THERAPEUTIC AREAS


New cell and gene therapies hold much hope for many currently incurable diseases, particularly in the field of regenerative medicine and immunomodulation, and also, increasingly, for cancer treatment. apceth Biopharma is one of the global leaders in this rapidly-progressing new field of medicine.

Learn more about our therapeutic products in clinical trials:

Clinical Pipeline

  • Cancer is the second highest cause of death globally, with approximately 14 million new cases and 8.2 million cancer-related deaths in 2012 (WHO, NIH).
  • The global cancer incidence is steadily increasing: 22 million patients can be expected in 2030 (WHO, NIH).
  • Despite significant medical progress, the majority of cancer patients remain chronically ill. In addition, systemic toxicity still presents a major problem for standard therapies, such as radiation and chemotherapy.

apceth Biopharma’s engineered MSC could offer a new therapeutic modality in cancer treatment, with high tumor-specificity, low systemic toxicity and few or no side effects. Our engineered MSC can be used as a primary therapy or as a bridging and relapse cancer therapy.
Our oncology program is currently in the clinical stage of testing. It is, globally, the first cancer therapy based on genetically engineered MSC to receive approval for application for patients within clinical settings.

  • Currently, 64 million people suffer from COPD and more than 3 million people died of the condition in 2012 (WHO). By 2030, COPD could become the third highest cause of death worldwide.
  • IPF leads to irreversible scarring of lung tissue, pulmonary deficiency and death 3-5 years after diagnosis. With a five-year survival rate of just 20-40 %, IPF is deadlier than many cancers. The EU incidence is 7-10 new cases per 100,000 people.
  • There is, currently, no cure for either COPD or IPF, and, today, only limited treatment options exist for each condition.

apceth Biopharma develops engineered MSC for the treatment of two severe lung conditions: Chronic Obstructive Pulmonary Disease (COPD) and Idiopathic Pulmonary Fibrosis (IPF). The proposed mode of action of this cell drug includes the modification of the inflammatory process; disease modification; and prevention or reversal of pathological lung remodeling.

  • CKD is a major health problem worldwide, affecting mainly elderly patients. It is a progressive condition, characterized by the gradual loss of kidney function. In the final disease stages, dialysis or kidney transplantation are currently the only available therapies.
  • In Europe, the prevalence of CKD varies greatly between countries, affecting between 3 % and 17 % of the population (Bruck et al. 2015). In the USA, an estimated 1 in 10 people suffer from some form of CKD. Current studies predict an increase in CKD prevalence both in Europe and America.

By incorporating a novel therapeutic transgene into our MSC, apceth Biopharma is currently developing genetically engineered MSC for the treatment of chronic kidney disease (CKD).

  • Type 1 diabetes is characterized by destruction of pancreatic beta cells – which normally produce insulin – culminating in complete insulin deficiency. The majority of cases are attributed to an autoimmune destruction of pancreatic beta cells.
  • Type 1 diabetes accounts for 10-15 % of all diabetes cases. It often begins in childhood, before the age of 15, making it one of the most common endocrine and metabolic conditions among children.
  • A 2-3 % increase in the incidence of type 1 diabetes worldwide has been reported lately.

apceth Biopharma’s genetically engineered MSC for the treatment of type 1 diabetes are expected to modulate the autoimmune process that leads to the onset of the disease.

  • Graft vs. Host Disease (GvHD) is a severe complication arising after allogeneic hematopoietic stem cell or bone marrow transplantation. It has a very high mortality rate.
  • Refractory GvHD was the first indication to demonstrate extremely positive response to therapy with native MSC.

Due to the low immunogenicity of MSC, no HLA-antigen matching between the MSC-donor and the MSC-recipient (patient) is necessary. This allows apceth Biopharma to use the donor-derived native MSC as an off-the-shelf clinic-ready cell therapy for severe cases of refractory GvHD.