Congenital diseases

Our strategic planning of therapeutic areas for development of new and generic drugs, as well as cellular products is based on the in-depth analysis of the patient population needs in the Russian Federation for various medical products and treatments. Currently, we are pursuing research and development of therapies for the following diseases and pathological conditions.

Rare (orphan) congenital diseases. Congenital or acquired defects of coagulation factors.

Rare (orphan) congenital diseases

Treatment of rare diseases represents a significant socio-economic problem. The prevalence of these diseases is less than 1–5 per 100 000 in the general population. Development of drugs for the treatment of orphan diseases is complex and not always proceeds from the sale of respective drugs can cover the costs of their development and manufacturing. Therefore, treatments for orphan diseases should be maximally effective and cheap. As part of our commitment to social responsibility, the company is developing a generic medication for the treatment for a rare Gaucher’ s disease — a deficiency of the enzyme glucocerebrosidase. The ongoing project is in the stage of the laboratory production process implementation.

Congenital or acquired defects of blood coagulation factors

Haemophilia is a disease characterized by the lack of the blood clotting factors; most commonly are being factor VIII or factor IX. It is among the most common congenital disorders of blood coagulation. Patients with haemophilia require frequent administration of the therapeutics, containing the missing factors. This demand in Russia has been covered by the generic medications Octofactor® (Factor VIII) and Innonafactor® (Factor IX), which were developed by our company. Following initial treatment, patients may develop a loss of feedback to coagulation factor inputs, which will require the use of alternative drugs that affect blood clotting. Such alternative medication is Coagyl®-VII (coagulation factor VII), that has also been developed by our company.

Our products:

GNR-001 Protein based Biosimilar Registered
GNR-002 Protein based Biosimilar Registered
GNR-003 Protein based Biosimilar Registered
GNR-008 Protein based Biosimilar Phase I clinical studies
GNR-021 Protein based Innovative Preclinical trials in vivo
GNR-039 Protein based Biosimilar Preclinical trials in vivo