Introduction

Over the past five years, six gene therapy products have been approved by the US FDA and/or the European Commission, and the development pipeline has increased from around 300 to nearly 3,000 candidates. The extraordinary blossoming of this scientific field in recent years comes after several decades of dogged research and development in academic laboratories and to a fluctuating degree by industry. Those years were beset by setbacks, with patient deaths and commercial challenges rightfully giving rise to circumspection and skepticism, but continuing research and growing understanding has led us to the point where we can celebrate the launch of life-changing treatments for conditions including a rare form of blindness and spinal muscular atrophy.

As gene therapy comes of age, companies in the space are proliferating and big pharma is squarely back in the game, evidenced by the high number of M&A and partnership deals centered on such technology and the commercialization of approved therapies by the likes of Novartis and Roche.

Gene therapy encompasses multiple technical approaches to altering the genetic code in target cells, and this diversity of technology offers hope that previously intractable problems in many different diseases may ultimately be addressed therapeutically. With the recent approval of new gene therapies, new debates have opened up over their pricing and acceptable payment models, especially as their scope promised to expand from a limited number of rare diseases to a broader array of orphan indications and potentially more common conditions.

This eBook brings you a selection of key articles from our publications, ranging from a broad overview of the field from In Vivo to commercial strategy and R&D analysis from Scrip and policy and regulatory considerations from Pink Sheet, covering key markets from the US and EU to Japan and China.

Eleanor Malone Editor In Chief, Europe | Scrip, Pink Sheet, In Vivo, Generics Bulletin

Informa Pharma Intelligence