Gene Therapy Market – Trends Forecast Till 2031

Market Snapshot

Market Overview

The global Gene Therapy Market size was estimated at USD 6,540 million in 2024 and is projected to reach USD 19,394.45 million in 2031 at a CAGR of 16.8 % during the forecast period 2025-2031.

Gene therapy is an innovative medical approach that involves altering or replacing faulty genes within a patient's cells to treat or prevent disease. This technique can address genetic disorders by introducing, removing, or modifying genetic material within a person’s cells. Gene therapy has gained traction in recent years due to its potential to provide long-term or even permanent cures for certain conditions that were previously difficult or impossible to treat using traditional therapies. It holds promise in treating a variety of conditions, including genetic disorders, cancers, and some viral infections, by targeting the root causes at a molecular level.

The scope of the gene therapy market is expanding rapidly, as advances in biotechnology and genetic research open new possibilities for treatments. The market includes the development of viral and non-viral gene delivery systems, gene editing tools like CRISPR, and the creation of new therapeutic products. It also encompasses various application areas, including oncology, rare genetic diseases, cardiovascular conditions, and infectious diseases. As research continues, gene therapy is likely to expand to other therapeutic areas, creating opportunities for biotech and pharmaceutical companies to develop novel therapies that could transform the landscape of medicine.

Key trends in the gene therapy market include the increasing focus on personalized medicine, the rise of gene-editing technologies like CRISPR, and growing investment from both public and private sectors. With personalized medicine, therapies can be tailored to an individual’s genetic makeup, improving effectiveness and minimizing side effects. Meanwhile, CRISPR and other gene-editing tools are providing more precise ways to alter genes, making gene therapy more feasible and cost-effective. The market is also witnessing collaborations between academic institutions, biotech companies, and pharmaceutical giants, driving the development and commercialization of new gene therapies.

The drivers of the gene therapy market include advancements in genetic research, increasing prevalence of genetic disorders, and rising demand for innovative treatments that offer more durable solutions compared to conventional drugs. As technology improves and regulatory frameworks evolve to accommodate new treatments, gene therapy is becoming more accessible to a wider patient population. Additionally, the growing understanding of the genetic basis of many diseases has sparked new interest in gene therapies, with governments and private investors recognizing the potential of these therapies to address unmet medical needs. This has resulted in increased funding and regulatory support for gene therapy research and development.

Delvens Industry Expert's Standpoint

Experts in the Gene Therapy Market highlight several key trends driving growth and innovation. The gene therapy market is rapidly growing due to advances in biotechnology and genetic research, offering potential cures for genetic disorders, cancers, and viral infections by targeting the root causes of diseases. With the rise of gene-editing technologies like CRISPR, personalized medicine, and growing investment from both public and private sectors, gene therapy is becoming more feasible and cost-effective. The market spans various therapeutic areas, including oncology, rare genetic diseases, and infectious diseases, driven by the increasing prevalence of genetic disorders and a demand for more durable treatments. As technology evolves and regulatory frameworks adapt, gene therapy is poised to revolutionize healthcare, offering long-term solutions for previously untreatable conditions.

Market Portfolio

Key Findings

• The AAV segment has seen significant revenue contributions, with many biopharma companies leveraging their viral vector platforms for the development of AAV-based gene therapies. For example, Lonza entered into an exclusive partnership with Massachusetts Eye and Ear to support their Anc-AAV gene therapy platform for next-generation gene therapies. Similarly, RegenxBio signed agreements with AveXis and Biogen, allowing both companies to utilize RegenxBio’s AAV vector platform for gene therapy development. In addition, Biogen Inc. and Capsigen Inc. formed a strategic partnership to engineer novel AAV capsids, aiming to deliver gene therapies for neuromuscular and CNS disorders. Furthermore, a collaboration between NIST, NIIMBL, and USP was established to assess analytical methods and develop standards for AAV, which will help advance gene therapies for better patient outcomes.

• Spinal muscular atrophy (SMA) has been a dominant segment, as it is a rare yet highly fatal inherited disease. The success of Zolgensma (AVXS-101), a gene therapy addressing the underlying cause of SMA, marked a breakthrough. The FDA approved Zolgensma, and it remains the only gene therapy in this space with approval. Its effectiveness in altering the disease's phenotype showcases the growing potential of gene therapies to treat serious inherited diseases like SMA, emphasizing the increasing use of gene-based treatments for rare, hereditary conditions.

• The Beta-Thalassemia Major and Sickle Cell Disease (SCD) segment is expected to grow rapidly. Gene therapy for these conditions generally involves the transplantation of gene-modified hematopoietic stem cells. While clinical and preclinical studies confirm the efficacy and safety of this approach, challenges such as suboptimal gene expression, gene transfer efficiency, and the toxicity of certain treatments remain. Despite these obstacles, bluebird Bio’s Zynteglo received conditional approval in Europe for β-thalassemia, with expectations for U.S. FDA approval. Additionally, Vertex Pharmaceuticals and CRISPR Therapeutics expanded their partnership to advance the development of CTX001 for SCD and beta-thalassemia, further strengthening the potential for gene therapies in this segment.

• The intravenous route of administration has dominated the global gene therapy market due to its effectiveness in delivering treatments directly into the bloodstream, ensuring broad distribution and rapid uptake by target cells. This method is preferred for a wide range of conditions, and its prominence is further enhanced by advancements in vector design and delivery systems. The intravenous segment is expected to maintain its leadership and emerge as the most lucrative route throughout the forecast period.

• The market is also divided into various regions such as North America, Europe, Asia-Pacific, South America, and Middle East and Africa. North America region is projected to hold the largest market share. Factors such as increasing prevalence of genetic disorders, advancements in gene editing technologies, growing biotech investments, high demand for personalized medicine, regulatory approvals, rising R&D activities, awareness and acceptance, expanding clinical applications, collaborative partnerships, and positive clinical trial results have triggered the Gene Therapy Market in the region.

Regional Analysis

North America to Dominate the Market

• The gene therapy market has been experiencing rapid growth, with significant regional variations in development and adoption. North America, particularly the United States, is a leader in the market, benefiting from a robust regulatory framework that supports innovation and commercialization. The U.S. FDA has already approved several gene therapies, providing a clear pathway for further advancements in the field. Strong investment from both private and government sectors, along with a well-established healthcare infrastructure, has facilitated the rapid development and clinical application of gene therapies. This makes North America a hub for gene therapy research and treatment availability.

• Europe is progressively catching up with North America in the adoption of gene therapies. The European Medicines Agency (EMA) has also approved several therapies, creating momentum in the region. However, regulatory and reimbursement complexities still present challenges in some countries, slowing the pace of adoption. Despite these hurdles, nations like Germany, France, and the UK are leaders in integrating gene therapies into healthcare systems. Moreover, European countries are increasingly engaging in collaborative research efforts and partnerships between academic institutions and the biotech industry, driving innovation in the field.

• In the Asia-Pacific region, countries such as China, Japan, and South Korea are becoming key players in the gene therapy market. The region is seeing rapid advancements in both research and clinical applications, backed by government initiatives that prioritize biotechnology. While Japan has a more established regulatory pathway for gene therapy approvals, other countries in the region are still developing their systems, which can sometimes create challenges in the market. However, the growing number of clinical trials conducted in the region signals the increasing importance of Asia in the global gene therapy landscape, particularly for genetic disorders and cancer treatments.

• Latin America is still in the early stages of adopting gene therapies, and the market remains less mature compared to North America and Europe. Countries like Brazil and Mexico are beginning to show interest in biotechnology and gene therapy development, but regulatory approvals and infrastructure for the widespread implementation of gene therapies are still limited. Furthermore, the high costs associated with gene therapies present a significant barrier to access for many patients in the region. As Latin American countries continue to improve their healthcare systems and regulatory frameworks, the market for gene therapies may expand in the future.

• The Middle East and Africa represent a niche market for gene therapies, with the region still in its infancy in terms of adoption and availability of these treatments. However, countries such as the UAE and Saudi Arabia are making significant investments in healthcare infrastructure, which could potentially drive growth in the gene therapy market in the coming years. The prevalence of genetic disorders in the region also creates a potential demand for gene therapy solutions. Despite this, limited access to healthcare resources, regulatory challenges, and financial constraints continue to impede the widespread availability of gene therapies across much of the region. However, as these challenges are addressed, the Middle East and Africa may emerge as an important market in the future.

Competitive Landscape

• Amgen Inc.
• Novartis AG
• F. Hoffmann-La Roche
• Gilead Sciences, Inc.
• bluebird bio, Inc.
• Bristol-Myers Squibb Company
• Legend Biotech
• BioMarin
• uniQure N.V.
• Merck & Co.
• Sarepta Therapeutics, Inc.
• Krystal Biotech, Inc.

Recent Developments

• In January 2024, Biogen and Ginkgo Bioworks announced the successful completion of their gene therapy partnership focused on AAV-based vectors, which is anticipated to drive the demand for gene therapies in the upcoming years.

• In December 2023, the Swiss Agency for Therapeutic Products approved Libmeldy for the treatment of early-onset metachromatic leukodystrophy.

• In May 2023, Krystal Biotech received approval for its VYJUVEK gene therapy to treat Dystrophic Epidermolysis Bullosa.

• In June 2023, the U.S. FDA approved Sarepta's ELEVIDYS gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children aged 4-5 years.

• In January 2023, Voyager Therapeutics and Neurocrine Biosciences formed a strategic collaboration for the development and commercialization of Voyager's GBA1 program and other next-generation gene therapies for neurological disorders.

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