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Gene Therapy For Inherited Genetic Disorders Market Landscape 2025: Forecast Data and Growth Strategy Insights

09-05-2025 07:59 AM CET | Health & Medicine

Press release from: The Business research company

Gene Therapy For Inherited Genetic Disorders Market

Gene Therapy For Inherited Genetic Disorders Market

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What Is the Expected CAGR for the Gene Therapy For Inherited Genetic Disorders Market Through 2025?
In recent years, there has been swift growth in the market size for gene therapy for inherited genetic disorders. An increase from $3.39 billion in 2024 to $4.05 billion in 2025, corresponding to a compound annual growth rate (CAGR) of 19.4% is projected. Factors contributing to the growth during this historic period include enhanced government initiatives and policies encouraging gene therapy, expanded availability of advanced gene delivery systems, increased demand from patients for potent and curative treatments, and rising execution of clinical trials aimed at inherited disorders. Additionally, there's an uptick in awareness about the benefits of gene therapy among healthcare professionals.

What's the Projected Size of the Global Gene Therapy For Inherited Genetic Disorders Market by 2029?
There is an anticipated rapid expansion in the market size for gene therapy for inherited genetic disorders in the forthcoming years. The market is projected to reach $8.15 billion by 2029, with a compound annual growth rate (CAGR) of 19.1%. This predicted growth in the forecast period is due to factors such as the rising incidences of inherited genetic disorders, increased investment and funding in gene therapy research, improved awareness and diagnosis of rare genetic diseases, increasing endorsements of new gene therapy products by regulatory bodies, and the expanding use of personalized medicine. The impending period will witness key trends such as the incorporation of state-of-the-art delivery systems for targeted gene therapy, evolution in viral and non-viral vector technologies, advancements in next-generation sequencing for accurate diagnosis, technological upgrades in the production and scalability of gene therapies, and the application of artificial intelligence to refine gene therapy design.

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Top Growth Drivers in the Gene Therapy For Inherited Genetic Disorders Industry: What's Accelerating the Market?
The growth of the gene therapy market for inherited genetic disorders is anticipated to be fueled by the increasing demand for personalized medicine. Personalized medicine is the process of adjusting medical treatments according to an individual's genetic and personal traits to fine-tune care. The surge in personalized medicine can be attributed to advancements in genetic technology, which have made it simpler and more cost-effective to tailor treatments to the unique genetic structures of individuals. By personalizing treatments to each patient's specific genetic profile, gene therapy is enhanced, which ensures exact gene targeting and delivery, amplifies effectiveness, and diminishes side effects. For instance, in February 2024, the Personalized Medicine Coalition, a non-profit organization in the US, reported that in 2023, the US Food and Drug Administration (FDA) approved 16 new personalized treatments for rare disease patients, an increase from six in 2022. The approved personalized treatments for 2023 also encompass seven drugs for cancer and three for other diseases and conditions. Thus, the escalating demand for personalized medicine is promoting the expansion of the gene therapy market for inherited genetic disorders.

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Which Emerging Trends Are Transforming the Gene Therapy For Inherited Genetic Disorders Market in 2025?
Leading businesses in the market for gene therapy for inherited gene disorders are working toward creating evolved solutions, including the establishment of gene therapy firms, aimed at developing targeted, safer and more potent treatments targeting the fundamental causes of gene diseases. In the context of gene therapy establishment, we refer to bioscience or pharmaceutical firms that devote their work to the research, development, and commercialization of gene therapy treatments intended to correct or edit faulty genes causing genetic disorders. For example, in November 2022, a US-based firm focusing on genome writing, Replay, introduced Telaria, a large DNA HSV gene therapy company. This is the second firm to take advantage of its high delivery capacity herpes simplex virus (HSV) vector, synHSV, with its main focus being recessive dystrophic epidermolysis bullosa (RDEB), a severe inherited skin disorder. Replay, established by leading HSV scientists and genetic skin disease experts, aspires to devise transformative gene therapies for such rare skin ailments. The company's previous venture into gene therapy was Eudora, focusing on retinal disorders. Replay's innovative model segregates technology evolution from therapy development, with separate product companies emphasizing on specific disease areas.

What Are the Main Segments in the Gene Therapy For Inherited Genetic Disorders Market?
The gene therapy for inherited genetic disorders market covered in this report is segmented

1) By Type: Viral Vector-Based Therapies, Gene Editing Techniques, Ribonucleic Acid-Based Therapies, Ex Vivo Gene Therapy
2) By Application: Inherited Metabolic Disorders, Neurological Disorders, Cardiovascular Disorders, Musculoskeletal Disorders, Ophthalmic Disorders
3) By End-User: Hospitals, Specialty Clinics, Research Institutes, Other End-Users

Subsegments:
1) By Viral Vector-Based Therapies: Adenovirus Vectors, Adeno-Associated Virus (AAV) Vectors, Lentivirus Vectors
2) By Gene Editing Techniques: Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs), Meganucleases
3) By Ribonucleic Acid-Based Therapies: Small Interfering Ribonucleic Acid, Antisense Oligonucleotides (ASOs), Messenger Ribonucleic Acid (mRNA) Therapies
4) By Ex Vivo Gene Therapy: Hematopoietic Stem Cell Gene Therapy, Mesenchymal Stem Cell Gene Therapy, Induced Pluripotent Stem Cell (iPSC) Therapy

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Which Top Companies are Driving Growth in the Gene Therapy For Inherited Genetic Disorders Market?
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.

Which Regions Will Dominate the Gene Therapy For Inherited Genetic Disorders Market Through 2029?
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 the gene therapy for inherited genetic disorders market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

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4.Consultants & Analysts - To support market entry, expansion strategies, and client advisory work.

Learn More About The Business Research Company
With over 15000+ reports from 27 industries covering 60+ geographies, The Business Research Company has built a reputation for offering comprehensive, data-rich research and insights. Armed with 1,500,000 datasets, the optimistic contribution of in-depth secondary research, and unique insights from industry leaders, you can get the information you need to stay ahead.
Our flagship product, the Global Market Model (GMM), is a premier market intelligence platform delivering comprehensive and updated forecasts to support informed decision-making.

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Learn More About The Business Research Company
With over 15000+ reports from 27 industries covering 60+ geographies, The Business Research Company has built a reputation for offering comprehensive, data-rich research and insights. Armed with 1,500,000 datasets, the optimistic contribution of in-depth secondary research, and unique insights from industry leaders, you can get the information you need to stay ahead.
Our flagship product, the Global Market Model (GMM), is a premier market intelligence platform delivering comprehensive and updated forecasts to support informed decision-making.

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