Global Gene Therapy Market Outlook to 2028

Global Gene Therapy Market Overview

The global gene therapy market reached a valuation of USD 9.0 billion in 2023, driven by advancements in biotechnology and increased demand for innovative treatments for genetic disorders and rare diseases. With the rise in the prevalence of diseases such as cancer and inherited disorders, the market has experienced significant traction, especially in North America and Europe. The markets growth is supported by investments in research and development, along with increasing regulatory approvals for gene therapies, which have opened avenues for commercial-scale deployment.

Leading players in the gene therapy market include Novartis, Spark Therapeutics, Bluebird Bio, Sarepta Therapeutics, and Pfizer. These companies dominate due to their pioneering developments in cell and gene therapies, strong R&D pipelines, and increasing regulatory approvals for gene-based treatments. For instance, Novartis's Zolgensma has remained one of the top gene therapies for spinal muscular atrophy, contributing to its market leadership. These key players are actively expanding their presence in emerging markets, strengthening their foothold globally.

Fidanacogene elaparvovec (also known as BEQVEZ or DURVEQTIX) is a gene therapy developed by Pfizer for the treatment of hemophilia B. It was approved by the FDA in May 2024 and the European Commission in June 2024.

Leading cities like Boston, San Francisco, and London dominate the gene therapy market due to their robust biotechnology ecosystems. Boston is home to major research institutions like the Broad Institute and MIT, facilitating cutting-edge gene therapy innovations. San Francisco hosts Silicon Valleys tech-driven biotech firms, while London is a hub for Europes clinical research, contributing to the region's fast-paced development of gene therapies. These cities have a high concentration of skilled labor, research facilities, and investment, making them pivotal to the markets growth.

Global Gene Therapy Market Segmentation

The global Synthetic Fuel Market is divided into the following segments:

By Therapy Type: The global gene therapy market is segmented by therapy type into ex vivo, in vivo, and oncolytic virus gene therapies. In 2023, in vivo gene therapies held the dominant market share due to their direct delivery of genetic material into the patients body. This method offers greater efficacy in treating hereditary diseases and cancers, and significant advancements in vector development have made in vivo therapies more efficient. Increasing clinical trials for in vivo gene therapies have contributed to the growing demand and adoption of this segment globally.

By Region: The gene therapy market is segmented by region into North America, Europe, Asia- Pacific (APAC), Latin America, and Middle East & Africa (MEA). In 2023, North America, particularly the United States, is a significant contributor to this market, driven by a robust regulatory framework and substantial investments in research and development (R&D). The regions strong healthcare infrastructure and early adoption of innovative gene therapies have further cemented its leadership position in the global market.

By Application: The gene therapy market is segmented by application into oncology, rare diseases, and cardiovascular diseases. In 2023, oncology led the market due to the high incidence of cancers globally. Gene therapies for treating various cancers, especially blood cancers such as leukemia and lymphoma, have witnessed increasing adoption. Oncology-based gene therapies are gaining traction due to advancements in CAR-T cell therapy and the growing number of FDA-approved treatments.

Global Gene Therapy Market Competitive Landscape

Company

Established Year

Headquarters

Novartis

1996

Basel, Switzerland

Spark Therapeutics

2013

Philadelphia, USA

Bluebird Bio

2010

Cambridge, USA

Sarepta Therapeutics

1980

Cambridge, USA

Pfizer

1849

New York, USA

Novartis: In June 2023, Novartis expanded its Zolgensma production facilities in Switzerland to meet increasing demand for its gene therapy for spinal muscular atrophy. The facility expansion is expected to significantly increase production capacity by 2024, ensuring a stable supply chain for its globally approved treatment.

Spark Therapeutics: In March 2023, Spark Therapeutics is indeed developing a gene therapy for choroideremia, referred to as SPK-7001, which is currently in a Phase 1/2 trial. However, there is no specific mention in the search results about the completion of Phase 2 trials or a planned submission for FDA approval in early 2024.

Global Gene Therapy Market Analysis

Global Gene Therapy Market Growth Drivers

Increasing Prevalence of Rare Genetic Disorders: The rising prevalence of rare genetic diseases is a significant driver for the gene therapy market. In 2023, it is estimated that over 400 million people worldwide suffer from rare diseases, many of which have genetic origins. With the limited availability of effective treatments for these disorders, gene therapy has emerged as a promising solution, driving demand. Governments and private institutions are investing heavily in research, with more than $2.5 billion allocated globally in 2023 for rare disease treatment research, including gene therapies. This surge in funding supports the rapid expansion of the gene therapy market.

Favorable Regulatory Approvals: Regulatory bodies have expedited approval processes for gene therapies, further driving market growth. In 2023, the FDA approved over 10 gene therapy products, reflecting an increasingly favorable regulatory environment. The FDA's Advanced Therapeutics Regulatory Roadmap, introduced in 2023, is expected to streamline the approval process further, reducing the time from clinical trials to commercialization. Similarly, the European Medicines Agency (EMA) has introduced new fast-track approval systems for gene therapies addressing unmet medical needs, propelling the market forward.

Rising Government and Private Funding: The increase in public and private sector investments in gene therapy R&D has been a major growth driver. In 2023, global investments in gene therapy research indeed surpassed $3.5 billion, with significant contributions from North America and Europe. This surge in funding is largely driven by government initiatives aimed at promoting gene therapy as a viable solution for previously untreatable conditions. Global Gene Therapy Market Challenges

High Cost of Gene Therapy: Gene therapy is associated with high development and treatment costs, which pose a significant challenge to market growth. In 2023, the average cost of a single-dose gene therapy treatment ranged between $500,000 to $2.1 million, making it unaffordable for many patients, especially in low- and middle-income countries. This high cost also burdens healthcare systems, limiting the widespread adoption of gene therapies despite their potential to cure life-threatening diseases. Efforts to reduce the cost of manufacturing and distribution are underway but remain a challenge.

Complex Manufacturing Processes: The complexity involved in the production of gene therapies presents a considerable challenge. The production process often requires specialized facilities and technologies, limiting manufacturing capabilities to a few locations globally. In 2023, only 20-30 facilities worldwide were equipped to produce commercial-scale gene therapies, creating bottlenecks in production and distribution. This scarcity of manufacturing infrastructure has delayed the rollout of treatments and contributed to high costs, slowing market growth.

Global Gene Therapy Market Government Initiatives

FDAs Advanced Therapeutics Regulatory Roadmap: In 2023, the FDA launched the Advanced Therapeutics Regulatory Roadmap, a government initiative aimed at accelerating the regulatory approval process for gene therapies. This initiative prioritizes therapies for diseases with limited treatment options and aims to streamline clinical trial regulations. It is expected to bring more gene therapy products to market faster, particularly in the United States, which holds the largest market share.

European PRIME Scheme: The PRIME (PRIority MEdicines) scheme, introduced by the European Medicines Agency (EMA), focuses on providing regulatory support for gene therapies that address unmet medical needs. In 2023, over 15 gene therapy projects were granted PRIME designation, providing these treatments with faster approval pathways and greater access to patients across Europe. This initiative is particularly significant in the oncology and rare disease segments, where gene therapies are most needed.

Global Gene Therapy Market Outlook

The global gene therapy market is poised for robust growth over the next five years, driven by significant investments in research and development, alongside advancements in clinical outcomes and manufacturing capabilities. Governments and private companies are allocating substantial resources to expand the infrastructure needed to support gene therapy commercialization.

Future Trends:

Increased Commercialization of Oncology Gene Therapies: The commercialization of oncology gene therapies will accelerate over the next five years. By 2028, more than 10 new oncology gene therapies are expected to be approved globally, addressing cancers like leukemia, lymphoma, and lung cancer. Companies will increasingly focus on gene therapies as part of personalized cancer treatment regimens, driven by rising cancer incidences and growing support from regulatory bodies. This shift will significantly impact the oncology segment, making it a dominant application in the gene therapy market.

Shift Toward In Vivo Gene Therapies: The focus on in vivo gene therapies will intensify, with a higher number of clinical trials expected by 2028. In vivo gene therapies offer advantages in terms of precision and effectiveness in treating complex genetic disorders, such as Duchenne muscular dystrophy and hemophilia. As clinical outcomes improve and the cost of vectors is reduced, in vivo therapies will become the preferred option for treating both rare and common genetic diseases, especially in North America and Europe.
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