ATMPs

Oncology & Haematology

Acute Graft versus Host Disease

Disease indication

Acute Graft versus Host Disease (aGvHD) is a serious complication following bone marrow transplantation. It occurs in about 50% of patients which receive bone marrow from a donor (allogenic transplantation). Transplanted immune cells attack recipients’ (patients’) tissue, predominantly in gastrointestinal track, liver and skin. High inflammatory environment causes tissue damage which can be often fatal. In acute steroid-refractory GvHD, mortality rate approaches 85%.
Currently, there are no approved standard therapies for acute steroid-refractory GvHD patients (with exception of Canada, New Zealand and Japan). However, mesenchymal stem cells (MSC) offer new treatment possibilities for these patients, due to their immunomodulatory capability.

ImmunoArt technology platform

ImmunoArt a unique technology platform for the preparation of cell therapy product was developed. The manufacturing process consists of ex-vivo culture of allogeneic bone-marrow mesenchymal stem cells, according to highest GMP standards. For the treatment of acute steroid-refractory GvHD, product is delivered intravenously in a multiple dose regime. ImmunoArt product is safe and has already demonstrated immunomodulatory properties and a consequent improvement or disease remission in patients. Technology platform was developed in close consultation with the regulatory authorities.

Gastroenterology

Crohn’s disease

Disease indication

Crohn's disease is a long-term condition that causes inflammation of the lining of the digestive system. Inflammation is due to patients’ over reactive immune system. White blood cells invade the intestinal lining and produce inflammatory toxins causing chronic tissue swelling, injury and ulceration. The precise cause of this abnormal immune response is unknown. Such inflammation causes abdominal pain, diarrhoea and a range of other symptoms and complications. One of the common complications is development of fistulas, which do not heal and often require invasive surgical procedures. The disease usually appears for the first time in young adults and highly decreases their life quality.
Approximately 15% of the patients are unresponsive or intolerant to existing treatments, which include corticosteroids, immunosuppressive agents and biologics. Currently, there are no alternative standard treatments for these patients. However, mesenchymal stem cells offer new treatment possibilities for these patients, due to their immunomodulatory and regenerative capacity.

CrohnArt technology platform

We have developed a unique cell therapy technology platform for preparation of CrohnArt product. The manufacturing process consists of ex-vivo culture of autologous or allogeneic bone-marrow mesenchymal stem cells, according to highest GMP standards. CrohnArt is delivered intravenously for the systemic treatment of Crohn’s disease. Technology platform was developed in close consultation with the regulatory authorities.

Orthopaedics

Osteochondral lesions

Disease indication

Crohn's disease is a long-term condition that causes inflammation of the lining of the digestive system. Inflammation is due to patients’ over reactive immune system. White blood cells invade the intestinal lining and produce inflammatory toxins causing chronic tissue swelling, injury and ulceration. The precise cause of this abnormal immune response is unknown. Such inflammation causes abdominal pain, diarrhoea and a range of other symptoms and complications. One of the common complications is development of fistulas, which do not heal and often require invasive surgical procedures. The disease usually appears for the first time in young adults and highly decreases their life quality.
Approximately 15% of the patients are unresponsive or intolerant to existing treatments, which include corticosteroids, immunosuppressive agents and biologics. Currently, there are no alternative standard treatments for these patients. However, mesenchymal stem cells offer new treatment possibilities for these patients, due to their immunomodulatory and regenerative capacity.

ChondroART 1D and ChondroART 2D technology platform

Products ChondroART 1D and 2D are two products for regeneration of articular cartilage which base on cultivated autologous chondrocytes. At arthroscopical determination of suitable lesion, a small piece of healthy cartilage is taken from a non-weight bearing region of the joint and sent to laboratory, where the chondrocytes are isolated and cultivated to appropriate number. For osteochondral lesions ChondroART 1D is used together with highly effective osteochondral material. The product is implanted during the second procedure, usually 4-5 weeks after the first arthroscopy. Our results after 10 years follow up show more than 85 % successful treatments which can prolong active life even for a decade or more.

Urology

Vesicourethral reflux

Disease

Haemodialysis is a life-saving procedure, however being forced to spend an afternoon three times a week in dialysis centre is a huge burden for person’s quality of life. On the other hand complications can take place because of continuous dialysis treatments.
Vesicoureteral reflux (VUR) is a condition in which urine is leaking back to the kidney, inflaming and eventually destroying it. Even though loss of kidney is treatable by kidney transplant, the patients with VUR are not eligible for transplantation since the VUR condition could lead to a destruction also of the new, transplanted kidney. Therefore, any product, that would enable these patients to be transplanted, would hugely improve lives of these patients.

UroART technology platform

UroART is a product intended to treat high grades of vesicoureteral reflux mainly in adult population. A small biopsy of auricular (from the patient’s ear) cartilage is taken and sent to laboratories, where the chondrocytes are expanded to proper quantity. The autologous cell suspension is then applied in outpatient procedure by injecting them together with autologous plasma under ureteral orifice, where they gel, remain localized and form an autologous natural bulking agent which greatly reduces the grade of VUR, thus enable the patient to be eligible or kidney transplant.