Summary

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024 growing at a CAGR of 19.25% during the forecast period 2024-2035.
A diverse range of disorders arising from congenital abnormalities and inherited genetic mutations, including autoimmune disorders, genetic disorders, neurological disorders and oncological disorders significantly impacts individuals’ health. These disorders are consequences of certain inherited genetic anomalies that disrupt the normal functioning of genes and adversely affect the process of translation. The statistics presented by the World Health Organization (WHO) states that 10 out of every 1,000 individuals are affected by genetic disorders, contributing to 70 million people globally. In addition, more than 40% of infant mortality globally is associated with various genetic disorders. Therefore, to address such challenges, gene therapies have emerged as a revolutionary approach and have proven to be a promising modality in treating a wide array of diseases, focused on delivering targeted therapies directly to affected organs or cells.
The objective of gene therapy is to comprehend the underlying genetic cause of a disease, followed by either introduction of a healthy version of the mutated gene or by inactivation of the faulty / disease-causing genes in the body. It is worth noticing that in 2003, Gendicine® (Sibiono GeneTech) became the world’s first gene therapy to receive regulatory approval from the China FDA. In the past two decades, this has led to several breakthroughs related to the development of gene therapies. For instance, in December 2017, the USFDA approved Luxturna® (Spark Therapeutics), the first gene therapy intended for the treatment of inherited retinal disorders. Additionally, in May 2019, the USFDA approved ZOLGENSMA® (Novartis), the first gene therapy for the treatment of spinal muscular atrophy in pediatric patients. It is noteworthy that the therapy received multiple designations, namely breakthrough therapy, fast track, orphan drug and priority review designations resulting in the accelerated approval of the drug in the US. Further, in 2023, five gene therapies received the USFDA market approval for various therapeutic indications.

In addition, the month of April 2024 witnessed a significant increase in the number of active clinical trials (1,100 active clinical trials) for evaluating gene therapies across various phases of clinical studies. Moreover, three gene therapies have already received regulatory approval in 2024, and according to predictions made by USFDA in 2019, it is estimated that 10 to 20 would gain market access, each year, till 2025. It is noteworthy that the favorable results from ongoing clinical research initiatives have spurred investments from both government and private sectors to support the development of these therapeutic products. With the progressing domain, gene therapies are anticipated to be utilized for the treatment of more than 65.6 million patients suffering from a myriad of disease indications, by 2034.

The rapidly evolving gene therapy product portfolio tends to attract an increasing number of startups and big pharma players, marking their presence in the gene therapy market. At present, 345 gene therapy companies are involved in the development of various early and late-stage therapies worldwide. The recent years have observed a significant increase in the integration of novel technologies, such as gene modification, genome editing, genome sequencing and manipulation technologies (molecular / gene switch), in conjugation with advanced gene delivery methods. Additionally, next-generation delivery platforms, including nanoparticles and hybrid vector systems, have been proven to facilitate an effective, precise and safe delivery of gene-based therapeutics, thereby enabling more targeted treatment approaches. With the integration of advancements in technology and growing preference for single-dose effective therapeutics, the gene therapy market is poised to witness substantial market growth during the forecast period.


Key Market Segments
Therapeutic Area
 Cardiovascular Disorders
 Dermatological Disorders
 Genetic Disorders
 Hematological Disorders
 Metabolic Disorders
 Muscle Disorders
 Oncological Disorders
 Ophthalmic Disorders
 Other Disorders
Type of Vector
 Adeno-associated Virus Vectors
 Adenovirus Vectors
 Herpes Simplex Virus Vectors
 Lentivirus Vectors
 Non-viral Vectors
 Retrovirus Vectors
 Other Viral Vectors
Type of Therapy
 Gene Augmentation
 Gene Editing
 Gene Regulation
 Oncolytic Immunotherapies
 Other Therapies
Type of Gene Delivery Method
 Ex vivo Gene Delivery
 In vivo Gene Delivery
Route of Administration
 Intramuscular Route
 Intratumoral Route
 Intravenous Route
 Subretinal Route
 Other Routes of Administration
Key Geographical Regions
 North America
 Europe
 Asia-Pacific
 Latin America
 Rest of the World
Leading Players
 Amgen
 Krystal Biotech
 Novartis
 Orchard Therapeutics
 Sarepta Therapeutics
Report Coverage:
 The chapter discusses evolutionary studies on gene therapies, along with the classification based on various attributes. Additionally, the chapter features a detailed discussion on the mechanism of action of gene therapies, prominent routes of administration and highlights the various advantages and disadvantages associated with this form of therapeutic intervention, providing an insight on the genome editing techniques, their evolution and applications in the biopharmaceutical industry.
 A thorough discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors has been comprehended in the report.
 The chapter provides a detailed input on the regulatory landscape related to gene therapies across various geographies, namely North America (US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing information on treatment-related reimbursement scenarios for gene therapies, highlighting existing challenges associated with such processes.
 A cross-examined report comprising the marketed and clinical stage gene therapies, based on several relevant parameters, such as stage of development (marketed, registration, phase III, phase II and phase I), drug designations (advanced therapy medicinal products, breakthrough therapy, fast track, orphan drug, priority medicines, priority review, regenerative medicine advanced therapy and rare pediatric disease designation), therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscle disorders, neurological disorders, oncological disorders, ophthalmic disorders, rare disorders and others), target gene (GM-CSF, 4-1BBL gene, anti-VEGF gene, DMD gene, F8 gene, F9 gene, HGF gene, HPV antigens, IL-12 gene, PH20 hyaluronidase, SMN1 gene, TMZ-CD40L gene and others), type of vector used (adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors), type of therapy (gene augmentation, gene editing, gene regulation, immunotherapy and oncolytic immunotherapy), type of gene delivery method (ex vivo and in vivo), route of administration (intracisternal, intradermal, intramuscular, intratumoral, intravenous, intravitreal, subcutaneous, subretinal and other routes) and dosing frequency (single dose and multiple doses). Furthermore, the chapter presents a detailed analysis of the current landscape of discovery and preclinical stage gene therapies based on parameters. These parameters include stages of development (discovery and preclinical), therapeutic area (cardiovascular disorders, genetic disorders, hematological disorders, hepatic disorders, infectious diseases, metabolic disorders, muscle related disorders, neurological disorders, oncological disorders, ophthalmic disorders and others), target gene (ABCA4 gene, antigen, anti-VEGF gene, caveolin-1 gene, DMD gene, F8 gene, F9 gene, FGF 21 gene, FXN gene, JUMP70 gene and others), type of vector used (adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors), type of therapy (gene augmentation, gene editing, gene regulation, immunotherapy and oncolytic immunotherapy) and type of gene delivery method (ex vivo and in vivo).
 A comprehensive assessment of the market landscape of gene therapy developers featuring information on year of establishment, company size (in terms of number of employees), location of headquarters and most active players (in terms of the number of gene therapies developed) has been provided in the report.
 A comprehensive set of profiles of prominent players involved in developing gene therapies. Each profile features a brief overview of the company (including information on year of establishment, location of headquarters and number of employees, leadership team and annual revenues (if available)), gene therapy portfolio, recent developments and an informed future perspective.
 The chapter provides a detailed exploration of marketed gene therapies, along with information on the development timeline of the therapy, mechanism of action, type of vector used, dosage and manufacturing details, target indication, status of development, as well as details related to the developer company.
 A comprehensive report comprising various commercialization strategies that have been adopted by drug developers engaged in the gene therapy domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.
 An intricate set of profiles of late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, mechanism of action, type of vector used, affiliated technology, dosage and manufacturing details, as well as details related to the developer company.
 A cross-examined analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. Further, it also provides an insight on competitive benchmarking analysis of the patent portfolios of leading industry / non-industry players with an emphasis on patent valuation. We have a separate detailed study available on gene therapy and vectors IP Landscape.
 An elaborate discussion on the various mergers and acquisitions undertaken in the gene therapy domain, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product, target therapeutic area and deal multiples has been provided in the report.
 A brief context of the funding and investments made in the gene therapy market (seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings), at various stages of development in companies that are engaged in this domain has been offered in the report.
 An analyzed report of the completed, ongoing and planned clinical studies, based on several relevant parameters, which includes, trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
 A detailed report focusing on the factors that are likely to affect the pricing of gene therapies, featuring different models / approaches that may be adopted by developers / manufacturers to decide the prices of these therapies.
 The report provides an overview encompassing the startup companies engaged in this domain (since 2017) based on year of experience.
 A brief review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting the trend across parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used. Furthermore, it provides a detailed exploration of the benchmarking analysis across key parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.
 An informed estimate of the global annual demand for gene therapies, considering the marketed gene-based therapies and clinical trials evaluating gene therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.
 A cross-examined report comprising the analysis of the factors that can impact the growth of the gene therapy market. It also features identification and analysis of key drivers, potential restraints, emerging opportunities, and existing challenges.
 A detailed gene therapy market forecast to estimate the current market size and future opportunity over the next 11 years. It is based on multiple parameters, likely adoption trends and through primary validations, we have provided an informed estimate on the market size, till 2035.
 An in-depth insightful projection of the current size and future opportunity within the gene therapy industry across different therapeutic areas, namely cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle disorders, oncological disorders, ophthalmic disorders and other disorders.
 A comprehensive report carrying details about the projections of the current size and future opportunity within the gene therapies market across different types of vectors, namely adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors.
 A substantial report focusing on the detailed projections of the current size and future opportunity within the gene therapies market across different types of therapy, namely gene augmentation, gene editing, gene regulation, oncolytic immunotherapies and other therapies.
 An enumerated report having insights on the detailed projections of the current size and future opportunity within the gene therapy market across different types of gene delivery method, namely ex vivo gene delivery and in vivo gene delivery.
 Detailed projections of the current size and future opportunity within the gene therapies market across different routes of administration, namely intramuscular, intratumoral, intravenous, subretinal and other routes of administration.
 Comprehensive projections of the current size and future opportunity within the gene therapy market across geographical regions, namely North America, Europe, Asia-Pacific, Latin America and rest of the world.
 The report includes detailed projections of the current and future revenues from the sales of gene therapies developed by the leading players, namely Novartis, Sarepta Therapeutics, and other players.
 An insightful projection of the current and future revenues from the sales of marketed and phase III gene therapies developed by the leading players within the gene therapy market.
 A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that are being used for the development of gene therapies.
 A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing viral vectors used in gene therapy products. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
 A refined report discussing the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.
Key Benefits of Buying this Report
 The report offers market leaders and newcomers valuable insights into revenue estimations for both the overall market and its sub-segments.
 Stakeholders can utilize the report to enhance their understanding of the competitive landscape, allowing for improved business positioning and more effective go-to-market strategies.
 The report provides stakeholders with a pulse on the Gene Therapy Market, furnishing them with essential information on significant market drivers, barriers, opportunities, and challenges.
Leading Market Companies
 Amgen
 Artgen Biotech
 BioMarin Pharmaceutical
 bluebird bio
 CRISPR Therapeutics
 CSL Behring
 Ferring Pharmaceuticals
 Kolon TissueGene
 Krystal Biotech
 Novartis
 Orchard Therapeutics
 Pfizer
 PTC Therapeutics
 Sarepta Therapeutics
 Shanghai Sunway Biotech
 Sibiono GeneTech
 Spark Therapeutics