1. Trinity College Dublin 12 April 2018 Hugh McMahon
Introduction to ATMPs
QP Forum
Trinity College Dublin 12 April 2018
Hugh McMahon
Principal Consultant/QP

2. Advanced Therapy Medicinal Products - Definitions
Tissue engineered (TE) products - growth and development of biomaterials using engineering principles and techniques, especially the synthesis of body tissues and whole organs for the restoration or replacement of a damaged or diseased body part.
Gene therapy (GT) medicinal products - the treatment of certain medical disorders, especially those caused by genetic anomalies or deficiencies, by introducing specific engineered genes into a patient's cells.
Somatic Cell therapy (SCT) - where living cells are injected into patient e.g. T- cells capable of fighting cancer cells via Cell –Mediated immunity may be injected in the course of immunotherapy
Combined ATMPs contain one or more medical devices as an integral part of the medicine, such as cells embedded in a biodegradable matrix or scaffold.
FDA defines ATMPs as: Cellular therapy products (that) include cellular immunotherapies and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.
Significant guideline …potential will set the global regulatory tone for ATMPs
General sections such as Personnel, Premises, Equipment, Documentation, Starting and Raw Materials, Production, Qualification and Validation, QP and Batch Release, QC, Quality Defects and Recalls follow fairly standard and well established GMP principles. However the following slides highlight some unusual aspects.

3. Gene Therapy – Mechanism of Insertion of
Functional Gene
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4. Luxturna - Gene Therapy for RPE65 Related Vision Loss
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5. Chimeric Antibody Receptor - CAR-T Cell Therapy
How CAR-T cells work
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6. Composition of EU ATMP Orphan Drug Designations
Since 2001 (185)
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7. CAR-T Cell Clinical Trials Over Time and Geographical Distribution
Jessica Hartmann et al. EMBO Mol Med. 2017;emmm
CAR T cell trials over time and geographical distribution (A) Timeline of cancer CAR T cell trials as listed in Datasets EV1 and EV2 distinguishing between ongoing number (dark blue bars) and newly initiated trials in the indicated year (light blue bars). (B) Geographical distribution of worldwide ongoing CAR T cells clinical trials (left) and distribution of trial sites of the ongoing European studies (right). Five studies are multi‐centric, of which four are multi‐country trials in Europe (Dataset EV5). Long‐term follow‐up studies are not included. Color code indicates the prevalence of trials from low (green) to high (red).
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8. Cost of ATMP Therapies and Potential Patient Populations
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9. Moore’s LAW VS Cost OF Genomic SEQUENCING
Cost of ATMP Therapies and Potential Patient Populations

10. Table of ATMP’s Approved by EMA
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11. Overview of First 12 Months of EMA Priority Medicine Designation
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12. Thank you for your attention
Questions?