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Atlas/Cell therapy/Zemcelpro/Safety margin

Zemcelpro: what safety margin did regulators accept?

dorocubicel · UM171-expanded cord blood CD34+
Check my plan vs. these
Safety margin at NOAEL
UM171: no toxicity in rat/monkey at doses 8,000-20,000x estimated human exposure
“Intravenous administration at doses 8,000-20,000 times higher than the estimated human exposure to UM171 demonstrated no toxicity in rats (single-dose study) or cynomolgus monkeys”
✓ VerifiedEMA assessment report, p. 45Source ↗
NOAEL / key findings
No toxicity from UM171 at HED 8,000-20,000x human exposure (8 ng/kg); no treatment-related histopathology in NSG mice
“Intravenous administration at doses 8,000-20,000 times higher than the estimated human exposure to UM171 demonstrated no toxicity in rats (single-dose study) or cynomolgus monkeys”
✓ VerifiedEMA assessment report, p. 45Source ↗

Same question, other cell therapies

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

spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroids
Minipig 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 cells
Mouse target dose ~two log above proposed clinical dose; this dose caused 100% GvHD mortality (no NOAEL)

Abecma

idecabtagene vicleucel · BCMA CAR-T
Not stated in public reviews

Alofisel

darvadstrocel · Allogeneic adipose MSC
Not stated in public reviews

Amtagvi

lifileucel · TIL therapy
Not stated in public reviews

Aucatzyl

obecabtagene autoleucel · CD19 CAR-T
Not stated in public reviews

Breyanzi

lisocabtagene maraleucel · CD19 CAR-T
Not stated in public reviews

Carvykti

ciltacabtagene autoleucel · BCMA CAR-T
Not stated in public reviews

ChondroCelect

characterised viable autologous cartilage cells · Autologous chondrocytes
Not stated in public reviews

Ebvallo

tabelecleucel · EBV-specific allogeneic T cells
Not stated in public reviews

Gintuit

allogeneic cultured keratinocytes and fibroblasts in bovine collagen · Allogeneic keratinocytes + fibroblasts
Not stated in public reviews

Holoclar

autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cells
Not stated in public reviews

Kymriah

tisagenlecleucel · CD19 CAR-T
Not stated in public reviews

Lantidra

donislecel · Allogeneic pancreatic islets
Not stated in public reviews

Laviv

azficel-T · Autologous fibroblasts
Not stated in public reviews

MACI

autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytes
Not stated in public reviews

Omisirge

omidubicel · Expanded cord blood HPC
Not stated in public reviews

Provenge

sipuleucel-T · PAP-GM-CSF (autologous APC)
Not stated in public reviews

Rethymic

allogeneic processed thymus tissue · Allogeneic thymus tissue
Not stated in public reviews

Ryoncil

remestemcel-L · Allogeneic BM-MSC
Not stated in public reviews

StrataGraft

allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblasts
Not stated in public reviews

Tecartus

brexucabtagene autoleucel · CD19 CAR-T
Not stated in public reviews

Tecelra

afamitresgene autoleucel · MAGE-A4 TCR-T
Not stated in public reviews

Tregzi

allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + Tcon
Not stated in public reviews

Yescarta

axicabtagene ciloleucel · CD19 CAR-T
Not stated in public reviews

Summary across all 27 cell therapies →