Robust Cell And Gene Therapy Pipeline Targets A Range Of Diseases

Cell and gene therapies (CGTs) are quickly making their way from theory to clinical practice, primarily because they target specific disease drivers rather than the broad targets of small-molecule therapies. This specificity is possible because cell and gene therapies target genes and cells to change the way tissues function. The goal for both types of treatment is to introduce healthy cells or correct mutations on patients’ genes.

Just four years ago, the first CGT treatment was approved by the FDA. Today, nine therapies are approved, and, according to PhRMA, the number of treatments in clinical trials increased by 25% over the past year, going from 289 to 362. Even more treatments are in preclinical stages and are expected to enter the clinical trials pipeline.