Module 9 2021

List of abbreviation and acronyms

Acronym Full name AAV

Adeno Associated Virus

Acronym Full name IR

ADA

Adenosine Deaminase Deficiency

ImmuneReconstitution

ADME

Administration Distribution Metabolism and Excreaction

LV

LentiVirus

MA

Market Authorization Monoclonal Antibody

AdV ALL

AdenoVirus

mAb

Acute Lymphoblastic Leukaemia Advanced Therapy Medicinal Product

MABEL

Minimal Assessable Biological Effect Level

ATMPs

MoA MOI MSC MW NCA

Mechanism of Action Multiplicity of infection

BM

Bone Marrow

CAR CAS CAT

Chimeric Antigen Receptor

Mesenchimal Cells Molecular Weight

Committee for Advanced Therapies

National Competent Autority NOD/SCID Non-Obese Diabete Severe Combined ImmunoDeficient (Mouse) OBA Office of Biological Affairs PD Pharmacodynamics PK PharmacoKinetics PMF Plasma Master File PoC Proof of Concept Pox Pox Virus RCR Replication Competent Retrovirus RCV Replication Competent Virus RNAi Interfering RNA RV Retrovirus SAWP Scientific Advice Party sCMP Somatic Cell Based Medicinal Product SME Small and Medium Enterprise TAA Tumour Associated Antigen TEP Tissue Engineered Product Treg Regulatory T cells TSE Transmissible Spongiform Encephalopathy VCN Vector copy Number 64

CBER CHMP

Centre of Biological Evaluation and Research

Committee for

CoA

Certificate of Analisys

COMP

Committee for Orphan Medicinal Products

CoO

Certificate of Origin

CRISPr CTD

Common Technical Document

DC

Dendritic Cell

DLI

Donor Limphocyte Infusion

DP DS

Drug Product

Drug Substance

EDQM

European Directorate of Quality Medicine

EM

Electron Microscope

ERA FDA GLP

Environmental Risk Assessment Food and Drug Administration

Good Laboratory Practice

GTMP GvHD

Gene Therapy Based Medicinal Product

Graft versus Host Disease

HC DNA Novartis Pharma AG- Business use only- not for further distribution

Host Cell DNA Host Cell Protein

HCP

HSPC

Hematopoietic Progenitor Stem Cells