Gene Therapy Market (By Indication: Acute Lymphoblastic Leukemia, Large B-cell Lymphoma; By Vector Type: Lentivirus; By Route of Administration) - Global Industry Analysis, Size, Share, Growth, Trends, Revenue, Regional Outlook and Forecast 2024-2033

The global gene therapy market size was estimated at around USD 8.69 billion in 2023 and it is projected to hit around USD 51.69 billion by 2033, growing at a CAGR of 19.52% from 2024 to 2033.

Key Pointers

• North America dominated the market with the largest market share of 66% in 2023.
• Asia Pacific region is anticipated to grow at the noteworthy CAGR from 2024 to 2033.
• By Vector Type, the AAV segment held the largest revenue share of 23% in 2023.
• By Indication, the spinal muscular atrophy (SMA) segment emerged as the dominant force in the market during 2023.
• By Route of Administration, the intravenous segment generated the maximum market share in 2023.

Gene Therapy Market Growth

The burgeoning growth of the gene therapy market can be attributed to several pivotal factors. Advances in molecular biology and gene-editing technologies have propelled the development of innovative therapeutic interventions, fostering a conducive environment for market expansion. The increasing prevalence of genetic disorders and the potential for gene therapy to address their root causes underscore its significance in the healthcare landscape. Collaborations between industry leaders and academic institutions further accelerate research and development efforts, propelling the market forward. Additionally, evolving regulatory frameworks, though stringent, provide a structured pathway for the commercialization of gene therapies, instilling confidence in investors and stakeholders. This confluence of scientific progress, strategic collaborations, and regulatory support positions the gene therapy market on a trajectory of sustained growth and transformative impact.

Gene Therapy Market Dynamics

Drivers

• Advancements in Molecular Biology and Gene Editing: Rapid progress in molecular biology and gene-editing technologies has significantly enhanced the precision and efficacy of gene therapies, driving innovation and expanding treatment possibilities.
  
  
• Diverse Therapeutic Applications: The versatility of gene therapy across a wide range of therapeutic applications, from addressing rare genetic disorders to tackling specific types of cancer, contributes to its increasing adoption and market growth.

Restraints

• High Development Costs: The extensive research and development required for gene therapies, coupled with the intricate nature of genetic disorders, contribute to high development costs. This financial barrier poses a challenge to the widespread affordability and accessibility of gene therapies.
  
  
• Limited Commercialization Success: Despite promising advancements, not all gene therapies successfully transition from the research phase to commercialization. Some therapies face hurdles in demonstrating consistent efficacy and safety in larger patient populations, hindering their successful market entry.

Opportunities

• Increased Investment and Funding: Growing interest and confidence in gene therapy have led to increased investment and funding. This financial support creates opportunities for research and development, facilitating the exploration of novel therapies and technologies within the gene therapy space.
  
  
• Advancements in Gene Editing Technologies: Ongoing advancements in gene editing technologies, such as CRISPR-Cas9, present opportunities for more precise and efficient gene therapies. Harnessing these technologies can lead to the development of targeted and personalized treatments with enhanced efficacy and safety profiles.

Vector Insights

In 2023, the AAV segment played a crucial role, contributing a significant 23% to the overall revenue. Various biopharmaceutical companies have been actively leveraging their viral vector platforms for the advancement of AAV-based gene therapy products. For example, Lonza, in September 2016, entered into an exclusive agreement with Massachusetts Eye and Ear to support the development and commercialization of next-generation gene therapies using their AAV platform, known as Anc-AAV gene therapy platform. Likewise, RegenxBio established agreements with AveXis and Biogen in March 2014 and May 2016, respectively, enabling both companies to utilize RegenxBio’s AAV vector platform for gene therapy molecule development.

In a notable collaboration, Biogen Inc. and Capsigen Inc. joined forces in May 2021, entering into a strategic research partnership aimed at engineering novel AAV capsids. These capsids hold the potential to deliver transformative gene therapies, addressing the fundamental genetic causes of various neuromuscular and CNS disorders. Another significant development occurred in July 2021, when the U.S. Department of Commerce’s National Institute of Standards and Technology (NIST), the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and the United States Pharmacopeia (USP) announced a collaborative effort.

Indication Insights

The spinal muscular atrophy (SMA) segment emerged as the dominant force in the market during 2023. Despite being a rare disorder, SMA stands out as one of the most prevalent and fatal inherited diseases affecting infants. The advent of Zolgensma (AVXS-101) has showcased remarkable efficacy in addressing SMA, causing a paradigm shift in the disease's phenotype. The FDA granted approval to Novartis for Zolgensma in May 2019, marking a milestone in treating the root cause of SMA. Presently, Zolgensma stands as the sole gene treatment within this domain to have received regulatory approval, underscoring the escalating use of gene therapies for tackling severe hereditary conditions like SMA.

The Beta-Thalassemia Major/SCD segment is poised to exhibit the most rapid Compound Annual Growth Rate (CAGR) throughout the forecast period. Gene therapy for Sickle Cell Disease (SCD) and β-thalassemia revolves around transplanting gene-modified hematopoietic stem cells. Clinical and preclinical studies affirm the efficacy and safety of this therapeutic approach. Nevertheless, challenges persist, encompassing factors such as suboptimal gene expression levels, limited gene transfer efficiency, stem-cell dosage and quality constraints, and the toxicity associated with myeloablative regimens, impeding optimal effectiveness. Despite these hurdles, bluebird Bio's Zynteglo (formerly LentiGlobin) obtained conditional approval in Europe for β-thalassemia treatment in June 2019, with anticipated U.S. FDA approval in August 2022. Zynteglo has also received Orphan Drug status from the U.S. FDA for sickle cell disease (SCD) treatment.

Route of Administration Insights

In 2023, the intravenous segment asserted its dominance in the global gene therapy market. This was primarily attributed to a substantial number of approved products and a robust pipeline for intravenous (IV) candidates, positioning the segment as a frontrunner. The sheer volume of approved therapies administered intravenously, coupled with the promising pipeline, underscores the significant influence of this segment.

Moreover, the intravenous segment is anticipated to continue its ascendancy, emerging as the most lucrative sector over the forecast period. The strong product presence and future potential in the intravenous category affirm its pivotal role in shaping the trajectory of the global gene therapy market.

Regional Insights

In 2023, North America asserted its dominance in the gene therapy market, holding the largest revenue share at 66%. This regional stronghold is projected to extend further, positioning North America as the leading routine manufacturer of gene therapy in terms of both approvals and revenue generation throughout the forecast period. The robust investment in Research and Development (R&D) by companies, both large and small, underscores the region's commitment to developing optimal therapeutic drugs, further propelling market growth.

Meanwhile, Europe is anticipated to be the fastest-growing regional segment from 2024 to 2033. This growth is attributed to a sizable population with unmet medical needs and an escalating demand for novel technologies, especially in the treatment of rare yet increasingly prevalent diseases.

The Asia Pacific region is poised for substantial growth in the commercial application of genetic therapies during the forecast period. This growth can be attributed to the region's abundant resources, the local presence of major companies, and increased government investments. The availability of resources, coupled with a conducive business environment, positions Asia Pacific as a key player in the global expansion of genetic therapies, showcasing the region's potential impact on the industry landscape.

Gene Therapy Market Key Companies

• REGENXBIO, Inc.
• Oxford BioMedica plc
• Dimension Therapeutics, Inc.
• Bristol-Myers Squibb Company
• SANOFI
• Applied Genetic Technologies Corporation
• F. Hoffmann-La Roche Ltd
• bluebird Bio, Inc.
• Novartis AG
• Taxus Cardium Pharmaceuticals Group, Inc. (Gene Biotherapeutics)
• UniQure N.V.
• Shire Plc
• Cellectis S.A.
• Sangamo Therapeutics, Inc
• Orchard Therapeutics
• Gilead Lifesciences, Inc.
• BENITEC BIOPHARMA
• Sibiono GeneTech Co., Ltd
• Shanghai Sunway Biotech Co., Ltd.
• Gensight Biologics S.A.
• Transgene
• Calimmune, Inc.
• Epeius Biotechnologies Corp.
• Astellas Pharma Inc.
• American Gene Technologies
• BioMarin Pharmaceuticals, Inc.

Recent Developments

• In December 2023, Swiss Agency for Therapeutic Products granted approval to Libmeldy for treatment of early-onset metachromatic leukodystrophy.
  
  
• In May 2023, Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment
  
  
• In June 2023, the U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years

Gene Therapy Market Segmentations:

By Indication

• Acute Lymphoblastic Leukemia (ALL)
• Inherited Retinal Disease
• Large B-cell Lymphoma
• ADA-SCID
• Melanoma (lesions)
• Beta-Thalassemia Major/SCD
• Head & Neck Squamous Cell Carcinoma
• Peripheral arterial disease
• Spinal Muscular Atrophy (SMA)
• Others

By Vector Type

• Lentivirus
• AAV
• RetroVirus & gamma RetroVirus
• Modified Herpes Simplex Virus
• Adenovirus
• Non-viral Plasmid Vector
• Others

By Route of Administration

• Intravenous
• Others

By Region

• North America
• Europe
• Asia Pacific
• Rest of the world