GENE THERAPY

DNA strands with a person in a lab coat in the background.

Gene Therapy Manufacturing Solutions

Gene therapy – modifications on a patient’s genome, e.g. by integration and activation of new genetic material or the deactivation of disease-causing or faulty genes via a viral carrier – have allowed to set milestones in the therapy of a variety of even rare diseases. But although being applied in a more and more experienced manner also beyond clinical trials, genetic therapies will keep requiring the highest possible diligence.

Single Use Support GmbH provides biotechnology companies with specialized single-use solutions for gene therapies to optimize ATMPs (advanced therapies) manufacturing processes – from sterile single-use systems and customizable liquid management systems up to freezing and storing solutions.

Preview of a guide about viral vector manufacturing

Download guide

Guide: Challenges in Viral Vector Manufacturing

Guide about Viral Vector Manufacturing with regards to fluid management challenges. In detail:

  • Scalability in Cell Line Development 
  • Accuracy in Bag Aliquoting
  • Viscosity and Homogeneity in Liquid Transfer
  • Product Viability after Freezing & Thawing
  • Bag Breakages in viral vector storage and shipping

Learn more to navigate 5 these overlooked challenge in the Viral Vector Guide.

Guide: Challenges in Viral Vector Manufacturing
 

Primary packaging for gene therapies

After gene therapy products such as viral vectors have undergone a gene editing process and have been cultured, they are not usually administered immediately. Instead, they must be safely stored ex vivo before they can perform the genetic modification for which they were designed, such as the replacement of a mutated gene or gene transfer.

To ensure the quality and safety of gene therapy products until they are transported to readministration, Single Use Support has developed single-use solutions in different sizes: For instance, RoSS.KSET is designed for the protection of smaller bioprocess containers, such as those needed for gene therapy, where smaller volumes need to be handled.

Solutions

Aseptic Filling and Draining of gene therapy products

To make sure that gene therapy works safely and effectively, the products that patients are provided with must be handled in a secure and sterile manner. This also applies to the filling and draining process, where the risk of cross-contamination and exposure to the environment must be avoided at all costs. Therefore, it is advisable to automate fluid management in gene therapies.

Single Use Support has developed dedicated gene therapy liquid solutions for safe and efficient filling and draining processes – the RoSS.FILL platform is the result of these efforts. The devices are available in different sizes, allow for high flexibility while speeding up turnaround time in gene therapy production.

Solutions

Controlled Freeze and Thaw processes for genetic therapies

Gene therapy products have very specific requirements for their freeze/thaw conditions, and an uncontrolled freezing process might cause irreparable damage to the product. Viral vectors, for example, are not only best stored at ultra-cold temperatures around -80 °C, but also need to be brought to this temperature at controlled cooling rates as they, too, are crucial to their quality.

Single Use Support has been innovating in this field and developed the RoSS.pFTU platform for optimized freezing and thawing of drug products. Based on plate freezing techniques, our freezers are available in different sizes, therefore suitable for various fields of application – be it the freezing of small volumes with RoSS.pFTU Lab Scale or the handling of larger volumes with the RoSS.pFTU Large Scale plate freezer, achieving temperatures as low as -80 °C. If even lower temperatures are required, the cryogenic freezer RoSS.LN2F can freeze products down to -150 °C.

Solutions

Gene therapy products – transport solutions

Gene therapy products have very specific requirements for their freeze/thaw conditions, and an uncontrolled freezing process might cause irreparable damage to the product. Viral vectors, for example, are not only best stored at ultra-cold temperatures around -80 °C, but also need to be brought to this temperature at controlled cooling rates as they, too, are crucial to their quality.

Single Use Support has been innovating in this field and developed the RoSS.pFTU platform for optimized freezing and thawing of drug products. Based on plate freezing techniques, our freezers are available in different sizes, therefore suitable for various fields of application – be it the freezing of small volumes with RoSS.pFTU Lab Scale or the handling of larger volumes with the RoSS.pFTU Large Scale plate freezer, achieving temperatures as low as -80 °C. If even lower temperatures are required, the cryogenic freezer RoSS.LN2F can freeze products down to -150 °C.

Solutions

More Information

Gene therapy manufacturing - 5 requirements to scale up

Gene therapy manufacturing - 5 requirements to scale up

Gene therapy manufacturing continues to cause a stir in the biopharma industry. Gene therapies have become a highly promising field of groundbreaking next generation treatment approaches that use personalized therapies for rare diseases and allow ...

Michael Mühlegger
Michael Mühlegger
Single Use Support providing solutions for gene therapies

Single Use Support providing solutions for gene therapies

Biopharma solution provider Single Use Support announces an order from Genezen, a cell and gene therapy Contract Development and Manufacturing Organization (CDMO). The Indianapolis-based CDMO has ordered RoSS.FILL CGT to fill lentiviral vectors in...

Michael Eder
Michael Eder