The global gene therapy market was valued at USD 5.54 billion in 2023 and is projected to experience a robust compound annual growth rate (CAGR) of 18.88% from 2024 to 2030. This growth is driven by several key factors, including the expanding field of advanced therapies, continuous advancements in gene delivery technologies, and the growing competition among major players in the market. These companies are aggressively pursuing strategies such as acquisitions, mergers, collaborations, and partnerships to bolster their in-house expertise, strengthen their product pipelines, and accelerate the development and commercialization of gene therapies.

However, the COVID-19 pandemic has had a considerable negative impact on the gene therapy market's growth. The pandemic disrupted multiple aspects of the sector, including the supply of essential materials, manufacturing processes, and logistics operations. Companies faced significant delays in the delivery of key components and experienced shortages in clinical trial supplies, particularly when contract manufacturing organizations (CMOs) were forced to halt operations. These disruptions exposed critical vulnerabilities in the market’s supply chain and created hurdles for the timely development and distribution of gene therapies.

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Indication Segmentation Insights

In 2023, the spinal muscular atrophy (SMA) segment was the dominant driver of the gene therapy market. Although SMA is a rare genetic disorder, it is one of the most common fatal inherited diseases affecting infants. The approval of Zolgensma (AVXS-101), developed by Novartis, has revolutionized the treatment of SMA by targeting the underlying genetic cause of the disease. Zolgensma, which was approved by the U.S. Food and Drug Administration (FDA) in May 2019, has proven to be highly effective in altering the disease’s phenotype, effectively halting the progression of SMA in treated patients. As of now, Zolgensma is the only gene therapy that has been FDA-approved for SMA, marking a significant milestone in the use of gene therapies for treating serious inherited diseases.

Looking ahead, the Beta-Thalassemia Major and Sickle Cell Disease (SCD) segment is anticipated to register the fastest growth rate during the forecast period. Gene therapy for these conditions typically involves the transplantation of gene-modified hematopoietic stem cells, which can address the underlying genetic defects causing the disorders. Clinical and preclinical trials have demonstrated the safety and efficacy of these gene therapies, though challenges remain. Issues such as suboptimal gene expression levels, limited stem-cell dose and quality, inefficiencies in gene transfer, and toxicity related to myeloablative regimens continue to hinder the full potential of gene therapy in treating these blood disorders.

Despite these challenges, significant progress has been made in this segment. In June 2019, bluebird Bio's Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of beta-thalassemia. Zynteglo is also expected to receive FDA approval in the U.S. in August 2022. The product has already been granted Orphan Drug status by the FDA for the treatment of Sickle Cell Disease (SCD), underscoring its potential as a game-changing treatment for these conditions. Additionally, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics expanded their partnership to develop, produce, and commercialize CTX001, a gene therapy aimed at treating both sickle cell disease and beta-thalassemia. This collaboration and the achievements within the segment are expected to drive further adoption of gene therapies for these blood disorders, fueling the growth of this market segment. 

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