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...
Tag: gene therapy 3 results
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...
Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for...