Research and Development in new gene therapies (including genetically modified cell therapies e.g. CAR-T) is reportedly at its highest level to date. A recent 2025 analysis (ASCGT/Citeline) indicated ~2,200 gene therapy programs are active globally (ranging from preclinical through pre-registration) with ~800 being either in vivo or ex vivo clinical assets (mostly in Phase 1 and Phase 2). Rare diseases and anticancer are key areas in the gene therapy pipeline with rare diseases itself having oncology and non oncology sub categories. The most common non oncology rare diseases include: Beta thalassemia | Duchenne Muscular Dystrophy | Retinitis Pigmentosa | Amyotrophic lateral sclerosis | Scleroderma | Lupus | Myasthenia Gravis | Hemophilia A. Some of these indications already have FDA-approved gene therapy products e.g. Elevidys (delandistrogene moxeparvovec.rokl) marketed by Sarepta Therapeutics. There are important issues with first generation gene therapies with regarding to safety and efficacy which necessitates further research and development.
VacZine Analytics has conducted a separate new review of the gene therapy pipeline for the aforementioned 8 non oncology rare/orphan disease indications. Our analysis finds that 436 trials are registered globally with around 50% currently active/ongoing. 50% of the trials are operated by the industrial sector. 25 countries are represented in our analysis and overall, USA-based companies such as Pfizer, Spark Therapeutics Inc, PTC Therapeutics Inc, BioMarin and others still dominate the field. Gene therapy modalities varied by disease indications with adenovirus-associated (AAV) vectors, autologous approaches, and stem cell therapies predominating. Competitive and platform intensity varies widely across indications.
This MarketVIEW product is a comprehensive Executive Presentation (.pdf) plus data worksheet (.xls) outlining an up-to-date review of the gene therapy R&D sector. 8 rare disease indications are profiled along with key gene late stage therapy programs and their mechanism of action (MOA)/technology. This review digs deeper and investigates current drivers/potential whitespace areas for any client wishing an opportunity assessment of this exciting future area of transformative medicine.
THIS PRODUCT IS A SUMMARY PRESENTATION (~150 slides, .pdf) + interactive MS Excel Workbook(s) (~15 worksheets)
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