截至2023年底,FDA批准的细胞和基因疗法清单

1,052

ABECMA (idecabtagene vicleucel) for refractory myeloma, Celgene Corporation.
ADSTILADRIN for unresponsive bladder cancer, Ferring Pharmaceuticals
ALLOCORD (Cord Blood), SSM Cardinal Glennon Children’s. “For use in unrelated donor hematopoietic progenitor cell transplantation.”
BREYANZI CAR-T-cell therapy for adults with relapsed or refractory large B-cell lymphoma, Juno Therapeutics.
CARVYKTI for refractory myeloma, Janssen
CASGEVY, Vertex.
CLEVECORD, Cleveland Cord Blood Center. Similar to ALLOCORD.
Ducord, Cord Blood, Duke Med School, Similar to ALLOCORD.
ELEVIDYS for DMD, Sarepta Therapeutics
GINTUIT for receding gums, Organogenesis.
HEMACORD (cord blood), New York Blood Center. This and the four that follow are similar to ALLOCORD.
Cord Blood, Clinimmune Labs.
Cord Blood – MD Anderson.
Cord Blood – LifeSouth.
Cord Blood – Bloodworks
IMLYGIC An oncolytic gene therapy product for treatment of melanoma, BioVex.
KYMRIAH CAR-T cell therapy for B-cell acute lymphoblastic leukemia (B-Cell ALL), Novartis.
LANTIDRA for Type 1 diabetes, CellTrans Inc.
LAVIV Treatment of deep wrinkles on the sides of our noses, Fibrocell Technologies.
LUXTURNA for Leber congenital amaurosis, Spark Therapeutics.
LYFGENIA, bluebird bio.
MACI for damaged cartilage, Vericel.
OMISIRGE for blood cancers, Gamida Cell.
PROVENGE (sipuleucel-T) for prostate cancer, Dendreon.
RETHYMIC for athymia, Enzyvant Therapeutics.
ROCTAVIAN for severe hemophilia A, BioMarin Pharmaceutical.
SKYSONA for CALD, bluebird bio.
STRATAGRAFT for burns, Stratatech.
TECARTUS, CAR-T therapy for treatment of adults with relapsed/refractory mantle cell lymphoma, Kite Pharma.
YESCARTA CAR-T for large B-cell lymphoma that hasn’t responded to other therapy, Kite Pharma.
ZOLGENSMA (Nusinersen), AveXis.
ZYNTEGLO for ß-thalassemia, bluebird bio.

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