Index A Antibiotic agents, 75 Abatacept, 557, 586, 590 Antibody dependent cellular cytotoxicity (ADCC), 30, 160, 604 Absorption, 167–168 Antibody-dependent cellular phagocytosis (ADCP), 160 Absorption, distribution, metabolism, elimination (ADME), 361 Antibody-drug conjugates (ADCs), 166, 489, 501 Absorption rate-limited kinetics, 441 Antibody fragments, 151 Accelerated degradation, 86 Antibody-mediated cytotoxicity, 544 Acceptable Operating Ranges, 79 Antibody-mediated rejection (AMR), 537, 547, 548 Acellular pertussis vaccines, 293 Antibody pharmacodynamics, 151 Actimmune®, 633 Antibody pharmacokinetics, 166–180 Active pharmaceutical ingredient (API), 83 Antibody structure, 151, 158–162 Actual wholesale price (AWP), 550 Anti-CD19, 371 Acute rejection, 544, 550 Anti CD20 antibodies, 491–498 Adalimumab, 557, 585 Anti CTLA4 antibodies, 512 Adaptive immune response, 285 Anti-drug antibodies (ADAs), 139, 159 Adaptive immune system, 281, 370 Anti-EGFR, 502–507 Adeno associated virus (AAV), 370 Anti-EGFR-strategies, 502–507 Adenosine deaminase severe combined immunodeficiency Anti-fibrotic, 368 disease (ADA SCID), 370 Antigen-presenting cells (APCs), 286 Adenovirus (Ad), 292, 370 Antigen processing, 287 Adipocytes, 365 Anti growth factor receptor antibodies, 502–504 Adjuvants, 286 Anti growth factor receptor antibodies anti-EGFR2, 507–508 Ado-Trastuzumab-/Trastuzumab Emtanside, 507 Anti-hGH antibodies, 446 Adsorption, 87, 89, 90 Anti-idiotypic IgE, 544 Adsorption chromatography, 69 Anti-IL-1β, 588 Advanced therapies, 357 Anti-IL-5, 603 Advanced therapy medicinal products (ATMPs), 357 Anti-IL-6 receptor, 589 Aerosol, 479 Anti-IL-12/IL-23, 589, 597 Affinity chromatography, 70 Anti-IL17A, 588 Aflibercept, 509 Anti-integrin, 597 Age related macular degeneration (AMD), 375 Antioxidants, 90, 393 Aggregation, 83, 85, 89, 90, 141 Anti PD1, 512–514 Alefacept, 593 Anti PD-L1 antibodies, 512–514 Alemtuzumab, 497, 538, 544 Antisense oligonucleotides (ASO), 309 Alkaline phosphatase (AP), 44 Antithrombin, 452, 463 Allergy-specific immunotherapy, 297 Anti-TNFα agents, 584 Alloantibodies, 538 Apheresis, 384 Allograft rejection, 537 Application of CRISPR-Cas9 in gene therapy, 340 Allometric scaling, 173 Aptamers, 307 α-helix, 19, 23, 24 Arthritides, 584 α2-Plasmin inhibitor (α2-PI), 452 Arthritis, 548 Alzheimer’s disease, 357 Aseptic conditions, 384 Anakinra, 586, 606, 634, 635 Aseptic manufacture, 361 Analytical techniques, 85 Assays for antibodies, 143–144 Anaphylactic reactions, 141, 544 Assisted reproductive technologies (ART), 429 Anaphylaxis, 603 Asthma, 584, 602 Anemia, 526 Asymmetric field flow field, 46 Angiogenetic, 368 Atelectasis, 482 Animal models, 140 Atopic dermatitis (AD), 584, 604 Anion exchange chromatography (AEX), 65 Atypical hemolytic uremic syndrome, 548 Ankylosing spondylitis (AS), 584 Autocrine, 98, 620 Anti-angiogenic antibodies, 508–509 Auto-fluorescence, 394 Anti-apoptotic, 368 Autoimmune disease, 291, 369, 546 © Springer Nature Switzerland AG 2019 645 D. J. A. Crommelin et al. (eds.), Pharmaceutical Biotechnology, https://doi.org/10.1007/978-3-030-00710-2 646 INDEX Autoimmune hyperthyroidism, 546 C Autologous, 359 Campath, 544 Autologous transfusion, 523 Canakinumab, 588, 606 Avacincaptad pegol, 308 Canakinumab, rilonacept, 557 Avonex®, 632 Cancer vaccines, 281 Capillary electrophoresis sodium dodecyl sulfate (CE-SDS), 45–46 B Capillary Zone Electrophoresis (CZE), 40 Bacille Calmette-Guérin (BCG), 291 CAR T cells, 364, 607 Bacterial contamination, 62 Cardiomyocyte, 372 Bacterial polysaccharides, 287 Cationic exchange chromatography (CEX), 65 Bacterial vector, 292 Cationic lipids, 319 Basiliximab, 538, 543, 549 Caveolin, 319 B cell acute lymphoblastic leukaemia, 371 CD3 receptor, 540 B-cell epitopes, 288 CD4+ epitopes, 294 B-cell memory, 291 CD4+ T-cells, 140, 287 B-cell receptor (BCR), 285 CD8+ epitopes, 294 B-cells, 140, 281, 285, 366 CD8+ T-cells, 287 B-cell tolerance, 139 CD19+, 364 Belatacept, 538 CD20, 546 Belimumab, 557, 592 CD25, 538, 542 Benralizumab, 557, 602, 604 CD28, 538, 549 Benzonase, 391 CD34+ cell, 364 Betaferon®, 632 CD52, 544 Betaseron®, 632 Cell and tissue transplantation, 359–364 β-Sheet, 19, 24, 25 Cell bank, 365 β-Strands, 24 Cell harvest, 384 β-Turn, 20 Cell immortalization technologies, 359 Bevacizumab, 509 Cell-mediated immunity, 281, 540 Big data, 196–198 Cell migration, 382 Bimekizumab, 609 Cellomics, 214 Binding assays, 52–53 Cell-penetrating peptides (CPPs), 319 Bioassays, 52–55 Cell plasticity technologies, 359 Biobanks, 195–196, 375 Cell surface marker, 373 Biodegradable microspheres, 99 Cell therapy, 357 Biofeedback system, 99 Cellular-mediated rejection, 538 Bioinformatics, 196–198 Cellular, Tissue and Gene Therapies Advisory Committee, 398 The Biologics Price Competition and Innovation Act Center for Biologics Evaluation and Research (CBER), 398 of 2009, 266 Certolizumab, 557 Biomarkers, 151, 201 Certolizumab pegol, 586 Biopharming, 223 Cetuximab, 141, 503 Bioreactor, 60, 389 Charge heterogeneity, 37–39 Biosensor, 99 Chemistry, manufacturing, and controls (CMC), 266 Biosensor-pump combinations, 99, 100 Chemogenomics, 229, 230 Biosimilars, 260, 261, 265–274, 523 Chemokines (CK), 368, 619 Biotechnological production, 57 Chimeric antigen receptor (CAR), 607 Bispecific antibodies, 180 Chimeric antigen receptor T cells (CART-T cells), 364 Bispecific T cell enhancer, 500 Chimeric MABs, 159 BiTE, 500 Chimeric mice, 373 Black-Box warning, 602 Chimeric monoclonal antibody, 543 Blastocyst, 365 Chronic idiopathic urticaria (CIU), 584, 605 Blinatumumab, 500 Chronic kidney disease (CKD), 444 Blood-brain barrier, 168 Chronic lymphocytic leukemia (CLL), 372, 546 Blood coagulation, 451 Chronic renal insufficiency (CRI), 444 Blood disorder, 363 Circular dichroism (CD), 36 Blotting, 43 Clarification, 63, 391 B-lymphocyte stimulator (BLyS), 592 Clathrin, 319 Bone/cartilage repair, 368 Clearance, 169–172 Bone marrow, 365 Clinical phases I–IV, 174 Bone morphogenetic protein (BMP), 374 CliniMACs Prodigy, 385 Bovine spongiform encephalopathy (BSE), 63 Closed-loop insulin pumps, 421 BRCA1, 196 Cluster of differentiation (CD), 364, 491 Brentuximab vedotin, 502 Clustered regularly interspaced short palindromic repeats Brodalumab, 557, 596 (CRISPR), 215, 315, 372 Buffer selection, 90 Collapse temperature (Tc), 93 INDEX 647 Colloidal aluminum salts, 300 Diafiltration (DF), 65, 391 Combination vaccine, 291 Dicer, 313 Combinatorial chemistry, 228 Differentiated cell, 363 Combined ATMP, 363 Diffuse large B-cell lymphoma, 371 Committee for Advanced Therapies (CAT), 398 3D-cell cultures, 225, 226 Committee for Medicinal Products for Human Use (CHMP), 3D technologies, 379 398 Dimethyl sulfoxide (DMSO), 382 CompacT SelecT, 399 Diphtheria toxoid, 293 Comparative genomics, 195–196 Direct-to-consumer genomic tests, 206 Complementarity determining regions (CDRs), 144, 159 Discontinuous epitopes, 288 Complement-dependent cytotoxicity (CDC), 160 Disease modelling, 365 Complement factors, 285 Disease-modifying antirheumatic drugs (DMARDs), 584 Complement-mediated cell lysis, 544 Distribution, 168–169 Conjugated antibodies, 489 Disulfide bond shuffling, 30 Contaminants, 73–76 Disulfide bridge formation, 30 Continuous subcutaneous insulin infusion (CSII), 421 DNA, 73 Corifollitropin, 433 DNA microinjection, 218, 219 Cost of goods, 389 DNase treatment, 391 Cost of illness (COI), 257, 262 DNAzymes, 309 Cost-benefit analysis (CBA), 258, 262 Dornase alfa, 471 Cost-effectiveness analysis (CEA), 258, 259, 262 Dosing regimen, 166 Co-stimulation blockade, 549 Double-stranded DNA, 473 Co-stimulatory molecules, 286 Downstream processing (DSP), 57, 391 Cost-minimization analysis (CMA), 258, 262 Drisapersen, 317 Cost-utility analysis (CUA), 259, 260, 262 Drug development, 117, 119, 122, 131 CpG motifs, 306 Druggable genome, 199 Creutzfeldt-Jakob disease, 437 Drug repositioning, 230 CRISPR-associated protein-9 nuclease (Cas9), 217, 315, 372 Drug reprofiling, 230 CRISPR-associated proteins (Cas), 217 Drug repurposing, 230 CRISPR-Cas9 system, 339–341 Drug screening, 365 CRISPR-Cpf1, 217 Duchenne’s muscular dystrophy (DMD), 316 Critical Material Attributes (CMAs), 78 Dupilumab, 557, 605 Critical Process Parameters (CPPs), 78 Critical Quality Attributes (CQAs), 78 Crohn’s disease (CD), 366, 596 E Crohn’s Disease Activity Index (CDAI), 597 Economic considerations, 253–262 Cryopreservation, 365 Ectoderm, 366 Cryoprotectants, 91, 393 Eculizumab, 538, 548 Cryopyrin-associated periodic syndrome (CAPS), 584, 606 Edifoligide, 312 Current good manufacturing practice (cGMP), 266 Efalizumab, 158, 593 CYP enzymes, 172 Electric dipole moments, 24 Cystic fibrosis (CF), 471 Electrochemiluminescence, 395 Cytokine engineering, 637–638 Electronic health records (EHRs), 197 Cytokine release syndrome (CRS), 371, 541, 584, 607 Electrophoresis, 34 Cytokines, 286, 365, 548, 619 Electrospray ionization (El), 35 Cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4), 549, 590 Electrostatic interactions, 28 Cytotoxic T-lymphocytes (CTLs), 285, 366 Elotuzumab, 498 Elotuzumb safety, 498–500 Elutriation, 384 D Emapticap pegol, 308 Daclizumab, 538, 542, 545 Embryogenesis, 373 Daratumumab, 498–500 Embryonic stem cells, 363 Darbepoetin alfa, 522 Empyema, 482 Deamidation, 37–39 Endocrine, 98, 620 De-differentiation, 374 Endoderm, 366 Delivery of proteins, 95 Endogenous antigens, 288 Denaturation, 86 Endothelial cell, 379 Dendritic cells (DCs), 286, 359 Endotoxins, 62, 75, 395 Deoxyribonuclease I (DNase I), 471 Engineered TCR cell therapies, 371 Deoxyribonucleic acid (DNA),