Gene Therapy Market Research Report Information by Type (Simple Generics and Super Generics), by Brand (Pure Generic and Branded Generic), by Indication (Central Nervous System (CNS), Cardiovascular, Urology, Oncology, Respiratory, and Others), by Route of Administration (Oral, Topical, Parenteral, and Others), by Distribution Channel (Hospital Pharmacy, Retail Pharmacy, and Online Pharmacy), and by Region (North America, Europe, Asia-Pacific, and Rest of the World - Forecast till 2032

Global Gene Therapy Market Overview

The Gene Therapy Market Size was valued at USD 9.4 billion in 2023 and is projected to grow from USD 10.8 billion in 2024 to USD 43.1 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 19.87% during the forecast period (2024 - 2032). The growth of the gene therapy market is influenced by major factor such as rise in the prevalence of chronic disease including cancer, genetic disorder, rare disorder and others, increase in government support, rise in ethical acceptance of generic drugs for treatment for various disease, presence of strong product portfolio and rise in the investment in the research and development activities to develop gene therapy drugs are the major ley factor drives the growth of market.

Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review

Gene Therapy Market Trends

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  Rise in the Prevalence Of Chronic Disease.

  
  

Rise in the prevalence of chronic disease such as cancer, neurological disorders, rare disorders, genetic disorder and others across the world is the major factor that drives the growth of market. For instance, according to the International Agency for Research on Cancer (IARC) in 2022, it was estimated that 20 million new cases of cancer diagnosed across the world and expected to be reach 35 million by 2035. In addition, according to Rare Diseases International in 2022, about 300 million people living with a rare disorder out of which 72% of rare disorders are genetic and approximately 50% of the people affected by rare disorders are children across the world.

Furthermore, the increasing number of drugs being developed for the treatment of chronic diseases has also fueled the growth of the gene therapy market. Many pharmaceutical companies are investing heavily in gene therapy research and development to develop gene therapy drugs.

For instance, in 2023, bluebird bio, Inc. has announced that U.S. Food and Drug Administration (FDA) approved LYFGENIA™ (lovotibeglogene autotemcel) for the treatment of sickle cell disease in patients ages 12 and older who have a history of vaso-occlusive events (VOEs). LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Additionally in 2024, Pfizer Inc. (NYSE: PFE) has announced that U.S. Food and Drug Administration (FDA) approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage. BEQVEZ is a one-time gene therapy that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.

Thus, rise in the prevalence of the chronic disease and increasing number of drugs being developed for the treatment of chronic diseases across the world drives the growth of market.

Significant Increase in the Number of Drugs In Development For Gene Therapy

Gene therapy is a cutting-edge medical treatment that involves altering or replacing faulty genes in order to treat or prevent diseases. In recent years, there has been a significant increase in the number of drugs in development for gene therapy, which has driven the growth of the gene therapy market.

The pipeline of gene therapy drugs includes a wide range of treatments for various genetic and acquired diseases, such as rare genetic disorders, cancer, and autoimmune diseases. The potential of these drugs to provide targeted and personalized treatments for patients with complex medical conditions has generated significant interest from biopharmaceutical companies, investors, and healthcare providers.

As more gene therapy drugs advance through clinical trials and receive regulatory approval, the market for gene therapy is expected to continue to grow. For instance, according to American Society of Gene and Cell Therapy in 2022 it was estimated that about 3,633 therapies in the pipeline out of which 2,024 gene therapies (including genetically-modified cell therapies such as CAR T-cell therapies) are in development —from preclinical through pre-registration in the second quarter of 2022. In the gene therapy pipeline and in the clinic, oncology and rare diseases remain the top areas of development. To illustrate this trend, below is a comparison table showing the increase in the pipeline of gene therapy drugs.

Thus, increase in the pipeline of gene therapy drugs is driving the growth of the gene therapy market and is expected to revolutionize the treatment of diseases in the coming years.

Gene Therapy Market Segment Insights

Gene Therapy Type Insights

By therapy type, the market is classified into ex vivo therapy and in vivo therapy. The in vivo therapy segment held the largest market share in 2022, owing to increase in the adoption of in vivo gene therapies globally. As vivo gene therapy can reach multiple sites or organs throughout the body, it allows for systemic treatment. This is especially beneficial for diseases that affect multiple organ systems or have systemic manifestations, allowing for a more comprehensive therapeutic approach. For instance, in 2024 the U.S. Food and Drug Administration (FDA) approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) an in vivo gene therapy for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage.

However, the ex vivo therapy segment is expected to grow at a significant CAGR during the forecast period, as ex vivo therapy allows for the manipulation and modification of cells outside of the body, enabling more precise and targeted delivery of therapeutic genes. This approach also allows for the expansion of modified cells before reinfusion into the patient, potentially increasing the efficacy of treatment. For instance, in 2024, the U.S. Food and Drug Administration (FDA) has approved LENMELDY Ex vivo gene therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

GENE THERAPY MARKET SIZE, BY TYPE, 2019 & 2032 (USD Billion)

Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review

Gene Therapy Application Insights

Based on Application, the gene therapy market segmentation includes Oncology, Genetic disorder, Neurological disorder, and others.  The oncology segment held the largest market share in 2022, owing to increase in prevalence of cancer, rise in development of gene therapy drugs specific to oncology. For instance, in 2022, the US Food and Drug Administration (FDA) approved Adstiladrin® (nadofaragene firadenovec-vncg) in December 2022, new long-term follow up data has been revealed.

Adstiladrin is the first FDA-approved intravesical gene therapy to be authorized for adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.

However, the genetic disorder segment is expected to grow at a significant CAGR during the forecast period, owing to rise in the prevalence of genetic disease across the world. For instance, according to FDA in 2022, rare diseases affect 30 million people in the USA and many rare conditions are life-threatening and most do not have treatments.

Gene Therapy Distribution Channel Insight

Based on Distribution Channel, the gene therapy industry includes Hospital Pharmacies, Online Pharmacies and Retail Pharmacies. The hospital pharmacies segment held the largest market share in 2022, owing to increase in the demand of gene therapy drugs for the treatment of various chronic diseases across the world

Gene Therapy Regional Insight

By Region, the study provides the market insights into North America, Europe, Asia-Pacific and Rest of the World. North America held the largest share in the gene therapy market, owing to rise in the prevalence of chronic disease, availably of reimbursements for the gene therapy and rise in healthcare expenditure and research development activities for gene therapy drugs across the region. For instance, in 2023, the US federal government spent approximately $190 billion on R&D. This was a roughly 13 percent increase over fiscal year 2022, making it one of the largest R&D spending jumps in recent history.

However, Asia-Pacific is anticipated to witness notable gene therapy market opportunity, owing to a sizable population base, heightened healthcare awareness, and a surge in cases of cancer. These factors collectively drive the demand for gene therapy in the region. Additionally, pharmaceutical market expansion, government initiatives, and a surge in research and development activities contribute to the region's accelerated growth. Furthermore, a rise in research activities as well as the well-established presence of domestic companies in the region are expected to provide notable opportunities for market growth.

Gene Therapy Key Market Players & Competitive Insights

Competitive analysis and profiles of the major players in the gene therapy market that operate in the market are provided in the report, such as Novartis AG, Gilead Sciences., Jazz Pharmaceuticals, Orchard Therapeutics., Bristol-Myers Squibb., Amgen Inc., Shenzhen SiBiono Genentech, Shanghai Sunway Biotech Co., Ltd., Adaptimmune Therapeutics plc, Adverum Biotechnologies, Inc., Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, F Hoffman la Roche., Astellas Therapeutics, Inc., Bluebird Bio, Inc., CRISPR Therapeutics AG, Intellia Therapeutics, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Unique N. V., Pfizer inc., Merck and Co Inc., and others. The key players have adopted strategies such as acquisition, agreement, strategic alliance, collaboration, clinical trial, expansion, joint venture, new product development, and product approval to expand their product portfolio.

For instance, in 2023, Sarepta and Roche has announced that food and Drug Administration (FDA) in the United States (US) has granted accelerated approval for the Duchenne muscular dystrophy (DMD) gene therapy treatment SRP-9001 (delandistrogene moxeparvovec). SRP-9001 (also known as delandistrogene moxeparvovec, or dela mox) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene. It is a one-time treatment designed to treat the underlying cause of DMD.

Key Companies in the Gene Therapy Market include

• Amgen Inc. (US)


• Spark Therapeutics Inc. (US)


• Bristol-Myers Squibb Company (US)


• Novartis AG (Switzerland)


• Gilead Sciences Inc. (US)


• Pfizer Inc (US)


• F Hoffman La Roche (Switzerland)


• Merck Coo & Inc (US)


• CRISPR Therapeutics AG (Switzerland)


• Orchard Therapeutics plc (UK)

Gene Therapy Industry Developments

April 2024: the U.S. Food and Drug Administration (FDA) approved Abecma (idecabtagene vicleucel; ide-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma.

October 2023: Spark Therapeutics announced a partnership with Splice Bio to licence their proprietary splicing platform to develop a novel gene therapy for renal disease.  

April 2023: Bristol Myers Squibb acquired Novartis' manufacturing facility in Illinois, US to further augment its manufacturing capabilities for hard to manufacture gene therapies. 

June 2023: The US FDA approved Elevidys, the first ever gene therapy for treating Duchenne Muscular Dystrophy. The product is manufactured by Sarepta Therapeutics, Inc..

December2022: the FDA approved nadofaragene firadenovec-vncg (brand name Adstiladrin) for adult patients with high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors.

May 2022: Novartis AG (Switzerland) received FDA approval for KYMRIAH for adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

Gene Therapy Drug Market Segmentation

Gene Therapy Type Outlook

• Ex-Vivo


• In-Vivo

Gene Therapy Vector Type Outlook

• Viral Vector


• Non-Viral Vector

Gene Therapy Application Outlook

• Oncology


• Genetic Disorders


• Neurological Disorders


• Other

Gene Therapy Distribution Channel Outlook

• Hospital Pharmacies


• Retail Pharmacies


• Online Pharmacies

Gene Therapy Market Regional Outlook

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  Middle East
  
  


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  Africa
  
  


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  Latin America