Genome Editing

Genotype vs Phenotype - The impact of new insights from human genetics on the future of drug development

by Maria Schacker

The genetics of disease inheritance is becoming more and more important for the pharmaceutical industry. Until the early 2000s, the only way to study genetic diseases was through family studies and linkage analysis in families with high occurrences of disease. However, these studies are extremely tedious as they can only be done on a small scale and they are by and large only applicable to diseases that follow mendelian inheritance, not multifactorial diseases. In this article, Maria Schacker provides an overview on how the insights from human genetics can be used for the development of new drugs. 

How should we regulate genome editing for pharmaceutical applications?

by Dr. Diane Seimetz

Genome editing technologies are not only having a game-changing effect on research. They also have the potential to transform health care by curing diseases for which there are currently no or no satisfactory therapies available.

In particular with the emergence of novel gene editing technologies such as CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) in the pharmaceutical arena the question arises how to best regulate these technologies.

On 18 October 2017 the European Medicines Agency (EMA) organized an expert meeting on genome editing technologies used in medicinal product development. Diane Seimetz contributed to the meeting as invited expert and has summarized the discussion and critical aspects in this article.