How Advanced Therapies are Changing the Landscape of Rare Disease

After years of innovative research, advanced therapies are dramatically changing the way patients are treated for a wide variety of diseases particularly for patients with rare diseases and orphan indications. New developments in advanced therapy medicinal products (ATMPs) comprising cell and gene therapy products show the potential to modify or even cure severe chronic conditions for which previously no treatment existed. As a consequence, the dynamic market for innovative therapies such as CAR-T, immuno-oncology, and gene and cell-based therapies grows across the globe. Academic institutes and biotech companies are contributing to these developments both in terms of scientific discovery and undertaking clinical trials aiming for regulatory approvals to treat specific patient populations. While we might be in a golden age of medicine due to advancements in ATMPs, innovative clinical research and development remain crucial for getting treatments to patients.