Gene Therapy Market to Reach US$ 35.91 Billion by 2033 | CAGR 23.1% | North America Leads with 45% Share | Key Players: Novartis, Vertex, Bluebird Bio, Alnylam, Spark Therapeutics, Pfizer

Market Overview

The Global Gene Therapy Market reached US$ 4.80 billion in 2024 and is projected to grow to US$ 35.91 billion by 2033, expanding at a CAGR of 23.1% during the forecast period 2025-2033. The market is witnessing rapid growth, driven by advancements in genomics, biotechnology, and personalized medicine, and is transforming the treatment landscape for genetic disorders, rare diseases, and cancer. Significant R&D investments, strategic partnerships, and regulatory approvals have fueled market expansion globally. For example, in January 2024, Vertex Pharmaceuticals received Saudi Food and Drug Authority (SFDA) marketing authorization for CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in patients aged 12 years and older.

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Gene therapy is also revolutionizing oncology, with therapies targeting various cancers. In January 2023, Ferring Pharmaceuticals' ADSTILADRIN (nadofaragene firadenovec-vncg) became fully available across the U.S. for adult patients with high-risk BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Approved by the FDA, ADSTILADRIN is the first and only intravesical gene therapy for adults with NMIBC who no longer respond to standard therapies, underscoring the market's expansion into innovative cancer treatments.

Recent Developments

✅ January 2026: Bluebird Bio announced expanded clinical trials for its gene therapy candidates targeting beta-thalassemia and sickle cell disease, aiming to increase patient enrollment in North America and Europe and accelerate regulatory approvals.

✅ December 2025: Novartis received FDA breakthrough therapy designation for its CAR-T gene therapy in multiple myeloma, highlighting advancements in oncology-focused gene therapy products.

✅ November 2025: REGENXBIO partnered with a major pharmaceutical company to co-develop AAV-based gene therapies for rare retinal disorders, improving access to advanced therapies for underserved patient populations.

✅ September 2025: Pfizer initiated Phase III trials for its gene therapy targeting hemophilia B, demonstrating strong efficacy and safety in early-stage studies and preparing for global regulatory submissions.

✅ June 2025: Sangamo Therapeutics launched an in vivo gene-editing program using zinc finger nuclease technology for treating metabolic disorders, expanding its pipeline in rare genetic diseases.

Mergers & Acquisitions

✅ January 2026: Novartis acquired a gene therapy-focused biotech startup specializing in AAV-based delivery systems, enhancing its pipeline for rare genetic disorders and strengthening its global manufacturing capabilities.

✅ December 2025: Vertex Pharmaceuticals completed the acquisition of a CRISPR/Cas9 gene-editing company, expanding its capabilities in developing treatments for sickle cell disease and transfusion-dependent beta-thalassemia.

✅ November 2025: Pfizer partnered with and later acquired a gene therapy company targeting hemophilia, integrating in vivo gene-editing technology into its rare disease portfolio.

✅ September 2025: Roche acquired a cell and gene therapy services firm, boosting its research, manufacturing, and regulatory support for oncology-focused gene therapies.

✅ July 2025: Sangamo Therapeutics entered a strategic merger with a clinical-stage gene therapy company to accelerate development of treatments for metabolic and rare genetic disorders.

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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.

Key Highlights:

• Alnylam Pharmaceuticals, Inc. - Holds an estimated 12% share, driven by its RNAi-based gene therapies and strong pipeline for rare genetic diseases.

• Spark Therapeutics, Inc. - Estimated 10% share, specializing in AAV-based gene therapies for inherited retinal disorders and hemophilia.

• Novartis AG - Holds around 14% share, fueled by its CAR-T and gene therapy platforms targeting oncology and rare genetic disorders.

• bluebird bio, Inc. - Estimated 11% share, supported by ex vivo gene therapies for sickle cell disease, beta-thalassemia, and oncology applications.

• Ferring Pharmaceuticals Inc. - Holds approximately 6% share, focusing on gene therapy solutions for urological and gastrointestinal disorders.

• Vertex Pharmaceuticals Incorporated - Estimated 13% share, driven by CRISPR/Cas9 therapies for sickle cell disease and transfusion-dependent beta-thalassemia.

• Sarepta Therapeutics, Inc. - Holds around 8% share, specializing in gene therapies for muscular dystrophies.

• CSL Behring LLC - Estimated 5% share, providing gene therapy solutions in rare hematology and genetic disorders.

• Amgen, Inc. - Holds approximately 7% share, leveraging its biotechnology expertise for oncology and rare disease gene therapies.

• Orchard Therapeutics Group - Estimated 6% share, focused on ex vivo gene therapies for rare pediatric disorders.

• Krystal Biotech, Inc. - Holds around 4% share, developing topical and systemic gene therapy products for rare skin disorders.

Market Segmentation:

➥By approach, In-Vivo therapies dominate the market with an estimated 60% share, driven by their ability to directly deliver therapeutic genes into patients' cells with minimal invasiveness. Ex-Vivo therapies account for approximately 40%, widely used in CAR-T and hematopoietic stem cell-based gene therapies where cells are modified outside the body before reinfusion.

➥By vector type, Viral Vectors hold the largest share at 70%, owing to their high transfection efficiency and proven track record in AAV, lentivirus, and retrovirus-based therapies. Non-Viral Vectors account for roughly 30%, preferred in applications requiring reduced immunogenicity and simpler manufacturing processes, such as lipid nanoparticles and plasmid DNA delivery systems.

➥By technique, Gene Addition leads with an estimated 45% share, used extensively in therapies that replace or supplement missing or defective genes. Gene Silencing accounts for around 25%, particularly in RNAi-based therapies targeting overexpressed or harmful genes. Gene Editing holds approximately 30%, driven by CRISPR/Cas9, TALEN, and zinc finger nuclease technologies that allow precise modifications of the genome for rare genetic disorders and oncology applications.

➥By application, Rare Diseases dominate with 40%, including inherited metabolic and hematological disorders. Oncology accounts for roughly 25%, reflecting the growing use of CAR-T and targeted gene therapies. Blood Disorders such as hemophilia and sickle cell disease contribute around 20%, while Musculoskeletal Conditions and Other applications make up the remaining 15%, driven by research in regenerative medicine and emerging gene therapies for chronic conditions.

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Regional Insights:

North America leads the market with an estimated 45% share, driven by strong R&D infrastructure, early adoption of advanced gene therapies, and supportive regulatory frameworks in the U.S. and Canada.

Europe accounts for approximately 25%, fueled by increasing approvals for gene therapies, strong pharmaceutical and biotechnology presence, and government incentives across countries such as Germany, the U.K., and France.

The Asia-Pacific region holds around 20%, supported by rising investments in biotech research, growing awareness of genetic disorders, and expanding healthcare infrastructure in countries like China, Japan, and India.

Market Dynamics:

The expansion into rare diseases is a key driver of growth in the global gene therapy market. Rare diseases, often genetic in origin, have limited or no effective treatment options, creating substantial opportunities for gene therapies to provide curative solutions where traditional treatments fail. For example, Luxturna treats inherited vision loss and is the first directly administered gene therapy approved in the U.S. for a disease caused by mutations in a specific gene. Similarly, Zolgensma, used for Spinal Muscular Atrophy (SMA), offers a one-time treatment that significantly improves survival and motor function in children. Hemophilia also benefits from gene therapy; in February 2025, CSL Behring reported four-year results from the pivotal HOPE-B study, confirming the long-term safety and durability of HEMGENIX (etranacogene dezaparvovec-drlb) for adults with hemophilia B. As more gene therapies for rare diseases gain approval, the market is expected to diversify further, addressing a broader range of conditions.

However, high costs associated with gene therapies pose a significant restraint on market growth. Many gene therapies are priced in the millions, including Zynteglo and Skysona at US$ 2.8 million and US$ 3 million per dose, and Zolgensma at US$ 2.1 million. The lengthy and expensive development process, extensive clinical trials, and regulatory approvals contribute to these high prices. For instance, Luxturna took over 10 years to develop and costs approximately $850,000 per patient. Additionally, many therapies are personalized, requiring bespoke production tailored to a patient's genetic makeup. CAR-T therapies such as Yescarta and Kymriah involve complex modification of a patient's own T-cells and can cost $300,000-$400,000 per patient. This combination of high development costs, individualized treatment processes, and premium pricing limits widespread accessibility and adoption, constraining overall market growth.

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