Gene Therapy at a Strategic Inflection Point
In 2025, gene therapy advanced with intention.
Innovation continued to enter the pipeline, yet progression through clinical development remained measured. Early-stage programs dominated, reflecting a field focused on optimisation, validation, and long-term durability rather than rapid late-stage expansion.
Clinical momentum remained strong, delivery platforms demonstrated structural stability, and editing technologies continued to evolve beneath the surface. At the same time, geographic concentration of new trial activity signalled a more focused and competitive global landscape.
Part 1 of the 2025 Landscape Review examines the Drug and Trial Landscape in depth – providing a clear view of where the science stood at the close of 2025.
Report Contents
The Drug Landscape
A view of how the gene therapy pipeline evolved in 2025, where innovation concentrated, which indications sustained momentum, and how platform technologies stabilised across the landscape. This section explored the balance between pipeline expansion and measured clinical progression, revealing where true advancement occurred beneath the surface growth.
The Trial Landscape
A closer examination of global clinical activity in motion. From early-stage optimisation to geographic concentration, this section unpacked how trials progressed throughout the year and what shifting patterns suggested about the field’s strategic direction.
The Commercial Market
An assessment of a maturing commercial environment. As capital discipline reshaped investment behaviour, this section explored how partnerships, deal structures, and company strategy evolved, highlighting what confidence looked like in a more selective market.
The Future of Gene Therapy
A forward-looking perspective on the milestones set to shape 2026. With pivotal readouts, regulatory decisions, and competitive inflection points ahead, this section outlined where momentum was building and where the next breakthroughs could emerge.
Regulatory Announcements
Insight into the regulatory pulse of 2025. From development-enabling interactions to late-stage filings, this section examined how programmes engaged with agencies and what those trends signalled about readiness for commercial transition.
Previous Gene Therapy Landscape Reviews
Gene Therapy 2024 H1 Landscape Review
An analysis of the Gene Therapy landscape in H1 of 2024
Gene Therapy 2024 Landscape Review
An analysis of the Gene Therapy landscape in 2024
Gene Therapy 2025 H1 Landscape Review
An analysis of the Gene Therapy landscape in H1 of 2025
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- The Most Comprehensive Way to Search the Gene Therapy Development Landscape: Unique search ontologies based on Gene Therapy drug and trial characteristics
- Market Leading Approach to Gene Therapy Preclinical & Clinical Data Curation: Manual curation of key Gene Therapy research and development data points by Gene Therapy research scientists
- Seamlessly Integrate Commercial & Scientific Data all in One Place: Search Gene Therapy company and deal data based on their pipeline and technologies.
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