Since the 1986 approval of Muromonab, the first therapeutic monoclonal antibody (mAb), used to treat steroid-resistant transplant patients, mAbs have rapidly evolved and gained clinical ground become the largest class of biopharmaceuticals. During this period, mAbs have garnered a reputation for safety, favourable PKPD, and high levels of specificity that have made them a preferred drug modality in many therapeutic applications.
Antimicrobial resistance (AMR) is on the rise. By 2050, AMR may be killing more people than cancer does now. Already, the mortality rates and economic impact are alarming. According to the Centre for Disease Control the total cost of AMR in the USA is estimated at $55bn and results in over 35,000 deaths each year. The worldwide death toll is ticking over 700,000.
Little more than 10 years ago, the prospects for gene therapy were bleak. Early clinical trials had served to highlight the risks. In particular, the 1999 death of Jesse Gelsinger proved a turning point, and clinical progress stalled for years. The risks are now better understood and controlled, and in recent years so much has changed. The FDA's 2017 approval of the first human gene therapy drug, Luxurna, heralded a new era with a further twenty gene therapies approved by 2019 with 1000 more in clinical trials.
16 years on from the groundbreaking development of induced Pluripotent Stem Cells (iPSCs), the scientific community has generated an explosion of applications in the areas of high throughput drug discovery and developmental biology research. Personalised regenerative medicine and cell-based therapies are also on the horizon. But after all these years, iPSC-based therapy remains in its infancy. What are the future prospects?