Streamlining Genetically Modified Organism Regulatory Classification in UK Gene Therapy Clinical Trials: A UK...

Area of Science:

• Biotechnology and Genetic Engineering
• Clinical Trial Regulation
• Gene Therapy Development

Background:

• Accurate classification of Genetically Modified Organisms (GMOs) is crucial for gene therapy clinical trials.
• Current UK regulatory processes for GMO risk assessments are fragmented, causing delays and inconsistencies.
• Patient access to novel gene therapies is hindered by complex trial initiation procedures.

Purpose of the Study:

• To develop a decision tree for streamlining GMO classification of Gene Therapy Investigational Medicinal Products (GTIMPs).
• To address challenges in clinical trial setup by providing a structured, evidence-based classification approach.
• To clarify international regulatory misalignments in hazard classification for GTIMPs.

Main Methods:

• Development of a novel decision tree tool for GMO classification.
• Focus on practical guidance for *in vivo* and *ex vivo* GTIMPs.
• Harmonization of decision-making and containment requirement clarification.

Main Results:

• The decision tree offers a structured approach to GMO classification for GTIMPs.
• It aims to reduce variability and duplication in risk assessments across trial sites.
• The tool provides guidance for common *in vivo* and *ex vivo* GTIMPs.

Conclusions:

• Adoption of the decision tree can accelerate gene therapy clinical trial setup in the UK.
• Harmonized classification can reduce variability in regulatory approaches.
• Implementing this tool strengthens the UK's competitiveness in GTIMP research and development.