Gene therapy in 2026 is entering a more commercially important phase.

The industry is no longer asking whether genetic diseases can be treated by modifying or replacing faulty genes.

The bigger questions are now:

Can gene therapies deliver durable benefits? Can manufacturers produce them consistently at scale? Can healthcare systems afford them? And which platforms will become commercially sustainable?

The answers are creating opportunities across the gene therapy market, AAV gene therapy market, cell and gene therapy CDMO market, manufacturing, CRO services, bioanalytical testing and genomic medicine.

In 2026, the FDA has already added important milestones to the gene and cell therapy landscape. Regeneron’s Otarmeni became the first FDA-approved gene therapy for genetic hearing loss and the first approved dual-AAV gene therapy. Ionis received approval for Zanvastro (zilganersen) for Alexander disease, while uniQure submitted its Huntington’s disease gene therapy for FDA review. At the same time, safety and manufacturing challenges continue to expose the risks of developing one-time genetic medicines.

The opportunity is therefore expanding – but so is the complexity.

Gene Therapy Market in 2026: From Scientific Promise to Commercial Market

The broader cell and gene therapy market is being supported by increasing numbers of clinical programs, regulatory approvals, investment and demand for specialized manufacturing.

One current market estimate puts the global cell and gene therapy market at $12.21 billion in 2025, rising to $16.45 billion in 2026 and potentially reaching $143.55 billion by 2034, representing a projected CAGR of 31.1%.

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Market estimates vary considerably depending on what is included – for example, whether the market includes only therapeutic products or also manufacturing, services and supporting technologies.

That distinction matters.

The commercial opportunity is not limited to gene therapy products.

It also includes:

• Viral-vector manufacturing
• AAV production
• Plasmid DNA
• Cell processing
• Gene editing
• Bioanalytical testing
• CRO services
• CDMO services
• Quality control
• Regulatory consulting
• Companion diagnostics
• Genomic testing
• Cold-chain logistics

This creates a much larger cell and gene therapy ecosystem.

2026 FDA Gene Therapy Approvals: The Market Is Expanding

Regeneron’s Otarmeni: A New AAV Milestone

In April 2026, the FDA approved Otarmeni (lunsotogene parvec-cwha) from Regeneron for pediatric and adult patients with severe-to-profound or profound sensorineural hearing loss associated with biallelic OTOF variants.

The FDA described Otarmeni as the first-ever dual-AAV vector-based gene therapy.

In the efficacy population, 80% of 20 evaluable patients experienced improved hearing. The FDA granted accelerated approval, meaning continued approval is dependent on additional evidence to verify and describe clinical benefit and durability.

This approval is important for several reasons:

AAV delivery is expanding beyond traditional systemic applications.

It demonstrates the potential for gene therapy to address sensory disorders.

And it creates additional demand for:

AAV vector design + manufacturing + analytical testing + regulatory support.

Ionis and Zanvastro: The Pricing Question

In September 2026, the FDA approved Zanvastro (zilganersen) from Ionis Pharmaceuticals as the first disease-modifying treatment for Alexander disease.

The treatment is priced at approximately $285,000 per dose in the United States, according to Reuters.

Alexander disease affects fewer than 1,000 people in the U.S., illustrating one of the central commercial challenges of rare-disease therapies:

How do companies build sustainable economics when the addressable patient population is extremely small?

For investors and pharma strategy teams, this makes pricing, reimbursement, patient identification and treatment-center infrastructure as important as clinical efficacy.

uniQure Pushes Huntington’s Disease Gene Therapy Forward

Another major 2026 development came from uniQure.

In September, the company announced submission of a Biologics License Application to the FDA for ifezuntirgene inilparvovec (AMT-130) for Huntington’s disease, supported by three-year Phase I/II data. The company also submitted a marketing authorization application in the U.K.

This is significant because neurological disorders remain one of the most challenging frontiers for gene therapy.

The central challenge is not simply getting a vector into the body.

It is:

getting the right genetic payload to the right cells, at the right level, for long enough — without creating unacceptable safety risks.

REGENXBIO Shows the Other Side of the Story

Gene therapy development also continues to demonstrate why safety monitoring remains critical.

In August 2026, the FDA placed a clinical hold on REGENXBIO’s RGX-121 program for MPS II after asymptomatic spine MRI findings were identified in five participants.

REGENXBIO said it did not expect to resubmit the BLA in the near term while longer-term follow-up and additional analysis were conducted.

This follows earlier 2026 regulatory concerns around REGENXBIO’s MPS programs.

The commercial lesson is straightforward:

A successful gene-therapy platform must demonstrate not only efficacy, but also durable and manageable safety.

The Gene Therapy Market Is Not Just About AAV

AAV remains one of the most important platforms, but the market is becoming more diverse.

1. AAV Gene Therapy

AAV continues to dominate many in-vivo gene therapy programs because of its ability to deliver genetic material to specific tissues.

The key development areas include:

• Engineered capsids
• Tissue-specific targeting
• CNS delivery
• Retinal delivery
• Muscle delivery
• Lower-dose approaches
• Reducing immunogenicity

However, AAV also brings challenges involving:

Pre-existing immunity + manufacturing yield + dose requirements + durability + re-dosing limitations.

2. Gene Editing

Gene editing is moving toward more precise genetic intervention.

The FDA published a June 2026 draft guidance on leveraging prior knowledge for human gene therapies incorporating genome editing and another draft focused on safety assessment using next-generation sequencing.

This reflects a broader industry movement toward:

more precise editing + better characterization + stronger genomic safety assessment.

Companies working in this area include:

• Intellia Therapeutics
• CRISPR Therapeutics
• Editas Medicine
• Beam Therapeutics
• Prime Medicine
• Sangamo Therapeutics

The commercial opportunity extends into delivery systems, analytical testing, sequencing and manufacturing.

3. In-Vivo Gene Therapy

In-vivo approaches aim to deliver genetic medicines directly inside the patient’s body.

This could reduce some of the complexity associated with individualized ex-vivo manufacturing.

The major development question is:

Can in-vivo therapies achieve sufficient targeting and durability while maintaining an acceptable safety profile?

If they can, they could significantly change the economics of cell and gene therapy.

4. Neurological Gene Therapy

Neurology is becoming one of the most closely watched areas.

The pipeline now includes programs targeting:

• Huntington’s disease
• Alexander disease
• Parkinson’s disease
• Alzheimer’s disease
• Rett syndrome
• Angelman syndrome
• MPS disorders
• Other rare neurological diseases

The reason is straightforward:

Many neurological diseases are caused by defined genetic abnormalities, but conventional treatments often manage symptoms rather than the underlying cause.

The challenge is equally clear:

The brain is one of the hardest organs to reach safely.

5. Rare Disease Remains a Major Driver

Rare diseases continue to provide an important pathway for gene-therapy development.

A single genetic defect can create a clearly defined therapeutic target.

This creates advantages in:

• Patient identification
• Biomarker development
• Natural-history studies
• Orphan-drug incentives
• Regulatory pathways

But rare disease also creates commercial challenges.

Small patient populations can mean extremely high prices per treatment.

Therefore, successful commercialization requires more than regulatory approval.

It requires:

diagnosis + reimbursement + treatment-center access + patient identification + long-term follow-up.

The Manufacturing Market May Become the Bigger Opportunity

This is where the cell and gene therapy manufacturing market becomes particularly important.

A therapy can demonstrate excellent clinical efficacy and still struggle commercially if it cannot be manufactured consistently and economically.

The manufacturing ecosystem includes:

AAV → Lentivirus → Plasmid DNA → Cell processing → Gene editing → Fill-finish → Analytical testing → QC → Logistics

One estimate places the global cell and gene therapy CDMO market at $5.2 billion in 2025, increasing to approximately $6.2 billion in 2026 and potentially reaching $27.1 billion by 2033, with a 23.4% CAGR.

Another 2026 market estimate puts the broader cell and gene therapy CDMO market at $10.2 billion in 2026, demonstrating how dramatically market estimates can vary based on market definition and methodology.

For companies making strategic decisions, the key point is not which estimate is “right.”

The key point is:

Outsourced CGT manufacturing is becoming a major commercial opportunity.

What Do Gene Therapy CDMO Clients Actually Need?

Pharma and biotech companies are increasingly looking beyond basic manufacturing capacity.

They want:

Vector development

Can the CDMO help optimize the vector and production process?

Process development

Can the process move from laboratory scale toward clinical and commercial manufacturing?

Analytical development

Can the provider establish robust methods for identity, potency, purity and safety?

GMP manufacturing

Can the company produce clinical-grade material consistently?

Scale-up

Can manufacturing capacity grow alongside clinical demand?

Technology transfer

Can the process be transferred between facilities without losing product quality?

Regulatory CMC support

Can the CDMO help prepare the manufacturing package for regulators?

Commercial manufacturing

Can the platform support long-term supply?

This is why cell and gene therapy CDMO market research should not simply compare facility square footage.

The more valuable question is:

Which CDMOs have the capabilities that future gene-therapy programs actually need?

Cell and Gene Therapy CRO Market: Another Growth Opportunity

Clinical development creates another major service opportunity.

Gene therapy trials often require specialized expertise in:

• Patient recruitment
• Rare-disease clinical trials
• Natural-history studies
• Biomarker testing
• Long-term follow-up
• Immunogenicity monitoring
• Specialized clinical sites
• Regulatory submissions
• Data management

This creates demand for specialized cell and gene therapy CRO services.

The CRO opportunity is particularly attractive for companies that can combine:

clinical operations + biomarker expertise + regulatory knowledge + rare-disease patient access.

Bioanalytical Testing Is Becoming Critical

One of the most underestimated opportunities in gene therapy is cell and gene therapy bioanalytical testing services.

As regulatory expectations increase, sponsors need increasingly sophisticated analytical packages.

Important areas include:

• Vector genome analysis
• Biodistribution
• Transgene expression
• Neutralizing antibodies
• Cellular immune responses
• Potency
• Product-related impurities
• Host-cell proteins
• Residual DNA
• Replication-competent virus testing
• Genomic integration
• Off-target assessment for gene editing

The FDA’s 2026 guidance activity demonstrates the growing importance of CMC, potency and safety characterization across CGT development.

For testing laboratories, CROs and analytical technology providers, this creates a growing addressable market.

Companies to Watch in Gene Therapy

The competitive landscape now includes large pharmaceutical companies, specialist biotech companies, vector developers, gene-editing companies and CDMOs.

Regeneron

Regeneron is now directly associated with the commercial gene-therapy market through Otarmeni.

Its 2026 approval demonstrates how major pharmaceutical companies can combine advanced vector technology with regulatory and commercial infrastructure.

Novartis

Novartis continues to commercialize Zolgensma, while its broader cell and gene therapy capabilities remain strategically important.

Zolgensma group sales reached approximately $1.23 billion in 2025.

Sarepta Therapeutics

Sarepta generated approximately $898.7 million in 2025 ELEVIDYS net product revenue, contributing to total 2025 net product revenue of $1.86 billion.

The company demonstrates both the commercial potential and complexity of rare-disease genetic medicines.

REGENXBIO

REGENXBIO remains important for AAV technology and has programs across neurological, muscular and retinal diseases.

The company reported positive Phase III results for RGX-202 in Duchenne muscular dystrophy in 2026, while simultaneously facing regulatory challenges around RGX-121.

uniQure

uniQure is one of the most closely watched companies in neurological gene therapy, particularly following its 2026 Huntington’s disease BLA submission.

Sangamo Therapeutics

Sangamo is advancing gene therapy and genome-engineering approaches, including its Fabry disease program and zinc-finger technology platform.

Experts Shaping the Gene Therapy Industry

For companies conducting competitive or technology intelligence, following scientific leaders can be as important as tracking corporate announcements.

R. Jude Samulski, PhD

A pioneer of AAV vector technology at the University of North Carolina, Samulski received the 2026 ASGCT Founders Award for contributions including the original cloning of AAV, chimeric AAV capsids and AAV-mediated gene delivery.

James M. Wilson, MD, PhD

A major figure in AAV gene therapy and genetic medicine, Wilson has been involved in foundational AAV development and translation into human therapies.

Beverly L. Davidson, PhD

Davidson was recognized by ASGCT in 2026 for major contributions to understanding inherited brain diseases and developing gene-based treatment strategies.

Ronit Mazor, PhD

At the FDA, Mazor works on gene-transfer immunogenicity and CMC review and leads research focused on evaluating and mitigating immunogenicity of gene-therapy products.

For market participants, these scientific and regulatory perspectives help explain where the technology is heading – beyond company press releases.

What Are the Biggest Gene Therapy Challenges in 2026?

Safety

Long-term safety remains one of the most important concerns.

Companies must understand:

immunogenicity + toxicity + biodistribution + off-target effects + genomic integration + durability.

Manufacturing

AAV and other vectors can be difficult to manufacture at high quality and scale.

Key challenges include:

• Yield
• Purity
• Potency
• Batch consistency
• Empty/full capsid ratios
• Scale-up
• Raw materials
• Facility capacity
• Cost of goods

Cost

A one-time therapy can carry a very high upfront price.

The commercial challenge becomes:

Can payers justify a large upfront payment for a therapy that may provide benefits over many years?

Reimbursement

Gene therapy requires new payment models in some cases.

Potential approaches include:

• Outcomes-based agreements
• Installment payments
• Risk-sharing models
• Annuity-style reimbursement

Patient Identification

A therapy cannot generate revenue if patients remain undiagnosed.

This makes:

genetic testing + newborn screening + biomarker testing

important components of the commercial ecosystem.

Long-Term Follow-Up

One-time treatment does not mean one-time monitoring.

Sponsors and regulators need long-term data to understand:

durability + safety + clinical benefit.

The Most Important 2026 Trends

The gene therapy market is moving toward several clear themes.

AAV 2.0

Next-generation capsids are being developed to improve targeting, reduce dose and overcome delivery limitations.

In-vivo gene editing

The industry is moving toward direct genetic modification inside the patient.

CNS delivery

Brain and spinal-cord diseases are becoming an increasingly important development area.

Better potency assays

Regulators and sponsors are placing greater emphasis on meaningful, reproducible potency measurements.

Manufacturing flexibility

Sponsors increasingly need CDMOs capable of moving programs from early development to commercial production.

Integrated diagnostics

Patient identification and biomarker testing are becoming increasingly connected to therapy commercialization.

AI and computational biology

AI and computational approaches are increasingly being used for:

vector design + target identification + patient stratification + biomarker analysis.

Regulatory modernization

The FDA issued multiple 2026 CGT guidance documents covering development, CMC flexibility, potency and genome-editing safety.

This is important because regulatory clarity can directly affect development timelines and investment decisions.

What Do Clients Actually Want to Know?

For organizations entering or expanding in gene therapy, a market-size number is rarely enough.

Pharma companies ask:

Which technologies are clinically validating?

Which indications have the strongest commercial potential?

Who are the emerging competitors?

Which companies should we partner with or acquire?

Biotech companies ask:

Who can manufacture our vector?

Which CDMO has the right capabilities?

What does our competitive landscape look like?

Which geographies should we enter?

CDMOs ask:

Where will future manufacturing demand come from?

Which biotech companies are approaching clinical milestones?

Which vector types require additional capacity?

Which customers are most likely to outsource?

CROs ask:

Where are clinical-trial opportunities emerging?

Which rare diseases have growing pipelines?

Which sponsors need specialized trial infrastructure?

Diagnostics and bioanalytical companies ask:

Which biomarkers will become commercially important?

What testing capabilities will regulators require?

Where is outsourcing demand increasing?

Investors ask:

Which platforms are technically differentiated?

Which companies have realistic commercialization pathways?

What are the safety, manufacturing and reimbursement risks?

These are the questions that turn market research into commercial intelligence.

What Towards Healthcare Research & Consulting Can Provide

Towards Healthcare Research & Consulting can help organizations turn the expanding gene-therapy ecosystem into actionable opportunities.

Market opportunity

TAM / SAM / SOM + market sizing + growth forecasting

Competitive intelligence

Company benchmarking + pipeline tracking + technology comparison

Manufacturing intelligence

AAV + viral-vector + CDMO capacity + outsourcing analysis

Buyer intelligence

Target companies + decision-makers + customer segmentation

Partnership intelligence

Licensing + collaborations + M&A + strategic opportunities

Clinical intelligence

Trials + indications + endpoints + regulatory milestones

Commercial strategy

Market entry + geographic prioritization + GTM + opportunity mapping

Supporting markets

Intelligence across:

• Gene therapy market
• AAV gene therapy market
• Cell and gene therapy CDMO market
• Cell and gene therapy manufacturing market
• Cell and gene therapy CRO market
• Cell and gene therapy bioanalytical testing services market

The objective is simple:

Not just telling clients how large the market is – but showing them where the opportunity is, who controls it, who buys it and how they can enter it.