Abecma
idecabtagene vicleucel · BCMA CAR-TNo relevant species; NSG mouse xenografts and in vitro only
Omisirge: which tox species, and why?
omidubicel · Expanded cord blood HPC
Species
Immunodeficient irradiated NOD/SCID mouse only (xenogeneic human cells)
“In vivo pharmacology, pharmacokinetic, and toxicology studies were conducted to evaluate the activity, distribution, and safety of the product in immunocompromised, irradiated mice.”
Why that species
NOD/SCID chosen for human cell engraftment over other strain in pilot; limited relevance to human homing
“Results showed that NOD/SCID mice were preferable over mice based on cell engraftment levels.”
Surrogate molecule
No surrogate; clinical-like human CF + NF cells tested in immunodeficient mice
“Like omidubicel-onlv, this test article consisted of cells that were expanded for with cytokines and NAM”
Species: general tox
Irradiated NOD/SCID mouse only (single-dose GLP toxicity study)
“The following toxicology study was conducted to evaluate the safety of the test article following administration in the NOD/SCID mouse.”
Species: DART
Not conducted; no reproductive organ findings in mouse toxicity study and conditioning regimen affects fertility
“Per the applicant, developmental and reproductive toxicology studies were not conducted because there were no test article-related toxicity findings in reproductive organs as assessed by histopathological analyses”
Species: carcinogenicity
Not conducted (not warranted based on product characteristics and safety profile)
“Carcinogenicity and developmental and reproductive toxicity studies were not conducted with omidubicel-onlv. These studies are not warranted based on the product characteristics and safety profile.”
Surrogate used in
No surrogate used; human CF + NF cells expanded with cytokines and NAM tested in mice
“Like omidubicel-onlv, this test article consisted of cells that were expanded for with cytokines and NAM”
Same question, other cell therapies
Alofisel
darvadstrocel · Allogeneic adipose MSCImmunocompromised athymic nude rats (GLP); nude mice for tumourigenicity
Amtagvi
lifileucel · TIL therapyNo animal studies with product; published murine and human TIL literature only
Aucatzyl
obecabtagene autoleucel · CD19 CAR-TNo relevant species; no in vivo tox. NSG xenograft (pharmacology) and human tissue cross-reactivity only
Breyanzi
lisocabtagene maraleucel · CD19 CAR-TNo relevant species; in vitro and NSG mouse xenograft only
Carvykti
ciltacabtagene autoleucel · BCMA CAR-TNo relevant species; NCG mouse xenografts, non-GLP NHP pilot with autologous NHP CAR-T
ChondroCelect
characterised viable autologous cartilage cells · Autologous chondrocytesNude mouse (ectopic), sheep and goat (orthotopic); non-GLP combined PD/PK/tox
Ebvallo
tabelecleucel · EBV-specific allogeneic T cellsSCID mice (EBV+ lymphoma xenograft, literature/PD); no tox species
Encelto
revakinagene taroretcel · CNTF (encapsulated RPE cell implant)Minipig, pig and rabbit (ocular tox/PK); rcd1 dog for pharmacology; rat and rabbit for rhCNTF DART
Gintuit
allogeneic cultured keratinocytes and fibroblasts in bovine collagen · Allogeneic keratinocytes + fibroblastsAthymic (nude) mice for pharmacology; rabbits, guinea pigs, mice for biocompatibility
Holoclar
autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cellsNo animal species; abridged programme of in vitro tumorigenicity assays and literature
Kymriah
tisagenlecleucel · CD19 CAR-TNo relevant species; immunodeficient mouse B-ALL xenograft (non-GLP) only
Lantidra
donislecel · Allogeneic pancreatic isletsNo studies with product; literature only (diabetic mice, rats, NHP, pigs)
Laviv
azficel-T · Autologous fibroblastsNo preclinical or toxicology studies conducted with the product
MACI
autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytesRabbit and horse, with analogous MACI (species-matched autologous cells on ACI-Maix)
Provenge
sipuleucel-T · PAP-GM-CSF (autologous APC)No conventional tox species; rat and mouse proof-of-concept studies with rodent surrogate products
Rethymic
allogeneic processed thymus tissue · Allogeneic thymus tissueNo relevant species; nude rat pharmacology studies only, no tox
Ryoncil
remestemcel-L · Allogeneic BM-MSCRat, baboon and pig using species-specific surrogate ceMSC
Spherox
spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroidsImmunodeficient (SCID/NSG) mice, Goettingen minipig, Merino sheep; no conventional tox
StrataGraft
allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblastsAthymic nude mice only (immunodeficient wound model)
Tecartus
brexucabtagene autoleucel · CD19 CAR-TNo relevant species; in vitro and syngeneic mouse surrogate CAR-T only
Tecelra
afamitresgene autoleucel · MAGE-A4 TCR-TNo relevant species; in vitro cross-reactivity/alloreactivity and murine xenograft pharmacology only
Tregzi
allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + TconNo tox species; sublethally irradiated immunodeficient mice (xenogeneic GVHD proof-of-concept) plus published murine literature
Yescarta
axicabtagene ciloleucel · CD19 CAR-TNo relevant species; syngeneic mouse lymphoma model with murine surrogate CAR-T (published, non-GLP)
Zalmoxis
allogeneic T cells modified with HSV-TK / dLNGFR (nalotimagene carmaleucel) · HSV-TK suicide-gene T cellsImmunodeficient NOD/SCID mice (GvHD xenograft models); no conventional tox species
Zemcelpro
dorocubicel · UM171-expanded cord blood CD34+Female sub-lethally irradiated NSG mice (human cells); UM171 compound PK/tox in NSG mouse, rat, cynomolgus monkey