Moving into the Future with Advanced Therapies

ATMPs offer new therapeutic approaches for previously incurable diseases  

In the last decade, many novel medicinal products, also known as advanced therapy medicinal products (ATMPs), have been further developed and achieved breakthrough success.  

Numerous gene therapeutics, cell therapeutics and bioengineered tissue products are already approved in the EU and the US, and many clinical trials are in progress worldwide.

This group of heterogenous medicinal products represents according to the German Medicines Act (AMG) and European Medicines Agency (EMA) a heterogeneous group of preparations. By definition, they are recombinant nucleic acids, cells or tissues which are used as gene therapeutics, somatic cell therapeutics or bioengineered tissue products. They are primarily isolated from blood, bone marrow or other tissues as cells or nucleic acids, are modified for therapeutic purposes and subsequently used autologously or allogeneically.
 

ATMPs – modified to heal

Unlike a transplantation of tissue, such as supporting tissue, skin or hematopoietic stem cells, mostly all ATMPs have in common that their starting materials are substantially modified. Gene therapeutics (manipulated nucleic acids), genetically engineered cell therapeutics or customized and/or stimulated expanded cells are created.

This means that cells are isolated, stimulated, expanded, and/or genetically modified specifically for the corresponding clinical picture. In this way, not only tumor diseases can now be treated, but also genetic defects, inborn errors of metabolism and autoimmune diseases, as well as tissue injured in accidents that cannot be regenerated in any other way. Furthermore, the therapeutic approach is no longer designed to improve the clinical picture in the classical sense, but to heal it.

Tailor-made treatments in small batches

Novel preparations are primarily developed in scientific research institutions and clinics and are usually produced in small batches and for individually designed therapy. On the one hand, this has the enormous advantage that the latest findings and innovations can be incorporated into development, but on the other hand it also brings the disadvantage that regulatory knowledge and financial resources for GMP-compliant pharmaceutical development are often not fully available in the facilities. Often even the manufacturing process is still under development.