Industry Research | Cell and Gene Therapy
Release Date:
2023-02-21 10:48
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I. Cell and gene therapies are maturing and have entered a phase of rapid development.
(1) Cell and gene therapies directly target genetic material, offering broad prospects for clinical application.
Cell and gene therapy refers to an emerging therapeutic approach that involves introducing exogenous genetic material into target cells to modify or manipulate gene expression, thereby altering the biological properties of the cells and achieving a therapeutic effect. Its mechanisms of action primarily encompass the following three aspects:
(1) Gene replacement: substituting the disease-causing gene with a normal gene;
(2) Inactivation: rendering functionally abnormal genes inactive;
(3) Insertion: Introducing a new or modified gene into the body.
Unlike small-molecule and antibody-based therapeutics, cell and gene therapies have the potential to directly target genetic material, making them particularly promising for treating many diseases for which no druggable targets have yet been identified. Since the 1990s, research in the field of cell and gene therapy has shown a steady upward trend, with growing scientific interest and attention.
According to Drug Discovery Today, as of July 2020, a total of 2,106 clinical trials in cell and gene therapy were underway worldwide, with the United States, China, and the European Union accounting for roughly half of these trials. The United States boasts a more comprehensive policy and regulatory framework, which has accelerated the rapid development of gene therapy clinical research. Meanwhile, China has been progressively refining its relevant policies and regulations in recent years and increasing investment in both research and clinical practice, thereby emerging as a key hub for the initiation and conduct of gene therapy clinical trials. In terms of indications, cancer remains the leading disease category for gene therapy, accounting for 65.2% of all trials; ongoing studies cover a wide range of malignancies, including hematologic, cutaneous, central nervous system, prostate, gastrointestinal, breast, pulmonary, and thyroid cancers. Other major areas of investigation include inherited rare diseases, cardiovascular disorders, and infectious diseases.
Although the concept of cell and gene therapy has a long history, the first clinical trial was not conducted until 1990 at the NIH, targeting a rare immunodeficiency disorder. Since then, cell and gene therapy has gone through three distinct phases: initial rapid development, a period of relative dormancy, and a cautious resurgence. As an emerging therapeutic modality that can complement conventional treatments, cell and gene therapy has demonstrated tremendous potential in the treatment of numerous diseases, particularly cancer, genetic disorders, and infectious diseases. With ongoing advances in technology and industrialization, the number of approved products is expected to continue to grow in the future.
To date, a total of 19 cell and gene therapy products have been approved for market launch worldwide, including CAR-T therapies, stem cell therapies, oncolytic virus therapies, and gene therapies. The first gene therapy drug was launched in China in 2003, while two others—Zalmoxis and Glybera—have since been withdrawn from the market. Since their launch, CAR-T therapies such as Kymriah and Yescarta, as well as the gene therapy Zolgensma, have posted robust sales growth and steadily increasing market penetration. According to Novartis’s interim report for 2021, Zolgensma generated $634 million in sales in the first half of 2021, representing a year-on-year increase of 69.07%. According to Drug Discovery Today, the commercialization of cell and gene therapy products worldwide has accelerated steadily since 2017, with projections indicating that 40 such products will be approved for marketing by 2022.
To date, China has only two cell and gene therapy products approved by the NMPA for marketing, both of which are oncology drugs: Anke Rui and Agilence Injection. Driven by factors such as increased domestic R&D investment, continuous technological maturation, and the ongoing refinement of relevant regulatory frameworks, it is anticipated that more products will enter the Chinese market in the future.
1. Cell and gene therapies can be classified, based on their delivery method, into ex vivo therapies and in vivo therapies.
Extracorporeal therapy This refers to a therapeutic approach in which cells are harvested from a patient, genetically engineered in an ex vivo system, and then infused back into the patient. Such therapies typically require a vector capable of delivering genes and integrating them into the genome—such as lentiviral or retroviral vectors—and rely on advanced equipment for performing a range of cellular manipulations. Ex vivo gene therapy primarily encompasses two main categories: therapies targeting T cells and those targeting hematopoietic stem cells. Among these, CAR-T cell therapy is the most mature and well-developed; Novartis’ Kymriah, Kite’s Yescarta and Tecartus, Juno’s Breyanzi, and Celgene’s Abecma have all been approved for market launch in recent years. In China, Fosun Kite’s axicabtagene ciloleucel injection has also been approved, and WuXi AppTec’s relmacabtagene mafarucel injection is expected to receive approval in the second half of this year, becoming the country’s second CAR-T therapy.
In vivo gene therapy involves directly administering gene therapy within the patient’s body to compensate for or suppress defective genes. In this approach, genetic material can be delivered into the body either directly or indirectly. Although in vivo gene therapy is relatively straightforward to perform, it places more stringent requirements on the delivery vector, which must exhibit tissue tropism, stable expression, and low immunogenicity.
2. Cell and gene therapies can be classified into five product types.
According to the FDA’s classification of cell and gene therapy products, these therapies can also be categorized into plasmid DNA, viral vectors, bacterial vectors, gene-editing systems, and ex vivo edited-cell products.
Plasmid DNA: Genetically engineered circular DNA molecules can carry therapeutic genes and be delivered into human cells.
Viral vector: Because viruses inherently possess the ability to deliver genetic material into mammalian cells, some gene therapy products are derived from viruses. By means of genetic engineering, the pathogenicity of these viruses can be eliminated, enabling them to serve as vectors for delivering therapeutic genes into human cells.
Bacterial vector: Similar to viruses, bacteria can also be engineered to lose their ability to cause infectious diseases and then used as vectors for delivering genetic material into cells.
Gene-editing system: Unlike viral vectors, which can only mediate gene supplementation, gene-editing systems function as molecular scissors, enabling a wide range of powerful genetic manipulations, including gene addition, gene deletion, and even precise gene correction.
Ex vivo cell-editing products: A therapeutic approach in which cells are harvested from a patient, genetically modified, and then infused back into the patient. This approach is particularly widely used in CAR-T therapy for cancer.
(2) Cell and gene therapy delivery vectors: Adeno-associated virus vectors are the most widely used in clinical applications.
Vectors for cell and gene therapy primarily include viral and non-viral vectors. Viral vectors are widely used in drug development due to their high delivery efficiency, tissue specificity, and ability to integrate into the host genome; approximately 70% of clinical trials in cell and gene therapy employ viral vectors. In contrast, non-viral vectors offer advantages such as simple operation, low cost, and low immunogenicity, and are therefore attracting increasing attention.
1. Viral vectors remain the mainstream delivery vehicles for cell and gene therapies.
Retroviral vectors, adenoviral vectors, lentiviral vectors, and adeno-associated viral vectors are currently the most widely used viral vectors in clinical applications. Among these, retroviral and lentiviral vectors, owing to their ability to integrate into the host cell genome, are frequently employed in ex vivo cell and gene therapies to deliver therapeutic genes into stem cells or T cells, thereby achieving long-term gene expression. In contrast, adeno-associated viral vectors and adenoviral vectors are typically utilized in in vivo cell and gene therapies to minimize the risk of ectopic transgene integration.
Retroviral vector: Retroviruses are enveloped, single-stranded RNA viruses with a diameter of 100–120 nm. Gamma-retroviral vectors are capable of integrating into the host cell genome and were first approved by the FDA in 1990 for use as viral vectors in clinical trials targeting ADA-SCID. Both of Kite Pharma’s commercially available CAR-T products employ retroviral vectors. The genome of gamma-retroviruses is relatively simple, encoding only three structural proteins: gag (encoding the capsid protein), pol (encoding replication-associated enzymes), and env (encoding the envelope glycoprotein).
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