Gene therapies for the eye often lead the field of advanced therapeutics, introducing exciting new technologies and techniques to the clinic. Topics include the first FDA approved gene therapy (Luxturna), gene editing trials for inherited vision loss, and how a protein that helps algae move is being repurposed into vision saving technology.
Pharmacy is often the first port of call when an organisation wishes to use a gene therapy and needs to provide pragmatic guidance to facilitate the introduction of these innovative products. This document is designed to give practical advice to centres wishing to undertake gene therapy trials and need to understand the requirements and recommendations for governance and, in particular to have in place a Genetic Modification Safety Committee. The document also gives practical advice for centres to help them understand operational requirements including deciding the optimal location for preparation.
This guidance summarises the authorisation process for Genetically Modified Organisms (GMO) as Investigational Medicinal Products (IMP) for contained use (CU) within the United Kingdom (UK). These submissions are unique to GMOs, in addition to Clinical Trial Authorisation (CTA) and Ethics
HSE (Health and Safety Executive) guidance to help organisations to choose the most appropriate regulatory procedure, particularly those wishing to undertake studies involving the administration to humans of substances based upon genetically modified microorganisms (GMMs)
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