Gene Therapy Market

The gene therapy market is anticipated to grow from US$ 5.84 billion in 2024 to US$ 29.36 billion by 2031, reflecting a projected CAGR of 14.3% from 2025 to 2031.

Market Insights and Analyst Perspective:Gene therapy involves treating diseases by either inactivating a harmful gene, replacing it with a healthy version, or introducing a new or modified gene to aid in disease treatment and prevention. This therapy can be categorized into in vivo and ex vivo methods. The primary goal of gene therapy is to replace or correct defective genes with normal ones, enabling the body to produce the necessary proteins or enzymes for proper functioning, which may potentially address the root causes of various diseases. The rising incidence of genetic disorders and cancer globally, along with an increasing number of FDA approvals for gene therapies, is propelling the growth of the gene therapy market. Furthermore, advancements in gene therapy technologies are expected to further enhance market growth in the future.

Growth Catalysts:Recent advancements in biotechnology have facilitated the creation of treatments for a broad spectrum of conditions. Gene therapies are now being utilized to address various ailments, including cancer, neurological disorders, and genetic diseases. The global adoption of gene therapies is on the rise, largely due to the availability of FDA-approved products. Here are some notable examples of gene therapy products that have received FDA approval in recent years:

In December 2023, the FDA approved two cell-based gene therapies for sickle cell disease: Bluebird Bio's Lyfgenia (lovotibeglogene autotemcel) for patients aged 12 and older with a history of vaso-occlusive events, alongside Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics.

In June 2023, the FDA approved Roctavian, a gene therapy utilizing an adeno-associated virus vector for adults with severe hemophilia A who do not have pre-existing antibodies to adeno-associated virus serotype 5. This one-time treatment addresses a gene mutation responsible for producing factor VIII (FVIII), a crucial protein for blood clotting.

In June 2023, the FDA also approved Elevidys, marking the first gene therapy for treating Duchenne muscular dystrophy in pediatric patients aged 4 to 5 years with a confirmed mutation in the Duchenne muscular dystrophy gene, provided they do not have any pre-existing medical conditions that would hinder treatment.

In November 2022, the FDA approved HEMGENIX, a recombinant Adeno-associated virus type 5 gene therapy developed by CSL Behring LLC, for adult patients with specific types of Hemophilia B.Thus, the increasing number of approved gene therapies is significantly driving the growth of the gene therapy market.

Strategic Insights

Report Segmentation and Scope:The analysis of the gene therapy market has been conducted by examining various segments: vectors, indications, delivery modes, and geographical regions. The market is divided into non-viral and viral vectors. In terms of indications, it includes neurological diseases, cancer, Duchenne muscular dystrophy, hepatological diseases, and other conditions. The delivery modes are categorized into in vivo and ex vivo. The report encompasses the gene therapy market across North America (the US, Canada, and Mexico), Europe (France, Germany, the UK, Spain, Italy, and the Rest of Europe), Asia Pacific (China, Japan, India, Australia, South Korea, and the Rest of Asia Pacific), the Middle East & Africa (Saudi Arabia, South Africa, the UAE, and the Rest of Middle East & Africa), and South & Central America (Brazil, Argentina, and the Rest of South & Central America).

Segmental Analysis:The gene therapy market is segmented by vectors into non-viral and viral vectors, with the viral vectors segment holding a substantial market share in 2023 and expected to achieve a higher CAGR from 2023 to 2030.

By indication, the market includes neurological diseases, cancer, Duchenne muscular dystrophy, hepatological diseases, and other conditions. The cancer segment represented a significant share of the gene therapy market in 2023 and is projected to experience the highest CAGR during 2023 to 2030.

In terms of delivery mode, the market is divided into in vivo and ex vivo, with the in vivo segment holding a considerable market share in 2023 and anticipated to register a higher CAGR from 2023 to 2030. In vivo gene therapy allows for systemic treatment, reaching multiple sites and organs throughout the body, which is particularly beneficial for diseases that affect various areas or exhibit systemic symptoms, enabling a comprehensive therapeutic approach. Additionally, advancements in delivery technologies, such as viral vectors, nanoparticles, and lipid-based carriers, have enhanced the effectiveness and specificity of in vivo therapies, improving the targeted delivery of genetic material and the overall safety and efficacy of treatments.

Regional Analysis:Regionally, the gene therapy market is divided into North America, Europe, Asia Pacific, South & Central America, and the Middle East & Africa. In 2023, North America held a significant market share, with the US leading the gene therapy market in this region. The growth in North America is driven by the increasing prevalence of genetic disorders, a rising number of cancer patients, enhanced government funding, greater adoption of advanced gene therapies for treatment, and a growing number of product approvals.

According to the Centers for Disease Control and Prevention (CDC), approximately 1,603,844 new cancer cases were diagnosed in 2020, resulting in 602,347 cancer-related deaths in the US. This translates to 403 new cancer cases per 100,000 individuals. Furthermore, the International Agency for Research on Cancer projects that new cancer cases could reach 30.2 million by 2040. Estimates from the US Government Accountability Office published in October 2021 indicate that around 25 to 30 million people in the US suffer from rare diseases, with nearly 50% of these patients being children. Many rare diseases stem from genetic mutations, with approximately 80% classified as genetic disorders.

As of October 2021, the National Institutes of Health reported that 10 pharmaceutical companies and 5 non-profit organizations are collaborating to expedite the development of gene therapies for the 30 million Americans affected by rare diseases. The US FDA has approved 7 cell and gene therapy drugs, with a pipeline of around 1,200 experimental therapies, half of which are in Phase 2 clinical trials. Annual sales growth estimates suggest a 15% increase for cell therapies and approximately 30% for gene therapies, according to the Chemical & Engineering News report from 2023. All these factors contribute to the expanding gene therapy market in the region.

Gene Therapy Market Report Scope

Industry Developments and Future Opportunities:Below are some initiatives undertaken by key players in the global gene therapy market:

In January 2022, Ori Biotech Ltd raised over US$ 100 million in an oversubscribed Series B funding round to launch an innovative cell and gene therapy manufacturing platform.

In January 2020, Astellas Pharma Inc. acquired Audentes Therapeutics, Inc., positioning the combined entity as a global leader in AAV-based genetic medicine.Competitive Landscape and Key Companies:The gene therapy market forecast provides valuable insights for stakeholders to strategize their growth plans. Key players profiled in the gene therapy market report include Novartis AG, Astellas Pharma Inc., Bristol-Myers Squibb Company, Bluebird Bio Inc., CSL Behring, Sanofi, F. Hoffmann-La Roche Ltd, Daiichi Sankyo, Biogen, and Oxford Biomedica. These companies are focused on launching new high-tech products, enhancing existing offerings, and expanding geographically to meet the increasing global consumer demand.