ReviewMolecular therapy : the journal of the American Society of Gene Therapy2025
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed.
- Editing the rulebook: building the policy environment for children to access gene therapy.Pediatric research · 2026Article
- Unlocking the curative potential of gene transfer and editing for hematopoietic disorders.Experimental hematology · 2026Review
- Preclinical efficacy of a gene therapy forMolecular therapy. Advances · 2026Article
- Advances in targeted and cellular therapies for relapsed/refractory mantle cell lymphoma: immunotherapeutic strategies and challenges.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2026Review
- Unifying the odyssey: artificial intelligence for rare disease diagnosis and therapy.Health and technology · 2026Article
- Leptin Receptor b (LEPRb) Mutations Disrupt Hypothalamic Control of the Reproductive Axis.International journal of molecular sciences · 2026Review
- Macrophage Extracellular Vesicles: Therapeutic Strategies for Corneal Fibrosis in Rare Diseases.Biomolecules · 2026Review
- SAVI: molecular mechanisms, clinical spectrum and precision medicine approaches beyond type-I IFN.Frontiers in immunology · 2026Review
- Melanoma vaccines: current R&D landscape, translational hurdles, and future outlook-a perspective drawn from 442 clinical trials.Frontiers in immunology · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene and cell therapies offer transformative potential for patients with rare and ultra-rare diseases. However, many treatments stall despite demonstration of safety and efficacy in pre-clinical studies and early-stage clinical trials. This market failure creates a barrier where otherwise successful therapies are unable to reach patients due to commercial non-viability. In March 2025, ASGCT held a workshop, "Establishing and Maintaining Access to Gene and Cell Therapy for Rare and Ultrarare Diseases," focusing on developing actionable paths forward to ensure that successful gene therapies reach patients regardless of commercial viability. The field faces ongoing and deep-seated challenges; addressing them will require coordinated action across private companies, regulatory agencies, and non-profit organizations to explore non-traditional business models. During the workshop and described here, ASGCT reviewed a matrix of solutions encompassing regulatory innovations, manufacturing efficiencies, financial modeling, and patient-focused frameworks to ensure that efficacious gene and cell therapies reach the patients who need them. Strategies included the creation of a temporary repository for deprioritized gene and cell therapy programs offering support while working to identify new sponsors to continue clinical trials and the creation of a consortium of developers focused on accelerating timelines and reducing costs.
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.