Gene therapy

Drug Discovery in a Multi-Omics World By Mike Furness Collectively rare diseases affect millions of people worldwide, but the number of people with any one condition is very small. Finding a cure is difficult as the market is not large enough to justify a big investment in drug development. This is all about to change […]

Chimeric Antigen Receptor T-cells (CAR-T): Looking beyond the hype By Christoph Oldenburg, Dr Markus Thunecke and Dr John Herrmann While Chimeric Antigen Receptor T-cells (CAR-T), have already demonstrated convincing efficacy in a specific setting, the jury is still out on whether they are a ‘game-changer’ for oncology as a whole and can justify current valuations […]

Therapeutic Drug Repurposing, Repositioning and Rescue Part IV: Financial model and analysis By David M. Kauppi and Dr Stephen Naylor There is a growing consensus that Drug Repurposing, Repositioning and Rescue (DRPx) impacts all stakeholders involved in the therapeutic drug sector. In part, this is due to the fact that the pharmaceutical industry accrues ~25% […]

By Dr Jean- Pierre Wery Optimal treatment for any disease is one that can cure or prevent spreading with minimal impact on the patient’s quality of life. In the case of cancer, therapeutic agents were initially designed to kill rapidly dividing cells. These traditional treatments, namely chemotherapy and radiotherapy, remain the backbone of current therapy. […]

Valuation In Pharma Portfolio Management By Dr Michel Azoulay Throughout the course of my career, I have been involved in a wide range of portfolio decisions and continue to be involved in them today. Over the years, it has been observed that despite cultural differences, pharma teams face similar problems. Portfolio management includes multiple challenges […]

Phenotypic Drug Discovery: Striving towards the highest level of biological relevance By Dr John Comley Phenotypic drug discovery (PDD) implies screening where the molecular mechanism of action is not assumed and does not require knowledge of the molecular target. As such phenotypic drug discovery is comparable to empirical screening, which was historically used in drug […]

How Neutron Science Has Enabled Innovation in Drug Design and Delivery By Dr Giovanna Fragneto Meeting global health challenges, from cancer to viral infections, requires a profound understanding of living processes – not just at the level of cells but right down at the molecular level. And designing the most efficient therapeutics requires an in-depth […]

The California Institute for Regenerative Medicines Human iPSC Initiative By Dr Thomas J. Novak This article discusses large-scale generation of patient-derived induced pluripotent stem cells (iPSCs) to accelerate the understanding of disease biology and drug discovery. In 2012, the California Institute for Regenerative Medicine (CIRM) announced its Human iPSC Initiative, an ambitious effort to generate […]

The Increasing Impact of Precision Research Models on Drug Discovery and Development Dr Amar Thyagarajan Animal models are the standard by which we gain insight into the complex biological processes that underlie normal human physiology and disease states. Rodent models such as rats and mice have been used to understand various aspects of human physiology, […]

mRNA Will Revolutionise the Biopharmaceutical Industry By Dr Ingmar Hoerr Messenger RNA (mRNA) – the naturally occurring molecule honed over thousands of years of evolution to translate accurately and efficiently the information encoded in a cell’s DNA into the proteins essential for maintaining cell function and ensuring an organism’s survival, will revolutionise the biopharmaceutical industry. […]