Gene Therapy Market by Vector (Non-Viral Vectors and Viral Vectors), Indication (Neurological Diseases, Cancer, Duchenne Muscular Dystrophy, Hepatological Diseases, Others), Delivery Mode (In-Vivo and Ex-Vivo): Global Market Size Estimates and Forecast (2022-2030)

The market crossed the US$ 8.9 billion mark in 2022 and is expected to hit US$ 29.5 billion by 2030, recording a CAGR of 14.6% during the forecast period

The gene therapy market has been significantly growing with the increase in technological advancements

Gene therapies potentially have brought significant clinical benefits to patients by targeting the underlying cause of diseases. For example, instead of treating the symptoms of diseases, one-time gene therapy treatment has the potential to address the root cause. Nowadays, technology is making advancements based on numerous factors such as academic laboratories that have continued to pursue research for gene therapies. Also, significant progress has been made to develop a next-generation approach for numerous indications. For instance, In February 2022, Sarepta Therapeutics (US) announced a collaboration to use GenEdit’s NanoGalaxy platform and Sarepta’s gene editing technology to develop gene editing therapeutics to treat neuromuscular diseases.

Within the report, the market is segmented into vectors, indications, delivery mode, and geography. By vector, the market is further bifurcated into non-viral vectors and viral vectors. On the basis of indication, the market is segmented into neurological diseases, cancer, duchenne muscular dystrophy, hepatological diseases, and others. By delivery mode, the market is bifurcated into in-vivo and ex-vivo. Geographically, the market is subsegmented into North America, Europe, Asia Pacific, South & Central America, and Middle East & Africa.

Gene therapy is a technique that uses a gene to treat, prevent or cure a particular disease or medical disorder. Frequently, gene therapy works by adding new copies of a broken gene or replacing a defective gene in a patient's cells with a healthy version of that gene. As gene therapy is a direct way of treating both inherited genetic diseases and acquired disorders are treated with gene therapy. Gene therapy can work by several mechanisms, such as inactivating a disease-causing gene that is not correctly functioning or replacing a disease-causing gene with a healthy copy of a gene. Due to its advantages offered, many companies are developing gene therapy products. For instance, on March 26, 2021, the FDA approved idecabtagene vicleucel (brand name Abecma) for treating adult patients with relapsed or refractory multiple myeloma. This is the first FDA-approved cell-based gene therapy for multiple myeloma.

The rise in technology for gene therapy are the major factors accelerating market growth

Gene therapy offers a potentially life-long therapeutic effect without the need for repeated administration which is the main advantage of gene therapy technology. Gene therapy has been utilized to correct defective genes by introducing a functional copy of the gene, introducing a disease-modifying gene, or silencing a mutant allele using RNA interference. Neurodegenerative diseases are one of the indications of gene therapy. Neurodegenerative diseases are a heterogeneous group of multi-system disorders that affect the central nervous system, leading to neurodegeneration, for example, Frontotemporal dementia, Alzheimer's disease, and Parkinson's disease. These neurogenerative diseases are caused by mutation or cellular and circuit dysregulation.

Recent strategic developments in the gene therapy market

The gene therapy market has undergone several significant developments, and a few of these have been mentioned below:

• On  Aug 2022, Mayo Clinic, Hibiscus BioVentures, and Innoforce Pharma launched Mayflower BioVentures, a cell and gene therapy accelerator that is used to identify and form companies around technologies that address unmet patient needs. This academic and industry relationship will establish independent cell and gene therapy companies to advance the development of Mayo Clinic technologies through preclinical and early feasibility studies.
• On Sep 2022, Bayer’s new collaboration with Global Life Sciences Solutions aims to set industry standards for allogeneic cell therapy manufacturing. Combining the manufacturing expertise with Bayer’s expertise in cell therapy development will enable to the creation of a new generation of manufacturing solutions specifically designed to meet the developing needs of allogeneic cell therapy.
• In March 2022, the National Heart Lung and Blood Institute granted USD 12 million to an Indiana University School of Medicine researcher. The grant is for exploring major themes in a gene therapy approach to safer and potentially curative treatments for hemophilia.
• In May 2019, Locana, Inc., a leading RNA-targeting gene therapy company, received USD 55 million in Series A financing led by ARCH Venture Partners, with participation from Temasek and Lightstone Ventures (all existing investors). The company will utilize this fund for its RNA-targeting gene therapy pipeline programs.

The gene therapy market is driven by several players by implementing strategic activities such as investments, new launches, mergers & acquisitions, and partnerships with Biogen Inc., REGENXBIO Inc., Spark Therapeutics, Inc., MolMed S.p.A., Orchard Therapeutics plc., SIBIONO, Human Stem Cells Institute, F. Hoffmann-La Roche Ltd., Bluebird bio, Inc., Novartis AG are among the prominent players operating in the market.

The target audience for the report:

• Government bodies, such as regulating authorities
• End users
• Research institutes, organizations, and consulting companies

Scope of the report:

In this report, the market has been segmented on the basis of:

• Vector

• • Non-viral vectors
  • Viral vectors

• Indication

• • Neurological diseases
  • Cancer
  • Duchenne muscular dystrophy
  • Hepatological diseases
  • Other

• Delivery mode

• • In-vivo
  • Ex-vivo

• Region
  • North America
  • Europe
  • Asia Pacific
  • South & Central America
  • Middle East & Africa

• Companies Profiled

• • Biogen Inc.
  • REGENXBIO, Inc.
  • Spark Therapeutics, Inc.
  • MolMed S.p.A.
  • Orchard Therapeutics plc.
  • SIBIONO
  • Human Stem Cells Institute
  • F. Hoffmann-La Roche Ltd.
  • Bluebird bio, Inc.
  • Novartis AG

Companies profiled
- Biogen Inc.
- REGENXBIO, Inc.
- Spark Therapeutics, Inc.
- MolMed S.p.A.
- Orchard Therapeutics plc.
- SIBIONO
- Human Stem Cells Institute
- F. Hoffmann-La Roche Ltd.
- Bluebird bio, Inc.
- Novartis AG