Gene Therapy for Inherited Genetic Disorders Market Report 2025

The gene therapy for inherited genetic disorders market size has grown rapidly in recent years. It will grow from $3.39 billion in 2024 to $4.05 billion in 2025 at a compound annual growth rate (CAGR) of 19.4%. The growth in the historic period can be attributed to rising government initiatives and supportive policies for gene therapy, expanding availability of advanced gene delivery systems, increasing patient demand for effective and curative treatments, the growth of clinical trials targeting inherited disorders, and heightened awareness among healthcare professionals about the benefits of gene therapy.

The gene therapy for inherited genetic disorders market size is expected to see rapid growth in the next few years. It will grow to $8.15 billion in 2029 at a compound annual growth rate (CAGR) of 19.1%. The growth in the forecast period can be attributed to the rising prevalence of inherited genetic disorders, increasing investment and funding in gene therapy research and development, greater awareness and diagnosis of rare genetic diseases, growing regulatory approvals for novel gene therapy products, and expanding adoption of personalized medicine approaches. Major trends in the forecast period include the integration of advanced delivery systems for targeted gene therapy, innovations in viral and non-viral vector technologies, advancements in next-generation sequencing for precise diagnosis, improvements in manufacturing and scalability of gene therapies, and the use of artificial intelligence to optimize gene therapy design.

The rising demand for personalized medicine is anticipated to drive the growth of the gene therapy for inherited genetic disorders market in the coming years. Personalized medicine involves tailoring medical treatments to an individual’s genetic profile and personal characteristics to optimize care. This approach is gaining momentum due to advancements in genetic technologies, which make it easier and more cost-effective to customize treatments according to a person’s unique genetic makeup. Personalized medicine enhances gene therapy by enabling precise gene targeting and delivery, improving treatment effectiveness, and reducing side effects. For example, in February 2024, the Personalized Medicine Coalition, a US-based non-profit organization, reported that the US Food and Drug Administration (FDA) approved 16 new personalized treatments for rare disease patients in 2023, up from six in 2022. These newly approved treatments included seven cancer drugs and three for other diseases and conditions. Consequently, the growing demand for personalized medicine is fueling the expansion of the gene therapy for inherited genetic disorders market.

Leading companies in the gene therapy for inherited genetic disorders market are focusing on developing advanced solutions, such as gene therapy establishments, to create targeted, safer, and more effective treatments that address the root causes of genetic diseases. A gene therapy establishment is a biotechnology or pharmaceutical company specializing in researching, developing, and commercializing gene therapies aimed at correcting or modifying defective genes responsible for inherited genetic disorders. For instance, in November 2022, Replay, a US-based genome-writing company, launched Telaria, a major DNA HSV gene therapy company. Telaria is the second product company utilizing Replay’s high-payload herpes simplex virus (HSV) delivery vector, synHSV, with its lead indication targeting recessive dystrophic epidermolysis bullosa (RDEB), a severe genetic skin disorder. The company was co-founded by leading HSV scientists and genetic skin disease experts, aiming to develop transformative gene therapies for rare skin conditions. This launch followed Replay’s first gene therapy company, Eudora, which focuses on retinal diseases. Replay’s innovative structure separates technology development from therapeutic development within distinct product companies focused on specific disease areas.

In October 2024, Ocuphire Pharma Inc., a US-based biotechnology company, acquired Opus Genetics Inc. for an undisclosed amount. Through this acquisition, Ocuphire Pharma aims to become a leader in developing and advancing gene therapies for rare inherited retinal diseases by combining Opus’s innovative pipeline with its own development expertise. Opus Genetics Inc., also a US-based biotechnology company, specializes in gene therapy for inherited genetic disorders.

Major players 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 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in gene therapy for inherited genetic disorders market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the gene therapy for inherited genetic disorders market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the pharmaceutical companies contend with tariffs on APIs, glass vials, and lab equipment inputs with few alternative sources. Generic drug makers, operating on razor-thin margins, are especially vulnerable, with some reducing production of low-profit medicines. Biotech firms face delays in clinical trials due to tariff-related shortages of specialized reagents. In response, the industry is expanding API production in India and Europe, increasing inventory stockpiles, and pushing for trade exemptions for essential medicines.

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 provide a potential cure rather than merely managing symptoms. By introducing functional genes into a patient’s cells, it restores normal cellular function and improves overall health. This strategy holds significant potential for treating rare genetic disorders, offering the possibility of long-lasting or permanent therapeutic benefits.

The primary types of gene therapy for inherited genetic disorders include viral vector-based therapies, gene editing techniques, RNA-based therapies, and ex vivo gene therapy. Viral vector-based therapies employ genetically modified, non-replicating viruses to deliver therapeutic genetic material into patient cells to treat diseases. These therapies are utilized in various applications, including inherited metabolic disorders, neurological disorders, cardiovascular disorders, musculoskeletal disorders, and ophthalmic disorders, and serve multiple end users such as hospitals, specialty clinics, research institutions, and others.

The gene therapy for inherited genetic disorders market research report is one of a series of new reports 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 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.

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