Life Sciences Industry Report - Part 6

R&D
Life Sciences Industry Report - Part 6

There are some 7,000 known rare diseases in the world. Yet, despite determined recent endeavours to attempt to address unmet needs in numerous rare diseases – including the transformative period of rare disease therapeutics post-2010 – the very nature of these diseases means that many complex challenges remain in development of new therapies, as well as the ability to diagnose such diseases earlier.

In Part 6 of the Life Sciences Industry Report, we’ve collated key thought leadership from 2024 that looks at ways in which to navigate such complexities, dives into both the opportunities that are now available with advanced technologies and coming next generation technologies, and explores the challenges that must yet be overcome.

And, looking to the future of clinical trials in the rare disease space, Part 6 presents also an overview of the potential of gene therapies and of precision medicine in the ongoing search for rare disease solutions.

Part 6 of the Life Sciences Industry Report 2025 aims to provide informed insight into an industry very much at the height of its discovery and development capabilities, on the precipice of truly impressive, paradigm shifting innovation and, vitally, paving the way to broader access to new and earlier rare disease treatments that will permit better quality of life and patient outcomes overall.

The outlook for drug development to treat rare diseases looks encouraging, offering a glimmer of hope to the approximately 400 million people globally who live with these conditions.