Dataset Information

Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.

ABSTRACT: Advanced therapy medicinal products (ATMPs) have a massive potential to address existing unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may potentially provide cure for several genetic diseases. In Europe, the ATMP regulation was fully implemented in 2009 and, at this point, the Committee for Advanced Therapies was created as a dedicated group of specialists to evaluate medicinal products requiring specific expertise in this area. To date, there are three authorized GTMPs, and the first one was approved in 2012. Broad research has been conducted in this field over the last few decades and different clinical applications are being investigated worldwide, using different strategies that range from direct gene replacement or addition to more complex pathways such as specific gene editing or RNA targeting. Important safety risks, limited efficacy, manufacturing hurdles, or ethical conflicts may represent challenges in the success of a candidate GTMP. During the development process, it is fundamental to take such aspects into account and establish overcoming strategies. This article reviews the current European legal framework of ATMPs, provides an overview of the clinical applications for approved and investigational GTMPs, and discusses critical challenges in the development of GTMPs.

SUBMITTER: Carvalho M

PROVIDER: S-EPMC5662580 | biostudies-other | 2017

REPOSITORIES: biostudies-other

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Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.

Carvalho Marta M Sepodes Bruno B Martins Ana Paula AP

Frontiers in medicine 20171026

Advanced therapy medicinal products (ATMPs) have a massive potential to address existing unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may potentially provide cure for several genetic diseases. In Europe, the ATMP regulation was fully implemented in 2009 and, at this point, the Committee for Advanced Therapies was created as a dedicated group of specialists to evaluate medicinal products requiring specific expertise in this area. To date, there are three authorized G ...[more]

PMID: 29124055

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Project description:The aim of European water policy is to achieve good ecological status in all rivers, lakes, coastal and transitional waters by 2027. Currently, more than half of water bodies are in a degraded condition and nutrient enrichment is one of the main culprits. Therefore, there is a pressing need to establish reliable and comparable nutrient criteria that are consistent with good ecological status. This paper highlights the wide range of nutrient criteria currently in use by Member States of the European Union to support good ecological status and goes on to suggest that inappropriate criteria may be hindering the achievement of good status. Along with a comprehensive overview of nutrient criteria, we provide a critical analysis of the threshold concentrations and approaches by which these are set. We identify four essential issues: (1) Different nutrients (nitrogen and/or phosphorus) are used for different water categories in different countries. (2) The use of different nutrient fractions (total, dissolved inorganic) and statistical summary metrics (e.g., mean, percentiles, seasonal, annual) currently hampers comparability between countries, particularly for rivers, transitional and coastal waters. (3) Wide ranges in nutrient threshold values within shared water body types, in some cases showing more than a 10-fold difference in concentrations. (4) Different approaches used to set threshold nutrient concentrations to define the boundary between "good" and "moderate" ecological status. Expert judgement-based methods resulted in significantly higher (less stringent) good-moderate threshold values compared with data-driven approaches, highlighting the importance of consistent and rigorous approaches to criteria setting. We suggest that further development of nutrient criteria should be based on relationships between ecological status and nutrient concentrations, taking into account the need for comparability between different water categories, water body types within these categories, and countries.

Dataset Information

Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.

Publications

Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework.

Similar Datasets

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