The growing focus on real-world evidence has led to the systematic collection of longitudinal patient outcomes, with at least 10 established disease registries contributing valuable data. These registries track patient progress over extended periods, providing insights into the long-term effectiveness and safety of gene therapies beyond the controlled conditions of clinical trials. This emphasis on real-world data strengthens the credibility of gene therapy, demonstrating its practical benefits and helping inform clinical decision-making in everyday healthcare settings.

Technological Advances Revolutionizing Gene Therapy

The field of gene therapy continues to undergo a profound transformation driven by rapid technological advances, especially in genome-editing tools. These cutting-edge technologies enable the precise and specific correction of genetic mutations, addressing the root causes of many inherited and acquired diseases. By targeting the genome with unprecedented accuracy, genome-editing tools such as CRISPR-Cas systems have shifted gene therapy from a concept-based approach to one with tangible therapeutic potential.

The revolution in gene therapy is further accelerated by the integration of artificial intelligence (AI), bioinformatics, and high-throughput sequencing technologies. AI algorithms enhance vector design by predicting the most effective and safe ways to deliver gene-editing components to target cells. Meanwhile, bioinformatics tools analyze vast datasets to improve patient stratification, helping identify which patients are most likely to benefit from specific gene therapies. High-throughput sequencing allows for rapid and comprehensive assessment of genetic variations and editing outcomes, ensuring precision and minimizing off-target effects.

These technological innovations are collectively pushing gene therapy toward greater precision, durability, and scalability. Researchers are no longer limited to using genome-editing technologies as mere proof-of-concept demonstrations; instead, they are evolving into practical, long-lasting treatments that can be manufactured at scale. This progress promises to expand the range of treatable genetic disorders and improve patient outcomes by delivering more reliable and accessible gene correction therapies.

Gene Slicing Emerges as the Leading Segment in Gene Therapy

Gene slicing techniques, particularly CRISPR-Cas9 and RNA interference (RNAi)-based approaches, have surged ahead to become the most prominent segment in the gene therapy market, commanding over 45% of the market share. This rise is largely due to their exceptional precision and versatility in targeting and editing genetic material. These technologies allow for highly specific modifications at the DNA or RNA level, making them powerful tools for treating a wide range of genetic disorders with potentially fewer off-target effects compared to earlier methods.

As of 2024, more than 90 CRISPR-based gene editing programs are in various stages of clinical development worldwide, underscoring the technology’s strong and growing foothold in the market. The momentum continued in 2023 when the U.S. Food and Drug Administration (FDA) approved four new Investigational New Drug (IND) applications involving gene slicing techniques targeted at rare genetic disorders. This regulatory progress highlights increasing confidence in the safety and therapeutic potential of gene slicing approaches.

Several biotech companies reported important clinical advances in 2023. Editas Medicine shared promising interim results from its EDIT-101 trial, which enrolled 23 patients with Leber Congenital Amaurosis, a rare inherited retinal disease. The data demonstrated notable improvements in retinal function, offering hope for patients affected by this condition. Intellia Therapeutics advanced two in vivo CRISPR therapies—NTLA-2001 and NTLA-2002—through Phase I trials, collectively enrolling 58 patients. These trials represent critical steps toward validating in vivo gene slicing as an effective treatment modality.

In Vivo Delivery Leading the Gene Therapy Market

In vivo delivery has established itself as the dominant approach in the gene therapy market, capturing approximately 60% of the market share. This preference is driven by its ability to overcome several challenges commonly associated with ex vivo methods, such as prolonged manufacturing timelines and complicated cell expansion procedures.

As of 2024, there are at least 85 advanced clinical trials utilizing in vivo delivery techniques that focus on single-gene disorders. These include diseases such as hemophilia and Duchenne muscular dystrophy, which have long posed significant treatment challenges. The growing number of trials reflects both scientific progress and increasing confidence in the safety and efficacy of in vivo approaches.

In 2023, Intellia Therapeutics reported promising results from a trial involving 12 patients treated with NTLA-2001, a CRISPR-based in vivo therapy designed to combat transthyretin amyloidosis. The data demonstrated a durable reduction in transthyretin protein levels, marking a significant milestone for gene-editing therapies administered directly inside the body. Similarly, Pfizer has supported five new in vivo programs over the past year, focusing primarily on direct organ injection techniques aimed at treating metabolic diseases, highlighting the pharmaceutical industry’s growing investment in this delivery route.

North America’s Dominance in the Global Gene Therapy Market

North America continues to lead the global gene therapy market, commanding more than 54% of the total market share. This leadership is largely attributed to the region’s robust academic ecosystem, state-of-the-art manufacturing infrastructure, and proactive regulatory environment that fosters innovation and accelerates development. Together, these factors create a fertile ground for advancing gene therapy research and commercial applications.

As of 2023, North America hosts over 700 active gene therapy clinical trials, underscoring its pivotal role in driving the field forward. The United States is at the forefront, accounting for nearly 600 of these trials, reflecting the country’s intense focus and investment in this cutting-edge area. Canada complements this activity with at least 25 active research collaborations specifically in gene editing, while Mexico is increasingly engaging in cross-border studies, highlighting growing regional cooperation and participation.

The presence of world-renowned institutions such as the University of Pennsylvania and Stanford University significantly boosts fundamental research in gene therapy. These institutions contribute critical scientific advancements and training that feed into clinical and commercial progress. Additionally, the National Institutes of Health (NIH) lists over 200 ongoing Phase I or II trials in the US that target rare genetic disorders, demonstrating a strong commitment to addressing unmet medical needs through innovative gene therapies.

Key Players in the Gene Therapy Market

• Amgen, Inc.

• AnGes, Inc.

• Biogen Inc. (US)

• Bluebird Bio Inc.

• Dimension Therapeutics Inc.

• F. Hoffmann-La Roche AG

• Ferring B.V.

• Gilead Sciences, Inc.

• Johnson & Johnson

• Novartis AG

• Orchard Therapeutics PLC

• Pfizer Inc.

• Regenxbio

• Sangamo Therapeutics, Inc.

• Sarepta Therapeutics, Inc.

• Shanghai Sunway Biotech Co., Ltd.

• Sibiono Genetech Co., Ltd.

• Ultragenyx Pharmaceutical Inc.

• UniQure N.V.

• Vertex Pharmaceuticals Incorporated

• Other Prominent Players

Market Segmentation Overview:

By Type

• Gene Silencing

• Cell Replacement

• Gene Augmentation

• Other Therapies

By Vector

• Viral Vectors

• Non-Viral Vectors

By Therapeutic Area

• Oncology

• Neurology

• Hepatology

• Other Therapeutic Areas

By Delivery Method:

• In Vivo

• Ex Vivo

By Route of Administration:

• Intravenous

• Other Routes of Administration

By Region

• North America

• Europe

• Asia Pacific

• Middle East & Africa

• South America

For more information about this report visit: https://www.astuteanalytica.com/industry-report/gene-therapy-market

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