ATMP - Cell and Gene Therapy
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What is ATMP - Cell and Gene Therapy?

Under the generic hat of ATMP - Cell and Gene Therapies, we collect a recent category of several transformative drugs, sharing a common root: all of them are based on living human cells. Existing treatments involve extracting different types of cells from the patient (or a donor) and modifying them, in different specific ways, to provide a highly personalized therapy that is re-injected into the patient. They have all the potential to address complex diseases by restoring or modifying specific groups of cells or using cells to deliver therapy into the body. This often involves treating rare diseases for which there are no effective treatments, but also largely diffused conditions (like diabetes of hemophilia) for which there are great unmet needs.

What is the difference between Cell Therapy and Gene
Therapy?

Cell therapy aims to treat diseases by restoring or modifying certain groups of cells or by using cells to deliver therapy into the body. With cell therapy, cells are cultured or modified outside the body before being injected into the patient. The cells can come from the patient (autologous cells) or from a donor (allogeneic cells). Gene therapy aims to treat diseases by replacing, inactivating, or introducing specific genes into cells, in order to repair their wrong behavior either directly inside the body (invivo treatments) or by extracting, modifying, and re-introducing the cells into the patient outside (ex vivo treatments). Some therapies can be considered both cell therapies and gene therapies. These therapies work by modifying genes in specific cell types extracted from the patient and re-introducing them into the body.

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