1. Clinical trial material & ATMPs
By Dirk Francken
December 16, 2016

2. Index Introduction to ATMPs & Regenerative Medicine
Clinical trial material (supply chain)
Future perspectives
Challenges

3. ATMPs Advanced Therapy Medicinal Products Gene therapy medicines
Somatic-cell therapy medicines
Tissue-engineered medicines
Combined ATMPs
”  these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue”
Source”
EMA website on ATMPs
Source: EMA on regulation of ATMPs

4. Regenerative Medicine
“[The] process of replacing, engineering or regenerating human cells, tissues or organs to restore or establish normal function” Regenerative Medicine, 2008, 3(1), 1–5 [47]
Different methods:
Gene editing
(Stem) cells
Small molecules / biologicals
Scaffolds
Bioprinting

5. RM vs. Conventional medicine
Focus on the source of disease/damage instead of symptoms
Repair & reverse damage instead of alleviating damage
Vs.

6. Gene editing Different methods: Virus-based: Transduction
Lipid-based: Transfection
CRISPR/Cas system
Source: Biontex

7. Gene editing
Source: Reuters, Nature, MIT

8. Stem cells Different sources:
Embryonic
Adult
Induced pluripotent
Regenerate tissue trough stem cell differentiation and proliferation

9. Small molecules / biologicals
Expose damaged tissue to compounds which enhance the body’s capabilities to heal tissue
Growth factors
Immunosuppressive compounds

10. Scaffolds (Tissue engineering)
To create structures to ‘fill up’ damaged tissue or as a loading vessel for:
Compounds: Controlled release of a drug/factor
(Stem)cells: Support and protect cells while they regenerate tissue

11. 3D Bioprinting
Create custom tailored 3D models of organs, skin, bone, etc.
Source: Knowlton et al. 2015, Bioprinting for cancer research

12. Total market value of regenerative medicine
2012
2019
2012
Source: Transparency Market Research, Regenerative Medicine Market Report

14. Future perspectives API Production CS Team CRO CT sites CTM management
CTM mfg
SC management  Constant information exchange
Reconciliation

15. Future perspectives ATMP Production CS Team CRO CT sites
Will require novel facilities
CS Team
Will advanced facilities and experience
CRO
CT sites
CTM management
Will require advanced facilities and experience
CTM mfg
Will require new facilities & training/personnel
SC management of strict cold chain  Constant information exchange on product viablity & dataloggers
Reconciliation
Clinical trial sites require advanced facilities
Will require new training & experience
Will require new facilities

16. Challenges All have to comply to new GMP, GCP & GTP guidelines
Personnel at all locations will require training on ATMPs
CROs require sufficient knowledge
Short shelve lives
ATMP Production
Personnel
CTM mfg
Storage
CT sites