Rare Disease Day is observed on the 28th of February every year and aims to raise awareness and generate change for the 300 million people worldwide living with a rare disease, their families, and carers.
Rare diseases currently affect 5 percent of the worldwide population, and there are approximately 6,000 rare diseases that have been identified to date.
As 72 percent of all known rare diseases are genetic, cell and gene therapy has focused on rare diseases since the field’s earliest days.
Speaking with David McCall, Commissioning Editor at Cell & Gene Therapy Insights, Alan describes his career journey in the gene therapy space; the competitive landscape for rare disease gene therapies; the concept of gene therapy manufacturing platforms; the impact of accelerated clinical trial timeframes on CMC; challenges in clinical trial design for rare diseases, and how we can secure a bright future for gene therapies for rare diseases.
Click here to read the Viewpoint article: ‘Current trends in advanced therapy development and commercialisation for rare diseases.’ Cell & Gene Therapy Insights 2023; 9(1), 153–157 DOI: 10.18609/cgti.2023.023
To find out more about Rare Disease Day, visit: https://www.rarediseaseday.org/what-is-rare-disease-day/