Authored By: Sarah
11 Jul 2024

 Gene Therapy Market Size to grow by USD 6738 million between 2024-2028

According to a research report “ Gene Therapy Market” by Delivery Mode (In vivo, Ex vivo) Therapy Area (Oncology, CNS, Ophthalmology, Rare diseases, Others) Geography (North America, Europe, Asia, Rest of World (ROW))- Global Forecast to 2028 published by Technavio, the market size is estimated to grow by USD 6738 million, at a CAGR of  23.31% during the forecast period. Gene therapy programs have secured significant designations from regulatory bodies, including the US FDA, EMA, and others, for treating various disorders. Orphan drug designation, granted for drugs targeting rare diseases affecting under 200,000 Americans, offers pharmaceutical companies ten-year market exclusivity instead of the standard seven years. Additionally, orphan drug status provides tax incentives for subsequent research, providing substantial benefits to companies involved in gene therapy development.

Browse market data tables, figures, and in-depth TOC on “Gene Therapy Market” by Delivery Mode (In vivo, Ex vivo) Therapy Area (Oncology, CNS, Ophthalmology, Rare diseases, Others) Geography (North America, Europe, Asia, Rest of World (ROW)) Global Forecast to 2028. Download Free Sample


By Delivery Mode, the In vivo segment is projected to dominate the market size in 2024

In the global health landscape, cancer remains the second leading cause of mortality, with conventional treatments such as chemotherapy posing challenges due to their impact on healthy cells and limited efficacy. The oncology drugs market faces a significant unmet need for safer and more potent therapeutic modalities. Gene therapy, with its potential to target specific genetic mutations and offer personalized treatment, is an emerging solution. Pharmaceutical companies and research organizations are increasingly investing in gene therapy research for various cancer indications, driven by the rising prevalence of these conditions and the need to improve patient outcomes.

By Therapy Area, Oncology  segment is expected to hold the largest market size for the year 2024

In the realm of gene therapy, in vivo techniques represent a groundbreaking approach for delivering genetic material directly into target cells to effect therapeutic change. This method involves the use of vectors to transfer functional copies of faulty or absent genes into the affected tissue. In vivo gene therapy holds immense promise, particularly in the treatment of genetic disorders, as it allows for the correction of genetic defects at their source. The success of this approach is contingent upon the development of advanced gene delivery systems to ensure precise and efficient transfer of genetic material into target cells. Numerous scientific advancements have led to the approval of several in vivo gene therapies, underscoring the potential of this field. As research continues to advance, targeted in vivo gene therapy is poised to revolutionize the way we approach the treatment of various diseases.

North America is forecasted to hold the largest market size by region in 2024

The Gene Therapy Market represents a significant business opportunity for pharmaceutical companies, with its potential to revolutionize the healthcare industry through the treatment of genetic disorders and diseases. This emerging market is expected to experience robust growth due to advancements in gene editing technologies, increasing research and development investments, and a growing patient population. Companies that can effectively navigate regulatory hurdles and bring innovative gene therapy products to market will reap substantial rewards.

The Gene Therapy Market growth and forecasting report also includes detailed analyses of the competitive landscape of the market growth and forecasting and information about 20 market companies, including:

  • Abeona Therapeutics Inc
  • Adaptimmune Therapeutics plc
  • Adverum Biotechnologies Inc
  • Amgen Inc.
  • Astellas Pharma Inc.
  • Biogen Inc.
  • bluebird bio Inc.
  • Editas Medicine Inc.
  • ElevateBio
  • F. Hoffmann La Roche Ltd.
  • Generation Bio Co.
  • Gilead Sciences Inc.
  • Novartis AG
  • Orchard Therapeutics Plc
  • Poseida Therapeutics Inc.
  • Sangamo Therapeutics Inc
  • Sibiono GeneTech Co. Ltd.
  • Syncona Ltd.
  • uniQure NV
  • Voyager Therapeutics Inc

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Research Analysis Overview

Gene therapy is an innovative approach in the field of biological therapies, which aims to treat or prevent diseases by introducing new genetic material into cells. This technique holds great promise for various disease indications, particularly in Oncology and Neurology. The use of molecular carriers or vectors, such as viral and non-viral, plays a crucial role in delivering the therapeutic gene to the target cells. In Oncology, gene therapy modalities include gene replacement, gene editing, and gene silencing, using temperature control for enhanced efficacy. For instance, in gene editing, CRISPR-Cas9 system is used to target and modify mutated genes in cancer cells. In Neurology, indications like metachromatic leukodystrophy are being addressed through gene augmentation and cell replacement therapies. The regulatory approval process, clinical trials, and preclinical testing are essential steps in bringing gene therapy products to market. Traditional medicines are being challenged by these advanced targeted therapies in the genomics field. Shelf life and safety concerns are significant challenges, especially for viral vectors. The clinical pipeline is rich with promising gene therapy products, with ongoing clinical research initiatives driving progress.

Market Research Overview

The Gene Therapy Market is a rapidly growing sector in the healthcare industry, with a focus on developing innovative treatments for various target therapeutic areas such as Hepatology, Oncology, and Neurology. Big Pharma players like Pfizer, Sanofi, and Roche, as well as startup companies, are investing heavily in therapy development platforms and emerging technologies like gene editing therapies, immunotherapy, oncolytic viral therapy, and hybrid vector systems. The mechanism of action for gene therapy involves manipulating a patient's genes to treat or prevent diseases. Marketed gene therapies include Spark Therapeutics' Luxturna for inherited retinal disease and Novartis' Zolgensma for spinal muscular atrophy. Commercialization strategies include partnerships with hospitals, infrastructure development, and regulatory approval processes. The regulatory landscape for gene therapy is complex, with clinical studies, preclinical testing, and clinical pipeline stages requiring rigorous evaluation. Reimbursements and shelf life are also critical considerations. The route of administration and vector used vary, with viral vectors and non-viral vectors being the most common. Gene editing modalities like CRISPR-Cas9, gene replacement, and gene modification are gaining popularity, with applications in diseases like metachromatic leukodystrophy. Capital investments in gene therapy are on the rise, with advanced technologies like nanoparticles, molecular switches, and manipulation technologies driving innovation. The genomics field and clinical research initiatives are also key drivers of growth in the gene therapy market. Traditional medicines are being complemented by advanced targeted therapies, with temperature control and biological therapies playing crucial roles in gene therapy product development. The future of gene therapy holds promise for breakthroughs in disease indications, regulatory approval, and clinical trials.


Technavio Research
Jesse Maida
Media & Marketing Executive
US: +1 844 364 1100
UK: +44 203 893 3200

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