Gene Therapy Market Size & Share Analysis - Growth Trends and Forecast (2026 - 2031)

Global Gene Therapy Market Trends and Insights

Surge in Approvals of One-Shot Curative Therapies

Regulators accelerated timelines in 2024-2025, clearing therapies such as Waskyra for Wiskott-Aldrich syndrome, Itvisma for spinal muscular atrophy, and Casgevy for sickle-cell disease under priority pathways.^{[1]U.S. Food and Drug Administration, “Approved Cellular and Gene Therapy Products,” fda.gov} Clinical datasets underpinning these decisions demonstrate durable efficacy, with Casgevy keeping 93.5% of patients crisis-free over 12 months and Beqvez maintaining greater than 40% factor IX activity, thereby eliminating the need for hemophilia B prophylaxis. Payers acknowledge that one-time interventions can offset decades of chronic-care costs, triggering recalibrated budget models. Japan’s Sakigake pathway extension to gene therapy underscores the broader alignment in the Asia-Pacific region, reinforcing the global shift toward curative paradigms.

Expanding Reimbursement for Ultra-Rare Disorders

Outcomes-based models now govern roughly 40% of U.S. commercial contracts, and CMS’s Cell and Gene Therapy Access Model covers 35 Medicaid programs, sharing financial risk with manufacturers.^{[2]Centers for Medicare & Medicaid Services, “Cell and Gene Therapy Access Model,” cms.gov} Europe remains fragmented but is experimenting with installment payments, as France’s HAS spreads Zolgensma’s USD 2.1 million cost over five years. U.S. forecasts from the Congressional Budget Office indicate that sickle-cell therapies alone could generate USD 1.8 billion in annual Medicaid expenditures by 2028, prompting legislatures to develop pooled-purchase solutions. Registry data from real-world Zolgensma use indicate that 95% of treated children retain motor milestones at a five-year follow-up, reinforcing the value of pay-for-performance schemes.

Manufacturing Platform Standardization (AAV & LV)

Lonza’s USD 1 billion Portsmouth facility and Catalent’s USD 675 million Harmans expansion are purpose-built for modular bioreactors ranging from 50 to 2,000 liters, which can pivot across multiple campaigns without requiring process transfer.^{[3]Lonza, “Lonza Announces USD1 Billion Portsmouth Facility,” lonza.com} MilliporeSigma’s AAV-MAX system cuts upstream costs in half by eliminating adherent culture requirements, while Viralgen’s Pro10™ platform reduced batch-failure rates to below 5% across 1,500 runs. Samsung Biologics is adding Asia-Pacific’s first large-scale AAV plant, foreshadowing a geographically diversified supply chain. The 2024 joint FDA-EMA guidance on AAV analytics harmonizes release testing, directly reducing duplicated validation costs.

Growing Venture & SPAC Funding Pipelines

Venture investors deployed USD 3.2 billion in 2024, with 70% of the funds allocated to Phase 2+ assets, indicating a higher demand for clinical de-risking. Prime Medicine’s USD 315 million Series C and Tome Biosciences’ USD 213 million Series A exemplify oversized bets on differentiated editing. SPAC activity declined in 2024-2025 as public markets prioritized revenue visibility; however, corporate venture arms such as Novo Holdings and Takeda Ventures remained active, bringing regulatory and CMC expertise alongside capital.

High COGS and Six-Figure Price Backlash

Viral-vector batches cost USD 500,000–2 million, driving list prices such as Lenmeldy’s USD 4.25 million, provoking U.S. Senate Finance hearings and NICE rejections under GBP 100,000/QALY thresholds. Yield variability ranging from 30% to 70% per campaign forces overproduction and write-offs. Continuous-manufacturing pilots by Resilience and Fujifilm Diosynth promise a 50% cost reduction but will not reach commercial scale meaningfully before 2027. Bluebird’s 2024 withdrawal of Zynteglo from the EU, following reimbursement failures, illustrates the existential risk posed by HTA bodies' reluctance to accept prices.

Complex Long-Term Safety Monitoring Mandates

The FDA requires 15-year patient surveillance for integrating vectors, including annual to triennial visits, plus hematologic malignancy screening, which costs smaller firms USD 5-10 million per year. EMA’s risk plans further extend the burden until full marketing authorization is achieved, thereby delaying breakeven timelines. Zolgensma’s REMS and Lyfgenia’s leukemia monitoring illustrate the multi-decade post-market commitments that strain single-asset developers. ASGCT’s voluntary registry initiative could centralize data but lacks universal participation.

Segment Analysis

By Therapy Type: Ex Vivo Platforms Accelerate on Allogeneic Potential

The gene therapy market size for in vivo approaches accounted for 67.31% of the revenue in 2025, reflecting its dominance in liver and systemic applications. Ex vivo therapies accounted for the remainder but are projected to outpace the market at a 21.97% CAGR, fueled by CRISPR-enabled allogeneic CAR-T designs that bypass autologous bottlenecks. Allogene and CRISPR Therapeutics advanced Phase 2 trials in 2024, demonstrating rapid 14-day vein-to-vein workflows. FDA draft CMC guidance clarified batch testing and donor-screening criteria, reducing regulatory ambiguity.

Adoption hinges on cost-effective point-of-care manufacturing. Lonza and Miltenyi Biotec piloted mobile clean rooms in 2024 that slash per-dose costs by 40%, positioning hospital sites as micro-factories. In vivo programs still encounter immunogenicity; up to 50% of candidates possess neutralizing antibodies, prompting the development of engineered capsids. Neurodegenerative disorders, led by uniQure’s AMT-130, show promise where ex vivo is impractical, keeping therapeutic diversity high.

Note: Segment shares of all individual segments available upon report purchase

By Vector Type: Non-Viral Systems Close Efficacy Gaps

Viral platforms generated 74.83% of 2025 revenue, anchored by AAV’s hepatocyte tropism and LV’s integration for hematologic disorders. Non-viral vectors are expected to expand at a 23.41% CAGR, narrowing the gap through optimized lipid nanoparticles that delivered 40%-60% editing rates in Moderna and BioNTech preclinical models. MaxCyte’s electroporation supports 50+ clinical trials, offering transient expression suitable for ex vivo editing.

Within viral categories, engineered AAV-PHP.B capsids entered first-in-human studies for Parkinson’s disease, highlighting next-generation tropism for CNS delivery. Touchlight’s enzymatic DNA competes as a plasmid alternative that avoids bacterial contaminants, broadening non-viral options. The FDA non-viral guidance standardized quality expectations, aligning oversight with irreversible editing risk rather than vector category.

By Indication: Neurology Gains Momentum

Oncology’s 44.15% contribution in 2025 is expected to continue as CAR-Ts advance into earlier-line settings, with Carvykti achieving 73% complete responses in multiple myeloma. However, neurology indications are forecast to grow at a 22.71% CAGR as intrathecal AAV delivery bypasses the blood-brain barrier. Successes include uniQure’s 79% huntingtin-protein knockdown and Neurocrine’s GBA1 Parkinson’s program. Ophthalmology remains a profitable niche thanks to the eye’s immune privilege, with Luxturna’s five-year durability data reinforcing payer confidence.

Rare genetic disorders continue to receive regulatory priority, with Waskyra and Itvisma obtaining approvals in 2025. Hematology stays competitive; eight hemophilia assets in late-stage development may squeeze pricing power, motivating differentiation through lower vector doses or outpatient administration settings.

Note: Segment shares of all individual segments available upon report purchase

By Delivery Method: Localized Administration Escalates

Systemic infusion represented 46.36% of revenue in 2025, but localized delivery is on a 25.18% CAGR trajectory. Intrathecal approaches require one-tenth the vector dose for spinal muscular atrophy, lowering immunogenic risk. Subretinal surgical protocols standardized by Luxturna are now in operation across 150 U.S. centers, ensuring reproducible outcomes. Intravitreal delivery is under evaluation for wet AMD, potentially reducing monthly anti-VEGF injections to annual schedules. Systemic regimens still dominate hepatocyte editing but face exclusion due to the development of neutralizing antibodies, prompting ongoing redesign of the capsid.

By End User: Academic Institutes Propel Early Innovation

Hospitals and specialty clinics captured 53.66% of 2025 revenue, functioning as commercial administration hubs equipped with GMP apheresis suites. Academic institutes, holding a smaller base, show a 26.64% CAGR, as 60% of active trials originate from investigator-initiated protocols. European Reference Networks exemplify cross-border harmonization, pooling cases of rare diseases for higher-powered studies. Hospital investments such as Massachusetts General’s USD 50 million cell-processing plant enhance domestic manufacturing, supporting decentralized point-of-care production.

Note: Segment shares of all individual segments available upon report purchase

Geography Analysis

North America maintained 41.36% of 2024 revenue, supported by 45 new RMAT designations and the CMS Access Model easing Medicaid barriers. Capacity built by Lonza, Catalent, and Resilience totaled 1.2 million liters of bioreactors, cementing the region’s supply dominance. Outcomes-based contracts now apply to 40% of U.S. therapies, exemplified by Lyfgenia’s coverage for 100 million lives. Canada trails with only two approvals in 2024, due to protracted provincial reimbursement negotiations.

The Asia-Pacific region, projected to grow at a 28.78% CAGR, is driven by China’s NMPA approvals of Roctavian and Yescarta, as well as over 200 active clinical trials. WuXi AppTec’s plasmid facility and Samsung’s AAV plant diversify global manufacturing. Japan’s PMDA extended Sakigake fast-track to gene therapy, and India green-lit its first CAR-T at one-tenth U.S. prices, showcasing cost innovation. Australia approved Casgevy but faces delays in reimbursement.

Europe grapples with fragmented HTA systems. Bluebird’s Zynteglo exit following reimbursement failures exemplifies the commercial risk associated with EMA approval. Germany’s IQWiG positive Hemgenix assessment and France’s installment payments for Zolgensma are isolated wins. EUnetHTA’s 2025 joint assessments aim to harmonize evidence, yet national payers still control pricing. The Middle East & Africa, as well as South America, remain nascent but are developing regulatory frameworks, with the UAE and Brazil approving select therapies for compassionate or public hospital use.