Encelto
revakinagene taroretcel · CNTF (encapsulated RPE cell implant)rhCNTF DART NOAEL margins of exposure above 100 vs modelled maximum blood rhCNTF from intraocular implant
Spherox: what safety margin did regulators accept?
spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroids
Safety margin at NOAEL
Minipig study up to 3.2-fold the maximum clinical dose; other studies at doses similar to the clinical dose
“Except for one study in the minipig, in which up to 3.2 fold the maximum clinical dose was applied, all studies used doses similar to the intended clinical dose.”
NOAEL / key findings
No NOAEL; no treatment-related adverse effects or signs of general/local toxicity in any study
“There were no indications of treatment related adverse effects and the implant was well tolerated.”
Same question, other cell therapies
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
Ebvallo
tabelecleucel · EBV-specific allogeneic T cellsNot 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