Andrew Finnerty General Manager - CCMI

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2 Andrew Finnerty General Manager - CCMI Manufacturing Human Mesenchymal Stem Cells for Clinical Trials Quality Considerations Biopharma and Pharma RQA Regional Forum Bioclin Laboratories, Athlone 13 May 2016

3 CCMI Location

4 Content Introduction to CCMI Overview of Facility Regenerative Medicine What are Stem Cells? Clinical Trials and CCMI/NUIG Regulations governing Advanced Therapy Medicinal Products (ATMPs) Production of stem cells Challenges Global perspective

5 Introduction Regenerative Medicine Institute (REMEDI) has developed strong R&D expertise over many years in adult stem cell technology. As part of REMEDI s translational mission the Centre for Cell Manufacturing Ireland (CCMI) was established as a site authorized in 2013 by the HPRA for the manufacture of human mesenchymal stem cells (hmscs) for use in clinical trials. The vision was to develop the potential to provide patients in Ireland with access to new, ground-breaking therapies. CCMI became the first facility on the island of Ireland to be licensed to manufacture cultured stem cells for human use. HPRA approval was then received in Oct 2014 for critical limb ischemia (CLI) trial - Autologous bone marrow derived mesenchymal stromal cells.

6 CCMI Facility 250m 2 cleanroom facility Qualified air-handling units Fully equipped for GMP processing

7 Facility Layout 7

8 Personnel Flow

9 Interlocking Doors in CCMI

10 CCMI Gowning

11 CCMI Management Team General Manager Andrew Finnerty Over 30 years in the pharmaceutical manufacturing industry Production Manager Aoife Duffy Over 10 years experience in GMP cell processing Quality Manager Margaret Desmond 10 years experience working in the medical device, pharmaceutical and biotech fields Directors Prof. Frank Barry Prof. Timothy O Brien

12 CCMI Organisation Chart

13 Regenerative Medicine There are many human diseases which have either inadequate or no treatment options. Regenerative medicine and cell therapies have the potential to impact many different areas of unmet medical need. For decades, blood transfusions have been used as a basic, temporary form of cell therapy. But time and technology has moved on and now the targeted delivery of cells to injured or diseased tissue is seen as a promising future method of treating a whole variety of illnesses and conditions. How does it work? - MSCs as pharmacological mediators in tissue repair. 13

14 Regenerative Medicine/Cell Therapies In 2012 regenerative medicine products generated over 1 billion dollars in revenue for the first time ever. Investment from public and private sources in regenerative medicine also exceeded 1 billion dollars. In terms of cell therapies, there are 1,900 clinical trials ongoing with 250 companies involved. There are already 40 cell therapy products on the market worldwide including the world s first stem cell-based product Prochymal, approved to treat graft versus host disease. 14

15 Commercial Therapy Products

16 What are Stem Cells? Stem cells are the progenitors that give rise to every type of adult cell Can give rise to specialized cells, in a process called differentiation Unspecialized cells lacking any tissue-specific structure Capable of dividing and renewing themselves for long periods

17 What are Stem Cells? Stem cells, sometimes called the body s master cells, are the precursor cells that develop into blood, brain, bones and all of your organs. Their promise in medical treatments is that they have the potential to repair, restore, replace and regenerate cells that could then be used to treat many medical conditions and diseases. Stem cells that come from bone marrow or blood are routinely used in transplant procedures to treat patients with cancer and other disorders of the blood and immune system. Umbilical cord blood is collected from a placenta with the birth mother s consent. Cord blood cells are then isolated, processed, and frozen and stored in a cord blood bank for future use. In the US Cord blood is regulated by FDA.

18 Types of Stem Cells Embryonic stem cells (ES Cells) Adult stem cells Bone marrow Umbilical cord blood Adult peripheral blood Adipose (fat) tissue

19 Spindle shaped cells: Stem Cells Dividing

20 What are Stem Cells? Adult stem cells are capable of surviving over long periods and divide to make additional stem cells. Human embryonic stem cells are unspecialized ( blank slates that can become specific types of cells). Induced pluripotent stem cells (ips) can develop into specialized cell types (cells that do specific work in the body). Adult stem cells can be found throughout the body. They are found as unspecialized cells among the specialized cells in tissues and organs as well as in umbilical cord blood and peripheral blood (that is, hematopoietic stem cells). An adult stem cell can either divide to make more adult stem cells, or differentiate to produce some or all of the major specialized cell types of the tissue or organ.