Cell and Gene Therapy

Understanding the Revolutionary Therapeutic Approach

Cell and gene therapy represent a significant advancement in medical science, offering hope for curing previously incurable conditions.

The success of these therapies relies heavily on precise and reliable manufacturing processes and quality assessments.

METTLER TOLEDO's advanced instruments and software solutions play a crucial role in supporting these innovative treatments, ensuring they are safe, effective, and of the highest quality.

Cell and gene therapy (CGT) represent transformative advancements in modern medicine, aiming to treat and potentially cure diseases by repairing or replacing malfunctioning cells or genes.

These therapies offer groundbreaking potential for conditions previously considered untreatable.

CGT addresses the root causes of diseases, offering long-term and potentially permanent solutions.

Gene therapy can introduce a healthy copy of a gene into cells, while cell therapy can replace damaged cells with healthy ones.

Cell Therapy

Cell therapy involves the introduction of new, healthy cells into a patient to replace damaged or missing ones.

These cells can be sourced from the patient (autologous) or a donor (allogeneic).

Gene Therapy

Gene therapy focuses on modifying or replacing defective genes to cure genetic disorders.

This involves inserting genetic material into cells to correct or replace faulty genes, often using viral vectors to deliver the therapeutic gene.

See Upstream and Downstream production processes on the scheme.

The Manufacturing Process

The manufacturing process for CGT involves several complex steps to ensure therapies are safe, effective, and high-quality.

Cell culture

Cell culture starts with preparing a nutrient-rich medium to grow and maintain healthy cells.

Monitoring pH and conductivity ensures optimal conditions. Cells are then expanded in a bioreactor until they reach the desired quantity.

Harvesting

Once cells have grown sufficiently, they are harvested by separating them from the growth medium through centrifugation and filtration to remove impurities and prepare them for further processing.

Purification and Formulation

Harvested cells are purified to remove contaminants, ensuring the therapy's safety and efficacy.

Finally, purified cells are formulated into a final product for patient administration.

Quality Assessment in CGT

Quality assessment is critical at every stage of CGT manufacturing to ensure therapies are safe and effective.

Various analytical methods monitor and verify the quality of raw materials, intermediate products, and final therapies.

METTLER TOLEDO offers a range of instruments and solutions supporting CGT development and manufacturing.

Parameter	What is to be measured?	Why is it measured?	MT Solution
UV/Vis	Absorbance/ Transmittance	To determine the concentration and purity to check contaminants, purification efficiency, and stability of the product.	UV/Vis Excellence	Offline
Cell count and viability	Cell count and viability	To determine the density of cell culture used during development of CGTs and the dose of the final product.	CytoDirect Cell counter	Offline
Characterizing cellular and biochemical parameters	Absorbance	High-throughput cellular assays aimed at determining toxicity, proliferation, or protein production of cultured cells	PlateDirect	Offline
pH	pH	Monitoring pH is required for the metabolism and growth of the cells. For pH optimization, cells and genes are pH-sensitive	pH sensor pH meter	Inline Atline
Dissolved oxygen	Dissolved oxygen (DO) level	Monitoring oxygen levels is required for the metabolism and growth of the cells	Dissolved oxygen sensor	Inline
Conductivity	Conductivity	Monitoring conductivity is essential to ensure product quality and consistency, check if the cells are dividing, growing, or dying.	Conductivity sensor	Inline
CO₂ accumulation	Dissolved CO₂ level	Measuring CO₂ concentration enables the adjustment of the nutrient delivery rate and waste removal to optimize the environmental conditions for maximum cell growth and productivity.	CO₂ sensor	Inline
Turbidity	Turbidity	Turbidity gives an idea about the expansion of bacterial culture and the absence of contaminants.	Turbidity sensor	Inline
Optical density	OD 600	Monitoring optical density helps track the growth of microbial cells and the quality of the product.	UV/Vis Excellence Spectrophotometer	Offline
Glucose	Enzymatic determination with UV/Vis	Quantification of glucose is needed to ensure adequate nutrients for cell growth and maintain metabolic processes, including producing proteins and other cellular components.	UV/Vis Excellence Spectrophotometer	Offline
Weight/Volume	Mass	Accurate weighing of each component added to ensure the product's safety and efficacy, consistency in the composition, and controlled addition of viral vector.	Analytical balances Precision balances	Atline/Inline
Temperature	Temperature	The process is temperature sensitive	-	-
Pressure	Pressure	Measuring pressure helps to control the flow of fluid and speed of reaction	-	-