1. Development of the scientific-technical platforms in a
field of biomedicine
Author:
Academician Genrikh Sofronov
Speakers:
Prof. Anton Orlov
Prof. Youry Scherbuk
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2. Scientific-technical platforms as mainstream approach for effective
innovative development
Ideology:
•
Approaches
Single product development
alternative
Scientific-research centers - Invention & development
• Complex vehicle Education centers - Education
Experimental-industrial
- Test output
production
Clinical centers - Clinical studies
Healthcare system - Implementation
Developing Platforms:
Nosological Technological
group group
Oncology Recombinant
vaccines
Autoimmune diseases
Development of founded platforms will lead to wide-front breakthrough in
technologies creation and implementation 2

3. Institute of Experimental Medicine (IEM)
of the North-West Branch
of Russian Academy of the Medical Sciences
• Over 120-years history (since 1890)
• The most authoritative center of the biomedical science in Russian Federation
Major goal of the studies An investigation of molecular mechanisms of
conducted in IEM: dysfunctions of the human organs and systems
and search of the ways for their correction
Staff: more than 600 employees, including:
• 7 academicians, members of RAMS;
• 24 professors;
• 53 doctors of sciences (Dr.Sc);
• 102 PhDs.
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4. Institute of Experimental Medicine (IEM)
of the North-West Branch
of Russian Academy of the Medical Sciences
Structure include:
11 research Clinic Scientific- Educational
departments practical centers unit
- diagnosis, study, treatment and prophylaxis of human diseases
• Mass-spectrometers,
Research • Real time PCR equipment,
• Confocal microscopy,
facilities • Microarray systems,
• Sequencers,
• Cytofluorymeters, etc.
International cooperation:
USA, Sweden, Slovakia, Vietnam, Germany, Australia, Italy, Poland, Hungary,
China, Czech Republic, Japan and others
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5. Modern Clinical facilities of Saint-Petersburg
Saint-Petersburg State Healthcare complex
• Sustainable development of new clinical capacities
• Sustainable city Expenses on top-class equipment
Outstandingly equipped RAMS
clinical complexes Scientific
• oncology; capacity
• cardiology;
• infections; •
• multi profile; International
• others Scientific
capacity
Bases to implement
breakthrough technologies and methodics
Unique facilities for initial founding of scientific-technological platforms of a
State/Federal value 5

6. Modern Clinical facilities of Saint-Petersburg
Saint-Petersburg clinical scientific & practical center (oncological)
Launched – feb 2010
Properties:
78 000 sq.m. area Bright example of one of nosological
23 units platform Participant
14 surgeries GMP laminar coatings
Personnel – 1 200 employees.
Clinical capacity – 600 beds
Technical capacity:
Diagnostics Beam therapy
• Multispiral computed tomography 128 14 beam therapy complexes, including:
• combined PET-CT,
• MRT 3Tesla, • LEA 20 MeV complexes,
• Intraoperational MRT, • Brahitherapy complexes,
• Dual-head CT gamma camera, • Intraoperational X-Ray complexes.
• Dual-source CT angiograph,
• US complexes of expert and premium class,
• Digital X-Ray complexes.
Clinical complex of a top level in Russia relevant to a worldwide level
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7. Scientific-technical platform #1
Research & Development of the facilities for
personified diagnostics and treatment of
oncological diseases
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8. Project Participants
SPbSU
Saint-Petersburg State University
IEM
Institute of Experimental Medicine of the North - West
Branch of the Russian Academy of the Medical Sciences
CSPC
Saint-Petersburg clinical scientific & practical center (oncological)
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9. Research & Development tools for personified diagnostics and
treatment of oncological diseases
Project structure:
• The risk group identification, via genetic diagnostics of the oncology
predisposition
• General diagnostics of oncology
• Noninvasive extracting of tumor cells
• Differential diagnostics with optimal for therapy tumor cell surface
antigens recognizing
• R&D and production of medicines made of genetically engineered
complexes
• Target therapy of multiple origin tumors
First case in Russia of full-stages united system to be created: from diagnostics to
personified therapy 9

10. Project actuality
1
Russia: the disease rate increase
Annually Annual
Under monitoring detected new cases deaths
2,5 mln 490 000 new 290 000
Статистика онкологических заболеваний в РФ
patients cases patients
Вестник РОНЦ им. Н.Н. Блохина, 2009 г.
29 % growth 11 % growth 0 % growth
in 10 years in 5 years
• Cancer– the second in value cause of country population mortality
• While peoples life length is growing the following growth of disease rate is estimated
2 Low medicine consumption rate in Russia
65-70% of cancer patients need to be treated with medicines on multiple desease stages
Medicine therapy Medicine Deathes due to the
required therapy absence of medicine
provided therapy
Lung tumor 65% 10% >50%
Breast tumor 95% >50% >30%
Ovary tumor 95% >50% >30%
• Indexes show medicines demand unavoidable growth in close future 10

11. 1 Risk groups identification
Genetical diagnostics of hereditary cancer predisposition
Researches held
The predisposition to breast and ovary cancer was explored
• The risk of hereditary cancer development is near 90%.
IEM researches
• The hereditary breast cancer occurs mostly due to genes BRCA1 and BRCA2 mutations.
• Most frequently noted is the 5382 ins C mutation, which is responsible for breast cancer cases and 40% ovary
cancer cases. Also were found some other mutations associated with tumors of several types development.
• The risk of cancer development in young aged mutations holders is 10 times higher.
• It is reasonable to run tests on mutations presence (PCR, sequenation, restriction analysis) firstly for women of
families with breast or ovary cancer, and for all women from 20 to 50 years old, as routine healthcare campaign.
Over 90% of patients with determined tumors of breast in due time are able to run a valid life.
Original Methodics
2012-2013 г.
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12. 2 General diagnostics via tumor markers
Methods
• PCR with reverse transcription
(cDNA, detection on transcriptase level);
• ELISA
(detection on proteomics level).
Base
Universal tumor genes are found which are proposed as base for
diagnostics.
Start-up №1
Reverse transcription PCR analysis of these markers (cDNA) original methodics
shows sensibility up to 95% and specificity – 95%. 2012-2013
ELISA with purified proteins for antibodies used in blood Registration of medicines,
certification of methodics,
detection shows sensibility up to 94%, specificity – 98% launch on market
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13. 3 Extraction of tumor cells via non-invasive technique
Method
• The fraction of tumor cells in blood determination via stream cytologic fluorimetry
• Concentration of tumor cells via monoclonal antibodies based sorter
• Test of tumor cells RNA function of specific surface antigen encoding
Advantages:
• Biopsy avoidance
Original methodic
• High promptness 2012-2013
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14. 4 Differential diagnostics
Antigens specification – as targets for each individual case
• for monoclonal antibodies (МАb) and target-medicines created on their base
• for medicine resistance determination and its neutralization vehicles switching on
• for immunotherapy efficiency increasing
Method: Reverse transcription – real time PCR
Start-up №2
original methodics
2012-2013
Medicines registration,
Methodics sertification,
Product launch
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15. 5 Developing of biotechnological medicines based on genetically
engineered complexes
- Monoclonal antibodies (MAb)
• Specified linking to the transmembrane antigenes, expressed on the surfaces of tumor cells (onco-markers)
• Initiate a cascade of immunological reactions of the humans organism which leads to a lysis of onco-markers
expressing cells
• Fundamental difference of MAb opposite to chemo-medicines is its specificity of damage action on target-cells causing
minimal harmful influence on normal tissues and organs of host
• Nowadays in Russia some of himeric and humanized monoclonal antibodies (MAb) medicines which are totally foreign
made are approved for clinical implementation
MAb
Conventional MAb
action illustration:
receptor
Start-up №3
Invention 2 original MAb worldwide exclusive MAb pharmaceuticals
IEM
Particularly based on stage №4 achievements 2013 г.
(Definition of universal tumor markers)
Therapeutic efficiency studies using cell-models and Clinical studies,
clinical samples Pharmaceuticals registration,
Methodics certification, 15
Methodics of application studies Product launch

16. 5 Developing of biotechnological medicines based on genetically
engineered complexes
- Single-chain antibodies (mini-MAb)
• Single-chains are genetically engineered atnibodies consisting of only
Fv and Sc fragments of ordinary monoclonal antibodies.
Their size is 5 times less than that of ordinary antibodies (up to 20nm).
• Action mechanism is equivalent to one of ordinary MAb
• Small size allows it to penetrate the areas unreachable for МАb
Start-up №3
single-chain MAb
pharmaceuticals
2013
Clinical studies, Pharmaceutical registration,
Methodics certification,
Product launch
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17. 5 Developing of biotechnological medicines based on genetically
engineered complexes
- MAb conjugated with cytostatic agents (A-MAb)
• Complexes of MAb molecules (as nano carrier) conjugated with chemotherapy cytostatic molecules
• Action principles based on MAb’s ability to specifically conjugate with onco-markers expressed on tumor cells
surface
• Due to implementation of MAb as nano-carrier it allows to deliver cytostatic agents directly to cells expressing
onco-markers.
Clathrine-banded
vesicle
Clathrine
Start-up №3
conjugated MAb
pharmaceuticals
2013
Nucleus Clinical studies, Pharmaceutical registration,
Methodics certification,
Product launch
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18. Targeted R&D achievements
Field Matter Target
1. Risk groups identification Investigation of additional reasonable Original methodics;
mutations; Base for diagnosticums development
Population studies and commercialization
Extended studies of proposed Base for unified diagnosticums for
2. General diagnostics
tumor-specified genes onco-markers development and
commercialization. Start-
Studies of additional reliable tumor- up
specified genes Original methodics
Extended studies and proving the
3. Non-invasive tumor cells Original methodics;
precision of proposed method ,
extraction relevant to all types of tumors
Base for unified diagnosticums Start-
4. Differential diagnostics Extended studies and proving the
development and up
precision of proposed method
relevant to all types of tumors commercialization/
Original methodics
Developing new 4 genetically-
5. Development of genetically- Technologies of synthesis
engineered medicines: technology Start-
egineered pharmaceuticals of synthesis, therapeutic efficiency
of 4 original pharmaceuticals. up
studies Lab Protocols of
efficiency studies
6. Developed pharmaceuticals In framework of following start-up
appliance to cancer therapy development
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19. Project result
Within 5 years following aims will be reached:
Creation of technologies and tools for Russia’s first united full-stage
system in treatment of oncological diseases: from risk groups
identification to personified diagnostics and therapy, relevant to top
worldwide class
Creation of technologies for national innovative pharmindustry of
general and differential diagnostics facilities and target-therapy
pharmaceuticals for treatment of oncological diseases. Creation of
original treatment methodics.
This will lead to competence for developing start-ups purchased with
more than 7 original oncologic medicines of top worldwide level
Creation of scientific-technological platform aimed to facilitate quick
spinoff of start-ups for development and to put into healthcare
practice a new highly innovative pharmaceuticals and treatment
methodics in a field of oncology
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