PUBLISHER: The Business Research Company | PRODUCT CODE: 1955385
PUBLISHER: The Business Research Company | PRODUCT CODE: 1955385
Gene therapy for inherited genetic disorders is a medical approach that addresses the underlying cause of hereditary diseases by repairing or replacing defective genes. Its primary objective is to offer a potential cure rather than simply managing symptoms. This therapy introduces functional genes into a patient's cells, restores normal cellular function, and improves overall health. It holds significant promise for treating rare genetic disorders, providing the potential for long-lasting or even permanent therapeutic effects.
The main types of gene therapy for inherited genetic disorders include viral vector-based therapies, gene editing techniques, ribonucleic acid (RNA)-based therapies, and ex vivo gene therapy. Viral vector-based therapies employ genetically engineered, non-replicating viruses to deliver therapeutic genetic material into patient cells for disease treatment. These therapies are used in a range of applications, including inherited metabolic disorders, neurological disorders, cardiovascular disorders, musculoskeletal disorders, and ophthalmic disorders, and are utilized by various end users such as hospitals, specialty clinics, research institutes, and others.
Tariffs have impacted the gene therapy for inherited genetic disorders market by increasing costs of imported viral vectors, gene editing reagents, and stem cell therapy components. Segments like viral vector-based therapies and RNA-based therapeutics are most affected, particularly in North America, Europe, and Asia-Pacific regions such as China and India. These tariffs have led to higher therapy costs and potential delays in clinical trials. On the positive side, tariffs have encouraged domestic production, innovation in cost-effective gene therapy techniques, and local R&D investments.
The gene therapy for inherited genetic disorders market research report is one of a series of new reports from The Business Research Company that provides gene therapy for inherited genetic disorders market statistics, including gene therapy for inherited genetic disorders industry global market size, regional shares, competitors with a gene therapy for inherited genetic disorders market share, detailed gene therapy for inherited genetic disorders market segments, market trends and opportunities, and any further data you may need to thrive in the gene therapy for inherited genetic disorders industry. This gene therapy for inherited genetic disorders market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
The gene therapy for inherited genetic disorders market size has grown rapidly in recent years. It will grow from $4.05 billion in 2025 to $4.84 billion in 2026 at a compound annual growth rate (CAGR) of 19.3%. The growth in the historic period can be attributed to limited availability of advanced gene therapy techniques, reliance on conventional treatments for genetic disorders, increasing prevalence of inherited genetic diseases, early clinical research on viral vectors, rising awareness of rare disease management.
The gene therapy for inherited genetic disorders market size is expected to see rapid growth in the next few years. It will grow to $9.69 billion in 2030 at a compound annual growth rate (CAGR) of 19.0%. The growth in the forecast period can be attributed to development of novel viral vectors and gene editing technologies, expansion of rna-based therapies, increasing adoption of ex vivo stem cell therapies, rising investment in personalized gene therapies, growth in hospital and specialty clinic capabilities for gene therapy administration. Major trends in the forecast period include expansion of viral vector-based therapies for rare genetic disorders, growth in ex vivo gene therapy applications, increasing adoption of rna-based therapeutics, rising focus on treating neurological and ophthalmic disorders, integration of personalized medicine in gene therapy.
The growing demand for personalized medicine is expected to drive the expansion of the gene therapy for inherited genetic disorders market in the coming years. Personalized medicine involves tailoring medical treatment to an individual's genetic profile and personal characteristics to optimize care. Its adoption is increasing due to advancements in genetic technologies, which make it easier and more cost-effective to customize treatments to each patient's unique genetic makeup. Personalized medicine enhances gene therapy by enabling precise gene targeting and delivery, improving treatment efficacy while minimizing side effects. For example, according to the Personalized Medicine Coalition in February 2024, the U.S. Food and Drug Administration (FDA) approved 16 new personalized treatments for rare disease patients in 2023, up from six in 2022. These approvals included seven cancer drugs and three therapies for other diseases and conditions. This rising demand for personalized treatments is therefore supporting the growth of the gene therapy for inherited genetic disorders market.
Key companies in this market are focusing on innovative approaches, such as ex vivo CRISPR/Cas9-based gene-edited cell therapies, to meet the demand for potentially curative, one-time treatments that reduce the need for lifelong transfusions, repeated hospitalizations, and high chronic-care costs in inherited blood and metabolic disorders. Ex vivo CRISPR/Cas9 therapy involves harvesting a patient's hematopoietic stem cells, using CRISPR/Cas9 to precisely edit disease-causing genes (or regulatory elements that increase protective fetal hemoglobin), and reinfusing the modified cells to provide long-term therapeutic benefits. For instance, in December 2023, Vertex Pharmaceuticals, a U.S.-based biopharmaceutical company, received FDA approval for CASGEVY (exagamglogene autotemcel), a CRISPR/Cas9-based ex vivo gene-edited cell therapy for sickle cell disease. CASGEVY is derived from a patient's own hematopoietic stem cells, which are edited to increase fetal hemoglobin levels. The therapy features precise genome editing performed ex vivo, a one-time administration, and durable effects. Its primary application is treating severe sickle cell disease, and clinical trials have shown significant reductions in vaso-occlusive crises and transfusion dependence compared with previous standard-of-care treatments.
In October 2024, Ocuphire Pharma Inc., a U.S.-based biotechnology company, acquired Opus Genetics Inc. for an undisclosed sum. Through this acquisition, Ocuphire Pharma aims to strengthen its leadership in developing gene therapies for rare inherited retinal diseases by combining Opus's innovative pipeline with its own development expertise. Opus Genetics Inc. is a U.S.-based biotechnology company specializing in gene therapies for inherited genetic disorders.
Major companies operating in the gene therapy for inherited genetic disorders market are Sanofi S.A., Novartis AG, Astellas Pharma Inc., Vertex Pharmaceuticals Incorporated, Genentech, Biomarin Pharmaceutical Inc, Sarepta Therapeutics Inc., Krystal Biotech Inc., Rocket Pharmaceuticals Inc., Sangamo Therapeutics Inc., REGENXBIO Inc., Passage Bio Inc., Voyager Therapeutics Inc., Generation Bio Co., Avrobio Inc, Editas Medicine Inc., MeiraGTx Holdings Plc, uniQure N.V., Intellia Therapeutics Inc., Bluebird Bio Inc, 4D Molecular Therapeutics Inc., CRISPR Therapeutics AG
North America was the largest region in the gene therapy for inherited genetic disorders market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the gene therapy for inherited genetic disorders market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
The countries covered in the gene therapy for inherited genetic disorders market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The gene therapy for inherited genetic disorders market consists of revenues earned by entities by providing services such as genetic testing and diagnosis, personalized treatment planning, and gene delivery and administration. The market value includes the value of related goods sold by the service provider or included within the service offering. The gene therapy for inherited genetic disorders market also includes sales of zolgensma, luxturna, and strimvelis. Values in this market are 'factory gate' values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
Gene Therapy For Inherited Genetic Disorders Market Global Report 2026 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses gene therapy for inherited genetic disorders market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
Where is the largest and fastest growing market for gene therapy for inherited genetic disorders ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward, including technological disruption, regulatory shifts, and changing consumer preferences? The gene therapy for inherited genetic disorders market global report from the Business Research Company answers all these questions and many more.
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, total addressable market (TAM), market attractiveness score (MAS), competitive landscape, market shares, company scoring matrix, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.
Added Benefits available all on all list-price licence purchases, to be claimed at time of purchase. Customisations within report scope and limited to 20% of content and consultant support time limited to 8 hours.