What is Cell and Gene Therapy? | Cell and Gene Therapy Essentials

Explore the fundamentals of cell and gene therapy and uncover existing solutions to key challenges in meeting patient demand.

Cell and gene therapies represent a diverse, emerging class of drug products that work by directly modifying human cells and tissues or altering genetic material to treat disease at the molecular level. These therapies offer promising solutions for conditions that were previously considered untreatable.

Cell Therapy

A cell therapy product is composed of cells intended to replace, repair, or supplement existing cells or tissues to treat or prevent disease. These therapies can include a variety of cell types, among the most common are stem cells and immune cells. Among the most successful cell therapies are those treating blood disorders, such as sickle cell disease and beta-thalassemia, where stem cell transplants have shown significant success in curing or alleviating the symptoms of these conditions.

Gene Therapy

A gene therapy product is a nucleic acid-based therapy intended to regulate, repair, replace, add, or delete a genetic sequence. This can be achieved using various delivery methods, including viral vectors like adeno-associated viruses (AAVs) and lentiviruses (LVs), as well as non-viral vectors such as lipid nanoparticles. These vehicles deliver therapeutic genes to the patient's cells, where they can correct genetic defects, regulate gene expression, or provide new functions. Recently approved gene therapies, such as those for spinal muscular atrophy (SMA) and inherited retinal diseases, have demonstrated their potential to provide long-lasting and potentially curative treatments.

Differences Between Cell and Gene Therapies

Cell and gene therapies differ in their mechanisms and applications. Cell therapies focus on the use of living cells to restore or enhance tissue function, whereas gene therapies involve the manipulation of genetic material to achieve therapeutic effects. Despite these differences, the two approaches often overlap. For example, in gene-modified cell therapies, cells are engineered to express therapeutic genes before being administered to the patient.

Cell and Gene Therapy Manufacturing

The manufacturing processes for cell and gene therapies are distinct from those of more mature modalities, such as monoclonal antibodies (mAbs). These established bioprocesses are well-defined and standardized, allowing for consistent and scalable manufacturing. In contrast, cell and gene therapies are much newer, often more complex, requiring tailored production processes to overcome new challenges.

Cell and Gene Therapy Manufacturing Challenges

At Sartorius, we consider cell and gene therapy manufacturing challenges to fall into three categories.

• Performance – Poor reproducibility is caused by variable source material and highly sensitive, complex, and manual processes.
• Scalability – Scaling up is hindered by tedious revalidation, costly optimization, and lower yields, largely due to processes not being developed with scalable and cost-effective technologies.
• Regulatory compliance - Managing an efficient, high-quality chemistry, manufacturing, and controls (CMC) strategy to demonstrate control, robustness, and safety is often challenging and complex.

Bringing cell and gene therapy products to the market affordably requires careful consideration of each of these potential barriers and solutions required to overcome them in specific applications. Partnering with an experienced provider of end-to-end solutions for cell and gene therapy manufacturing can ensure the development of a process that performs consistently, delivers long-term scalability, and complies with evolving regulations.

Cell and Gene Therapy Solutions

From discovery to process development to clinical and commercial manufacturing, Sartorius’ portfolio of proven solutions helps cell and gene therapy manufacturers achieve faster time to market while saving costs.

Reproducible Performance

Starting with a process that delivers high performance, and ensuring the results can be consistently repeated, is critical to developing a reproducible process. Bioprocess developers can accelerate process development activities with high-throughput technologies that promote process efficiency, optimized critical raw materials that improve yield, and intuitive data analytics that inform decision-making.

Streamlined Scalability

Leveraging decades of demonstrated expertise in scalable upstream and downstream solutions can streamline revalidation and optimization from the start.

Designed with scalability in mind, our range of cell culture bioreactors, single-use bags, media, and reagents enable seamless process transfer to larger volumes. Clarification and purification technologies that improve yield and maintain quality throughout downstream processing can drive cost efficiency, while opting for preassembled storage solutions during shipping and handling can help protect product performance.

Simplified Route to Regulatory Compliance

Selecting products and services backed by rigorous quality-assurance processes and trusted to deliver approved therapeutics worldwide can ensure efficacy and safety standards are reliably met.

Sterile, single-use solutions that maximize closed, aseptic processing reduce risk, and serum- and xeno-free cell culture media and reagents protect against contamination. Finally, reliable testing and validation services, along with expert regulatory support, can simplify commercialization.

Setting the Standard in Cell and Gene Therapy, Together

We share the industry’s mission to develop life-changing cell and gene therapies. Cell and gene therapy manufacturers that partner with us can accelerate commercialization with end-to-end solutions that enable performance and scalability with a simplified path to regulatory approvals.