The global gene therapy market was valued at USD 3.61 billion in 2019 and is expected to grow to USD 35.67 billion by 2027, reflecting a compound annual growth rate (CAGR) of 33.6% during the forecast period. In 2019, the U.S. led the global market, accounting for 59.83% of the total share.
The gene therapy market is poised for remarkable growth, with a projected CAGR of 33.6%, fueled by rapid advancements in CRISPR-Cas9 and CAR-T-based gene editing technologies. Gene therapy represents a transformative approach, offering renewed hope for individuals suffering from rare and previously untreatable genetic disorders. By delivering functional genes through viral or non-viral vectors, gene therapy aims to correct defective, non-functional, or absent genes at their source.
Gene editing techniques can be applied to both somatic and germline cells, opening new frontiers in precision medicine. This cutting-edge technology has revolutionized treatment possibilities for chronic and life-threatening diseases, including cancer and spinal muscular atrophy (SMA). Celebrated for its potential as a single-dose cure, gene therapy works by directly modifying the underlying DNA, thereby addressing the root cause of disease rather than merely managing symptoms.
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Market Segmentation:
The gene therapy market is segmented by application into oncology, neurology, and others. In 2019, the neurology segment accounted for the largest market share, driven by the high cost and widespread use of treatments for spinal muscular atrophy (SMA). However, the oncology segment is anticipated to register significant growth, supported by a growing number of market participants focusing on cancer therapies and a strong pipeline of clinical and preclinical candidates.
By vector type, the market is classified into viral and non-viral vectors. Viral vectors led the market in 2019, with around 58% of gene therapies in development utilizing them — particularly Adeno-Associated Virus (AAV), recognized for its safety and therapeutic effectiveness. In contrast, non-viral vectors, such as bacterial vectors and plasmid DNA, are expected to grow at a slower pace.
From a distribution channel perspective, hospitals dominated the market due to superior availability and accessibility of gene therapy treatments. Meanwhile, clinics are projected to witness steady growth as more standalone facilities expand their offerings in advanced therapies for oncology and neurological conditions.
List Of Key Companies Profiled in Gene Therapy Market:
Novartis AG
Spark Therapeutics, Inc.
Biogen
Gilead Sciences, Inc
Amgen, Inc.
Jazz Pharmaceuticals, Inc.
Sarepta Therapeutics
GlaxoSmithKline plc
Other Prominent Players
Gene Therapy Market Driving Factors:
The global prevalence of rare genetic disorders continues to rise. According to the National Organization for Rare Disorders (NORD), spinal muscular atrophy (SMA) impacts approximately 1 in every 10,000 live births in the United States annually. Breakthroughs in gene therapy have opened new possibilities for treating conditions that were once deemed incurable. At the same time, cancer remains one of the most prevalent diseases worldwide. Recent data from the World Health Organization (WHO) and Globocan estimate that the five-year global cancer prevalence stands at approximately 43 million cases. Gene therapy is emerging as a vital tool in the prevention of certain cancers, especially for individuals with a genetic predisposition caused by inherited mutations.
Gene Therapy Market Restraining Factors:
Despite the increasing prevalence of chronic conditions like SMA and cancer, gene therapy remains one of the most expensive treatment options available. Promoted as a single-dose solution that addresses genetic defects at their source, the high cost continues to be a major barrier. For instance, Novartis AG prices a single dose of its cancer gene therapy drug, Kymriah, at USD 475,000 in the U.S. and USD 306,000 in Japan. Such substantial costs have led to hesitation among some insurance providers to offer reimbursement, which could potentially slow the growth of the gene therapy market in the years ahead.
Regional Analysis:
In 2019, the U.S. gene therapy market was valued at USD 2.16 billion, fueled by the high prevalence of chronic diseases, growing adoption of advanced therapies, and supportive reimbursement policies. As gene therapy remains unapproved in many developing nations, leading market players are focusing their product launches primarily in the U.S. Europe also shows strong acceptance of innovative treatments for rare diseases, backed by substantial research investments. According to the Alliance for Regenerative Medicine, over 100 companies in the region are actively involved in gene therapy development, further driving market growth. Meanwhile, the Asia Pacific region holds a smaller market share, largely due to lower adoption rates stemming from the high cost of treatment. However, countries like India are making progress by establishing regulatory frameworks aimed at facilitating the approval and commercialization of gene therapy products.
Key IndustryDevelopments:
June 2021 – Biogen Inc. reported positive Phase 3 STAR study results for timrepigene emparvovec (BIIB111/AAV2-REP1), an investigational gene therapy for choroideremia.
February 2021 – Spark Therapeutics announced the dosing of the first U.S. participant in an international gene therapy clinical trial for Pompe Disease.