EMA issues gene therapy draft guidance

EMA released for consultation draft guidance for gene therapies, saying the document was designed for smaller companies and academics unfamiliar with the regulatory environment.

The document describes the agency's thinking on quality and manufacturing of therapies, including specifications for obtaining vectors and genetic elements and ensuring the purity and stability of batches. The guidance also outlines practices for non-clinical development, with a goal of optimizing dose selection and methods of administration. ...