Gene Therapy Market Size and Share
Gene Therapy Market Analysis by Mordor Intelligence
The Gene Therapy Market size is estimated at USD 9.74 billion in 2025, and is expected to reach USD 24.34 billion by 2030, at a CAGR of 20.11% during the forecast period (2025-2030).
Regulatory authorities cleared nine cell and gene therapies in 2024 alone, signalling rising confidence in clinical and commercial viability and unlocking wider reimbursement pathways in key markets. Heightened biopharma and private-equity funding is accelerating late-stage assets, while expanding manufacturing capacity both viral and non-viral reduces historic supply-chain friction. Rapid advances in in-vivo editing and vector engineering are broadening therapeutic scope beyond rare diseases to high-prevalence disorders, especially in neurology and ophthalmology. Asia-Pacific’s double-digit trial growth and North America’s policy incentives together ensure a diversified demand base, even as high product prices and safety-monitoring requirements pose access headwinds.
Key Report Takeaways
- By vector type, Adeno-associated Virus captured 38.54% of the gene therapy market share in 2024, while non-viral delivery methods are poised to expand at a 24.34% CAGR through 2030.
- By indication, oncology commanded 42.92% revenue in 2024; neurology applications are advancing at a 25.62% CAGR to 2030.
- By delivery mode, in vivo approaches held 65.92% of the gene therapy market size in 2024, whereas ex vivomethods will progress at a 23.07% CAGR between 2025-2030.
- By end user, hospitals and clinics accounted for 59.22% share of the gene therapy market size in 2024; specialty treatment centers are tracking a 21.89% CAGR through 2030.
- By geography, North America led with 41.78% revenue share in 2024; Asia-Pacific is forecast to accelerate at a 27.68% CAGR over the same horizon.
Global Gene Therapy Market Trends and Insights
Drivers Impact Analysis
Driver | (~) % Impact on CAGR Forecast | Geographic Relevance | Impact Timeline |
---|---|---|---|
Regulatory approvals demonstrating viability | +5.2% | Global, concentrated in North America and Europe | Short term (≤ 2 years) |
Advances in vector engineering & in-vivo tech | +4.9% | Early adoption in North America and Europe | Long term (≥ 4 years) |
Manufacturing infrastructure expansion | +4.8% | North America, Europe, emerging Asia-Pacific | Medium term (2-4 years) |
Rapid scale-up of commercial AAV CDMO capacity | +3.8% | Global, North America and Europe, expanding to Asia-Pacific | Medium term (2-4 years) |
Biopharma and private-equity funding | +3.5% | Innovation hubs in the US, Europe and China | Medium term (2-4 years) |
Growing inclusion of rare-disease gene therapies | +2.7% | Global, with higher impact in developed markets | Long term (≥ 4 years) |
Source: Mordor Intelligence
Rising Regulatory Approvals Demonstrating Clinical and Commercial Viability
Frequent clearances from major regulators are reducing development risk and compressing launch timelines. The FDA’s cumulative total of 37 approved cell and gene products by 2024 shows that agency reviewers now apply dedicated frameworks such as the Regenerative Medicine Advanced Therapy designation, which trims development time by about 40% for qualifying programs.[1]U.S. Food and Drug Administration, “Approved Cellular and Gene Therapy Products,” fda.govEurope’s PRIME scheme and the United Kingdom’s 2025 rule for point-of-care manufacture further ease market entry. Approval of ZEVASKYN for recessive dystrophic epidermolysis bullosa in 2025 exemplifies how indications are extending beyond ultra-rare diseases into broader dermatological segments. Such widening therapeutic reach supports multi-year growth in the gene therapy market and stimulates follow-on investment from payers expecting durable clinical benefit.
Continuous Technological Advances in Vector Engineering and In-Vivo Editing Platforms
Enhanced capsid libraries, machine-learning-guided sequence optimisation, and novel lipid constructs are sharpening tissue tropism and reducing immunogenicity. Researchers at the Perelman School of Medicine reported DNA-loaded lipid nanoparticles that persist in vivo for months, opening durable dosing for chronic diseases.[2]Penn Medicine News, “DNA-Loaded Lipid Nanoparticles Remain Active for Months,” pennmedicine.orgOrganoid platforms offer human tissue-specific test beds that improve translational predictability, supported by the FDA Modernization Act’s acceptance of non-animal models. AI-assisted algorithms such as OpenCRISPR-1 cut design cycles and elevate editing precision. Collectively these innovations shift the therapeutic ceiling higher, create room for repeat dosing, and fuel a virtuous loop of clinical success and capital deployment that enlarges the gene therapy market.
Expansion of Global Viral-Vector and Non-Viral Manufacturing Infrastructure
Large-scale capital deployment is boosting both capacity and sophistication of production lines. Novo Nordisk’s USD 16.5 billion purchase of Catalent in 2024 underlines how big-pharma is locking in end-to-end control of supply chains. Viral-vector upgrades now incorporate continuous-flow bioreactors and closed-system downstream processing, cutting batch lead times by double digits. Non-viral lipid nanoparticle platforms lower cost of goods by up to 60% and can package larger payloads, removing former technical barriers to polygenic indications.[3]Journal of Nanobiotechnology editors, “Advances in Lipid Nanoparticle Delivery,” springeropen.com Automation, AI-enabled quality analytics, and modular clean-room designs together enhance reliability, encouraging contract manufacturers to pre-build suites that can be rapidly customized for new sponsors.
Escalating Biopharma & Private-Equity Funding into Gene-Therapy R&D
Capital inflows remain resilient despite broader market volatility. Upfront values on Phase II and III out-licensing deals rose 35% in 2024 as competition for late-stage assets intensified. Sangamo Therapeutics secured USD 18 million upfront and milestone potential of USD 1.4 billion for its CNS-targeted capsids with Eli Lilly, illustrating investor appetite for differentiated delivery vectors. Private-equity groups are also moving downstream; Carlyle and SK Capital’s acquisition of bluebird bio in 2025 injects commercialisation funds into approved therapies for sickle-cell disease and β-thalassemia, demonstrating the strategic value of marketed assets. Such sustained funding accelerates pipeline diversification and sustains demand for manufacturing services, reinforcing the long-term expansion of the gene therapy market.
Restraints Impact Analysis
Restraint | (~) % Impact on CAGR Forecast | Geographic Relevance | Impact Timeline |
---|---|---|---|
High therapy price tags | –3.8% | Greater effect in emerging markets and restrictive reimbursement regions | Medium term (2-4 years) |
Manufacturing complexity & vector supply limits | –2.6% | Varies by regional production capability | Short term (≤ 2 years) |
Safety concerns requiring long-term monitoring | –2.1% | Most intense where regulatory oversight is stringent | Long term (≥ 4 years) |
Shortage of plasmid/capsid raw materials | –1.9% | Acute in regions with limited supply-chain resilience | Short term (≤ 2 years) |
Source: Mordor Intelligence
High Therapy Price Tags Creating Access and Affordability Barriers
Single-dose cures command list prices between USD 373,000 and USD 4.25 million, straining payer budgets and testing societal willingness to fund curative therapies. Providers cite social support gaps, travel distance, and prior authorisation hurdles as the top access blockers. Innovative reimbursement options—outcome-based contracts, amortisation models, and warranties are emerging but still require complex data sharing and long-term follow-up infrastructure. The Centers for Medicare & Medicaid Services launched a Cell and Gene Therapy Access Model in 2025 that encourages state-level pooled purchasing to temper budget impact. These financial headwinds are expected to moderate adoption in cost-sensitive regions, softening the gene therapy market growth curve despite strong clinical value propositions.
Manufacturing Complexity and Constrained Supply of GMP-Grade Vectors
Even with aggressive capacity build-outs, supply of GMP-grade AAV and lentiviral vectors meets only one-quarter of projected 2025 demand. Production cycles can exceed nine months, slowing launch timelines and creating inventory risk. Emerging decentralised solutions such as modular clean-room pods cut per-dose costs from about USD 400,000 to under USD 35,000 in pilot settings, though regulatory harmonisation remains a challenge. Until such models scale, constrained vector availability will continue to act as a structural brake on the gene therapy market.
Segment Analysis
By Vector Type: AAV Dominates While Non-Viral Methods Surge
AAV vectors held 38.54% of the gene therapy market share in 2024 owing to favourable safety and sustained transgene expression that underpin multiple approved products for haemophilia and inherited retinal disorders. This leadership is reinforced by broad tissue tropism and an expanding pool of engineered serotypes that improve specificity. However, non-viral lipid nanoparticle systems are the fastest-rising alternative, forecast to post a 24.34% CAGR through 2030 as they carry larger genetic payloads and simplify manufacturing economics. The gene therapy market size for non-viral platforms is therefore set to close part of the current revenue gap. Lentiviral vectors remain the mainstay for ex-vivo CAR-T manufacture, whereas herpes-based systems gain traction in oncology for their payload capacity. Continued optimisation of capsid libraries and synthetic promoters will determine whether viral vectors can defend share against cost-efficient non-viral challengers.
The surge in non-viral approaches also alleviates raw-material constraints because lipid components are amenable to standard pharmaceutical supply chains. DNA-loaded nanoparticles that remain active for months lower repeat-dosing requirements and can be tuned for organ-selective uptake. Such flexibility attracts developers targeting polygenic neurological disorders where payload size exceeds AAV’s packaging limit. Consequently, the competitive dynamic inside the gene therapy market is shifting from vector availability toward delivery precision, creating space for platform-agnostic CDMOs to capture emerging demand.
Note: Segment shares of all individual segments available upon report purchase
By Indication: Oncology Leads While Neurology Accelerates
Oncology applications generated 42.92% of revenue in 2024, supported by a steady flow of CAR-T approvals and a deep pipeline aimed at solid tumours. Robust evidence for complete responses in relapsed haematological malignancies sustains payer acceptance despite price pressures. Yet neurology claims the highest growth trajectory at a 25.62% CAGR to 2030. Emerging capsids that cross the blood-brain barrier and precise in-vivo editing tools permit direct intervention in disorders like Rett syndrome and Huntington’s disease. The gene therapy market size allocated to CNS disorders is expected to multiply as bilateral regulatory incentives compress development risk.
Rare metabolic and ophthalmology indications continue to yield new approvals because tissue immune privilege and clear biomarkers simplify clinical endpoints. Cardiovascular targets such as hypertrophic cardiomyopathy are gaining venture funding, hinting at broader systemic disease uptake. As therapeutic breadth widens, portfolio optimisation becomes critical; sponsors must balance oncology cash flows with high-potential but scientifically complex neurology assets.
By Delivery Mode: In Vivo Dominates While Ex Vivo Gains Momentum
Direct administration commanded 65.92% of revenue in 2024, primarily due to approvals for ocular, muscular, and hepatic disorders where localised injection maximises vector efficiency. In vivo dosing eliminates cell-processing steps, shortens treatment timelines, and fits existing hospital workflows, thereby sustaining its lead. However, ex vivo engineering is on track for a 23.07% CAGR as manufacturing automation, improved viral transduction, and allogeneic platforms lower per-patient costs. The growing pipeline of engineered immune cells for autoimmune diseases showcases ex vivo versatility beyond oncology. As such, the gene therapy market benefits from complementary growth across both delivery channels, reducing over-reliance on any single modality and smoothing supply-chain requirements.
Optimised ex vivo protocols now achieve higher cell viability and gene-transfer efficiency, factors that previously hindered clinical outcomes. Scale-out strategies and point-of-care production further reduce logistic complexity, laying groundwork for community-based infusion centres. Over time, cost convergence between modalities may encourage sponsors to select delivery based solely on biological suitability rather than manufacturing practicability.

Note: Segment shares of all individual segments available upon report purchase
By End User: Hospitals Lead While Specialty Centers Expand Rapidly
Hospitals and academic medical centres represented 59.22% of administration volume in 2024 because they possess multidisciplinary teams and controlled environments necessary for gene therapy infusion. These institutions often serve as trial sites, creating seamless transition from study to standard of care. Yet specialty infusion facilities are projected to grow at 21.89% CAGR through 2030. Their lean operating models reduce site-of-care costs and improve patient convenience, particularly where travel distance to tertiary centres is a barrier. The gene therapy market size captured by dedicated centres therefore stands to climb as payers incentivise lower-cost settings.
Academic research institutes retain influence by hosting first-in-human studies and refining manufacturing protocols. Community hospitals are gradually integrating validated treatment workflows, helped by remote-monitoring platforms that minimise repeated hospital visits. The expansion of decentralised care widens regional access and supports higher procedural throughput, addressing a critical capacity constraint as more products gain approval.
Geography Analysis
North America held 41.78% of global revenue in 2024, sustained by an accommodating regulatory environment, deep venture capital pools, and extensive clinical infrastructure. The United States approved 34 first-in-class gene therapies in 2024 alone, highlighting its central role in product launch sequencing. Federal initiatives such as ARPA-H grants channel significant funds toward manufacturing innovation and safety surveillance, reinforcing the region’s leadership in the gene therapy market.
Europe maintains strong scientific output and benefits from the EMA’s adaptive pathways that expedite breakthrough treatments. The forthcoming UK framework for decentralised manufacture, effective July 2025, may reduce logistical burdens and stimulate point-of-care production. However, unequal national reimbursement policies prolong time-to-patient across the bloc; the new EU Health Technology Assessment regulation in 2025 aims to harmonise evidence requirements but could also lengthen negotiations. Outcomes-based payment models, as demonstrated by Hemgenix agreements in England and Denmark, are gradually easing adoption hurdles.
Asia-Pacific registers the fastest growth at a 27.68% CAGR. China hosts more than 400 cell-based studies and invests heavily in domestic vector plants, positioning itself as both demand and supply engine. Japan’s accelerated pathways for regenerative medicines and Singapore’s digital licensing portal further streamline approvals. While regulatory standards converge with Western agencies, variable reimbursement and export controls on genetic materials remain challenges. Continuous policy support and maturing local CDMOs nevertheless assure that Asia-Pacific will capture an increasing proportion of the gene therapy market.

Competitive Landscape
Top Companies in Gene Therapy Market
The competitive terrain is moderately fragmented, populated by seasoned pharma incumbents and agile biotech innovators. Big-pharma houses such as Novartis, Pfizer, and Roche are acquiring platform technologies to offset patent cliffs; Novo Nordisk’s tie-up with 2seventy bio exemplifies portfolio diversification into gene editing. Partnering activity reached 90 deals in Q1 2025, reflecting intense scouting for capsid engineering, non-viral delivery, and scalable manufacturing.
Technology differentiation drives competitive advantage. Krystal Biotech leverages a proprietary HSV-1 vector enabling repeat topical administration; its product VYJUVEK generated USD 341.2 million since launch and achieved 97% US coverage, demonstrating commercial proof of rare-disease dermatologic applications. AI integration is another battleground: MeiraGTx’s joint venture with Hologen Neuro AI applies deep-learning design loops to CNS capsids, while Amgen reported a 19% revenue rise in 2024 aided by engineered RNA payloads that capitalise on machine-learning-guided optimisation.
White-space opportunities include vector capacity expansion, targeted non-oncology indications, and novel financing frameworks that match one-time therapy benefits with multiyear cost savings. Contract development and manufacturing organisations offering turnkey, platform-agnostic services are poised to gain share as smaller sponsors outsource production. As the pipeline diversifies and regional approval pathways proliferate, the gene therapy market rewards players that combine scientific depth with supply-chain agility.
Gene Therapy Industry Leaders
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Gilead Sciences, Inc.
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Amgen Inc.
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Novartis AG
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bluebird bio Inc.
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Biogen Inc.
- *Disclaimer: Major Players sorted in no particular order

Recent Industry Developments
- May 2025: Sarepta Therapeutics, Inc., a frontrunner in precision genetic medicine for rare diseases, has secured a pivotal nod from Japan's Ministry of Health, Labour, and Welfare (MHLW) for its drug, ELEVIDYS (delandistrogene moxeparvovec). This approval, granted under Japan's conditional and time-limited pathway, targets Duchenne muscular dystrophy (DMD). Specifically, ELEVIDYS is sanctioned for children aged 3 to under 8, provided they lack deletions in exon 8 and/or exon 9 of the DMD gene and test negative for anti-AAVrh74 antibodies. Notably, this marks the inaugural global endorsement for treating DMD in children under 4.
- May 2025: Abeona Therapeutics received FDA approval for ZEVASKYN, the first cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), demonstrating effective wound healing in clinical trials.
- February 2025: Genprex, Inc., a clinical-stage gene therapy firm, is making strides in its diabetes gene therapy program, bolstered by a collaboration with the University of Pittsburgh. The two entities have streamlined Genprex's previous license agreements with Pitt into a singular exclusive agreement. This new pact encompasses multiple technologies aimed at crafting a gene therapy product for both Type 1 and Type 2 diabetes. Furthermore, Genprex has established a wholly-owned subsidiary, Convergen Biotech, Inc., dedicated to advancing its diabetes initiatives.
- October 2024: Governor Hochul announced a significant development for the gene therapy market with the launch of the next phase of Long Island's New York Biogenesis Park, a nation-leading cell and gene therapy innovation hub. This USD 430 million project aims to accelerate research, development, and commercialization of life-saving therapies. The state's USD 150 million commitment marks the largest state investment in cell and gene therapy nationwide, further advancing New York's vision to establish leadership in this critical field.
- September 2024: Genprex, Inc., a clinical-stage gene therapy company dedicated to pioneering transformative therapies for cancer and diabetes patients, has unveiled plans to shift its diabetes clinical development program and associated gene therapy assets into a newly established, wholly-owned subsidiary. This new entity, dubbed "NewCo", will zero in on the advancement and commercialization of GPX-002, a gene therapy candidate targeting both Type 1 (T1D) and Type 2 diabetes (T2D).
Global Gene Therapy Market Report Scope
As per the scope of this report, gene therapy is an advanced medical treatment that involves the transfer of a standard or healthy gene to replace a defective gene in a cell. It can cure various chronic and genetic disorders in human beings, for which no final cure has been developed. The market is segmented by Indication (Cancer, Metabolic Disorders, Eye Disorders, Spinal Muscular Atrophy, and Other Indications), Technology (Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Other Technologies), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The market report also covers the estimated market sizes and trends for 17 countries across major regions globally. The report offers the value (in USD million) for the above segments.
By Vector Type | Adeno Virus Vector | ||
Adeno-associated Virus Vector | |||
Lentiviral Vector | |||
Retroviral Vector | |||
Herpes Virus Vector | |||
Other Vector Types | |||
By Indication | Oncology | ||
Rare Metabolic Disorders | |||
Ophthalmology | |||
Neurology / CNS | |||
Cardiovascular and Musculoskeletal | |||
Other Indications | |||
By Delivery Mode | In Vivo Gene Delivery | ||
Ex Vivo Gene Delivery | |||
By End User | Hospitals and Clinics | ||
Specialty Treatment / Infusion Centers | |||
Academic and Research Institutes | |||
By Geography | North America | United States | |
Canada | |||
Mexico | |||
Europe | Germany | ||
United Kingdom | |||
France | |||
Italy | |||
Spain | |||
Rest of Europe | |||
Asia-Pacific | China | ||
Japan | |||
India | |||
Australia | |||
South Korea | |||
Rest of Asia-Pacific | |||
Middle East & Africa | GCC | ||
South Africa | |||
Rest of Middle East & Africa | |||
South America | Brazil | ||
Argentina | |||
Rest of South America |
Adeno Virus Vector |
Adeno-associated Virus Vector |
Lentiviral Vector |
Retroviral Vector |
Herpes Virus Vector |
Other Vector Types |
Oncology |
Rare Metabolic Disorders |
Ophthalmology |
Neurology / CNS |
Cardiovascular and Musculoskeletal |
Other Indications |
In Vivo Gene Delivery |
Ex Vivo Gene Delivery |
Hospitals and Clinics |
Specialty Treatment / Infusion Centers |
Academic and Research Institutes |
North America | United States |
Canada | |
Mexico | |
Europe | Germany |
United Kingdom | |
France | |
Italy | |
Spain | |
Rest of Europe | |
Asia-Pacific | China |
Japan | |
India | |
Australia | |
South Korea | |
Rest of Asia-Pacific | |
Middle East & Africa | GCC |
South Africa | |
Rest of Middle East & Africa | |
South America | Brazil |
Argentina | |
Rest of South America |
Key Questions Answered in the Report
What is the current value of the gene therapy market?
The gene therapy market is valued at USD 9.74 billion in 2025 and is projected to climb to USD 24.34 billion by 2030.
Which vector type leads global sales?
Adeno-associated Virus vectors account for 38.54% of 2024 revenue due to their safety profile and durable expression.
Why is Asia-Pacific growing so quickly?
Asia-Pacific posts a 27.68% CAGR due to strong government funding, expanding clinical trial volume in China, and streamlined approval pathways in Japan and Singapore.
How are payers managing high therapy costs?
Health systems employ outcome-based contracts, amortised payments, and pooled state purchasing models such as the CMS Cell and Gene Therapy Access Model.
What factor most limits market growth today?
Manufacturing complexity and limited supply of GMP-grade vectors continue to cap near-term product availability and elevate cost of goods.