Gene therapies are a type of Advanced Therapy Medicinal Product (ATMP) designed to deliver a functional gene or to permanently modify cell progenitors. “Classical” gene therapies utilize vectors such as adeno-associated virus (AAV), lentiviral vectors, or others for directly administered in vivo use or ex vivo engineered (CD34+) autologous hematopoietic stem cell products. These therapies aim to provide one-time treatments for long-term disease control or cure in monogenic disorders. Importantly, gene therapy approaches are favoured with new decision-making appointees at the USA FDA.
On the US market, 15 gene therapies* that meet the above “classical” classification are FDA-approved, with the first being Luxturna (voretigene neparvovec) in 2017/18, marketed by Spark Therapeutics for Inherited retinal disease (RPE65 mutation). Other gene therapies approved since this time target a range of indications, including transfusion-dependent beta-thalassemia (Zenteglo), sickle cell disease (Lyfgenia/Casgevy), and other inherited hematologic or immune disorders. Novartis Zolgensma/Itvisma (approved in 2019) for spinal muscular atrophy (SMA) is currently the bestselling gene therapy with ~$1.2bn in revenues in 2024.
Market analysts predict the gene therapy market (currently estimated by VacZine Analytics at $2.9bn in FY2024) will expand significantly to tens of billions of dollars by the mid-2030s as indications expand from ultrarare diseases into more prevalent non-orphan conditions. New-born screening and diagnosis and technological improvements in next-generation capsid and vector engineering will also drive growth. However, high product pricing (often >$1m per treatment) coupled with uncertain reimbursement policies still pose issues for the market outlook. Manufacturers are faced with constrained capacity, especially in the autologous space (CD34+). The threat of competition from non-viral delivery systems, e.g., lipid nanoparticles and off-the-shelf cell or antibody-based therapies, is likely to moderate uptake and keep access concentrated in specialized centers. Safety concerns that have arisen with certain products Elevidys/Skysona cast a shadow over the sector calling for tighter regulatory scrutiny.
The Approved Gene therapies Situation Review is a comprehensive Executive Presentation (.pdf) plus data worksheet (.xls), which provides a detailed overview of the currently FDA-approved “classical” gene therapy landscape. The analysis includes a disease profile for the 11 relevant indications, 15 gene therapy product audits, along with their competitor/commercial performance, and total market assessment. All the latest relevant issues are covered, ranging from adoption case studies/technological advancements. This analysis is the ideal starting point for any client that seeks to familiarize themselves with the gene therapy space for opportunity scanning, competitive benchmarking and portfolio strategy.
Product link: https://www.vaczine-analytics.com/products-marketviewVAMVG_gene_therapies_1.asp
*There is a total of 40-45 cellular and gene therapy products that are FDA approved as of early 2026
Photo credit: digitale.de (Unsplash)
1) US Food and Drug Administration. Approved Cellular and Gene Therapy Products. Available at: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed January 2026
VacZine Analytics is a strategic research publisher based in the United Kingdom since 2007. We provide high-quality disease and commercial analysis to those working within or in collaboration with the vaccine and gene therapy industries.
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