Everything to Know About Cell and Gene Therapy in 2025

Is cell and gene therapy the future?

Cell and gene therapy is widely regarded as a major part of the future of medicine, with ongoing innovation, increased approvals, and expanding indications (1-13). However, challenges, such as high costs, access, complex manufacturing, and regulatory uncertainties, still need to be addressed for these therapies to become mainstream. (14-22)

What diseases can be cured by gene therapy?

Gene therapy can currently cure or treat certain rare genetic disorders like sickle cell disease, spinal muscular atrophy (SMA), certain blood disorders, and inherited retinal diseases, as well as some forms of blood cancer (e.g., leukemia and lymphoma) through CAR T-cell therapy (23-33). Research is also expanding to investigate gene therapy for other conditions, including Duchenne muscular dystrophy, cystic fibrosis, and more (34-35).

Who is a good candidate for gene therapy?

Individuals who are well-suited for gene therapy usually have a genetic disorder stemming from a single-gene mutation, such as sickle cell disease or cystic fibrosis (23-26). Patients are particularly suitable when no effective treatments exist or when existing therapies only address symptoms rather than the underlying cause. Important considerations include confirming a genetic diagnosis, ensuring the patient fits the therapy’s approved age range, and verifying the absence of pre-existing antibodies against the therapy’s delivery vector, such as an adeno-associated virus (36,37).

How are cell and gene therapies manufactured?

Cell and gene therapy manufacturing is a highly intricate process that demands precision and control at every stage. For cell therapies, production typically begins with collecting cells from a patient or donor (5,38). These cells are then genetically modified or otherwise engineered to enhance their therapeutic properties, expanded in sterile culture conditions, and carefully processed, packaged, and shipped for clinical use.

Gene therapy manufacturing, on the other hand, centers on developing viral vectors or other delivery systems capable of transporting therapeutic genes into target cells (39). These vectors are produced, purified, and formulated under tightly regulated conditions to ensure consistency and safety.

Across both modalities, rigorous quality control and testing are essential throughout manufacturing—from raw material sourcing to final product release—to confirm identity, potency, purity, and overall efficacy (21,40-42).

How big is the cell and gene therapy manufacturing market?

The global market for cell and gene therapy manufacturing was valued at $18.13 billion in 2023 and is forecasted to soar to approximately $97.33 billion by 2033, reflecting a compound annual growth rate (CAGR) of 18.3% (43). This surge is fueled by rising demand for cutting-edge therapies, expanded government support, and technological advances such as increased automation.

What companies are in cell and gene therapy?

Several companies are actively engaged in cell and gene therapy, including Krystal Biotech, Intellia Therapeutics, and Beam Therapeutics, which primarily focus on gene editing and therapies (44,45). Other notable players, such as Allogene Therapeutics, Kite Pharma, and Novartis, specialize in CAR-T cell therapies, gene therapies, and manufacturing across various disease areas (45,46).

What are the challenges of CGT manufacturing?

The primary challenges in CGT manufacturing stem from the specialized, time-sensitive, and often personalized nature of these treatments (1,3,21). High production costs, scalability limitations, and complex regulatory pathways remain significant obstacles to broader commercialization (1-3,16,17,20,22). Additional difficulties include securing consistent, high-quality raw materials, maintaining stringent aseptic environments, and managing temperature-sensitive supply chains. The industry also faces a shortage of advanced manufacturing facilities and skilled professionals capable of meeting the technical demands of CGT production.

References

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