Clinical Research Pipeline

TICEBA's unique know-how and specialized scientific team leads to a successful development of stem cell based drugs for diseases with high and unmet medical need.

Active Ingredient
Mechanism
Indication
Pre-clinical discovery
Pre-clinical development
Phase I/IIa
Phase IIb
Phase III

allo-APZ2 1

Anti-Inflammation

RDEB a b

allo-APZ2 1

Anti-Inflammation

CVU 4

allo-APZ2 1

Anti-Inflammation

DFU

allo-APZ2 1

Anti-Inflammation

ACLF

LSC2 3

Regeneration

LSCD a b c

APZ2 2

Anti-Inflammation

CVU



1 Allogeneic ABCB5-positive mesenchymal stem cells
2 Autologous ABCB5-positive mesenchymal stem cells
3 Allogeneic ABCB5-positive limbal stem cells
4 Requested for the national marketing authorization in Germany for AMESANAR

a Orphan Drug Designation FDA
b Orphan Drug Designation EMA
c Fast Track Designation FDA

Epidermolysis Bullosa (EB): severe genetic pediatric disease

Non-healing chronic venous wounds

Non-healing diabetic foot ulcers

Acute-on-Chronic Liver Failure (ACLF)

Limbal Stem Cell Deficiency (LSCD)

Focusing on the most severe forms of Epidermolysis bullosa (RDEB and JEB) working together with leading international experts. Our efforts are dedicated to help those small patients who cannot expect any cure.

EB is a severe, inherited, clinically and genetically heterogeneous skin disorder. EB patients suffer from extremely fragile skin and mucosa, caused. Thus, even a minor mechanical trauma or friction can lead to blisters, erosions and open, slowly healing wounds. The life expectancy of EB patients is highly dependent on the inheritance pattern and severity of the disease. In its most severe forms - as a result of side effects (e.g. metastatic skin carcinoma) - EB can lead to death in early childhood or early adulthood.

For non-healing chronic venous wounds there is currently no sufficient cure. We are focusing on these patients to give therapeutic options and being able to participate in a "normal" life.

CVUs develop as a consequence of impaired venous drainage of the lower extremities, mainly due to venous reflux or venous outflow obstruction. The increased venous pressure causes micro-circulatory disturbances, initiating a cascade of pathophysiological events that lead to tissue breakdown and the development of painful wounds. To date there is no conclusive evidence that any particular dressing, topical agent or systemic agent can effectively improve venous ulcer healing. For severely affected patients, surgical interventions aimed at eliminating the cause of the venous hypertension are often the last hope.

Patients with non-healing diabetic foot ulcers are facing major health risks. We are focusing on these patients to give therapeutic options and to avoid complications e.g., amputation, by treating these ulcers with the most modern drug.

During the course of diabetes mellitus, (diabetic foot ulcers) DFUs represent the most common and severe complication, affecting 15% of diabetic patients in their lifetime. DFUs are the major cause of hospitalization of diabetic patients. It is estimated that approximately 50-70% of total lower limb amputations are due to DFUs. This number is expected to increase further, reflecting the increasing number of diabetic patients from 463 million in 2019 to 700 million by 2045 and obese patients, as predicted by the International Diabetes Federation (IDF).

ACLF is a condition characterized by acute hepatic decompensation in patients with chronic liver disease, which is accompanied by multi-organ failure and a profound systemic inflammatory response. Non-manageable organ damage and secondary infections account for poor short-term survival with 30-day lethality rates of up to 77%, depending on the number of extrahepatic organ failures. ACLF requires intensive care and in most cases, liver transplantation is the only effective treatment option. However, it is restricted by the limited availability of donor organs.

Patients with limbal stem cell deficiency are facing a life-long blindness. We are developing a stem cell based drug with the potential to help patients to be able to see again.

Limbal stem cells (LSCs) are crucial for the regular physiological regeneration of the transparent anterior part of the human eye, i.e. the corneal epithelium. Damage to the limbus, the area which forms the border between cornea and sclera, may cause a lack of LSCs leading to LSCD. Typical symptoms of LSCD involve corneal conjunctivalization, neovascularization and scarring as well as chronic ocular inflammation. This contributes to loss of corneal transparency, possibly culminating in profound visual impairment or even blindness.