Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. The Cell and Gene Therapy (CGT) field has unprecedented potential to effectively treat and/or cure...
Expertise: Cell & Gene Therapy 56 results
Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. In this blog, we will address common questions we receive from our clients and aspects to consider when designing those nonclinical...
Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus...
Cell & Gene Therapy
Operationalizing Gene Therapy Studies for Success
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...
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...
Rare Disease