In recent years, the pharmaceutical landscape for rare diseases has witnessed a remarkable evolution. Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than 300 million people worldwidei and account for a significant portion of the global clinical development pipeline. This blog delves into the rise of…
Kenneth Ndugga-Kabuye
Cell therapies, despite their transformative potential, face significant barriers that can hinder their development and commercialization. Safety concerns, manufacturing challenges, analytical complexities and scale-up difficulties present substantial risks. Competition from other drug modalities, coupled with the inherent complexity of clinical development, further complicates the pathway to market. Distribution and supply chain challenges add another layer…
Today there are over 10,000 rare diseases affecting more than 30 million people in the U.S.1 Many of these disorders are life-threatening, and more than 90% do not have a U.S. FDA-approved treatment2. Novel gene therapies offer hope for improving the health of patients afflicted with these conditions. Securing clearance for these treatments is…
Today there are over 10,000 rare diseases affecting more than 30 million people in the U.S.1 Many of these disorders are life-threatening, and more than 90% do not have a U.S. FDA-approved treatment2. Novel gene therapies offer hope for improving the health of patients afflicted with these conditions. Securing clearance for these treatments is…
Cell & Gene Therapy
Where Biomarkers and Gene Therapy Trials Intersect: Benefits Abound
We’re currently experiencing breakthrough times for clinical research in gene therapies. These complex treatments offer much-needed hope to more than 30 million U.S. patients affected by over 1000 rare diseases1, many of which are life-threatening and the vast majority of which have no available treatment. Gene therapy products must meet the same key regulatory…