Gene Therapy - Global Strategic Business Report

The global market for Gene Therapy was valued at US$1.7 Billion in 2024 and is projected to reach US$6.0 Billion by 2030, growing at a CAGR of 23.0% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions. The report includes the most recent global tariff developments and how they impact the Gene Therapy market.

Global Gene Therapy Market - Key Trends & Drivers Summarized

Revolutionizing Medicine: How Gene Therapy Is Redefining Treatment Paradigms?

Gene therapy represents a groundbreaking medical approach that involves modifying the genetic material within a person's cells to treat or prevent disease. This innovative therapy can potentially provide a cure for a range of disorders by correcting defective genes responsible for disease development. Techniques used in gene therapy include replacing a mutated gene that causes disease with a healthy copy, inactivating a mutated gene that is functioning improperly, or introducing a new or modified gene into the body to help treat a disease. These methods are primarily delivered through vectors, typically viruses that have been genetically altered to carry human DNA. The potential of gene therapy extends from treating hereditary diseases like cystic fibrosis and hemophilia to more complex conditions such as cancer, heart disease, and HIV/AIDS. Its development represents a significant leap from traditional treatments, offering hope for long-term or permanent therapeutic effects after a single treatment course.

Pushing the Boundaries: Next-Generation Techniques Enhancing Gene Therapy

Technological advancements have significantly improved the precision and efficacy of gene therapy. The advent of CRISPR-Cas9 and other gene-editing technologies has provided scientists with tools of unprecedented accuracy for editing DNA at specific locations, allowing for the correction of mutations at their source. These advancements not only increase the potential for successful treatment outcomes but also reduce the risks associated with older gene therapy methods, such as unintended effects on other parts of the genome. Additionally, innovations in vector technology, particularly in the development of non-viral delivery systems, are overcoming previous challenges related to safety and immune response management. These enhancements in delivery systems ensure that therapeutic genes reach the target cells more effectively and work as intended, which is critical for the treatment of diseases that require cellular-level interventions.

Navigating Complexities: The Challenges and Ethical Dilemmas of Modern Gene Therapy

Despite its promising potential, gene therapy faces several challenges and ethical considerations that influence its clinical application. One of the primary challenges is ensuring long-term safety and efficacy, as the long-term effects of gene editing are still not fully understood. Questions remain about the potential for off-target effects, where unintended parts of the genome may be edited, leading to unforeseen consequences. Additionally, the immune system's response to viral vectors can complicate delivery and effectiveness, particularly for patients who might have pre-existing immunity to the viruses used as vectors. Ethical concerns also play a significant role, particularly regarding germline editing, which could allow genetic changes to be passed down to future generations. The debate centers on the implications of making genetic modifications that could alter human evolution, underscoring the need for rigorous ethical standards and regulatory oversight in the development and application of gene therapy treatments.

Fueling a Medical Revolution: Key Drivers Behind the Expanding Gene Therapy Market

The growth in the gene therapy market is driven by several factors, including significant advancements in genetic research and biotechnological techniques. The increasing prevalence of genetic disorders and high unmet need for novel treatments in oncology, neurology, and rare diseases are major market drivers. The successful commercialization of several gene therapies in recent years has demonstrated the potential for substantial returns on investment, attracting more funding for research and development in this field. Additionally, regulatory incentives, such as orphan drug designations and accelerated approvals, encourage the development of gene therapies for rare and severe conditions. Consumer behavior, particularly the growing demand for personalized medicine and treatments that offer durable to permanent cures, also propels the market forward. Moreover, collaborations between biotech firms, pharmaceutical companies, and academic institutions are fostering innovation and speeding the translation of gene therapy from research to clinical settings. These factors, combined with the increasing global capacity for biopharmaceutical manufacturing, ensure continued growth and expansion of gene therapy solutions, making it one of the most dynamic and transformative fields in modern medicine.

Report Scope

The report analyzes the Gene Therapy market, presented in terms of units. The analysis covers the key segments and geographic regions outlined below.

Segments: Vector Type (Viral, Non-Viral); Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Diseases, Other Applications).

Geographic Regions/Countries: World; USA; Canada; Japan; China; Europe; France; Germany; Italy; UK; Rest of Europe; Asia-Pacific; Rest of World.

Key Insights:

• Market Growth: Understand the significant growth trajectory of the Viral segment, which is expected to reach US$5.5 Billion by 2030 with a CAGR of a 23.3%. The Non-Viral segment is also set to grow at 20.8% CAGR over the analysis period.
• Regional Analysis: Gain insights into the U.S. market, valued at $928.9 Million in 2024, and China, forecasted to grow at an impressive 29.5% CAGR to reach $216.3 Million by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific.

Why You Should Buy This Report:

• Detailed Market Analysis: Access a thorough analysis of the Global Gene Therapy Market, covering all major geographic regions and market segments.
• Competitive Insights: Get an overview of the competitive landscape, including the market presence of major players across different geographies.
• Future Trends and Drivers: Understand the key trends and drivers shaping the future of the Global Gene Therapy Market.
• Actionable Insights: Benefit from actionable insights that can help you identify new revenue opportunities and make strategic business decisions.

Key Questions Answered:

• How is the Global Gene Therapy Market expected to evolve by 2030?
• What are the main drivers and restraints affecting the market?
• Which market segments will grow the most over the forecast period?
• How will market shares for different regions and segments change by 2030?
• Who are the leading players in the market, and what are their prospects?

Report Features:

• Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030.
• In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa.
• Company Profiles: Coverage of players such as Amgen, Inc., Alnylam Pharmaceuticals, Inc., Bionomics Ltd., BioMarin Pharmaceutical, Inc., AnGes, Inc. and more.
• Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments.

Some of the 154 companies featured in this Gene Therapy market report include:

• Amgen, Inc.
• Alnylam Pharmaceuticals, Inc.
• Bionomics Ltd.
• BioMarin Pharmaceutical, Inc.
• AnGes, Inc.
• Amicus Therapeutics, Inc.
• Arrowhead Pharmaceuticals, Inc.
• bluebird bio, Inc.
• Advantagene, Inc.
• Biogen, Inc.
• Abeona Therapeutics
• Aptah
• Avrobio
• Arbor Biotechnologies, Inc.
• Biocardia, Inc.

Tariff Impact Analysis: Key Insights for 2025

Global tariff negotiations across 180+ countries are reshaping supply chains, costs, and competitiveness. This report reflects the latest developments as of April 2025 and incorporates forward-looking insights into the market outlook.

The analysts continuously track trade developments worldwide, drawing insights from leading global economists and over 200 industry and policy institutions, including think tanks, trade organizations, and national economic advisory bodies. This intelligence is integrated into forecasting models to provide timely, data-driven analysis of emerging risks and opportunities.

What’s Included in This Edition:

• Tariff-adjusted market forecasts by region and segment
• Analysis of cost and supply chain implications by sourcing and trade exposure
• Strategic insights into geographic shifts

Buyers receive a free July 2025 update with:

• Finalized tariff impacts and new trade agreement effects
• Updated projections reflecting global sourcing and cost shifts
• Expanded country-specific coverage across the industry