Gene therapies are a type of Advanced Therapy Medicinal Product (ATMP) designed to deliver a functional gene or to permanently modify hematopoietic progenitors.
They 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 and importantly, are in favour with new decision-making appointees at the USA FDA.
On the US market, 16 gene therapies that meet this 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 betathalassemia (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. The most recently approved gene therapy is Kresladi (marnetegragene autotemcel) in March 2026 for severe Leukocyte Adhesion Deficiency-I.
Market analysts predict the gene therapy market (currently estimated by VacZine Analytics at $2.9bn in FY2024) will expand greatly by the mid-2030s as indications expand from ultrarare diseases into more prevalent conditions. Newborn screening/diagnosis and technological improvements in next.generation capsid and vector engineering will also drive growth. However, high product pricing 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.
This MarketVIEW product is a comprehensive Executive Presentation (.pdf) plus data worksheet (.xls), which gives a detailed situation review of the currently FDA-approved 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 who wishes to familiarize themselves with the gene therapy space for opportunity scanning.
THIS PRODUCT IS A SUMMARY PRESENTATION (~150 slides, .pdf) + interactive MS Excel Workbook(s) (~15 worksheets)
Photo credit: digitale.de (Unsplash)
*Prices quotes are for 1 region license only. Regions are North America, or EU or ROW.
For UK orders VAT will be added at 20%. On-line prices may include discounts and are converted from list price in US dollars (subject to currency fluctuations).
Please review the TERMS and CONDITIONS of purchase.
