2. BioWin
• Advanced Therapy Medicinal Products (ATMPs)
= Medicinal products based on:
– gene therapy,
– somatic cell therapy,
– tissue engineering.
• ATMPs offer groundbreaking new treatment
opportunities for diseases and injuries of the
human body (regenerative medicine).
Laurence Timmermans
3. BioWin
• First ever ATMP cell-based product approved in Europe from
TiGenix (2009)
• ChondroCelect, commercially available in Belgium, the Netherlands,
Luxemburg, Germany, the United Kingdom, Finland, and Spain.
• Name of interest
•
TiGenixTiGenix
Cardio3 Bsc.Cardio3 Bsc.
Bone TherapeuticsBone Therapeutics
Beta-CellBeta-Cell
Promethera Bsc.Promethera Bsc.
4. R&D investments
Total 2011 = 27,1 M €
Investments in manufacturing represent about a third of the
total
Most are carried out in Belgium
4
BioWin
5. BioWin
510 members
Clinical & academic excellence
Industrial excellence (116 companies with global leaders)
Technological platforms excellence
6. BioWin
• Cell-based medicinal product based on expanded autologous
cartilage cells
• Indication: Repair of single symptomatic cartilage defects of
the femoral condyle of the knee (ICRS III or IV) in adults
• Challenge aborded :
vhigh complexity product
vEvolving science, little product benchmarks
vBiological variability (autologous therapy)
• Challenges for cell therapy product trials :
vSelection of comparator and blinding of the trial
vNumber of patients to enroll and trial compliance
vStandardization of surgery and rehabilitation
v5 years follow up
7. TiGenix
5 ans 118 patients, autologous therapy, high differentiated cells
Wilfried Timmermans
8. TiGenix
• Why Belgium? Belgium has already a big biocenter
The variability is a big issue in the quality of the product (depend on
the size of the sampling, of the organ and the patient)
TiGenix not economically viable now but thanks the consecutive
reimbursement it will be
• What other challenge?
The shelf life is a problem
70% viability because cells died with time, no serum used and
sterilization need twoo weeks
9. Promethera Bioscience
• Spin off : University Catholic of Leuven
• Focus on the liver disease (ultra orphan disease Crigler-
Najjar)
• 80 patients
• Challenge the time : on site in gmp accreditated van
• Challenge on the scale-up :
• Control and regulation of the cell culture environment
• Monitoring of the cell biomass
• Risks of contamination (try to have a system entirely
automated, for example close bioreactor like CEO did)
• Why Belgium? Talented people
•
–
Etienne Sokal
10. Cardio3
Application :
– Coronary Artery Disease (CAD)
– Hypertension
– Cardiac non-ischemic diseases
– Heart Failure
Target cell dose 600 -1,200 x 106 cells, endoventricular delivery
CHART-1 approved in Europe (Belgium and UK) : 240 patients
Christian Homsy
11. Bone Therapeutics
• Private biopharmaceutical company focused on cell therapy for
bone diseases (Principe is administering novel and biologically
active bone forming cells)
• 2 products :
– PREOB (autologous) for Osteonecrosis, Non-unions (phase
III) and Osteoporosis
– ALLOB (allogenic) for complex fracture (preclinical)
• 2 cell lines for the 2 products
• The challenge : the quality approval (indeed pesonnel have to
follow 6 certification stepps to be correctly certified
–
Gaetan Michel
12. MaStherCell
• The keys steps in the development of your cell therapy :
– Keep the product’s characterized (have product very
well characterized) : PCR, Elisa, BNA and RnA profil
– Regulation restrains ATMPs and CGMPs (March 2013)
– Quality difficult to reach because of variability of the
product
– If any change in process, verify nothing change for cell
characteristic
– Cells distribution have to efficient (shelf life is a critical
issue , cryopreservation is a solution)
Gisèle Deblandre
13. ATMI
• The challenge in the cell culture device are :
– Preserve the environment to keep the phenotype
– Sterility of the process
– Cell culture parameters control (pH, T, C….)
•
The reproducibility and the possibility of larger volume are also
important point to considerate
José Castillo
15. BiiOn
• Check the reproducibility : map biology, number of cells, which
type, still living or not, where they are in the life cycle. In brief
holographic finger prints of cells
• Less manipulation and allows the following of the cells
•
Mathieu and Anne Cassart
