Advanced therapies, and particularly gene therapies have gained increasing popularity over the recent years. Especially gene therapies offer a potential curative treatment that would eliminate the disease by correcting a misfunctioning or missing gene in a single treatment application. In 2018, the US FDA released six guidance documents that reflect the agencies current thinking based on the experience gained in clinical application.

Gene editing is one of the newer technologies that can be used to manipulate genes. However, the clinical experience to-date is limited and the increased residual risk is warrants additional safeguards and restrictions in clinical research. In November 2018, a Chinese scientist announced that the first gene edited babies were born. The revelation stirred a firestorm of controversy over the ethical acceptability of gene editing embryos for implantation due to the remaining uncertainty regarding safety and efficacy.

The presentation will summarize the development of gene therapy as promising treatment option and look at some of the techniques employed for gene therapies and gene editing. We will then look at the scientific and ethical implications of the birth of the Chinese gene-edited twins, Nana and Lulu.

Kirsten Messmer, PhD, RAC is a Principal Regulatory Affairs Specialist in the Regulatory Intelligence, Policy and Advocacy team at PPD. Dr. Messmer received her PhD in Neuroscience from University of Sheffield and a Biology Diplom from the Eberhard-Karls University in Tübingen, Germany. She completed post-doctoral research fellowships at the Royal Free Hospital and University College London and the University of Maryland, Baltimore before joining Theradigm to develop stem cell therapies for neurodegenerative diseases. In her position at ERA Consulting, Dr. Messmer was responsible for authoring regulatory intelligence newsletters, consulting on biopharmaceutical product development and including US FDA interactions. In her current position at PPD, she is part of a specialist team providing regulatory intelligence to clients and within PPD to support efficient, compliant and successful clinical research and drug development.