cell therapy: from idea to marketDaniela Couto

June 2015

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Phase 1 Phase 2 Phase 3Pre-clinical

Phase 1 Phase 2 Phase 3Pre-clinical

challenging to manufacture

tailor-made quality control regulatory uncertainty

challenging to manufacture

tailor-made quality control regulatory uncertainty

silver-lining: tough to raise capital

• design GMP facility • certify GMP facility • establish batch record

• design GMP facility • certify GMP facility • establish batch record • scale-up manufacturing

• design GMP facility • certify GMP facility • establish batch record • scale-up manufacturing • scale-out manufacturing

• design GMP facility • certify GMP facility • establish batch record

• design GMP facility • certify GMP facility • establish batch record • qualify operators • “tailor-made” quality control

Phase 1 Phase 2 Phase 3Pre-clinical

• design & design GMP facility • establish batch record • scale-up manufacturing • qualify operators • “tailor-made” quality control

• scale-out manufacturing

• fine balance between flexibi- lity versus clear requirements • design & discuss clinical trials • request ATMP certification • request SME status • request orphan designations • request scientific advice

Phase 1 Phase 2 Phase 3Pre-clinical

• ATMP certification • SME status • design clinical trials

• orphan designations • scientific advice

Key aspects in fundraising: • team • geographical location • product / technology • IP • University spin-off

Amusing limiting steps: • right door • tradition • additional travelling (location) • placement agents vs. personal

Key aspects in fundraising: • team • geographical location • product / technology • IP • University spin-off