Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products

4 Oct 2023

Abstract excerpt

In 2000, the European Union (EU) introduced the orphan pharmaceutical legislation to incentivize the development of medicinal products for rare diseases. The Committee for Orphan Medicinal Products (COMP), the European Medicines Agency committee responsible for evaluation of applications for orphan designation (OD), received an increasing flow of applications in the field of gene therapies over the last years....

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CAR-T cell therapy research
Share to CRISPR and Genetic Engineering
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 37794679
DOI: 10.1016/j.ymthe.2023.09.020

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal ProductsPMID 37794679Read ↗

Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutionsSemantic proximity Read ↗

Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare DiseasesSemantic proximity Read ↗

Development and Clinical Translation of Approved Gene Therapy Products for Genetic DisordersSemantic proximity Read ↗

Development of Cell and Gene Therapies for Clinical Use in the US and EU: Summary of Regulatory GuidelinesSemantic proximity Read ↗

Access to gene therapy for rare diseases when commercialization is not fit for purposeSemantic proximity Read ↗

Improving access to gene therapy for rare diseasesSemantic proximity Read ↗

Gene therapies development: slow progress and promising prospectSemantic proximity Read ↗

Clinical development of gene- and cell-based therapies: overview of the European landscapeSemantic proximity Read ↗

A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene TherapiesSemantic proximity Read ↗

BJCP-EMA commentaries on the guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells.Semantic proximity Read ↗

Strengthening health systems for access to gene therapy in rare genetic disordersSemantic proximity Read ↗

EMA commentary on the guideline on quality, nonclinical and clinical aspects of medicinal products containing genetically modified cellsSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.