IMVUCABTAGENE GELEUCEL

UNII:	PXY6S56878
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	IMVUCABTAGENE GELEUCEL
GSRS Full Record:	PXY6S56878 i

Imvucabtagene Geleucel
allogeneic T lymphocytes obtained from cryopreserved leukapheresis material of healthy donors, electroporated with CRISPR/Cas12a (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 12a) hybrid RNA-DNA (chRDNA) nucleoprotein complexes to knock out (i) the T cell receptor alpha chain constant (TRAC) and (ii) the beta-2 microglobulin (B2M) locus. The cells are also transduced with two recombinant adeno-associated virus serotype 6 (rAAV6) vectors to introduce (i) an anti-B-cell maturation antigen (BCMA, TNFRSF17, CD269) chimeric antigen receptor (CAR) flanked by TRAC homology regions that are inserted into the lymphocyte DNA by homology-directed repair, and (ii) a B2M-HLA-E peptide fusion protein flanked by beta-2 microglobulin (B2M) homology regions that are also inserted into the lymphocyte DNA by homology-directed repair. The anti-BCMA CAR expression cassette comprises in reverse orientation the synthetic MND promoter, Kozak sequence, CD8α signal sequence, anti-BCMA single chain variable fragment (scFv, clone PMC306), CD8 hinge region, CD8 transmembrane (TM) domain, 4-1BB co-stimulatory domain and CD3ζ signaling domain, followed by a bovine growth hormone (BGH) polyadenylation sequence. The B2M-HLA-E fusion protein expression cassette comprises a P2A skipping sequence, a B2M secretion signal, an HLA-G signal peptide sequence, the B2M peptide sequence followed by an HLA-E peptide sequence and a bovine growth hormone (BGH) polyadenylation sequence. The leukapheresis material is stimulated with anti-CD3 and anti-CD28 antibodies and cultured in the presence of interleukin 2 (IL-2) to enrich for T lymphocytes. The cells are subsequently electroporated with CRISPR/Cas12a chRDNA nucleoprotein complexes and transduced with the two AAV vectors followed by further cell expansion. Finally, residual TCRαβ+ lymphocytes are removed by magnetic depletion. The suspension consists primarily of T lymphocytes (CD4+/CD8+/CD3+ ≥70%) which are ≥32% CAR+, ≥20% B2M-HLA-E+ and are cytotoxic to BCMA expressing target cell
imvucabtagene geleucel [INN]

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UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

IMVUCABTAGENE GELEUCEL

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