Gene Therapy Market To Reach USD 49.3 Billion By 2032

Introduction

Global Gene Therapy Market size is expected to be worth around USD 49.3 Bn by 2032 from USD 7.0 Bn in 2023, growing at a CAGR of 25% during the forecast period from 2023 to 2032. In 2023, North America led the market, achieving over 65% share with a revenue of US$ 3.6 Billion.

Several factors are driving this rapid growth. A key driver is the rising prevalence of genetic disorders and chronic diseases, which increases demand for innovative treatments. Advances in technologies like CRISPR and new gene delivery methods have accelerated the development of effective gene therapies. Approved therapies, such as treatments for blood cancers and inherited retinal disorders, demonstrate their ability to address previously unmet medical needs.

Government support plays a critical role in this expansion. Regulatory frameworks like the Regenerative Medicine Advanced Therapy (RMAT) designation and FDA guidance on manufacturing and clinical development challenges foster innovation while ensuring safety and efficacy. These initiatives streamline the approval process for new treatments.

Funding from public and private sectors also supports research and development. Programs such as the Cell and Gene Therapy Access Model improve access to high-cost therapies for rare and severe diseases. By linking payments to treatment success, these models facilitate financing and reduce financial risks.

Strategic collaborations between companies and research institutions enhance market growth. These partnerships combine expertise and resources, accelerating the development of advanced therapies. Early regulatory advice programs further streamline the process, helping developers meet preclinical requirements more efficiently.

Recent advancements highlight the market’s potential. In June 2021, Biogen Inc. reported positive results from a Phase 3 study of timrepigene empacrvovec, a gene therapy for choroideremia, a condition causing progressive vision loss. Similarly, in February 2021, Spark Therapeutics began dosing participants in the RESOLUTE trial, testing SPK-3006, an adeno-associated viral vector gene therapy for Pompe disease. These breakthroughs emphasize gene therapy’s capacity to improve treatment options and patient outcomes.

Key Takeaways

• Market Size: The global gene therapy market is expected to reach USD 49.3 billion by 2032, growing from USD 5.6 billion in 2022.
• Market Growth: The gene therapy market is projected to grow at a CAGR of 25% from 2023 to 2032.
• Type Analysis: The viral vector segment is expected to hold the largest revenue share, exceeding 85%, during the forecast period.
• Gene Type Analysis: The antigen segment has held the largest market share and is expected to maintain its lead during the forecast period.
• Delivery Methods Analysis: The in-vivo method segment held the largest market share during the forecast period. The increasing adoption of in-vivo therapies globally is anticipated to drive market growth in the future.
• Disease Indication Analysis: The oncology segment dominated the global gene therapy market in terms of revenue during the forecast period and is expected to sustain its dominance.
• Distribution Channel Analysis: The hospitals segment is anticipated to hold the highest market share throughout the forecast period, driven by the accessibility and availability of treatment methods in these facilities.

Gene Therapy Statistics

• FDA Approvals in 2022: 27 cell and gene therapies received regulatory approval.
• Clinical Trials in 2022: Over 1,500 active trials, with 90% in early development stages.
• Investment Trends in 2022: Investments in gene therapy were estimated between $9.8 billion and $13.5 billion.
• Venture Capital Funding in 2022: Funding reached 40% of 2021’s total in the first half of the year.
• IND Applications in 2020: The FDA received more than 350 new investigational new drug applications.
• Clinical Holds (2020–2022): A total of 33 clinical holds were issued, with 26 resolved and 5 active by the end of 2022.
• Hold Duration: The average duration was 6.2 months, ranging from 2 to 19 months.
• FDA Response Time: Holds and sponsor responses were reviewed within 30 days on average.
• Protocol Amendments: Half of resolved holds required protocol changes, averaging 4.8 months to address.
• CMC Data Requests: 25% of resolved holds involved Chemistry, Manufacturing, and Controls data, resolved within 8.4 months on average.
• Adverse Event Resolutions: Holds related to adverse events took 6.5 months on average to resolve (25% of cases).
• Preclinical Data Issues: Represented 9% of holds, resolved in an average of 4 months.
• Product Types in Holds: CAR T cell therapies accounted for 27%, lentiviral-based therapies for 15%, AAV-based therapies for 45%, and other therapies for 13%.
• CAR T Therapy Holds: Most were resolved within 4.1 months, mainly due to safety concerns.
• Patient Deaths and Holds: Three holds related to patient deaths or adverse events were resolved within 2.3 months on average.
• Corporate Impacts: Three clinical holds significantly affected companies, disrupting partnerships and sponsor support.
• FDA’s Prediction for 2025: The FDA expects to approve 10–20 new gene therapies annually.
• 2023 Approvals Forecast: Up to 13 new therapies anticipated for approval in the US and Europe.
• EU Pharmaceutical Revisions: A proposal to revise legislation was expected in the first quarter of 2023.
• Asia-Pacific Developer Growth: The number of developers increased by 271% over five years.
• Growth in Clinical Trials: Trials expanded from 1,800 to over 5,000 in the last decade.
• Current Clinical Trials: Over 2,000 trials are ongoing, with 200 in Phase III.
• FDA Modernization: The Office of Tissues and Advanced Therapies transitioned to the Office of Therapeutic Products.
• EU Market Challenges: Seven out of 23 advanced therapy medicinal products approved in the EU were later withdrawn.

Emerging Trends in Gene Therapy

• Increased FDA Approvals: The FDA has implemented initiatives to accelerate gene therapy approvals, leading to a surge in recent years. Programs like Breakthrough Therapy Designation and Priority Review have been pivotal. Recent approvals for inherited retinal diseases and rare genetic disorders highlight the FDA’s commitment to fostering innovation. As more therapies demonstrate efficacy, this trend is expected to expand the availability of advanced treatments.
• Advancements in Gene Editing: Technologies such as CRISPR/Cas9 and TALENs have transformed gene therapy, enabling precise genetic modifications. These tools offer potential cures for previously untreatable diseases. Innovations in gene delivery methods, including advanced nanoparticles and viral vectors, are further enhancing precision and efficiency. Emerging technologies like base and epigenome editors promise improved accuracy with minimal off-target effects, advancing the field significantly.
• Focus on Rare Diseases: Gene therapy increasingly targets rare and orphan diseases, which are often underserved by conventional treatments. Incentives like orphan drug designations, providing market exclusivity and financial grants, mitigate high development costs. Therapies for conditions like spinal muscular atrophy and bubble boy disease underscore the transformative potential of gene therapy in addressing unmet medical needs in these areas.
• In-Vivo Therapy Development: In-vivo therapies, where therapeutic genes are delivered directly to cells within the body, are gaining traction. This approach avoids the complexities of ex-vivo methods and enhances treatment precision. Advances in adeno-associated viral vectors and targeted delivery techniques are improving safety and efficacy. Ongoing research aims to overcome immune response challenges and ensure long-term gene expression.
• Oncology Applications: Gene therapy is making significant progress in oncology, with innovations like CAR T-cell therapy achieving success in treating blood cancers such as leukemia and lymphoma. Efforts to expand these therapies to solid tumors are underway, focusing on enhancing specificity and efficacy. Advances in gene editing and immune modulation continue to drive breakthroughs in cancer treatments.
• Regulatory Frameworks and Guidance: Evolving regulatory frameworks are facilitating the rapid development of gene therapies. Programs like the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation streamline the approval process while addressing manufacturing and safety challenges. Updates to guidelines ensure that new therapies meet stringent safety and efficacy standards, fostering innovation in the sector.
• Public and Private Funding: Gene therapy research benefits from significant funding from public and private sources. Government initiatives like NIH funding and private investments from biotech firms and venture capitalists support clinical trials, scaling production, and expanding therapeutic applications. These investments are critical for advancing the development of new treatments.
• Strategic Collaborations: Collaborations between biotech companies, academic institutions, and healthcare organizations drive innovation in gene therapy. Partnerships facilitate resource sharing, regulatory navigation, and joint ventures for manufacturing and research. Such strategic alliances are essential for addressing challenges and translating research into clinical practice effectively.
• Rising Clinical Trials: The number of clinical trials in gene therapy continues to grow, reflecting a strong pipeline of innovative treatments. These trials explore new delivery methods, therapeutic targets, and long-term safety. The increasing volume of trials highlights the dynamic evolution of gene therapy and its potential to revolutionize treatment for various conditions.

Use Cases of Gene Therapy

• Sickle Cell Disease: Gene therapies like Casgevy and Lyfgenia address sickle cell disease by targeting the genetic mutation responsible for abnormally shaped red blood cells. Using CRISPR/Cas9 technology and lentiviral vectors, these therapies introduce functional beta-globin genes into the patient’s hematopoietic stem cells. This restores normal hemoglobin production, reducing pain episodes, improving blood flow, and enhancing quality of life. These therapies hold the potential for long-term relief or a cure for this debilitating condition.
• Duchenne Muscular Dystrophy (DMD): Elevidys targets Duchenne Muscular Dystrophy by delivering a micro-dystrophin gene via viral vectors to muscle cells. This gene encodes a shorter but functional version of the dystrophin protein, preserving muscle integrity and function. By slowing muscle degeneration, Elevidys improves mobility, extends life expectancy, and offers a significant advancement over traditional treatments for DMD.
• Hemophilia A: Roctavian offers a transformative solution for severe Hemophilia A by delivering a functional factor VIII gene to liver cells using adeno-associated virus vectors. This therapy reduces dependence on frequent clotting factor infusions, normalizes blood clotting, and minimizes bleeding risks. Roctavian moves the treatment toward a long-term or potentially permanent solution for Hemophilia A.
• Severe Combined Immunodeficiency (SCID): Gene therapy for ADA-SCID involves inserting a functional copy of the ADA gene into the patient’s stem cells, which are then reintroduced into the body. This approach restores immune function, offering a potential cure and eliminating the need for lifelong enzyme replacement therapy or frequent immunoglobulin infusions. It marks a major breakthrough in managing this life-threatening condition.
• Giant Axonal Neuropathy (GAN): Gene therapy for GAN, a rare nerve degeneration disorder, delivers functional copies of the defective gene into the cerebrospinal fluid. This aims to provide a continuous supply of the essential protein for nerve function, potentially slowing disease progression, preserving motor skills, and improving quality of life. While still experimental, this approach offers hope for mitigating this devastating condition.
• Leber Congenital Amaurosis (LCA): For LCA, a hereditary retinal disorder causing blindness, gene therapies introduce functional copies of defective genes into retinal cells. This approach restores or significantly improves vision, representing a milestone in treating inherited blindness. These therapies offer new possibilities for vision restoration where traditional treatments fall short.
• Cancer Treatments: CAR T-cell therapies have revolutionized oncology by genetically modifying T cells to express chimeric antigen receptors (CARs) targeting cancer cells. These therapies have achieved remission in conditions like acute lymphoblastic leukemia (ALL) and diffuse large B-cell lymphoma (DLBCL), especially in cases resistant to traditional treatments. Their specificity and effectiveness highlight their transformative potential in cancer care.
• Pompe Disease: Gene therapy for Pompe disease delivers a functional copy of the gene responsible for producing the enzyme alpha-glucosidase, essential for breaking down glycogen in muscle cells. Research aims to replace lifelong enzyme replacement therapy with a one-time treatment, significantly improving muscle strength and respiratory function.
• Beta Thalassemia: Gene therapy for beta thalassemia corrects genetic mutations or introduces a functional beta-globin gene into hematopoietic stem cells. This reduces the need for regular blood transfusions and offers a durable treatment solution. By addressing the underlying defect, these therapies significantly improve patient outcomes and quality of life.

Conclusion

The gene therapy market is witnessing rapid growth, driven by advancements in technology, increasing prevalence of genetic disorders, and strong regulatory and financial support. Innovations like CRISPR and in-vivo delivery methods enhance precision and efficacy, addressing previously unmet medical needs. Key drivers include rising clinical trials, strategic collaborations, and a growing focus on rare diseases and oncology applications.

Regulatory frameworks and funding are pivotal in streamlining development and accessibility. Promising breakthroughs in conditions like sickle cell disease, DMD, and cancer underscore the transformative potential of gene therapy, positioning it as a cornerstone of modern medicine with immense future possibilities.

Discuss your needs with our analyst

Please share your requirements with more details so our analyst can check if they can solve your problem(s)

Request a Sample Download PDF