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Index
Note: ‘Page numbers followed by f indicate figures and t indicate tables.’
A
Absorption, distribution, metabolism, and excretion
(ADME), 277, 426
Accelerating Medicines Partnership (AMP), 285
Active pharmaceutical ingredient (API), 4
Acute graft rejection, 326
Acute hypersensitivity, 73
Acute lymphoblastic leukemia (ALL), 107, 316
Acute myeloid leukemia (AML), 299, 321e322
Acute respiratory distress syndrome (ARDS),
294e295
Adaptive immune system, 296
Adeno-associated vectors/virus (AAVs), 258,
263e266, 282
gene therapies (GTs), 266, 291
serotypes, 293
transduction process, 265
Adenoeassociated virus receptor (AAVR), 265
Adenoviridae, 263e264
Adenovirus (AdV), 263e264
vector treatment, 290
Adjuvants, 210e211
advancements in adjuvant technologies, 189e192
arms of immune system, 180e184, 182te183t
regulation of vaccines, 184e185
United States Food and Drug Administration
(US FDA), 185e193, 186te188t, 190te191t
impacts on human health, 193
science and business of vaccinology, 193e199
serious infectious diseases, 201e205
antimicrobial resistant bacteria, 201e202, 204t
parasitic diseases, 204e205
therapeutic vaccines, 205e209
complexity from product quality perspective,
209e210, 210t
development using animal rule, 211e213, 212t
nonclinical testing, 210e211
platforms, 205e209, 208te209t
vaccines with, 245
World Health Organization’s (WHO) threats
to global health, 193e199, 202t
hesitancy and importance of vaccinology
education, 213e217
Adoptive T cell transfer (ACT), 316
Adoptive transfer T-cell receptor (TCR) -transduced
T cells, 336e337
Adult stem cells, 314e315, 401
Advanced Cell Technology, Inc. (ACT), 439
Adverse effects of special interest (AESI), 216
Adverse reactions, 96
Af
finity maturation, 336e337
Age-related macular degeneration (AMD), 46e47,
406, 439
Alanine transaminase (ALT), 292
Albumin, 140e141
Albumin fusion proteins, 25
Allogeneic cells, 318
Allogeneic chimeric antigen receptor T (AlloCAR T)
cell products, 335
therapies for cancer, 337e338
Allogeneic engineered T cells, 335e336
Allogeneic mesenchymal stem cells (MSCs),
406
Allogeneic mouse pregnancy model, 387
Allogeneic stem cells, 326
Allogeneic vaccines, 205
Allometric models, 286e287
Allometric scaling, 151
AlphaLISA, 157e158
Alpha-retroviruses, 266
Alzheimer’s vaccines, 245
American Society for Transplantation and
Cellular Therapy (ASTCT), 329
American Society of Gene and Cell Therapy
(ASGCT), 285
AMG X case study, 121
Amyloid-beta (Ab) peptides, 48
Amyloid precursor protein (APP), 48
Amyotrophic lateral sclerosis (ALS), 406
Anaphylaxis, 215e216
Anesthetics, 380
Angiopep-2 (AN2), 48
Animal models, 231e232, 240, 244, 247
cytokine release syndrome (CRS)
human disease, 274
neurotoxicity assessments, 331e332, 331f
451
Animal models (Continued )
nonclinical efficacy species studies, viral vector gene
therapies (GT), 274e275
selection of, 422e425, 422t
Animal protection data, extrapolation of, 212
Animal-sourced reagents, 409
Animals, physiological state of, 354
Animal tissues, 64e65
Antagonistic monoclonal antibodies, 88
Antemortem parameters, 243
Anthracycline-based cytotoxics, 376
Antibodies (Abs), 23, 102, 134e135, 248, 297
design, 104e106
development, 101
fragments (Fabs), 20e21, 135e136
modalities, 101e107
bispecific and multimer antibody modalities,
104e107
effector modified antibodies, 102e104, 104t
monoclonal antibody (mAb) therapies, 101e102,
103f
proteins, 107e108
Antibody-dependent cell-mediated cytotoxicity
(ADCC), 20, 45, 102, 140, 359
Antibody-dependent cellular phagocytosis (ADCP),
20, 102, 140
Antibody-drug conjugates (ADCs), 12e13, 20e21, 96,
101, 142e143
Anticancer drugs, 85
special considerations for, 84e86
Anticancer pharmaceuticals, 359
Antidrug antibodies (ADAs), 33e34, 71, 106, 108, 142,
354, 367e368
pharmacological activity and formation of, 42e43
reactions, 101
screening assays, 164e165
Antigen-presenting cells (APCs), 181, 246, 377
Antigens, 207
specific antibody responses, 247
specific immune response, 234
Antiinsulin antibodies, 108
Antimicrobial resistant (AMR), 201e202
bacteria, 201e202
vaccine pipeline, 203t
Antinuclear antibody (ANA), 379e380
Anti-TfR/BACE1, 48
Antitherapeutic antibodies, 8
Antivaccine, 214
Area under the curve (AUC), 4, 355
Aromatic
L-amino acid decarboxylase (AADC), 284
Array comparative genomic hybridization (aCGH),
413
Arthropod vectors, 234e235
Artificial intelligence (AI) approaches, 11
Artificial transport vehicle (ATV), 48
Aspartate transaminase (AST), 292
Assays, vaccines, 246e247
Assessment of ADAs, 362e363
Atezolizumab, 384
Attributable, legible, contemporaneous, original,
accurate (ALCOA), 232e233
Autoimmune disease, 242
Autologous cell vaccines, 205
Autologous mesenchymal stem cells, 325e326
Autologous stem cells, 326
Avastin, 369e387
Avelumab, 384
B
Bacillus CalmetteeGuérin (BCG) vaccine, 181e184
Base editing, 434
Basic fibroblast growth factor (bFGF),
133e134
B-cell maturation antigen (BCMA), 42, 316
BCL2L1 gene, 417e418
Bespoke Gene Therapy Consortium (BGTC), 285
Beta 2-microglobulin (B2M) gene, 328
Beta-retroviruses, 266
Bevacizumab, 369e387
case study, 120
related renal adverse effects, 373
trastuzumab, 374e377
Bioanalytics method, 152e166
Biodistribution (BD), 140e144, 427e428
analyses, 277
ex vivo and in vivo gene therapies in, 338e339
human-specific markers, 428
immunogenicity impact on, 145e146
metabolism and, 144e145
nonclinical vaccine development, 234
reporter genes, edited cells, and Y chromosomes,
428e429
species and model selections, 429e430
Biologic License Application (BLA), 320
Biologic therapeutic, 353
Biologic therapies, 3e4. See also Cellular therapies
Biological medicines
anticancer drugs, special considerations for,
84e86
carcinogenicity, 81e84
developmental and reproductive toxicology (DART),
356e358
embryoefetal development (EFD) and/or pre-and
postnatal development (PPND), 356e357
fertility, 357
species selection considerations, 356e357
Index452
embryo-fetal development (EFD) and enhanced
pre-and post-natal development (ePPND), 77f,
78e81, 79t
first-in-human (FIH) clinical trial, 86e89
no-observable-adverse-effect level (NOAEL)
considerations, 88
nonclinical data to clinical trial dose selection,
88e89
nonclinical safety program considerations, 87e88,
87t
genotoxicity, 81e84
immunogenicity, 71e73
nonclinical studies of biopharmaceutical candidates,
61t
repeat-dose studies, 66e71
dose selection, 68e69
recovery periods, 70e71
route of administration, 69e70
safety pharmacology endpoints, 68
reproductive and developmental toxicity, 74
species selection, 61e66, 63t
considerations, 74e76
identified relevant species, 65e66, 65t
relevant species, 62
screening paradigms, 62e64
tissue cross-reactivity (TCR) assay, 64e65
studies for assessment of reproductive toxicity,
76e81
fertility and early embryonic development (FEED),
77e78
Biologics, 14, 351, 360
Biologics License Application (BLA), 184
Bioluminescence, 429
Biomarkers, 163e164
cytokine release syndrome (CRS) and neurotoxicity
animal models, 331e332
utility for predicting, 331
in vitro models for biomarker assessment, 332
Biopharmaceuticals, 59, 132, 430
and comparison with small-molecule drug
development
carcinogenicity assessment, 9
classes, 11e14
nonclinical species for toxicology testing, 7e8
routes of administration, 6e7
small molecule and protein-based
biopharmaceutical therapeutics, 5t
toxicology program design in of coronavirus
disease 2019 (COVID-19) pandemic, 9e11
drugs, 246
differences in nonclinical safety assessment of,
248e249
product, 70
Biosimilar, 12
Biotechnology Innovation Organization (BIO), 9e10
Biotherapeutic-induced hematotoxicity,
mechanisms of, 117e120, 118te119t
Biotherapeutics, 7, 96
bispecific antibodies of endogenous receptor ligands,
47e48
drugs, 4
general safety considerations related to, 97e110
antibody-based modalities, 101e107
recombinant (nonantibody) protein therapeutics,
107e110
target liability assessment, 97e100
in vivo studies, 100e101
immunotoxicity, evaluation of, 110e116
hypersensitivity reactions, 116
immunostimulatory biotherapeutics, 112
immunosuppressive biotherapeutics, 111e112
tumor lysis syndrome (TLS), first dose effect and
cytokine release syndrome (CRS) induced by
monoclonal antibodies (mAbs), 112e116,
114fe115f
potential off-target toxicity, 116e122
hematologic toxicity, 117e122, 118te119t
safety profile for, 97, 98te99t
Bispecific antibodies, 43e44, 104e106
of endogenous receptor ligands for central nervous
system (CNS) delivery, 47e48
and multimer antibody modalities, 104e107
bispecific T-cell engager (BiTE) case study,
106e107
Bispecific T-cell engagers (BiTEs), 24e25, 96, 104e107
Bloodebrain barrier (BBB), 47
bispecific antibodies of endogenous receptor ligands,
47e48
Blood products, 58
Blood urea nitrogen (BUN), 373
Bone marrow/liver/thymus (BLT), 424
Botulism Antitoxin Heptavalent (BAT), 213
Bovine serum albumin (BSA), 373e374
Brain and b-secretase (BACE1), 48
5-bromo-2-deoxyuridine (BrdU), 428
Busulfan, 327
C
Cancer antigen-recognizing tumor (CAR-T) therapy,
207e209
Cancer, biological therapies for
bevacuzimab, 369e387
development of relatlimab as combination
immunotherapy, 385e387
endogenous human proteins, recombinant analogs
of, 387e390
Index 453
Cancer, biological therapies for (Continued )
global regulatory guidances, 351e352
immunotherapy, 350
marketed biologics, nonclinical development of, 364
monoclonal antibodies (MAbs), 366e367
nonclinical studies and principles of study design,
355e359
developmental and reproductive toxicology
(DART), 356e358
genotoxicity and carcinogenicity, 358e359
immunotoxicity, 359
safety pharmacology, 356
specialty toxicology assessments, 356
programmed cell death-1 (PD-1) and developmental
and reproductive toxicology (DART), 384e385
rituximab, 367e369
tissue-binding study, 359e363
pharmacokinetics (PK), immunogenicity, and
pharmacodynamics (PD), 360e363
toxicology assessments, general principles of,
352e355
Cancer immunotherapy, 350e351
Cancer vaccines, 245
Candida albicans, 111e112
Cap gene, 264e265
Capsid envelope immune responses, 297
Carcinogenesis, 9
Carcinogenicity, 81e84, 235, 282e283, 358e359.
See also Tumorigenicity
assessment, 9
studies, 249, 358e359
Carcinogenic potential, 434
Cardiac tissues, 231e232
Cardiotoxicity, 294e295
Cardiovascular (CV) diseases, 206, 235
Case-by-case approach, 319
Catumaxomab, 36e38, 41
CD47, 328
Cell-associated neurotoxicity syndrome (ICANS),
40e41
Cell-based product, 419
Cellebased therapies, 8, 314, 316, 407e408, 413e415,
419e420, 426, 433
Cell dose, 427e428
Cell fate, 325
Cell-labeling methods, 427e428
Cell line
characterization, 412e413
critical decisions in cell line selection, 410e412
donor screening and testing requirements, 411t
informed donor consent for pluripotent stem cell
(PSC) research, 412t
proposed cell line provenance checklist, 411t
master cell bank (MCB) characterization of
pluripotent stem cell (PSC) lines, 414t
working cell bank (WCB) characterization of
pluripotent stem cell (PSC) lines, 415t
Cell-mediated immune response assays, 247
Cell microarray technologies, 334
Cell products, 413e415
Cell proliferation, 82
Cell surface receptors, 325
Cell trafficking and persistence, 427e428
Cellular attributes, 417e418
Cellular cancer vaccines, 205
Cellular immunotherapies, 316
Cellular targets, 139e140
Cellular therapies, 319
advantages and disadvantages of stem cells, 329f
basic principles based on regulatory guidance,
316e321
species selection, 319e320
study design components, 320e321
development challenges, 321e339
challenges associated with chimeric antigen
receptor T (CAR-T)-cell therapies, 328e339
challenges associated with stem cell therapies,
321e328
cytokine release syndrome (CRS) and
neurotoxicity, 329e332
main types of stem cells, 314f
models for cytokine release syndrome (CRS) and
neurotoxicity, 331e332
clustered regularly interspaced short palindromic
repeats (CRISPR) and transcription activator-like
effector nuclease (TALEN), 338e339
graft-versus-host disease (GVHD) and
rejection with allogeneic engineered T cells,
335e336
insertional mutagenesis and clonal dominance,
337e339
models for cross-reactivity, 337
models for on-target, off-target, and off-tumor
toxicity, 333e334
on-target and off-target, off-tumor toxicity,
332e334
T-cell receptor cross-reactivity, 336e337
utility of biomarkers for predicting cytokine
release syndrome (CRS) and neurotoxicity, 331
Center for Biologics Evaluation and Research (CBER),
184, 284e285, 403, 437
Center for Devices and Radiological Health (CDRH),
403, 421e422
Center for Drug Evaluation and Research (CDER), 58,
403
Central nervous system (CNS), 68, 235
Index454
bloodebrain barrier (BBB), bispecific antibodies of
endogenous receptor ligands for, 47e48
disorders, 406
Cerebral adrenoleukodystrophy (CALD), 284
Certolizumab, 118te119t
Cetuximab, 41
Cetuximab-based bispecific T-cell engager antibodies
(C-BiTE), 41
Chemical cross-linking approaches, 23e24
Chemical immunosuppression models,
424e425
Chemistry, manufacturing, and controls (CMC),
209e210, 270e273
early decisions regarding, 270
producer cell line selection, 270e271
product characterization and potency assays,
272e273
product format and formulation, 271e272
toxicology studies, 277
vector development, 270
in vivo PKPD, 278
Chemotherapy, 350
Chimeric antigen receptor (CAR), 14
macrophages, 332
natural killer (NK) cells, 332
Chimeric antigen receptor T (CAR-T) cells, 268,
316e317, 332
adverse events associated with engineered T cells,
329f
challenges associated with, 328e339
therapy-harvested T cells, 316
Chromium (51Cr) -release assay (51Cr assay), 332
Chromosomal defect, 389
Chromosomal stability, 417e418
Chronic diseases, 69e70
Class II Major Histocompatibility Complex
Transactivator (CIITA) gene, 328
Climate changes, 202e204
Clinical Trial Application (CTA), 320
Clinical trial design, 287e289
Clonal dominance, 337e339
Clotting factors, 110
Cluster designation 19 (CD19), 316
Clustered regularly interspaced short palindromic
repeats (CRISPR), 12, 328
associated nucleases, 408e409, 433
clustered regulatory interspaced short palindromic
repeats (CRISPR) -Cas, 274e275
gene-editing system, 328, 338e339
for genotoxicity, 338e339
Coagulation disorder, 45e46
haemophilia, 45e46
Coagulation factor therapy, 112e113
Coalition for Epidemic Preparedness Innovations
(CEPI), 195
e196
Combination immunotherapy, development of
relatlimab as, 385e387
Combination therapy, 21e23
Combinatorial models, 424
Committee for Medicinal Products for Human Use
(CHMP), 88e89
Common light chains (CLCs), 26
Compact disc (CD) formats, 154
Comparability testing, ICH Q5E, 70
Compartmental pharmacokinetic modeling, 147
Competitive intelligence, 99e100
Complement activation-related pseudoallergy
(CARPA), 116
Complementarity determining regions (CDRs), 21, 64,
87e88
Complement-dependent cytotoxicity (CDC), 20, 140,
374e375, 378
Complement system, 296
Complex innovative trial designs (CIDs), 288
Controlled Fab-arm exchange (cFAE), 26
Controlled human infection models (CHIMs),
192e193
Conventional biotherapeutic approaches, 97, 99t
Conventional immunosuppressants, 298
Copy number variations (CNV), 417e418
Coronavirus disease 2019 (COVID-19) pandemic, 9,
76, 81, 240, 287
toxicology program design in wake of, 9e11
vaccines, 240
Coronavirus disease (COVID-19) vaccines global
access (COVAX), 199e200
Coxsackievirus and adenovirus receptor (CaR),
263e264
Critical quality attributes (CQAs), 272e273
Cross-reactive immunological material (CRIM),
297e298
Cross-reactivity, models for, 337
Cross-species immunogenicity, 319e320
CTLA-4, 366e367, 379
Current Good Manufacturing Practices (cGMP), 211,
409
Cyclophosphamide, 327
Cyclosporine, 298
Cytokine release assays (CRAs), 113e116, 359
Cytokine release syndrome (CRS), 106, 112, 328
animal models for, 331e332
induced by monoclonal antibodies (mAbs), 112e116
and neurotoxicity, 329e332, 330f
models for understanding, 331e332
utility of biomarkers for predicting, 331
Cytokines, 40
Index 455
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