Bristol Enterprise Networkthe Personalised Health Care Revolution

Getting to Personalised Health Care

8th October 2009 Clifton Pavilion, Bristol Zoo

Getting to Personalised HealthcareAgenda

• Introduction to Regenerative Medicine• Regenerative Medicine – Manufacturing

Challenges• Regenerative Medicine – Star Gazing!!

INTRODUCTION TO REGENERATIVE MEDICINEGetting to Personalised Health Care

Regenerative medicine replaces or regenerates human cells, tissue or organs, to restore or

establish normal function Mason & Dunnill, 2008

Stem Cells:Cardiac repairNeurological repairOrgan reconstruction

Tissue cells:Islet cells in diabetes treatmentHepatocytes in liver disease

T cells:Target cancersFight infectionRegulate immune activity

•Autologous A to A (person specific).•Allogeneic A to B (person specific) or A to many (bulk biopharma)•Cell Therapy / Gene Therapy / Tissue Engineering

Regenerative Medicine Product Lifecycle

Science Select Product Development Small scale

manufacture Trials

Approval Large scale manufacture Purchase Logistics Delivery

Person specific cellular products don’t fit the infrastructure currently in place for traditional pharmaceuticals and biopharmaceuticals

REGENERATIVE MEDICINE – MANUFACTURING CHALLENGES

Getting to Personalised Health Care

Typical T Cell Process

•Research/ Medical equipment•Manual process•2-3 weeks/therapy •Takes space•Beginning to focus on GMP/scale out

Guy’s Cell Therapy Facility Layout

Guy’s Hospital, LondonCell Therapy Trials Facility

Departments involved:•Nephrology & Transplantation•Oncology•Dermatology•Immunobiology

•Location•GMP Processes•Contractors•Funding•Staffing•Where to next?

KCH Cell Therapy Unit Layout

KCH Denmark Hill Clinical Research Facility

Diversity:•Bone marrow processing•Cell therapies•Gene therapies•Liver/pancreas cell isolation

Departments involved:•Diabetes•Liver Studies•Cardiology•Neurosciences•Haematology

REGENERATIVE MEDICINE – STAR GAZINGGetting to Personalised Health Care

Regenerative Medicine – Star Gazing!!

Patient Gene Mapping Diagnostics Interventions Follow-up

Direct Gene Therapy

Bedside Autologous

Remote Autologous

Person Specific

Allogeneic

Bulk Allogeneic

•Autologous A to A (person specific).•Allogeneic A to B (person specific) or A to many (bulk biopharma)

Regenerative Medicine Suppliers:

•R&D•Organs•Tissues•Cells•Viral vectors

Specialist Suppliers:•Diagnostic equipment•Process equipment•Raw materials/consumables•Assay / QC•Hospital / bedside equipment•Logistic providers

The Regenerative Medicine Journey

Now!

Future!

Regenerative Medicine - challenges

1. Science 2. Select Product 3. Development 4. Small scale

manufacture 5. Trials

6. Approval 7. Large scale manufacture 8. Purchase 9. Logistics 10. Delivery

•Step change.•Global, fast, broad.•Fragmented•Risk.

•How to value patient life-time benefit?

•Negligible process development or manufacturing capacity.•Process equipment not developed.•Confused regulatory environment.•Skill shortage.

•Expensive

•No capacity or capability at scale.•No supply chain.

•No appropriate contracts / relationships in place.

•More: local, smaller, diagnostic•Less: longer term chronic.•Public acceptance?

•Complex approvals process

•Fast, local•Low shelf life•Cold chain

Thank you!