Gene Therapy Market Size, Assessment, Value, Share, Growth, Trends, Competition, Industry Analysis, Actionable Insights Report; By Indication, By Vector Type, By Type of Therapy, By Delivery Method, By Route of Administration, By Region and By Country; Outlook and Forecasts, 2024 – 2030

Report Summary

Gene Therapy Market Assessment and Growth

The Global Gene Therapy Market size was valued at USD 9 billion in 2023, and it is projected to reach around USD 30 billion by 2030. This market growth is expected to be driven by a compound annual growth rate (CAGR) of 21% during the forecast period from 2024 to 2030.

The growth of the gene therapy market is driven by an increasing number of approvals from regulatory bodies. Furthermore, factors such as the expansion of clinical research initiatives in genomics, a growing emphasis on advanced targeted gene therapies, and the broadening scope of gene delivery technologies are contributing to the gene therapy market expansion. Competition among key players seeking to commercialize their gene therapies has also driven growth. Biotechnology companies are strategically investing in acquisitions, mergers, collaborations, and expansions to enhance in-house expertise and fortify product pipelines. However, gene therapy products are relatively high-priced, which may partially constrain market growth.

The gene therapy market is poised for significant growth in the forecast period, driven by several key factors. These include the increasing prevalence of genetic disorders, a rise in research and development activities, and the expanding geriatric population. Concurrently, elevated healthcare expenditure, heightened health awareness among the general population, and the growing focus of medical research organizations on finding potential cures for rare, genetic, and autoimmune diseases create lucrative prospects for market growth.

The presence of a robust gene therapy pipeline is expected to further propel gene therapy market expansion in the forecast period. Researchers are diligently working to make gene therapy readily available at clinics and hospitals. Numerous universities and institutes are showcasing a diverse portfolio of products in the pipeline, which is anticipated to drive revenue generation in the near future. According to the American Society of Gene & Cell Therapy (ASGCT), there are more than 2,000 gene therapy products in development pipeline, including CAR T-cell therapies and other genetically modified cell therapies. By mid-2023, around 280 gene therapies had entered Phase II, with about 35 in Phase III and 4 in the Pre-registration stage on a global scale. Notably, gene therapies predominantly target Oncology and Rare Diseases across all stages. Additionally, Neurological and Sensory indications have claimed the third position in gene therapy targeting, closely followed by Alimentary / Metabolic Diseases.

Furthermore, improving regulatory support presents growth opportunities for the gene therapy market in the forecast period. Several international regulatory organizations have made positive changes in how they promote therapies, with examples like the FDA's support for CAR-T technology. Regulators are showing flexibility in the usual hierarchy of conducting clinical trials, particularly in phase II and III studies. The FDA's projection of approving 10 to 20 new cell and gene therapies each year by 2025 further underscores the optimistic outlook for the gene therapy market.

What is Gene Therapy?

Gene therapy is an innovative treatment method that utilizes genes to treat diseases. This method involves the insertion of functional genes into cells, replacing faulty genes and targeting genetic disorders. Gene therapy is employed to rectify defective genes, ultimately aimed at either curing a disease or enhancing the body's ability to combat diseases. This is achieved through a range of ways, including replacing mutated genes with functional ones, repairing genes with mutations, and rendering diseased cells more recognizable to the immune system. Typically, a genetically engineered virus serves as a vector to carry and deliver the therapeutic gene into the patient's cells. When successful, this method results in the production of a functioning protein by the newly introduced gene. In the future, gene therapy holds the promise of treating various chronic diseases by directly inserting genes into a patient's cells, potentially reducing the reliance on drugs or surgical procedures.

Gene therapy presents a hopeful alternative for addressing a range of conditions, including inherited disorders, specific types of cancer, and certain viral infections. Gene therapy is a promising treatment approach for treating a wide range of diseases, such as cancer, diabetes, AIDS, cystic fibrosis, cardiovascular ailments, and hemophilia, among others. However, it remains an area of ongoing research to ensure safety and effectiveness. Several techniques are employed in gene therapy, such as gene augmentation therapy, gene inhibition therapy, suicide gene insertion, and other methods. Currently, gene therapy is primarily tested for diseases lacking other cures. With the advancements in therapeutic techniques and gene delivery technologies, the gene therapy market is expected to experience substantial growth. However, it is still a relatively new and complex technology, and it comes with various ethical and safety concerns which the industry players are trying to mitigate.

What are the Growth Drivers, Restraints, and Opportunities in Gene Therapy Market? 

Rising Prevalence of Genetic Disorders Drives Gene Therapy Industry Growth

The global gene therapy market is witnessing growth driven by the increasing prevalence of genetic and congenital diseases on a global scale. These genetic diseases, stemming from mutations in genes, disrupt the normal functioning of the body. Traditional treatments for genetic diseases, such as medication and surgery, offer only temporary relief and do not provide a cure. Gene therapy presents a promising alternative by potentially replacing or repairing faulty genes, offering a potential cure for genetic disorders. As the number of individuals affected by genetic diseases continues to rise, there is a growing demand for this therapeutic approach.

According to the Department of Health, as cited by the Government of Western Australia, approximately six out of ten people are likely to experience ailments with genetic ties. These genetic disorders can vary in severity, with an estimated 3 to 5% of newborns in Western Australia born with genetic disorders or birth abnormalities. Factors such as mutations, exposure to chemicals, and radiation can all contribute to genetic disorders. While some conditions have been treated with gene therapy, many genetic disorders lack effective treatments addressing the underlying genetic abnormalities. Therefore, the increasing prevalence of genetic abnormalities across various age groups and geographic regions is expected to drive the growth of the global gene therapy market.

Accelerated Regulatory Approvals Boosts Gene Therapy Market Growth 

Gene therapy products are subjected to stringent regulatory evaluations before receiving approval. These therapies work at the genetic level, providing therapeutic effects that are notably more effective and enduring than traditional medicines. The advantage of this longevity has led to an increasing number of approvals for gene therapy products. The US FDA, for instance, has granted approval to over 10 such products between 2021 and 2024, with a substantial number of products in the clinical pipeline anticipated to secure final approval in the forecast period. Consequently, this factor significantly propels gene therapy market growth during the forecast period.

Mid-2024 Status - 32 Gene Therapies Approved Worldwide for Clinical Use till date

Gene Therapy Approvals (Latest)

• April 2024, Beqvez (fidanacogene elaparvovec) from Pfizer received approval for adults with moderate to severe hemophilia B (congenital factor IX deficiency) who are either on factor IX prophylaxis therapy, have experienced life-threatening hemorrhage, or suffer from repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

• December 2023, Lyfgenia (lovotibeglogene autotemcel) from Bluebird Bio received approval for the treatment of patients aged 12 and above with sickle cell disease and a history of vaso-occlusive events (VOEs).

• November 2023, Casgevy (exagamglogene autotemcel) from CRISPR Therapeutics received approval for treating transfusion-dependent β-thalassemia (TDT) in patients aged 12 and above without a matched related HSC donor, as well as for treating sickle cell disease (SCD) in similar patient populations with recurrent vasoocclusive crises (VOCs) and specific genotypes.

• June 2023, Fucaso (equecabtagene autoleucel) from Nanjing IASO Biotechnology

•  received approval for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) who have progressed after at least three lines of therapy.

• June 2023, ROCTAVIAN (valoctocogene roxaparvovec-rvox) from BioMarin Pharmaceutical received approval for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

• June 2023, ELEVIDYS (delandistrogene moxeparvovec-rokl) from Sarepta Therapeutics received approval for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

• May 2023, VYJUVEK (beremagene geperpavec) from Krystal Biotech received approval for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

• December 2022, ADSTILADRIN (nadofaragene firadenovec-vncg) from Ferring Pharmaceuticals received approval for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors 

• November 2022, HEMGENIX (etranacogene dezaparvovec-drlb), an adeno-associated virus vector-based gene therapy from CSL Behring received approval for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

• November 2022, Upstaza (eladocagene exuparvovec) from PTC Therapeutics received approval to be used in adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis.

• September 2022, SKYSONA (elivaldogene autotemcel) from Bluebird Bio received approval for the treatment to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).

• August 2022, ZYNTEGLO (betibeglogene autotemcel) from Bluebird Bio received approval for treatment of adult and pediatric patients with ß-thalassemia who require regular red blood cell (RBC) transfusions.

• February 2022, CARVYKTI (ciltacabtagene autoleucel) from Janssen Biotech received approval for the Treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Groundbreaking Gene Editing Advancements to Drive Gene Therapy Market Expansion

The rapid progression of gene editing technology, exemplified by CRISPR-Cas9, has served as a significant catalyst for the gene therapy market. Gene editing has enabled precise and efficient modification or replacement of specific genes, giving rise to innovative gene therapies for a spectrum of conditions, encompassing rare genetic disorders, cancer, and infectious diseases. As ongoing research and development further refine gene editing technologies, the growth trajectory of the gene therapy market is expected to persist.

In November 2023, Exa-cel, branded as Casgevy, secured its inaugural regulatory approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). This gene therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, employs the CRISPR system to excise genes from DNA, facilitated by the enzyme Cas9. Casgevy specifically targets the BCL11A gene, offering treatment for sickle cell disease and transfusion-dependent beta-thalassemia.

Surging Investments Fuel Gene Therapy Industry Development

Increased investments in cell and gene therapy are poised to propel the growth of the gene therapy market. These emerging cell and gene therapy companies have become focal points for heightened public and private funding. Notably, private equity and capital expenditures in the life sciences sector have witnessed substantial growth in recent years. This market surge is primarily attributed to the remarkable increase in the clinical pipeline and the mounting regulatory approvals for advanced pharmaceuticals. To bolster the production of cell and gene therapies, various new techniques are being introduced, addressing the rising demand for cutting-edge treatments and intensifying market competition among participants. Moreover, the increasing prevalence of cardiovascular disorders is expected to further propel market growth. Additionally, improved healthcare infrastructure and increased healthcare expenditure stand as significant factors driving the gene therapy market's expansion.

Exponential Growth in Cell and Gene Therapies Drives Lucrative Opportunities for the Gene Therapy Industry

Research and development efforts in the field of cell and gene therapies have been expanding. This growth is driven by the significant potential of these therapies to address a range of challenges, particularly in the context of neurology indications where effective treatments remain elusive. This presents a promising area for pharmaceutical and biotechnology companies to invest in the development and commercialization of innovative cell and gene therapies. Furthermore, cell and gene therapies provide a targeted therapeutic effect, making them a preferred choice for reducing the need for repeated and costly treatments. Therefore, the rising demand for cell and gene therapies, attributable to the advantages they offer, represents a substantial opportunity within the gene therapy market.

Gene Therapy - Overcoming Cost Barriers, Unintended Mutations, and Side Effects for Wider Adoption

Extensive research and development are pivotal for cutting-edge therapies like gene therapy, demanding significant investments in time and finances. Costs encompass laboratory research, preclinical testing, clinical trials, and regulatory approval. Gene therapy products rely on vectors tailored to specific needs, heightening the complexity of the manufacturing process. Rigorous control and adherence to stringent quality standards escalate production costs. Moreover, these highly targeted therapies aren't mass-produced, further inflating expenses. Collectively, these factors result in high prices and significantly limit the widespread adoption of gene therapy products.

What are Gene Therapy Market Segmentations and Bifurcations? 

Gene Therapy Market Segmentation by Indication

• Cancer (Oncology)
  • Acute Lymphoblastic Leukemia (ALL)
  • Head & Neck Squamous Cell Carcinoma
  • Large B-cell Lymphoma
  • Melanoma (Lesions)
• Inherited Retinal Disease
• ADA-SCID
• Beta-Thalassemia Major / SCD
• Peripheral Arterial Disease
• Spinal Muscular Atrophy (SMA)
• Other Indications

In 2023, the spinal muscular atrophy (SMA) emerged as the leading segment, capturing a substantial share of over 40%. Despite its rarity, SMA stands as one of the most prevalent and fatal inherited disorders among infants. Notably, the advent of Zolgensma (AVXS-101) has showcased its efficacy in treating SMA, significantly altering the illness's phenotype. In May 2019, the FDA granted approval for Novartis' Zolgensma, targeting the root cause of SMA. Presently, Zolgensma stands as the sole approved gene treatment in this domain. This approval signifies the increasing adoption of therapies to combat severe hereditary ailments such as SMA. 

Looking ahead, the Beta-Thalassemia Major/SCD segment anticipates the fastest CAGR. Gene therapy for Sickle Cell Disease (SCD) and β-thalassemia revolves around gene-modified hematopoietic stem cell transplantation. Both clinical and preclinical studies validate the efficacy and safety of this therapeutic approach. Nevertheless, challenges persist, including suboptimal gene expression levels, limited stem-cell dosage and quality, inefficiencies in gene transfer, and the toxicity associated with myeloablative regimens, all of which hinder its efficacy.

Moreover, the inherited retinal disease segment foresees substantial growth owing to increased efforts by concerned entities to develop effective gene therapies for ocular diseases. 

Simultaneously, the oncological disorders segment is witnessing significant traction, propelled by the rising prevalence of cancer and the burgeoning demand for potent anti-cancer therapies.

Gene Therapy Market Segmentation by Vector Type

• Adeno-Associated Virus (AAV)
• Adenovirus
• Herpes Simplex Virus
• Lentivirus
• Retrovirus
• Non-Viral Vectors
• Others

The current market sees a dominant position held by the viral vectors segment, owing to the safety and high efficiency exhibited by most viral vectors in combating infectious diseases and viruses. Moreover, their ease of handling and adaptability further fuels the segment's growth in gene therapy market.

In 2023, the Adeno-Associated Virus (AAV) segment held around 20% revenue share. Numerous biopharma companies are leveraging their viral vector platforms for AAV-based gene therapy products. For example, Lonza inked an exclusive deal with Massachusetts Eye and Ear in September 2016, supporting the development and commercialization of advanced gene therapies utilizing their Anc-AAV gene therapy platform. Additionally, Biogen Inc. partnered with Capsigen Inc. in May 2021, aiming to engineer innovative AAV capsids capable of delivering groundbreaking gene therapies targeting genetic causes of various neuromuscular and CNS disorders.

In July 2021, the U.S. Department of Commerce’s National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaborative effort to evaluate analytical methods and establish standards for AAV. Through interlaboratory studies, several labs will measure crucial quality attributes, fostering the development of promising gene therapies that significantly enhance people's lives.

Gene Therapy Market Segmentation by Type of Therapy

• Gene Augmentation
• Gene Silencing
• Cell / Gene Replacement
• Others

In 2023, gene silencing therapy held the largest share among the various types within the gene therapy industry. The gene therapy market is categorized into gene silencing, cell replacement, gene augmentation, and other therapies. Gene silencing dominated the market due to the presence of numerous gene therapy products utilizing this mechanism, particularly noted for its effectiveness in combating neurological conditions, including neuromuscular diseases.

Furthermore, gene editing is poised to emerge as a prominent method for gene therapy in the forecasted period. According to the latest update, Casgevy (Exa-cel), a gene therapy utilizing CRISPR-Cas9 gene editing technology, obtained approval on November 16, 2023, from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. This groundbreaking approval is specifically for treating sickle cell disease and β-thalassemia.

Gene Therapy Market Segmentation by Delivery Method

• In Vivo
• Ex Vivo

The gene therapy market is categorized by delivery method into in vivo gene therapy and ex vivo gene therapy. In 2023, in vivo gene therapy held a substantial market share of approximately 70%, a trend anticipated to exhibit significant growth throughout the forecast period. In vivo gene therapy facilitates systemic treatment, allowing it to reach various sites or organs across the body. This attribute proves particularly advantageous for diseases with widespread impact or systemic manifestations, enabling a comprehensive and effective therapeutic approach. The dominance of in vivo gene therapy underscores its versatility and potential in addressing conditions that necessitate a holistic treatment strategy.

Gene Therapy Market Segmentation by Route of Administration

• Intravenous
• Others

On the basis of route of administration, the gene therapy market is segmented into intravenous, inhalation, and direct injection to the target site among others.

What are the Regional and Country Trends in Gene Therapy Market?

Global Gene Therapy Market - Regional Analysis

• North America
• Europe
• Asia Pacific
• Middle East & Africa
• South America

In 2023, North America emerged as the dominant region in the global gene therapy market, a trend expected to persist from 2024 to 2030. The region held over 60% market share in 2023, driven by advanced technologies facilitating patient eligibility assessments for gene therapy and robust hospital infrastructure. North America foresees continued leadership in routine gene therapy manufacturing, marked by increased approvals and revenue generation. Substantial R&D investments by both large corporations and smaller entities to develop optimal therapy drugs are poised to further elevate market growth.

The United States, within North America, boasts a robust gene therapy research and development ecosystem. It houses leading academic institutions, research centers, and biotech firms actively contributing to advancements in the field. This environment fosters innovation, attracting investments crucial for novel gene therapy development. Moreover, the prevalence of genetic disorders and complex ailments, like cancer, has fueled demand for innovative treatments, supported by payer willingness to endorse these therapies through reimbursement programs.

Europe, renowned for rapid clinical research, evolving healthcare infrastructure, and favorable reimbursements, is forecasted as one of the fastest-growing regions in gene therapy industry from 2024 to 2030. In Asia Pacific, increased investments, accessible resources, and government support promise significant market growth for genetic therapies in the coming years.

Report Scope & Snapshot

How is the Competition Scenario in Gene Therapy Market?

Gene Therapy Market - Competitive Landscape

More than 200 companies are actively engaged in various developmental phases within the global gene therapy industry. Some of the key companies in gene therapy market include American Gene Technologies, Amgen, AnGes, Astellas Pharma, AstraZeneca, BENITEC BIOPHARMA, Biogen, BioMarin Pharmaceutical, Bluebird Bio, Bristol-Myers Squibb Company, Cellectis, CRISPR Therapeutics, CSL Behring, Epeius Biotechnologies, Gene Biotherapeutics, Gensight Biologics, Gilead Lifesciences, Novartis, Orchard Therapeutics, Oxford BioMedica, Pfizer, REGENXBIO, Roche, Sangamo Therapeutics, SANOFI, Shanghai Sunway Biotech, Shenzhen Sibiono GeneTech, Vertex Pharmaceuticals, and Others.

Gene therapy market players bolster their position through investments in research and development activities, strategic partnerships, portfolio expansion, regulatory approvals, and company mergers and acquisitions. These proactive strategies enable them to maintain competitiveness and address the increasing demand for gene therapies.

Gene Therapy Market Developments

• In April 2024, Pfizer obtained approval from the US Food and Drug Administration (FDA) for its groundbreaking one-time gene therapy, BEQVEZ (fidanacogene elaparvovec-dzkt), designed to address adults with moderate to severe hemophilia B. BEQVEZ facilitates the internal production of FIX, offering the potential to eradicate the necessity for routine prophylactic infusions. This approval was grounded on the outcomes of the pivotal Phase III BENEGENE-2 trial.

• In November 2023, AstraZeneca announced a collaboration and investment pact with Cellectis. AstraZeneca intends to leverage Cellectis' gene editing technologies and manufacturing prowess to develop innovative cell and gene therapy products. The agreement exclusively reserves 25 genetic targets for AstraZeneca, exploring up to 10 potential products for development.

• In August 2023, the FDA granted fast track designation (FTD) for TSHA-102 gene transfer therapy from Taysha Gene Therapies, Inc., used in treating patients with Rett syndrome.

• In August 2023, Xcell Biosciences partnered with Labcorp to jointly develop and validate cell and gene therapies.

• In July 2023, Astellas Pharma Inc. completed the acquisition of IVERIC bio, Inc. for $5.9 billion, obtaining a pipeline of AAV gene therapies for rare ophthalmic diseases.

• In June 2023, Sangamo Therapeutics entered into a licensing agreement with Voyager Therapeutics for the development of epigenetic regulation treatment, a gene therapy targeting prion disease.

• In May 2023, Ray Therapeutics secured USD 100 million in a Series A funding round, aimed at advancing optogenetic gene therapies.

• In May 2023, Oxford BioMedica Plc launched the TetraVectaTM System, a 4th generation lentiviral vector delivery system facilitating gene therapy by delivering therapeutic genes into target cells.

• In December 2022, Primera Therapeutics (launched by Mayflower Bioventures) collaborated with Cellectis, aiming to develop gene editing therapies for mitochondrial disease treatment.

• In June 2022, Bristol-Myers Squibb Company and Immatics NV expanded their alliance for the development of multiple allogeneic off-the-shelf TCR-T and CAR-T programs, both gene therapy programs targeting cancer cells.

• In January 2022, Novartis AG partnered with Alnylam to explore target therapies for liver function restoration, utilizing Alnylam’s siRNA technology, a gene therapy for functional liver regrowth.

• In January 2022, US-based biotech firm 64x Bio secured $55.0 million in funding, aiming to enhance its gene therapy manufacturing platform.

• In July 2021, Bluebird Bio Inc. entered a strategic alliance with National Resilience Inc. to develop next-generation cell and gene therapies for treating various diseases by modifying cell materials.

• In July 2021, Orchard Therapeutics collaborated with Pharming Group for the commercialization and development of HSC gene therapy, focusing on treating hereditary angioedema.

• In June 2021, UniQure NV acquired Corlieve Therapeutics to implement Corlieve's AMT-260 program, a gene therapy employing miRNA silencing technology to address temporal lobe epilepsy (TLE) by targeting kainite receptors.