Encelto
revakinagene taroretcel · CNTF (encapsulated RPE cell implant)rhCNTF DART NOAEL margins of exposure above 100 vs modelled maximum blood rhCNTF from intraocular implant
Ebvallo: what safety margin did regulators accept?
tabelecleucel · EBV-specific allogeneic T cells
Safety margin at NOAEL
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.
NOAEL / key findings
No NOAEL; no overt toxicity (activity loss, weight loss) after single dose in immunodeficient mice
“Studies conducted in immunodeficient animal models for EBV+ PTLD revealed no overt signs of toxicity (e.g. loss of activity or weight loss) associated with a single dose of tabelecleucel.”
Same question, other cell therapies
Spherox
spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroidsMinipig study up to 3.2-fold the maximum clinical dose; other studies at doses similar to the clinical dose
Zalmoxis
allogeneic T cells modified with HSV-TK / dLNGFR (nalotimagene carmaleucel) · HSV-TK suicide-gene T cellsMouse target dose ~two log above proposed clinical dose; this dose caused 100% GvHD mortality (no NOAEL)
Zemcelpro
dorocubicel · UM171-expanded cord blood CD34+UM171: no toxicity in rat/monkey at doses 8,000-20,000x estimated human exposure
Abecma
idecabtagene vicleucel · BCMA CAR-TNot stated in public reviews
Alofisel
darvadstrocel · Allogeneic adipose MSCNot stated in public reviews
Amtagvi
lifileucel · TIL therapyNot stated in public reviews
Aucatzyl
obecabtagene autoleucel · CD19 CAR-TNot stated in public reviews
Breyanzi
lisocabtagene maraleucel · CD19 CAR-TNot stated in public reviews
Carvykti
ciltacabtagene autoleucel · BCMA CAR-TNot stated in public reviews
ChondroCelect
characterised viable autologous cartilage cells · Autologous chondrocytesNot stated in public reviews
Gintuit
allogeneic cultured keratinocytes and fibroblasts in bovine collagen · Allogeneic keratinocytes + fibroblastsNot stated in public reviews
Holoclar
autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cellsNot stated in public reviews
Kymriah
tisagenlecleucel · CD19 CAR-TNot stated in public reviews
Lantidra
donislecel · Allogeneic pancreatic isletsNot stated in public reviews
Laviv
azficel-T · Autologous fibroblastsNot stated in public reviews
MACI
autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytesNot stated in public reviews
Omisirge
omidubicel · Expanded cord blood HPCNot stated in public reviews
Provenge
sipuleucel-T · PAP-GM-CSF (autologous APC)Not stated in public reviews
Rethymic
allogeneic processed thymus tissue · Allogeneic thymus tissueNot stated in public reviews
Ryoncil
remestemcel-L · Allogeneic BM-MSCNot stated in public reviews
StrataGraft
allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblastsNot stated in public reviews
Tecartus
brexucabtagene autoleucel · CD19 CAR-TNot stated in public reviews
Tecelra
afamitresgene autoleucel · MAGE-A4 TCR-TNot stated in public reviews
Tregzi
allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + TconNot stated in public reviews
Yescarta
axicabtagene ciloleucel · CD19 CAR-TNot stated in public reviews