United States Gene Therapy For Rare Disease Market 2033 | Growth Drivers, Key Players & Investment Opportunities

Market Size and Growth

Gene Therapy for Rare Disease Market Size reached US$ 3.01 Billion in 2024 and is expected to reach US$ 24.54 Billion by 2033, growing at a CAGR of 26.5% during the forecast period 2025-2033.

Get a Free Sample PDF Of This Report (Get Higher Priority for Corporate Email ID):- https://www.datamintelligence.com/download-sample/gene-therapy-for-rare-disease-market?sb

Key Development:

United States: Recent Gene Therapy for Rare Disease Developments

✅ In December 2025, the U.S. FDA approved Waskyra (etuvetidigene autotemcel) as the first gene therapy for Wiskott-Aldrich syndrome. The one-time treatment targets the underlying genetic cause in pediatric and adult patients. This approval represents a major milestone for nonprofit-led gene therapy development.

✅ In November 2025, the FDA approved Itvisma (onasemnogene abeparvovec-brve) for spinal muscular atrophy patients aged two years and older. The intrathecal gene therapy expands access beyond infant populations. It strengthens the long-term treatment landscape for rare neuromuscular disorders.

✅ In September 2025, the FDA granted Fast Track designation to MVX-220 for Angelman syndrome, accelerating regulatory interactions and clinical development. The therapy addresses a severe neurogenetic disorder with limited treatment options. This move highlights growing regulatory support for rare disease gene therapies.

✅ In September 2025, MCO-010 received RMAT designation for Stargardt disease and inherited retinal dystrophies. The designation enables prioritized review and development guidance. It reflects increasing momentum in gene-based vision restoration therapies.

Buy Now & Unlock 360° Market Intelligence: https://www.datamintelligence.com/buy-now-page?report=gene-therapy-for-rare-disease-market

Japan: Recent Gene Therapy for Rare Disease Developments

✅ In December 2025, OTL-200 was designated a rare disease regenerative medicine product in Japan for adrenoleukodystrophy (ALD). The status enables priority regulatory review and development support. It strengthens Japan's pipeline for advanced gene therapies targeting neurological disorders.

✅ In October 2025, OTL-200 also received Orphan Drug and Priority Review designation in Japan for early-onset metachromatic leukodystrophy (MLD). The designation accelerates clinical and commercialization pathways. It reinforces Japan's commitment to rare pediatric gene therapies.

✅ In October 2025, Japan's Ministry of Health, Labour and Welfare reaffirmed support for conditional and time-limited approval pathways for regenerative and gene therapies. The framework enables earlier patient access while collecting confirmatory data. It continues to attract global gene therapy developers to Japan.

✅ In September 2025, Takara Bio completed regulatory acceptance for a clinical trial of its NY-ESO-1 siTCR gene therapy. The program prepares for patient enrollment in rare and advanced sarcoma indications. It marks progress in Japan's translational gene therapy ecosystem.

Key Players:

=> Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics Group., Krystal Biotech, Inc., and others.

Speak to Our Analyst and Get Customization in the report as per your requirements: https://www.datamintelligence.com/customize/gene-therapy-for-rare-disease-market?sb

Key Segments:

By Vector Type

Viral Vectors dominate with 65% share, driven by high transduction efficiency and strong clinical success in approved gene therapies. Adeno-associated virus (AAV), lentivirus, and retrovirus vectors are widely used in oncology, rare genetic disorders, and ophthalmology. Non-Viral Vectors account for 35%, supported by lower immunogenicity, cost advantages, and growing adoption of lipid nanoparticles, plasmid DNA, and polymer-based delivery systems.

By Technique

Gene Addition leads with 45% share, owing to its extensive use in treating inherited disorders and blood-related diseases by introducing functional genes. Gene Silencing holds 30%, driven by rising applications of RNA interference (RNAi) and antisense oligonucleotides in oncology and neurological disorders. Gene Editing accounts for 25%, fueled by rapid advancements in CRISPR-Cas9, TALENs, and ZFNs for precise genome modification.

By Approach

In-Vivo gene therapy dominates with 60% share, supported by direct delivery of vectors into patients and increasing approvals for ocular, neuromuscular, and liver-based therapies. Ex-Vivo approaches hold 40%, driven by their effectiveness in hematological disorders, CAR-T cell therapies, and personalized cancer treatments.

By Application

Oncology leads with 30% share, fueled by strong investment in cancer gene therapies, CAR-T treatments, and immuno-oncology research. Blood Disorders account for 25%, supported by gene therapies for hemophilia, sickle cell disease, and beta-thalassemia. Ophthalmology holds 15%, driven by successful treatments for inherited retinal diseases. Musculoskeletal Conditions represent 15%, supported by applications in muscular dystrophy and orthopedic regenerative therapies. Others account for 15%, including neurological, cardiovascular, and metabolic disorders.

By Region

North America - 38% Share
North America leads with 38%, driven by strong clinical trial activity, favorable regulatory frameworks, and high adoption of advanced gene therapies in the U.S. and Canada.

Europe - 27% Share
Europe holds 27%, supported by robust R&D funding, expanding gene therapy approvals, and strong biotech ecosystems in Germany, the U.K., France, and Italy.

Asia Pacific - 22% Share
Asia Pacific accounts for 22%, fueled by rising healthcare investments, growing biotech manufacturing, and increasing clinical trials in China, Japan, South Korea, and India.

South America - 8% Share
South America holds 8%, driven by improving healthcare infrastructure, research collaborations, and gradual adoption of advanced therapies in Brazil and Argentina.

Middle East & Africa - 5% Share
Middle East & Africa represent 5%, supported by niche adoption, expanding medical research initiatives, and growing investment in advanced healthcare solutions.

FAQ

What is the current size of the Gene Therapy for Rare Disease Market?

A: In 2024, the Gene Therapy for Rare Disease Market was valued at US$ 3.01 Billion , reflecting its strong industry presence.

Q2: How large is the Gene Therapy for Rare Disease Market expected to be by 2033?

A: By 2033, industry forecasts suggest the Gene Therapy for Rare Disease Market will grow to around US$ 24.54 Billion, demonstrating significant expansion.

Q3: What is the growth rate of the Gene Therapy for Rare Disease Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 26.5% during the forecast period from 2025 to 2033.

Unlock 360° Market Intelligence with DataM Subscription Services: https://www.datamintelligence.com/reports-subscription?sb

Power your decisions with real-time competitor tracking, strategic forecasts, and global investment insights all in one place.

✅ Competitive Landscape
✅ Sustainability Impact Analysis
✅ KOL / Stakeholder Insights
✅ Unmet Needs & Positioning, Pricing & Market Access Snapshots
✅ Market Volatility & Emerging Risks Analysis
✅ Quarterly Industry Report Updated
✅ Live Market & Pricing Trends
✅ Import-Export Data Monitoring

Have a look at our Subscription Dashboard: https://www.youtube.com/watch?v=x5oEiqEqTWg?sb

Contact Us -

Company Name: DataM Intelligence
Contact Person: Sai Kiran
Email: Sai.k@datamintelligence.com
Phone: +1 877 441 4866
Website: https://www.datamintelligence.com

About Us -

DataM Intelligence is a Market Research and Consulting firm that provides end-to-end business solutions to organizations from Research to Consulting. We, at DataM Intelligence, leverage our top trademark trends, insights and developments to emancipate swift and astute solutions to clients like you. We encompass a multitude of syndicate reports and customized reports with a robust methodology.

Our research database features countless statistics and in-depth analyses across a wide range of 6300+ reports in 40+ domains creating business solutions for more than 200+ companies across 50+ countries; catering to the key business research needs that influence the growth trajectory of our vast clientele.

This release was published on openPR.