1

Symposium

Raw Materials for the Production of Cell-based and

Gene Therapy Products3 April 2013

Strasbourg, France

03/04/13 ©2013 EDQM, Council of Europe. All rights reserved.

Session 2

User, Manufacturer & Academic Viewpoints

03/04/13 ©2013 EDQM, Council of Europe. All rights reserved.

04/04/2013

7

Decell scaffold release criteria

Frozen H&E Fixed H&E Fixed �DMHC

Pre

Po

st

04/04/2013

8

CD45 CD34 CD14 CD90 CD73 CD105

CD45 CD34 CD14 CD90 CD73 CD105

MSC Monolayer (P0) Day 14

CD45 CD34 CD14 CD90 CD73 CD105

Passage 1 Day 22

Urgently needed EDQM reagents

• Recombinant cytokines

• Monoclonal antibodies for “in process” cell selection

• Trypsin

• RNAse

• Specialised media for – MSC

– Epithelia

– Chondrocytes

04/04/2013

9

Regulators

Academic clinician

University / Hospital

/ CMO

Patient

Industry

All are interdependent:

1. Clinician wants to innovate treatments and NEEDS patients

2. University NEEDS high impact publication and IP but not to fund phase III trials for MA

3. Industry NEEDS clinician to innovate and University to support proof-of-principle

4. University NEEDS Industry to buy its IP and commercialise it after clinical trials

5. Regulators NEED to ensure patient safety and maximum availability of novel therapies and commercialisation

6. Patients need access to new, safe and effective treatments from Clinicians AND Industry

The ATMP stakeholders

The virtuous circle