ReviewMolecular therapy. Methods & clinical development2021
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs.
Review in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
22 citing papers in PubMed.
- Nanocarrier-Based Gene Delivery Systems: Mechanisms, Clinical Translation, and Future Perspectives.Biotechnology and bioengineering · 2026Review
- Microbial medicines: Unlocking the therapeutic potential of the microbiome in cancer treatment.Journal of controlled release : official journal of the Controlled Release Society · 2026Review
- Cell and Gene Therapy in Equine Ocular Disease.Veterinary ophthalmology · 2026Review
- FDA first to global follow-on: alignment in expedited oncology approvals across EMA, TGA, and PMDA.Frontiers in pharmacology · 2026Article
- Developing and validating a regulatory science competency framework for immune cell therapy: a mixed-methods study.Frontiers in medicine · 2026Article
- Policies to promote affordability and access across the life cycle of costly new drugs.BMJ (Clinical research ed.) · 2025Article
- Cell-engineered technologies for wound healing and tissue regeneration.npj biomedical innovations · 2025Review
- Artificial Intelligence in Personalized Medicine for Diabetes Mellitus: A Narrative Review.Cureus · 2025Review
- Blood and urine-based biomarkers in prostate cancer: Current advances, clinical applications, and future directions.The journal of liquid biopsy · 2025Review
- Review
- The Application of Epicardium in Heart Failure Treatment: Opportunities and Challenges.International journal of medical sciences · 2025Review
- Development of Cell and Gene Therapies for Clinical Use in the US and EU: Summary of Regulatory Guidelines.Current gene therapy · 2025Review
- The Impact of COVID-19 on RNA Therapeutics: A Surge in Lipid Nanoparticles and Alternative Delivery Systems.Pharmaceutics · 2024Review
- Advances and Challenges in Gene Therapy for Inherited Retinal Dystrophies: A Comprehensive Review.Cureus · 2024Review
- Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape.Healthcare (Basel, Switzerland) · 2024Article
- Search, identification, and curation of cell and gene therapy product regulations using augmented intelligent systems.Frontiers in medicine · 2023Article
- Planning for progress: A US regulatory approach to advancing the clinical development of gene therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Gene therapy in haemophilia: literature review and regional perspectives for Turkey.Therapeutic advances in hematology · 2022Review
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
- Advanced Therapy Medicinal Products' Translation in Europe: A Developers' Perspective.Frontiers in medicine · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Various factors exacerbate the issues in commercializing products across regions, including the lack of established regulatory frameworks for developing gene therapy products in many jurisdictions. While some countries have established separate regulatory frameworks for advanced therapies/regenerative medicine products, differences exist between them. Recommended solutions to overcome these hurdles include fostering convergence among countries with separate regulatory frameworks for these products and utilizing reliance and recognition for countries without such frameworks. Additionally, regulators who choose to establish new dedicated frameworks for regulating gene therapies should consider the inclusion of key elements such as expedited regulatory pathways that offer early engagement with regulators, innovative clinical trial design, and adequate post-market confirmatory studies. Increasing the alignment of regulatory pathways across countries will be crucial to facilitating the development of, and access to, gene therapies on a global scale.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.