deep understanding of the properties of the specific vector is critical, because characteristics like stability and isoelectric point will determine the optimal parameters and conditions. To minimize the number of steps in the purification of your specific biopharmaceutical and achieve maximum yield, you will need an experienced team. We can be your partner.

Experience has taught us a few guiding principles. Commonly used downstream capabilities include:

  • Clarification, depth filtration
  • Tangential flow filtration
  • Chromatographic purification techniques
  • Final formulation
  • Sterile Filtration
  • Viral Inactivation Techniques
  • Viral clearance support