What's in a word? Defining "gene therapy medicines"

Area of Science:

• Pharmacology
• Genetics
• Regulatory Science

Background:

• Gene therapy medicinal products (GTMPs) represent a significant advancement in medicine.
• Accurate definitions are crucial for regulatory frameworks and scientific understanding.
• The European Commission proposed new pharmaceutical legislation in April 2023.

Discussion:

• The proposed legislation introduces a broader definition of GTMPs.
• This includes genome editing medicines and nucleic acids, regardless of source.
• The definition covers interventions regulating, replacing, or adding genetic sequences via transcription or translation.

Key Insights:

• The new definition is comprehensive, applying to any "genetic" intervention.
• It is agnostic to the mechanism, duration, location, or risk associated with the therapy.
• Terminology and definitions have profound implications for regulation, science, and public health.

Outlook:

• The broad definition necessitates careful consideration of its impact on drug development and approval.
• Clear definitions are vital for navigating the evolving landscape of genetic medicines.
• Future regulatory strategies must adapt to the expanding scope of GTMPs.