A Data Driven View of Late-Stage Gene Therapy Progress
This report examines how leading developers, emerging technologies, and evolving regulatory frameworks are shaping the next generation of gene therapies approaching approval. Drawing on Beacon Gene Therapy’s expertly curated dataset, it provides detailed analysis of late-stage clinical progress, approval submissions, and recent setbacks across non-oncology gene therapy programs.
From metabolic and neurological disorders to rare and ultra rare diseases, the report explores how scientific innovation and regulatory adaptation are influencing development strategies. Whether your focus is scientific advancement, competitive positioning, or strategic planning, this report offers the insights needed to navigate an increasingly complex late-stage environment.
Key insights:
- Comprehensive late-stage landscape: Analysis of 2,700+ tracked gene therapy assets, with only 20 approved therapies, highlighting the narrow funnel from development to approval.
- Clinical momentum and risk: Coverage of 23 ongoing pivotal trials, with strong activity in metabolic and neurological indications alongside notable clinical holds and delays.
- Regulatory and submission activity: In-depth review of recent BLA and MAA submissions, withdrawn applications, and CMC-driven regulatory challenges shaping timelines.
- Technology evolution: Insights into the continued dominance of viral gene replacement alongside growing momentum in CRISPR-based gene editing and emerging delivery platforms.
- Regulatory innovation: Examination of the FDA’s new plausible mechanism pathway and its potential impact on personalized and small-population gene therapies.
- What’s next: A forward-looking view of expected approval milestones through 2026 and their implications for the competitive landscape.
You May Also Be Interested In…
Learn More About Gene Therapy
- 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.
- Unrivalled Granularity in Reports & Analysis: Our search ontology and technology indexing enable comprehensive analysis not seen elsewhere.
What is Beacon?
Beacon is a world leading data analysis tool for preclinical and clinical trial information. With unrivalled granularity of search function, data quality, and exhaustiveness, Beacon provides timely information and gives you confidence to progress your targeted drug therapies to patients.
Learn more about how our clinical trials and drug database solutions can be tailored to your research requirements
