Gene- and cell-based therapies (GCTs) offer promising prospects for treating previously incurable diseases. They can also support regenerative processes in the body. Gene-based therapies use nucleic acids (DNA or RNA) to regulate or supplement biological functions. Cell-based therapies produce and/or transfer cells in an organism for therapeutic purposes, often using genetically modified cells as a combination of both approaches.
GCTs go beyond purely symptomatic treatment, as they specifically target the causes of disease— potentially enabling curative effects and tissue regeneration. Their range of applications extends from rare genetic diseases (e.g., hemophilia A and B) to common complex conditions (e.g., type 1 diabetes), for some of which no effective therapies are currently available. Despite ongoing research questions regarding safety and efficacy, millions of patients in Germany alone could benefit from GCTs.
Gene- and cell-based therapies include the following approaches:
Therapy approaches with advanced therapy medicinal products (ATMPs):
Therapy approaches with novel biological products, e.g., mRNA-based and other nucleic acid-based procedures, extracellular vesicles, or exosomes used in the context of a GCT approach
Development through to approval of ATMPs
ATMPs differ significantly from conventional drugs in terms of their manufacturing, mechanisms of action, and regulatory assessment. Their development involves, among other things, several sequential stages—from initial basic research through preclinical and clinical development to approval, market launch, and long-term follow-up.
The following interactive graphic illustrates the key stages in the development and manufacturing of an ATMP and shows the pathway from scientific research through clinical development and approval to use in patients.