Advanced gene therapy resources

Gene therapy is part of an innovative class of treatments known as advanced therapy medicinal products (ATMPs), which has been developing internationally for over a decade and has demonstrated scientific and technological advances that are favorable for treating hematological and ocular diseases and some types of cancer.

Some gene therapies based on CRISPR technology are already being tested in clinical trials with patients, with very positive results. In Brazil, this category is still nascent, but there is already legislation regulating the production chain and clinical studies.

The current objective in research is to develop gene therapy products based on gene editing technology, sch as CRISPR/Cas9, CRISPR prime editing, and base editing in order to safely and effectively correct genetic mutations in hematopoietic stem cells in patients. Significant efforts are also being made in clinical biomanufacturing toward safe and effective therapeutic application for human genetic diseases that do not yet have medical alternatives.