Cell Therapy Formulation Development

Unlike traditional drug therapies, cell therapy uses living cells to treat or cure diseases. It provides new therapeutic ideas for many diseases related to aging, such as tumor, metabolic syndrome, autoimmune disease, infection, cardiovascular and cerebrovascular diseases and neurodegeneration.

Successful cell therapy formulation not only would guarantee viabilities and functionalities of the therapeutic cells but also meet the concerns regarding stability, scalability, and even patient safety. Proper formulation is critical for long-term storage, effective delivery, and potency consistency in cell therapies. BOC Sciences support cell therapy formulation development with professional services to help in creating high-quality formulations meeting regulatory standards.

Gene vs cell therapy

Gene Therapy: Gene therapy involves inserting a normal gene into the cells of a person with a genetic disorder who lacks the normal gene. This technique is called gene insertion therapy or insertional gene therapy. This usually involves the delivery of genetically derived material into cells via vectors, usually viruses, that have been modified to deliver human DNA.

Cell Therapy: Cell therapy refers to the injection of viable human cells into a patient's body to treat disease. The goal is to restore or replace diseased tissues and cells. The method requires healthy autologous or allogeneic cells to replace diseased cells. At present, cell therapy is mainly divided into two categories: immune cell therapy and stem cell therapy. Stem cells are used in cell therapy because of their potential for multi-cell type differentiation. Genetically modified CAR T-cells are able to recognize and attack cancer cells.

Injection of CAR T cell. (Hosseinkhani, N., 2020)

Our cell therapy formulation development services

Formulation design and optimization

The properties of different cell types (stem cells, immune cells, somatic cells) affect the formulation process, and BOC Sciences customizes the formulation according to the specific needs of the cell types provided by its customers. For example, stem cells often require specific growth factors and a defined extracellular matrix in order to maintain their pluripotency and differentiation potential. For immune cells, such as T cells, careful cytokine supplementation is required to support their activation, expansion, and persistence after infusion.

At BOC Sciences, formulation optimization also includes adjustments to media and supplements. The development of customized media covering essential nutrients, growth factors, and cytokines can significantly promote cell growth and therapeutic potential. In addition, supplemental growth factors such as EGF (epidermal growth factor) and FGF (fibroblast growth factor) help promote cell survival and proliferation during culture expansion.

Cryopreservation and storage solutions

Cryopreservation provides a means of preserving the integrity of the treatment cells for long-term storage and subsequent use. Among the most critical tasks in cryopreservation is the maintenance of cell survival through both the freezing and subsequent thawing processes. This requires the appropriate formulation of a cryoprotectant solution that has an optimized refrigeration protocol. Cryopreservation of cell therapy products at BOC Sciences is executed in conditions that ensure the activity of therapeutic cells and functional capability is maintained right after their defrosting. Parts cryoprotectants, such as DMSO, are prepared in such a way to reduce intracellular tendencies toward ice crystal formation and cellular stress. Large-scale freezing protocols also need to be developed, especially in manufacturing plants where the consistency and repeatability among various batches of production have to be maintained.

Stability and release dynamics research

Cell therapy preparations will be effective only if they are stable during storage. For this reason, stability testing is a necessary means to evaluate the performance of different formulations under various environmental conditions. The impact on various factors including temperature, humidity, pH, and oxygen concentration on cell survival and therapeutic potential must be made.

At BOC Sciences, full stability testing on all formulations is executed, including cell viability, proliferative capacity, and therapeutic efficacy, to ensure the final product remains effective and safe during storage.

Release kinetics and biological effect studies

For therapies in which cells secrete therapeutic molecules, such as cytokines or growth factors, release kinetics studies are required. Determine the dynamics of the release of these bioactive molecules from cells and their effects on target tissues.

Understanding the relationship between release rates and biological effects is critical to designing therapies with an ideal therapeutic window. In CAR-T cell therapy, cytokine release may be a key factor in determining the effectiveness of the treatment. At BOC Sciences, we use advanced in vitro and in vivo models to evaluate the efficacy and safety of formulations by monitoring cell migration and cytokine release curves.

Functional testing and toxicity evaluation

Functional testing of cell therapy involves assessing its ability to perform specific therapeutic functions, such as immune response activation, tissue regeneration, or direct targeting of tumors. In addition to functional efficacy, BOC Sciences performs cytotoxicity and immune response assessments. This is key to ensuring that cell therapy agents do not trigger an adverse immune response or excessive inflammation, undermining the effectiveness of the treatment or raising safety concerns.

Quality research and verification

Rigorous sterility testing and endotoxin testing are the basis for ensuring that preparations do not contain any contaminants that could compromise patient safety. Microbiome load monitoring is particularly important during the cell culture and cryopreservation phases, as these processes can introduce contamination risks if not performed properly. At BOC Sciences, we implement comprehensive quality control protocols, including biometric load testing and process monitoring, to ensure that cell therapy formulations meet regulatory standards and are free of contaminants.

Preclinical safety evaluation

Preclinical safety studies are critical to evaluate the safety and efficacy of cell therapies prior to human trials. This includes the use of animal models to evaluate therapeutic effects, such as tumor regression or tissue regeneration. In addition, immunogenicity studies are performed to assess whether the therapy may trigger immune rejection when administered. BOC Sciences addresses safety concerns and provides the data needed for clinical trial applications by designing comprehensive preclinical protocols.

Scalability and process validation

When cell therapy agents are optimized at the laboratory scale, processes need to be developed that can be mass-produced without compromising quality.

BOC Sciences works closely with customers to ensure that cell therapy formulations can be manufactured at scale and to verify that the manufacturing process can be adapted to larger batches, addressing issues such as batch variance, cost effectiveness and consistent quality control.

Why choose BOC Sciences?

Professional knowledge and industry experience: With more than 20 years of experience in the biochemical and pharmaceutical industries, BOC Sciences understands cell biology, formulation science, and regulatory requirements. This expertise enables us to help our clients meet complex challenges in the development and optimization of cell therapy agents.

Time efficiency and faster time-to-market: One of the biggest advantages of working with BOC Sciences is our ability to accelerate the development process. By applying leading-edge technologies and streamlining workflows, we help our customers shorten development cycles and get cell therapy products to market faster.

Compliance and global standards: It is critical to ensure that cell therapy agents meet the strict regulatory standards of agencies such as the FDA and EMA. BOC Sciences helps clients navigate complex regulatory environments, providing the necessary documentation and support to ensure compliance throughout the development process.

Seamless transition from lab to mass production: Our expertise in scale production ensures a smooth transition from a small laboratory environment to large-scale commercial production. This ability is essential to ensure that cell therapies are not only effective, but can broadly benefit the patient population.

FAQ

1. What is cell therapy?

Cell therapy is the treatment of disease by administering living cells to patients. These cells can come from the patient's own body (autologous) or a donor (allogeneic).

2. How to prepare cell therapy?

Cell therapy preparations are designed to maintain cell activity and function. This involves suspending the cells in a biologically compatible medium to support their survival and activity during storage and application.

3. What are the key parameters in cell therapy agents?

Key parameters include cell viability, potency, concentration, purity, and stability of the formulation medium.

4. How to ensure the sterility of cell therapy products?

Aseptic treatment, sterilizing filtration and rigorous quality control testing ensure that cell therapy products are not contaminated.

5. What are the storage requirements for cell therapy products?

Cell therapy products may require cryopreservation (e.g. liquid nitrogen storage) or refrigeration, depending on the characteristics of the cell and the formulation.

6. What types of cells does BOC Sciences deal with in the development of cell therapeutics?

We deal with multiple cell types, including stem cells, immune cells (such as T cells, CAR T cells), and somatic cells, and each cell type requires a customized formulation strategy to optimize its therapeutic potential.

7. How does BOC Sciences ensure the safety of cell therapy agents?

We ensure that preparations meet safety standards prior to clinical use through comprehensive toxicity testing, immune response assessment and preclinical safety assessment.