Pharmaceutical Quality Control

Cell and gene therapies have been undergoing exciting developments recently, including expansion into second line use for existing products. This means that cell and gene therapies are no longer treatments of last resort, and studies are currently underway for front line use.

The landscape of advanced therapy medicinal products (ATMPs) continues to evolve rapidly, revolutionizing modern medicine to help provide additional treatment options for patients with rare diseases, cancers, and autoimmune disorders.

Cell, gene, and tissue-based therapies, also referred to as Advanced Therapy Medicinal Products (ATMPs), present unique opportunities to address some of the most problematic diseases, like cancer autoimmune diseases, cardiac diseases, etc., including those with limited treatment options and genetic disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease.

Parkinson’s disease, diabetes, muscular dystrophy: these diseases all lack cures, but they also share another important similarity: the quest for their cures is playing out in the field of ATMPs.

Scientific discoveries, however important, are not enough to improve public health. Manufacturing infrastructure and innovation are needed to allow pharmaceutical companies to successfully and safely produce products at scale and make them available to clinicians and their patients.