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R&D AREAS AND PROJECTS

In order to find new opportunities to expand the applications of existing products and technologies, as well as to develop new diagnostic and therapeutic products, Human Stem Cells Institute (HSCI) conducts its own research and cooperates with a number of reputable scientific and clinical organizations.

The Сompany's efforts are focused on developing products for the diagnosis and treatment of widespread, socially significant and age-related diseases.

Research and development, preclinical studies and clinical trials, conducted by Human Stem Cells Institute, lays a significant foundation for the Company’s future sustainable development.

The following R&D projects are implemented both by parent company - PJSC "HSCI" and its subsidiary companies, some of which are Skolkovo* residents.


THERAPEUTIC ANGIOGENESIS

Expansion of the range of indications for applying Neovasculgen®(the first-in-class gene therapy drug for therapeutic angiogenesis) and the development of new methods for ischemia treatment

Pre-clinical studies and clinical trials to determine the applicability of Neovasculgen® in the treatment of various diseases, beyond PAD/CLI, related to ischemia (currently the work is underway for the therapy of IHD /Ischemic Heart Disease/, Diabetic Foot Syndrome, trauma-induced peripheral nerve damage).
 

TISSUE ENGINEERING

Gene-activated materials

In cooperation with our partners, we develop a pipeline of gene-activated materials – a new class of medical products intended for the effective treatment of patients suffering from alveolar ridge atrophy, skeletal bone defects or skin lesions.

A gene-activated material is a complex of a scaffold and nucleic acid (gene construct) combined using different methods. Detailed information is available in the review: Deev R.V., Drobyshev A.Y., Bozo I.Y., Isaev A.A. Ordinary and Activated Bone Grafts: Applied Classification and the Main Features. Biomed Res Int. 2015; 2015:365050. doi: 10.1155/2015/365050 (https://www.hindawi.com/journals/bmri/2015/365050/).

Among other gene constructs, plasmid DNA encoding vascular endothelial growth factor (VEGF), the active substance of the Neovasculgen®, is used for the creation of gene-activated materials. This distinctive product component provides a number of pronounced inductive properties neither ordinary nor other groups of activated materials have.

The advanced part of the project is aimed at the development of standardized and personalized (3D-printed) gene-activated bone substitutes containing plasmid DNA encoding VEGF  to cover a bone reconstruction in any clinical cases. As of now, R&D is in progress for the first product in a line – “Histograft”, a complex of octacalcium phosphate granules with the plasmid DNA. Its clinical trial started in March 2017 and is expected for completion in early 2018. 20 patients with acquire and congenital maxillofacial bone pathology including alveolar ridge atrophy will be enrolled in the study.

Personalized tissue-engineered biocomposites for the periodontal hard tissue restoration (SPRB – therapy)

The set of personalized diagnostic and treatment procedures for the restoration of periodontium hard tissue by applying biocomposite osteoplastic material and autologous gingival fibroblasts.

SPRB-therapy (Service for Personal Regeneration of Bone) is a cell-based technology for treating patients with pathologies affecting periodontal hard tissues (i.e. bone tissues lost due to infection or other pathological processes). It includes isolation of the patient’s gingival cells, cell processing and placement of cell cultures in bioresorptive media to grow personal tissue-engineered product for transplantation into the same person during surgical treatment of periodontal pathologies.

Currently preclinical studies and pilot clinical trials have been completed and shown positive results.

GENETIC DIAGNOSTICS AND CORD BLOOD HSC TRANSPLANTATION

PGD (Preimplantation Genetic Diagnosis) and transplantation treatment for the families with inherited diseases accompanied by hematological syndrome – the Program for the prevention of GID (Genetic Immunodeficiency Disorders).

Preventative treatment program for primary immunodeficiency diseases is a special program for the birth through IVF with PGD of a healthy child to be a donor for UCB stem cell transplantation to the elder brother/sister having inherited disease. PGD makes it possible to ensure the birth of a sibling who is not only free of the genetic mutation causing the disease but also is an appropriate donor match for the sick child.

GENE THERAPY AND DNA REPAIR

Myogenesis

Gene therapy of some inherited diseases, in particular those related to skeletal muscle tissue lesion, remains an unsolved problem. HSCI, together with partner organizations, is conducting research on the creation of a molecular toolkit for the abnormal DNA repair in patients with limb-girdle muscular dystrophy (type 2B). This work is at an early preclinical stage and is aimed at the formation of the scientific and technical basis for the creation of gene and cell-based technologies for the treatment of such diseases in the future.

DEVELOPMENT OF DIAGNOSTIC PCR PANELS

HSCI develops a number of diagnostic PCR panels based on microfluidics technology for the early detection and prevention of hereditary diseases. These panels will help identify the carriage of mutations associated with the risk of serious diseases.

Currently under development:

              • Monogenic diseases carrier status
              • Neonatal screening for early detection of curable diseases with a genetic component
              • Infertility (male and female) testing panel
              • Cancer prevention (familial oncology)


INTRODUCTION AND TRANSFER OF NON-INVASIVE PRENATAL TESTING TECHNOLOGY

Development of bioinformatics algorithm analysis of primary genetic data is underway, clinical trials of the technology have been carried out. The result of this work will be the introduction in Russia of a new NGS method for prenatal testing of fetal chromosome abnormalities using maternal venous blood (NIPT).

TRANSPORTATION OF NATIVE SPERM

It is supposed to develop a processing and transportation protocol as well as the transport medium for sperm. This will allow for long transportation of native sperm, for at least 24 hours, without substantial loss of viability.

* The Skolkovo Innovation Center is a high technology business area in Moscow, set up with the strategic goal to concentrate intellectual capital, thereby stimulating the development of break-through projects and technologies. Companies engaged in innovative development, after a thorough selection process, may become project participants of the center to be provided with all assistance necessary for development. Project participants using the general taxation system are granted a number of incentives. Members of the Skolkovo Foundation may be eligible for a grant to fund their projects. Within the Skolkovo project, five clusters are created, each one developing innovative projects. These are the information, biomedical, energy efficiency, nuclear and space technologies clusters.