Gene Therapy Market

Gene Therapy Market Outlook (2024 to 2034)

The global gene therapy market size is calculated at a value of US$ 10.34 billion in 2024. Increasing cases of rare diseases is contributing to the rising need for cell and gene therapies. Expanding at a CAGR of 14.1%, the market is forecasted to reach US$ 38.52 billion by the end of 2034.

Ex-vivo gene therapy is becoming more popular for neurological treatments, which is expected to drive gene therapy market growth through 2034. Over the last few years, the world has seen a significant increase in focus on healthcare, which is resulting in a massive surge in research and development activities.

Increasing use of gene modification in a variety of treatments, as well as the rising use of genome editing in biotechnology applications, are also expected to boost the demand for advanced gene therapies in the coming 10 years. Genetic testing plays a vital role in preventive healthcare driven by its rising awareness and knowledge among people. The rising prevalence of chronic disorders is boosting the use of genetic testing technologies. Furthermore, the growing popularity of DNA testing is generating profitable opportunities for genealogy product and service providers.

Gene delivery systems can effectively improve the treatment procedure and lead to effective results. Cell and gene therapies are expected to be vital in the treatment of diabetes, cancer, and cardiovascular diseases. Also, as the prevalence of hyperthyroidism and other chronic disorders is increasing in newborns, parents are widely opting for genetic testing services.

• The East Asian gene therapy market size is forecasted to reach US$ 8.42 billion by 2034.
• North America is estimated to account for 34.8% of the global gene therapy market share by 2034.

Report Attribute	Detail
Gene Therapy Market Size (2024E)	US$ 10.34 Billion
Forecasted Market Value (2034F)	US$ 38.52 Billion
Global Market Growth Rate (2024 to 2034)	14.1% CAGR
South Korea Market Value (2034F)	US$ 2 Billion
India Market Growth Rate (2024 to 2034)	22.4% CAGR
Key Companies Profiled	American Gene Technologies BioMarin Bluebird Bio Inc. Orchard Therapeutics Limited uniQure NV BioNTech Oxford Biomedica Plc. Celgene Corporation Shanghai Sunway Biotech Co., Ltd. Sibiono GeneTech Co. Ltd. Biogen Dendreon Pharmaceuticals LLC Spark Therapeutics Inc. Amgen Inc. Gilead Sciences Inc. Enzyvant Therapeutics GmbH Novartis AG Astellas Pharma, Inc.

Which Market Trends are Influencing Demand for Gene Therapy?

“Research and Development Activities Leading to Production of Novel Gene Therapy Solutions”

Ongoing research and development (R&D) activities are expected to have a significant influence of the sales of gene testing technologies. Several companies are aiming to build a gene therapy platform with a strategy centered on establishing a transformational portfolio through in-house faculty and enhancing those capabilities through strategic collaborations, R&D expansion, potential licensing, and merger & acquisition activities.

• The Cystic Fibrosis Foundation announced an investment in SalioGen Therapeutics in January 2022 to support the company's preclinical research into novel gene therapy for cystic fibrosis. SalioGen's Gene Coding approach is intended to activate, deactivate, or modify the function of any gene in the genome.

“Rigorous Clinical Trials Leading to Development of Novel Cell and Gene Therapies for Sickle Cell Disease”

For sickle cell disease, previously bone marrow transplant was the only and limited treatment procedure. Sickle cell disease is considered as a lifelong condition causing blockages in the blood vessels. This deadly disease if not treated effectively can lead to organ damages and strokes.

Researchers are highly focusing on the novel treatment procedures for sickle cell disease. The use of gene therapy and CRISPR technology is set to be effective for treating sickle cell disease.

• In 2023, after rigorous clinical trials carried out at Children’s Hospital of Philadelphia (CHOP) the United States Food and Drug Administration approved CASGEVYTM by Vertex Pharmaceuticals Inc. any LYFGENIATM by Bluebird Bio Inc. as first two gene therapies for the sickle cell disease for patients of 12 years and older.

Lyfgenia is a cell-based gene therapy while Casgevy is a type of novel genome editing technology. Thus, introduction of these technologies is representing and innovative advancements in the field of gene therapy.

Which Factors are Limiting Sales of Genealogy Products and Services?

“High Cost of Gene and Cell Therapies”

Gene therapies are expensive medical procedures that necessitate significant capital investment on the part of both the provider and the patient. Gene modifications necessitate precision equipment, which significantly raises the cost of cell and gene therapy and hinders their demand growth. Thus, high treatment cost is leading to the lower adoption of viral vector gene therapies.

Start-up Ecosystem

• StrideBio is a United States-based start-up that provides adeno-associated virus-medicated gene therapy solutions. The start-up’s portfolio includes drugs for genetic conditions such as Reavet Syndrome, Rett syndrome, and Froedrich’s ataxia.
• Generation Bio is an American start-up that offers non-viral gene therapy solutions. The company’s solution uses closed-ended deoxyribonucleic acid (ceDNA) system, which is capable of delivering multiple genes to a cell. The company also offers solutions for retina and liver disorders, which are in pre-clinical development and early stages.

Country-wise Analysis

What is the Demand Outlook for Gene Therapy in the United States?

“Strong Regulatory Frameworks and Positive Reimbursement Policies”

Attribute	United States
Market Value (2024E)	US$ 3.14 Billion
Growth Rate (2024 to 2034)	13.4% CAGR
Projected Value (2034F)	US$ 11.05 Billion

Presence of strong regulatory frameworks for promoting the development of cellular therapy and the existence of major biopharma companies are contributing to the growth of the United States market. The United States government is strongly funding centers and institutes who are engaged in gene therapy research and development efforts. Furthermore, the rising prevalence of chronic diseases, the presence of advanced healthcare infrastructure, and the availability of reimbursement policies are expected to boost the adoption of gene therapies in the coming decade.

Will India Be a Profitable Market to Invest In?

“Investments in R&D to Offer Lucrative Opportunities for Gene Therapy Solution Providers”

Attribute	India
Market Value (2024E)	US$ 569.9 Million
Growth Rate (2024 to 2034)	22.4% CAGR
Projected Value (2034F)	US$ 4.30 Billion

Optimization of vectors such as retroviruses and lentivirus, the introduction of new techniques such as induced pluripotent stem cells in conjunction with current gene editing models, and even trials in germ cells are set to boost the popularity of gene therapies in India over the coming 10 years. Increasing investments in healthcare infrastructure by central government are emerging as a notable gene therapy market trend in India. The country is also witnessing an increase in cancer cases, which is further encouraging the use of gene therapy in oncology treatments.

Which Aspects are Pushing Sales of Gene Therapy Solutions in Germany?

“Strong Presence of Regenerative Medicine Companies”

Germany is the best market for manufacturing cell and gene therapy products in Europe. Germany is at forefront in innovations and healthcare sector is no exception. Europe is home to more than 238 regenerative medicine companies, nearly to a quarter of the world's regenerative medicine therapeutic developers.

Category-wise Analysis

Which Vector Type is Gaining Traction in This Business Space?

“Adeno-Associated Virus (AVV) Safe and Well-tolerated Vector for Gene Therapy”

Attribute	Adeno-Associated Virus
Segment Value (2024E)	US$ 2.6 Billion
Growth Rate (2024 to 2034)	15.9% CAGR
Projected Value (2034F)	US$ 11.37 Billion

Vector is particularly a particle acting as a vehicle to carry any foreign nucleic sequence usually DNA. Adeno-associated virus (AAV) is gaining high attention as a vector for gene therapy especially in clinical-stage experimental therapeutic strategies. The ability to regenerate recombinant AAV particles lacking any viral genes is proving to be fastest move for gene therapies.

Which Disease is Gene Therapy Widely Used to Treat?

“Effective and Widespread Use of Gene Therapy to Treat Spinal Muscular Atrophy (SMA)”

Attribute	Spinal Muscular Atrophy (SMA)
Segment Value (2024E)	US$ 3.6 Billion
Growth Rate (2024 to 2034)	8.9% CAGR
Projected Value (2034F)	US$ 8.43 Billion

Spinal muscular atrophy (SMA) is a rare disease but very common fatal inherited diseases of infancy. Gene therapies are found to be very effective in treating SMA.

• In 2019 the United States Food and Drug Administration approved onasemnogene abeparvovec, SMN1 gene replacement therapy (brand name Zolgensma), for all children with SMA younger than two years of age.

Zolgensma is a recent advancement in the field of gene therapy to treat spinal muscular atrophy. Zolgensma is administered in a one-time infusion to patients less than 2 years of age.

Competitive Landscape

Some of the key players in the gene therapy market are Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd., Spark Therapeutics (Roche AG), and UniQure N.V. To secure a major position in the global market, various strategic alliances such as collaborations, acquisitions, and the launch of advanced products are employed by these industry leaders.

• In October 2021, the National Institutes of Health, the United States Food and Drug Administration, ten pharmaceutical companies, and five non-profit organizations announced a collaboration to accelerate the development of gene therapies for the 30 million Americans who suffer from rare diseases. Such initiatives are expected to boost demand for gene therapies in the coming decade.
• Novartis acquired Gyroscope Therapeutics in December 2021, adding a one-time gene therapy that is expected to transform care for geographic atrophy, a leading cause of blindness.
• Abecma (idecabtagene vicleucel), a cell-based gene therapy, was approved by the United States Food and Drug Administration in March 2021 to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy. Abecma is the first FDA-approved cell-based gene therapy for the treatment of multiple myeloma.

Key Segments of Gene Therapy Market Research

• By Vector Type :

  • Lentivirus
  • Adeno-Associated Virus (AAV)
  • Retrovirus & Gamma Retrovirus
  • Modified Herpes Simplex Virus
  • Adenovirus
  • Non-Viral Plasmid Vector
  • Others

• 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 Route of Administration :

  • Intravenous
  • Others

• By Region :

  • North America
  • Latin America
  • Europe
  • East Asia
  • South Asia & Oceania
  • Middle East & Africa