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Single-Use Technology for Gene Therapy

 Gene therapies target diseases by replacing, inactivating or introducing genes into cells— either inside the body (in vivo) or outside of the body (ex vivo). This technique treats a disorder by replacing a faulty gene or adding a new gene into a patient's cells instead of using drugs or surgery. By altering the genetic material of a patient, gene therapies allow the patient to begin producing healthy cells on their own.

FDA Gene Therapy Definition

A treatment that “seeks to modify or manipulate the expression of a gene or to alter the biological properties 
of living cells for therapeutic use.”

FDA Source

 Gene Therapies: Hope for the Prevention of Diseases

Gene therapies are looking to change medicine from being a treatment-based to a prevention-based practice. With new and ever-evolving genetic engineering technologies, researchers are turning to gene therapies as answers for preventing and treating many genetic diseases that currently have only symptomatic treatment or no treatment standard. Gene therapies offer promise for treating and curing a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.   

The ultimate goal, and hope, is that every genetic disease has a gene therapy treatment. While still an experimental technique, recent gene therapy approvals from the FDA and EMA along with more than 1,000 different types of gene therapies in clinical trials, have seen a rapidly growing industry and patients potentially receiving long awaited treatment.

Viral Vectors Deliver Genetic Materials to Cells

The production of viral vectors is an important component of gene therapy success. A gene is inserted directly into cells by using a carrier, called a vector. The most common gene therapy vectors are viruses. Viral vectors identify certain cells and carry genetic material into the cells' genes. In fact, these viral vectors act as Trojan horses to deliver the genetic materials to cells.

Other Types of Vectors in Clinical Trials:

  • Stem cells – cells from which all other cells with specialized functions are generated, providing new cells for the body as it grows. For gene therapy, stem cells can be trained in a lab to become cells that can help fight disease. 
  • Liposomes – fatty particles that can carry the new, therapeutic genes to the target cells and pass the genes into the cells' DNA. Liposome-based vectors are designed to target specific cells actively and safely by targeting molecules. 
  • Bacterial vectors – carry a plasmid-based gene to a new host cell. The genetic material is delivered by the bacteria entering into the target cells. 
  • Plasmid vectors – vehicles used to drive modified DNA (recombinant) into a host cell where it reproduces in cells, creating a group of cells with the same DNA strand.  

Lean on CPC’s Cell and Gene Therapy (CGT) Expertise

By collaborating with gene therapy manufacturers and partnering with equipment suppliers, CPC helps provide these evolutionary gene therapies in a faster time frame. CPC’s single-use technology for gene therapy helps in making sure efficacious, repeatable and standardized processes are maintained.

CGT manufacturers often struggle with the high costs of production including labor, supply chain and low yields. That’s where CPC comes in. Not only providing single use connection technologies for gene therapy, but CPC also provides guidance in critical areas, such as best practices in closed system design. CPC’s guidance and aseptic connectors offer an advantage that may be necessary in the cost effective and reliable delivery of gene therapy treatments to patients who are counting on them. 

Using a Closed System for Gene Therapy’s Smaller Batches 

While any drug development program can face pitfalls and uncertainties, developing cellular and gene therapies (CGT) present unique challenges above and beyond those encountered for bioprocessing’s small molecule drugs. A closed system designed for gene therapy manufacturing provides the protection of a cleanroom against outside contaminants without the costs associated with maintaining it. Closed systems are becoming widely used in SUT (single-use technology) facilities for monoclonal antibodies, recombinant protein and vaccine production while the adoption of closed systems at lab scale for smaller batches for CGT manufacturing is gaining ground. 

Simplify Gene Therapy Processes with CPC Aseptic Connectors 

 Easy-to-use CPC aseptic connectors help reduce system complexity and production costs by offering enhanced process flexibility and reliability. The simple integration of SUT plug-and play assemblies with CPC aseptic connectors eliminate the complicated process for connecting tubes via tube welding. Using CPC aseptic connectors means there is no need to bring a tube welding cart and all the necessary components into the production suite, and then moved to the biosafety cabinet to be put together. Gene therapy manufacturers designing production processes with single-use sterile connectors no longer experience the costs and delays associated with the extra time needed for open manual processes.

Meeting Gene Therapy Expectations with CPC Aseptic Connectors

  • Broad chemical compatibility
  • Performance integrity down to -80°C
  • Sterilization by gamma or autoclaving methods
  • Reliable, repeatable performance
  • Validation reports available
  • Minimal operator training
  • Broad product portfolio from benchtop to cGMP production

Secure and Sterile with CPC Aseptic Connectors for Gene Therapy

With the labor-intensive and open nature of gene therapy manufacturing, production is highly susceptible to operator error and contamination risks. CPC aseptic connectors, attached to the SUT, ensure a terminally sterilized, closed system process where each biologic drop is secure and sterile.

CPC Single Use Connectors Used for Viral Vector Production

CPC aseptic connectors offer an easy-to-use method for flow path sterility and integrity during gene therapy manufacturing. These single-use connectors help deliver the necessary sterility protection to avoid costly failures from contamination.

Where CPC Aseptic Connectors Are Used in Viral Vector Production Processes

CPC Aseptic Connectors Benefits

  • Remove cumbersome steps in production
  • Minimize costs by closing operations that would normally have to performed under the hood
  • Save time and labor during set up, production and between batches
  • Eliminate cross contamination in between batches
  • Reduce processing area footprint 

CPC Aseptic Connectors: Decades of Experience, Forefront of Innovation

CPC has been delivering aseptic connectors to the biopharmaceutical market for decades. With this extensive biopharma experience, CPC engineers have applied their single-use technology for gene therapy to sterile connector innovation. This laser sharp focus directly led to CPC’s development of AseptiQuik®, Steam-Thru® and MicroCNX® Aseptic Connectors, exclusively designed and built for the complexities of upstream and downstream applications used in gene therapy manufacturing.

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