Gene Therapy Market Assessment, By Type [Gene Silencing, Cell Replacement, Gene Augmentation, Others], By Vector [Non-Viral Vectors, Viral Vectors], By Gene Delivery Method [Ex vivo and In vivo], By Application [Oncological Diseases, Neuromuscular Disease

Gene Therapy Market Assessment, By Type [Gene Silencing, Cell Replacement, Gene Augmentation, Others], By Vector [Non-Viral Vectors, Viral Vectors], By Gene Delivery Method [Ex vivo and In vivo], By Application [Oncological Diseases, Neuromuscular Diseases, Cardiovascular Diseases, Infectious Diseases, Genetic Diseases, Haematological Diseases, Others], By Route of Administration [Intravenous, Oral, Intramuscular, Others], By End-users [Hospitals, Cancer and Research Institutes, Clinics, Others], By Region, Opportunities and Forecast, 2017-2031F

Global gene therapy market size was valued at USD 7.64 billion in 2023, which is expected to reach USD 37.83 billion in 2031, with a CAGR of 22.13% for the forecast period between 2024 and 2031. The global gene therapy market is experiencing robust expansion, owing to various factors such as innovations in gene editing technologies, increasing volume of gene therapy, growing popularity of personalized medicine, advancements in the manufacturing process, and expediting the regulatory approval process.

The global gene therapy market is currently experiencing rapid expansion, driven by advancements in gene editing technologies, such as CRISPR-Cas9 and TALEN, which have revolutionized the precision and efficiency of gene therapy. The scope of treatable conditions continues to widen, with gene therapy candidates in development for an ever-expanding range of diseases, including rare genetic disorders, cancer, neurodegenerative conditions, and many more. The diversification of global gene therapy market is partly a result of financial support, as venture capitalists, pharmaceutical giants, and government agencies invest heavily in research, clinical trials, and commercialization.

The growing interest in personalized medicine is another driver, as gene therapy offers tailored treatment strategies based on an individual's genetic profile. It aligns with a broader trend in healthcare where patient-centric care is prioritized, increasing the demand for gene therapy. Collaborative efforts and partnerships have become pivotal in accelerating gene therapy development, with academic institutions, biotechnology firms, and pharmaceutical companies combining their resources and knowledge. The regulatory environment of global gene therapy market is becoming more friendly, with expedited review processes and flexible pathways for rare diseases and unmet medical needs. Consequently, improvements in manufacturing process have streamlined production processes, reducing costs and complexities associated with gene therapy products.

Growing Demand for Personalized Medicine

The rising interest in personalized medicine significantly boosts the global gene therapy market. Personalized medicine focuses on tailoring medical treatments to the specific genetic makeup and needs of individual patients. Gene therapy plays a pivotal role by offering precise targeted treatment strategies based on a patient's genetic profile. As the understanding of genetics and genomics continue to advance, there is a growing awareness among both healthcare providers and patients about the potential benefits of gene therapy in addressing a wide range of genetic and rare diseases. The increasing demand for treatments customized to an individual's unique genetic characteristics is a driving force behind the growth of the gene therapy market. Patients and healthcare professionals are increasingly seeking gene therapy solutions, which in turn, encourages research and development and investment, thus driving the growth of global gene therapy market.

Technological Advancements

Artificial intelligence plays a crucial role in the global gene therapy market. AI is being used in various segments of gene therapy R&D, encompassing tasks such as analyzing extensive genomic datasets, identifying potential gene targets, predicting treatment outcomes, and optimizing delivery methods. The speed at which AI can process and interpret intricate genetic data, expedites the discovery and advancement of gene therapies. Furthermore, AI-driven algorithms contribute to the refinement of personalized gene therapies, tailoring treatments to individual patients based on their unique genetic profiles. As AI advances, its integration into gene therapy research and clinical application is poised to foster innovation and market expansion.

In March 2023, Google unveiled that a group of researchers utilized Google's AI based technology, DeepMind, to create a bacterial injection system capable of directly delivering proteins, including cancer-fighting medications and gene therapies, into human cells.

Government Initiatives

Governments of many countries are increasingly taking proactive steps to promote and regulate the field of gene therapy. These initiatives encompass a range of activities, from funding research and development programs to establishing clear regulatory frameworks. Such government initiatives provide financial support for groundbreaking gene therapy research and foster an environment of safety and compliance, which is crucial for gaining public trust and industry investment. As a result, these efforts create a favorable landscape for gene therapy companies, encouraging innovation and accelerating the development of promising gene-based treatments. Government initiatives are acting as a catalyst, propelling the growth of global gene therapy market.

In February 2023, the Department of Health and Social Care and NHS England unveiled the 2023 England rare diseases action plan, demonstrating commitment towards assisting individuals affected by rare diseases. The government is introducing novel medications for those suffering from various rare conditions, encompassing life-saving gene therapies and advancements in genomic testing and sequencing technologies.

Growing Demand for Adeno-Associated Virus Vector

The increasing demand for adeno-associated virus (AAV) vectors in gene therapy is significantly propelling the global gene therapy market. AAV vectors are crucial delivery vehicles for introducing therapeutic genes into target cells and tissues. Their rising popularity can be attributed to their safety profile and effectiveness in transferring genetic material. As gene therapy continues to advance and diversify, AAV vectors are being used in different treatments for genetic and rare diseases, including inherited disorders and various forms of cancer. The growing reliance on AAV vectors is fueling the gene therapy market's growth, as researchers and biotech companies invest in their development and production.

In July 2023, Astellas entered into a substantial agreement worth USD 962 million with 4D Molecular Therapeutics, focusing on treating rare eye diseases. Under the partnership, Astellas secured the rights to utilize 4DMT's gene therapy vector for at least one specific therapeutic target. R100, an adeno-associated virus (AAV) vector developed by 4DMT, designed for intravitreal delivery.

Growth in Volume for Neuromuscular Diseases

The rising demand for gene therapy in treating neuromuscular diseases is a significant driver of the global gene therapy market. Neuromuscular disorders consist of a range of conditions affecting the nervous system and muscles, often with limited treatment options. Gene therapy offers a promising avenue for addressing these conditions by delivering functional genes to replace or repair faulty ones. As research and clinical trials continue to demonstrate the potential effectiveness of gene therapy in treating neuromuscular diseases, the demand for such therapies is growing. It in turn, fuels investment, innovation, and expansion in the gene therapy market as companies strive to meet the unmet medical needs of patients with these challenging conditions.

In June 2023, Astellas Pharma Inc. and Kate Therapeutics unveiled an exclusive licensing agreement to develop and commercialize KT430. KT430 is an advanced preclinical gene therapy, utilizing a novel MyoAAV capsid, designed to deliver a functional version of the MTM1 gene. The primary objective of the therapy is to address X-linked myotubular myopathy (XLMTM), an exceedingly severe and rare neuromuscular disorder characterized by profound muscle weakness, respiratory insufficiency, and a high risk of premature mortality.

Future Market Scenario

The future of the global gene therapy market appears promising, driven by several key factors. Ongoing advancements in gene editing technologies, such as CRISPR-Cas9 and emerging techniques, are enhancing the precision and efficiency of gene therapy. These breakthroughs enable scientists to target and modify specific genes with unprecedented accuracy, expanding the range of treatable diseases. Regulatory agencies worldwide are increasingly recognizing the potential of gene therapy and adopting more flexible and expedited review processes for these innovative treatments. It encourages greater investment and interest from pharmaceutical companies and biotech firms, spurring the development of a robust pipeline of gene therapy candidates across a wide spectrum of medical conditions. The expanding focus on personalized medicine, where treatments are tailored to an individual's genetic profile, drives gene therapy market growth.

Key Players Landscape and Outlook

In the global gene therapy market, major pharmaceutical and biotechnology companies are entering into partnerships and collaborations. These collaborations facilitate the sharing of resources, knowledge, and expertise, ultimately expediting the development and commercialization of gene therapies. For pharmaceutical giants, teaming up with innovative biotech firms allows them to diversify their portfolios and tap into innovative gene therapy solutions.

AstraZeneca disclosed on November 1, 2023, its plans to allocate USD 245 million in a strategic partnership with the French biotechnology firm Cellectis. The collaboration is focused on accelerating the advancement of therapeutics within sectors such as oncology, immunology, and rare diseases. By harnessing Cellectis' expertise in gene-editing technology and manufacturing, AstraZeneca aims to enhance its expanding portfolio of innovative cell and gene therapy products. The investment signifies AstraZeneca's commitment to fortify its presence in these critical therapeutic areas, leveraging Cellectis' capabilities to drive innovation and the development of novel treatments.