United States Gene Therapy Market to Hit $6510.45 Million by 2033 | Leading companies - Alnylam Pharmaceuticals, Inc., NOVARTIS AG, Sarepta Therapeutics, Inc.

Leander, Texas and Tokyo, Japan - Dec.10.2025
As per DataM intelligence research report" The US Gene Therapy Market Size reached US$ 2,812.92 million in 2024 and is expected to reach US$ 21,701.51 million by 2033, growing at a CAGR of 23.5 % during the forecast period 2025-2033." Advances in viral vectors and precision medicine are driving rapid progress in the US gene therapy market.

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United States: Recent Industry Developments

✅ In November 2025, The FDA approved a new gene therapy for a rare form of inherited retinal disease The one-time treatment restores vision by delivering a functional copy of the defective gene It offers hope to patients facing progressive blindness

✅ In October 2025, Bluebird Bio announced the commercial launch of its sickle cell gene therapy with a novel payment model The outcome-based pricing ensures insurers only pay if the therapy remains effective It addresses the high cost barrier of potentially curative treatments

✅ In September 2025, Vertex Pharmaceuticals expanded its manufacturing center in Boston for cell and gene therapies The facility uses automated processing to increase patient throughput It solves the manufacturing bottleneck for personalized medicines

✅ In August 2025, The NIH launched a "Gene Therapy for All" initiative to fund research on ultra-rare diseases The program provides templates and manufacturing support for "n-of-1" therapies It democratizes access to gene therapy for conditions with very few patients

Japan: Recent Industry Developments

✅ In December 2025, The PMDA approved the first domestically developed CAR-T therapy for leukemia The therapy offers a more affordable alternative to imported CAR-T products It strengthens Japan's national health security in advanced medicine

✅ In November 2025, Astellas Pharma opened a new gene therapy research center in Tsukuba The center focuses on developing AAV vectors with improved tissue targeting It aims to reduce the dosage and side effects of gene therapies

✅ In October 2025, Daiichi Sankyo licensed a novel gene editing technologyfrom a U.S. startup The technology allows for precise correction of mutations without double-strand breaks It expands the Japanese pharma giant's modality beyond ADCs

✅ In September 2025, The Japanese government updated its "Sakigake" designation to include gene therapies The designation grants priority review to innovative therapies developed in Japan It encourages global biotech firms to launch their products in Japan first

US Gene Therapy Market: Drivers
The US gene therapy market is witnessing robust growth driven by breakthroughs in gene editing, viral vector technologies, and personalized medicine. Gene therapies target rare genetic disorders, cancers, and monogenic diseases by delivering functional genes or modifying defective ones. Regulatory approvals and increasing investment in clinical trials are accelerating innovation, with CRISPR and viral vector-based approaches leading development. Integration of advanced manufacturing and quality systems ensures scalability, safety, and efficacy.

Patient-centric approaches, including companion diagnostics and long-term follow-up programs, enhance treatment outcomes. Collaborations between biotech startups, pharma companies, and academic institutions drive pipeline expansion and commercialization. The convergence of digital tools, AI for patient selection, and real-world data analytics further supports clinical development. With rising awareness of gene-based treatments, the US market remains a global leader in gene therapy innovation.

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US Gene Therapy Market: Major Players
Alnylam Pharmaceuticals, Inc., NOVARTIS AG, Sarepta Therapeutics, Inc., Krystal Biotech, Inc., CSL, Bluebird Bio, Inc., SPARK THERAPEUTICS, INC., Ferring, Vertex Pharmaceuticals Incorporated, Amgen, Inc., and Orchard Therapeutics Plc, among others.

Segment Covered in the US Gene Therapy Market:
By Approach
The market is segmented into In-Vivo and Ex Vivo approaches. In-vivo therapy leads adoption as it enables direct delivery into the patient's body for conditions requiring systemic correction, offering faster dosing and broader applicability. Ex vivo therapy remains crucial for personalized, cell-based interventions-especially for hematological disorders-where cells are modified outside the body before reinfusion. Both approaches are expanding due to regulatory support, growing clinical pipelines, and advancements in delivery technologies. In the U.S., in-vivo therapies are gaining traction due to streamlined administration and scalability.

By Vector Type
Vector types include Viral Vectors and Non-Viral Vectors, with viral vectors dominating due to their high transduction efficiency and established safety profiles in approved therapies. AAV, lentiviral, and adenoviral vectors remain core platforms for gene delivery in rare genetic disorders, oncology, and ophthalmology. Non-viral vectors-such as lipid nanoparticles (LNPs), polymer-based systems, and physical delivery methods-are rapidly emerging, driven by reduced immunogenicity, lower manufacturing cost, and compatibility with gene editing tools. Growing FDA approvals and funding continue to expand vector innovation in the U.S.

By Technique
Techniques include Gene Addition, Gene Silencing, and Gene Editing, each serving distinct therapeutic needs. Gene addition is widely used to introduce functional genes in cases of genetic loss-of-function, forming the backbone of many U.S. approvals. Gene silencing leverages RNA-based and regulatory mechanisms to suppress harmful gene expression, increasingly important in neuromuscular and metabolic disorders. Gene editing CRISPR, TALENs, base editing, and prime editing-is the fastest-growing segment, enabling precise correction of genetic mutations. Advances in delivery and high specificity continue to accelerate clinical adoption across these modalities.

By Application
Applications span Rare Diseases, Musculoskeletal Conditions, Blood Disorders, Oncology, Ophthalmology, and Others, with rare diseases representing the majority of approved therapies due to strong unmet need and regulatory incentives. Blood disorders, particularly hemophilia and sickle cell disease, show high clinical activity. Oncology remains a major growth driver with expanding gene-modified cell therapies. Ophthalmology benefits from localized, immune-privileged environments that support durable therapeutic effects. Musculoskeletal and broader chronic conditions are emerging areas as delivery technology and editing precision improve.

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