Gene Therapy Market

Market Overview

The global gene therapy market was valued at USD 5.17 billion in 2022. It is estimated to reach USD 39.95 billion by 2031, growing at a CAGR of 25.51% during the forecast period (2023–2031). According to reports, over a million patients will receive gene therapy treatments by the end of 2034, with treatment costs potentially amounting to $12.2 billion. The gene therapy market is expected to have sustained growth in the forecast period, primarily driven by the increasing prevalence of target disorders such as cancer. Surging R&D investments to support gene therapy research and development projects will further boost market expansion.

Gene therapy prevents disease progression by modifying the expression of mutated genes or the biological properties of living cells for therapeutic purposes. It comprises various items, including viral vectors, bacterial vectors, plasmid deoxyribonucleic acid (DNA), human gene-editing technology, and patient-derived cellular gene therapy. It entails inserting a healthy gene into an individual's genome to correct a mutation that causes a genetic disorder.

Gene therapy includes numerous treatments, including ex vivo, in vivo, and in situ gene therapy. Gene therapy functions by inhibiting, amplifying, and eliminating disease-causing cells. It can eliminate disease symptoms permanently and cure diseases that no medication can prevent. It also enhances the body's resistance to various diseases, including cancer, hemophilia, cystic fibrosis, diabetes, heart disease, and acquired immunodeficiency syndrome (AIDS).

Market Dynamics

Gene Therapy Market Drivers

Growing Prevalence of Target Diseases Like Cancer

In recent years, the prevalence of chronic diseases, such as cancer, cardiovascular diseases, and others, has increased significantly, significantly increasing people's healthcare expenditures. Gene therapy has exhibited greater efficacy than conventional medication, leading to its increasing adoption among individuals afflicted with cancer and other disorders. This trend has consequently contributed to the rise of the market for gene therapy.

According to the data released by the International Agency for Research on Cancer in 2020, it is predicted that approximately 20% of individuals will experience cancer at some point in their lifetime. The statistics indicate that the mortality rates for cancer are higher among men, with approximately one in eight succumbing to the disease, while for women, the mortality rate stands at approximately one in eleven. Thus, the growing burden of cancer increases the burden of diagnostic facilities, which will further contribute significantly to market growth.

Rising Investments in the R&D

With the increasing burden of various diseases, biopharmaceutical firms are investing heavily in R&D activities to develop novel and advanced therapeutics and drugs that are more effective against the disease or medical condition. This is further expected to expedite market expansion during the forecast period. According to the Global Research and Development Expenditures Fact Sheet by Congressional Research Services in September 2021, since 2000, total global R&D investments have more than tripled in current dollars, from USD 677 billion to USD 2.2 trillion in 2019.

Additionally, the innovations and investments in research and development of novel therapeutics and drugs by the key market leaders in the area, along with the new product launches or expansion of R&D facility for gene therapy, will also boost the market's growth. For instance, in order to support the manufacture of its pipeline of lentivirus and adeno-associated virus (AAV) gene treatments, Rocket Pharmaceuticals plans to build a new research and development and manufacturing plant in Cranbury, New Jersey, in February 2021.

Gene Therapy Market Restraints

High Price of Products

The cost of gene therapy is a major problem for patients worldwide. Gene treatments are exceedingly expensive to design and manufacture, and clinical studies and bringing goods to market add to the expense due to this fundamental reason why gene therapy is so costly. As per the article published in the Journal of Managed Care and Specialty Pharmacy in May 2021, titled "Gene therapy may not be as expensive as people think challenges in assessing the value of single and short-term therapies," the one-time gene treatment Onasemnogene Abeparvovec for spinal muscular atrophy, a rare neuromuscular illness that is usually deadly by the age of two if untreated, has been dubbed the "most expensive medication ever" with a price tag of USD 2.125 million. Thus, higher product prices will restrain market growth in underdeveloped countries over the forecast period.

Gene Therapy Market Opportunities

Technological Advancements

Rapid advancements in the biomedical sciences may result in the development of new and advanced strategies for gene therapy. Gene therapy is gradually moving toward excellence with various advanced technologies. It is currently poised to take advantage of promising revolutions in the biotechnology field.

As per the article published in the Frontier in Genome Edition in March 2021, titled "CRISPR/Cas: Advances, Limitations, and Applications for Precision Cancer Research," CRISPR/Cas is a technology capable of making specific genome modifications in living eukaryotic cells, making it one of the most important scientific breakthroughs of the twenty-first century. Advances in this technology have made it possible to drive into basic and clinical research applications in recent years. Thus, the technological advancement in CRISPR gene therapy will help diagnose various chronic diseases, likely creating opportunities for the market for gene therapy.

Regional Analysis

North America Dominates the Global Market

Based on region, the global gene therapy market is bifurcated into North America, Europe, Asia-Pacific, South America, and the Middle East and Africa.

North America is the most significant global gene therapy market by market share and is estimated to exhibit a CAGR of 24.81% during the forecast period. The North American gene therapy market is driven by the strong regulatory framework for supporting cellular therapy development and the presence of many biopharma companies. Gene therapy technologies are being given increasing approval by the organizations above, which is expected to drive market growth. For instance, AveXis, a Novartis company, announced in May 2019 that the US FDA had approved the use of Zolgensma (onasemnogene abeparvovec-xioi) to treat pediatric patients with spinal muscular atrophy (SMA) who are under two years of age and have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Additionally, according to the Canadian Agency for Drugs and Technologies in Health (CADTH), Canadian researchers are recently participating in or have previously participated in a large number of gene therapy trials, including two of the three trials for the drug alipogene tiparvovec for monogenic lipoprotein lipase deficiency, which has a high prevalence in Quebec owing to a founder effect (151–153). Hence, all the factors mentioned above will likely boost market growth in the future.

Europe is estimated to exhibit a CAGR of 25.87% over the forecast period. Europe is the world's second-largest gene and cell therapy market, with Germany and the UK leading. Germany is actively developing and testing innovative medicines to benefit patients and advance the gene therapy field. In addition, research and development are well funded in Germany, and the output of scientific publications and patents is comparable with that of the leading countries in gene therapy. This boosted market growth in Germany.

Moreover, new product development by market players is also likely to spur market growth. For instance, in October 2021, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, IP Group, and Oxford Biomedica announced that they had accelerated the development of first-in-class gene therapy for patients with cystic fibrosis. Hence, all the factors mentioned above boost the market growth of gene therapy in the United Kingdom. Hence, all the factors mentioned above boost market growth in the region.

Asia-Pacific is expected to grow at the quickest rate. The market growth in the region is boosted by factors like the flourishing geriatric population, surging product launches, and an improving regulatory framework. For instance, as per an article published in July 2020 titled "Spotlight on Gene Therapy in China," China has a population of more than 1.4 billion people, with an estimated 57 million people living with genetic diseases and more than 4 million new cancer cases each year, creating a pressing need for new therapeutics, of which gene therapies are becoming a critical component. Furthermore, as per the article published in March 2021 titled "Current status and future perspective of gene therapy products in Japan," as of January 2021, 10 regenerative medical products have been approved in Japan. Research and development of regenerative medical products and cell and gene therapy products are accelerating, especially for gene therapy products.

In the Middle East and Africa, the regional disease burden is increasing spending on research and development by the pharmaceutical industry, impacting the gene therapy market in this region. For instance, as per the statistics by GLOBOCAN 2020, in South Africa, in 2020, an estimated number of 108,000 new cancer cases were identified, estimated to reach 178,000 by 2040. In addition, collaborations and mergers among pharmaceutical companies to provide gene therapy will likely drive market growth. For instance, in January 2021, Orchard Therapeutics intends to expand its commercial reach in the Middle East and Turkey by partnering with GenPharm Services and GEN, two specialty pharmaceutical companies with extensive experience in rare genetic diseases.

Segmental Analysis

The global gene therapy market is segmented by indication and technology.

Based on indications, the global gene therapy market is bifurcated into cancer, metabolic disorders, eye disorders, spinal muscular atrophy, and other indications.

The cancer segment dominates the global market and is projected to exhibit a CAGR of 25.25% over the forecast period. The potential of gene therapy in treating different forms of cancer has led to huge investment in research and development activities by the government as well as non-government entities, which have fueled intensive research in the area, and results from these research activities are expected to have a significant impact on the growth of the cancer segment over the forecast period. For instance, according to the research study published by Shoushan Hu et al. in October 2021, academic attention from multiple subjects on cancer gene therapy grew at a significant pace from 2016 to 2020, in which materials science, nanoscience, and nanotechnology took a surging part in the research on cancer gene therapy.

With the advancement in delivery systems, gene therapy has emerged as one of the most potent tools for treating metabolic disorders due to its high accuracy in treating such disorders. For instance, according to the research study published in August 2020 by Carlos G. Moscoso and Clifford J. Steer, liver-targeted gene therapy is found to be a treatment option for monogenic genetic disorders (such as hemophilia A and B, urea cycle disorders, and familial hypercholesterolemia). There has been significant success at both the pre-clinical and clinical stages. According to the research study published in September 2020 by Berna Seker Yilmaz et al., gene therapy is gradually becoming a therapeutic option for patients with inherited metabolic diseases and safer delivery vectors, and successful results have been reported for various inherited rare diseases such as Leber's congenital amaurosis, which shows the potential of gene therapy in the treatment of various metabolic disorders.

Based on technology, the global gene therapy market is segmented into adenovirus vectors, adeno-associated virus vectors, lentiviral vectors, retroviral vectors, herpes virus vectors, and other technologies. 

The adeno-associated virus vector segment owns the highest market share and is estimated to exhibit a CAGR of 26.36% during the forecast period. The 4.7-Kb single-stranded DNA genome of the small (25 nm), non-enveloped virus known as adeno-associated virus (AAV) is frequently used as a platform for delivering genes to treat various human diseases. Its advantages, like long-term DNA persistence in target cells, minimal or no host inflammatory response, and high efficiency in gene delivery, along with pre-clinical and clinical successes in AAV-mediated gene replacement, gene silencing, and gene editing, have helped AAV gain popularity in the gene therapy segment as the ideal therapeutic vector for various diseases. Thus, the AAV segment is expected to grow significantly. Many research studies and clinical trials are underway for gene therapy using AAV virus vectors, which is expected to significantly impact the growth of the studied segment over the years.

Retroviral vectors are 7 to 10-kb-long single-stranded RNA vectors consisting of two copies of a single-stranded RNA genome with gag, pol, and env sequences, which encode viral structural and catalytic proteins required for viral functions. Retroviral vectors are commonly employed in gene therapy to facilitate the effective transfer of genes into cells. These vectors offer several advantages, including a straightforward manipulation process for incorporating therapeutic genes into the cellular genome, a relatively high concentration of recombinant retroviruses, the ability to infect a diverse range of target species and cells without causing noticeable harmful effects, and a simplified manipulation procedure for the recombinant virus. These advantageous characteristics have led to the widespread utilization of retroviral vectors in gene therapy, and this particular segment is expected to experience significant growth.

Recent Developments

• June 2023: Laurus Labs Ltd. announced that it signed a MoA with IIT Kanpur (IITK) for in-licensing and commercializing novel gene therapy assets using adeno-associated virus (AAV) vectors.
• June 2023: Sarepta Therapeutics, Inc. announced that the FDA accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.