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Atlas/Cell therapy/Tregzi/Species

Tregzi: which tox species, and why?

allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + Tcon
Check my plan vs. these
Species
No tox species; sublethally irradiated immunodeficient mice (xenogeneic GVHD proof-of-concept) plus published murine literature
“In the applicant’s POC studies using xenogeneic models, co-administration of human donor Tregs with Tcons demonstrated dose-dependent GVHD inhibition in sub-lethally irradiated immunodeficient mice.”
✓ VerifiedFDA pharmacology/toxicology review, p. 3Source ↗
Why that species
Immunodeficient mice allow human Tcon-induced xenogeneic GVHD and Treg mitigation; no relevant tox species stated
“evaluating the ability of human Tcons to induce xenogeneic GVHD”
✓ VerifiedFDA pharmacology/toxicology review, p. 3Source ↗
Surrogate molecule
Yes: published murine HSC, Treg, Tcon studies used as mechanism-of-action support; human cells tested in mouse xenograft
“Instead, in Module 4 of the BLA, the applicant submitted 45 publications delineating the MOAs of HSCs (mouse and human), Tregs (mouse), Tcons (mouse), and their combinations (mouse).”
✓ VerifiedFDA pharmacology/toxicology review, p. 8Source ↗

Same question, other cell therapies

Abecma

idecabtagene vicleucel · BCMA CAR-T
No relevant species; NSG mouse xenografts and in vitro only

Alofisel

darvadstrocel · Allogeneic adipose MSC
Immunocompromised athymic nude rats (GLP); nude mice for tumourigenicity

Amtagvi

lifileucel · TIL therapy
No animal studies with product; published murine and human TIL literature only

Aucatzyl

obecabtagene autoleucel · CD19 CAR-T
No relevant species; no in vivo tox. NSG xenograft (pharmacology) and human tissue cross-reactivity only

Breyanzi

lisocabtagene maraleucel · CD19 CAR-T
No relevant species; in vitro and NSG mouse xenograft only

Carvykti

ciltacabtagene autoleucel · BCMA CAR-T
No relevant species; NCG mouse xenografts, non-GLP NHP pilot with autologous NHP CAR-T

ChondroCelect

characterised viable autologous cartilage cells · Autologous chondrocytes
Nude mouse (ectopic), sheep and goat (orthotopic); non-GLP combined PD/PK/tox

Ebvallo

tabelecleucel · EBV-specific allogeneic T cells
SCID 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 + fibroblasts
Athymic (nude) mice for pharmacology; rabbits, guinea pigs, mice for biocompatibility

Holoclar

autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cells
No animal species; abridged programme of in vitro tumorigenicity assays and literature

Kymriah

tisagenlecleucel · CD19 CAR-T
No relevant species; immunodeficient mouse B-ALL xenograft (non-GLP) only

Lantidra

donislecel · Allogeneic pancreatic islets
No studies with product; literature only (diabetic mice, rats, NHP, pigs)

Laviv

azficel-T · Autologous fibroblasts
No preclinical or toxicology studies conducted with the product

MACI

autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytes
Rabbit and horse, with analogous MACI (species-matched autologous cells on ACI-Maix)

Omisirge

omidubicel · Expanded cord blood HPC
Immunodeficient irradiated NOD/SCID mouse only (xenogeneic human cells)

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 tissue
No relevant species; nude rat pharmacology studies only, no tox

Ryoncil

remestemcel-L · Allogeneic BM-MSC
Rat, baboon and pig using species-specific surrogate ceMSC

Spherox

spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroids
Immunodeficient (SCID/NSG) mice, Goettingen minipig, Merino sheep; no conventional tox

StrataGraft

allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblasts
Athymic nude mice only (immunodeficient wound model)

Tecartus

brexucabtagene autoleucel · CD19 CAR-T
No relevant species; in vitro and syngeneic mouse surrogate CAR-T only

Tecelra

afamitresgene autoleucel · MAGE-A4 TCR-T
No relevant species; in vitro cross-reactivity/alloreactivity and murine xenograft pharmacology only

Yescarta

axicabtagene ciloleucel · CD19 CAR-T
No 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 cells
Immunodeficient 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

Summary across all 27 cell therapies →