Gene Therapy Market Is Estimated To Witness High Growth Owing To Increasing Prevalence of Genetic Disorders

The gene therapy market is driven by the increasing prevalence of genetic disorders and growing R&D investment in gene therapy treatment. Over 7,000 rare genetic diseases have been identified to date, and only 5% of them have approved treatment options. Gene therapy offers potential for a one-time administration to provide a long-lasting therapeutic effect. Furthermore, drug manufacturers are focusing on developing novel gene therapies for various genetic diseases such as cancer, neurological disorders, and cardiovascular diseases. For instance, clinical trials are ongoing for gene therapies targeting Duchenne muscular dystrophy and hemophilia.

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Market Trends:

Viral vector-based gene therapy is gaining immense popularity in the market owing to its high efficiency. Retroviruses and lentiviruses are commonly used viral vectors to integrate the functional gene into the host cell genome. Chinese Hamster Ovary (CHO) cells are extensively utilized bioreactors for commercial-scale manufacturing of viral vectors for gene therapies.

Leading players are also focusing on developing non-viral gene therapies to overcome several challenges associated with viral vectors such as immunogenicity, insertional mutagenesis, and luggage size restrictions. For instance, CRISPR/Cas9 gene editing is a promising non-viral approach for correcting mutations associated with genetic disorders.

Market Opportunities:

The global gene therapy market was valued at US$ 9 billion in 2023 and is anticipated to witness a CAGR of 19.3% during the forecast period of 2024-2031. Gene therapy offers a potentially curative approach for various genetic disorders by correcting defective genes or replacing missing genes. Two major market opportunities in the rapidly growing gene therapy market are discussed below.

Viral vector gene therapy accounted for the largest share of the global gene therapy market in 2023. Within viral vectors, retroviral vectors dominated the market owing to their ability to efficiently and stably integrate transgenes into the genomes of dividing cells. Lentiviral vectors are also gaining popularity due to their ability to transduce both dividing and non-dividing cells.

The in vivo delivery method held the largest market share in 2023 due to the advantages it offers over ex vivo delivery methods. In vivo gene therapy involves direct administration of vector materials into target tissues and enables broad tissue distribution through systemic delivery methods like intravenous injections. This approach obviates the need for cell isolation, ex vivo manipulation and transplantation required for ex vivo delivery.

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Key Market Takeaways

The global gene therapy market is anticipated to witness a CAGR of 19.3% during the forecast period of 2024-2031, driven by the ability of gene therapy to provide long-term cure and prevent the progression of diseases.

On the basis of vector, viral vectors segment dominated the market in 2023 led by retroviral and lentiviral segments.

Based on delivery method, the in vivo segment held the largest share in 2023 due to advantages over ex vivo delivery.

In terms of indication, cancer was the leading segment in 2023 owing to rising cancer incidence and the curative potential of gene therapy for cancer.

Regionally, North America dominated the global market in 2023 owing to higher adoption and presence of major market players in the region.

Key Players Insights

• Merck KGaA

• REGENXBIO, Inc.

• Oxford BioMedica plc

• Dimension Therapeutics, Inc.

• Bristol-Myers Squibb Company

• SANOFI

Recent Developments

In May 2023, the FDA approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months and older with dystrophic epidermolysis bullosa (DEB) and mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
In April 2023, REGENXBIO Inc. announced that the FDA has given fast track designation to RGX-202, a possible one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne).

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