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Navigating Through Uncharted Waters

Gene-based therapies are members of a larger therapeutic category characterised as advanced therapy medicinal products (ATMP). Most stakeholders recognise the potential impact of new gene-based therapies: improved outcomes and quality of life for patients with some of the most complex and chronic genetic conditions, which can be presented with considerable clinical variation.

In particular, the industry witnessed several milestones in 2017, including approval by the FDA that allowed the first gene-based treatment for an inherited eye disease in the US (1). Earlier in 2017, the FDA also cleared two other gene-based treatments targeting advanced haematologic malignancies (2).

Such industry developments underscore a renewed readiness to advance gene-based clinical trials after a series of high-profile setbacks in the late1990s prompted the FDA and the EMA to develop additional guidance for clinical trials involving gene-based therapies − including oversight procedures emphasising short- and long-term patient safety. A layered and either sequential or overlapping review process includes institutional review boards, institutional biologic committees, and the National Institutes of Health assuring protocol adherence and proper handling of therapeutics.

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Dr Michael F Murphy’s professional career has spanned 30 years, and his positions emphasise the integration of medical and scientific acumen with operational excellence. He is board-certified in psychiatry, with a doctorate in pharmacology, training at Tulane University, Stanford University, and the Icahn School of Medicine at Mount Sinai, US. In 2017, he was the recipient of the Clinical Research and Excellence Lifetime Achievement Award and selected as one of the 100 Most Inspiring People in the life sciences industry. His supervisory responsibilities as Chief Medical and Scientific Officer at Worldwide Clinical Trials are international in scope.

Dr deMauri Mackie is a member of a unique fellowship programme at Worldwide Clinical Trials, which combines didactic and experiential training across R&D activities for novel chemical entities, biological products, and devices. She has a BA in biochemistry from the University of Pennsylvania, US, and a PhD in cellular and molecular medicine from Johns Hopkins University, US.

Sophie Humphrey has worked in the clinical research industry for over 10 years, five of which have been at Worldwide Clinical Trials, filling lead roles within regulatory affairs, business development, and study start-up in fast-paced CRO environments. Sophie has experience of submission management, as well as providing expertise on legislative framework, project representation, and strategy on regulatory and development activities of trials. Her regional experience extends to EU, the Middle East, Africa, and Asia-Pacific across a wide range of therapeutic indications.

Dinah Otieno has worked in global clinical study start-up and regulatory affairs for the past decade and has been the Global Start Up and Regulatory Manager at Worldwide Clinical Trials since 2016. Dinah is responsible for site regulatory compliance and drug release activities for country site activation. She has over 17 years’ experience within the academic and CRO industry, where she oversees preparation of various strategic and process improvements for streamlined working procedures in regulatory and clinical start-up submission to country-specific regulatory agencies. Dinah obtained her undergraduate degree in Psychology from the University of North Carolina at Greensboro, US.
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Dr Michael F Murphy
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Dr deMauri Mackie
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Sophie Humphrey
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Dina Otieno
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