Therapeutic Asset Ratings

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91,583 assets · 7,906 sponsors · 3,128 indications

Methodology v0.2

Asset	Sponsor	Phase	Rating
AUTOLOGOUS PERIPHERAL BLOOD MONONUCLEAR CELL (PBMC)-DERIVED T LYMPHOCYTES TRANSDUCED EX VIVO WITH A NON-REPLICATING, SELF-INACTIVATING (SIN) LENTIVIRAL VECTOR ENCODINGA CHIMERIC ANTIGEN RECEPTOR (CAR) TARGETING TWO EPITOPES OF THE HUMAN B CELL MATURATION ANTIGEN (BCMA) UNDER THE CONTROL OF A HUMAN ELONGATION FACTOR 1 ALPHA (EF1.ALPHA.) PROMOTER THE CODING SEQUENCE COMPRISES A HUMAN CD8 ALPHA SIGNAL PEPTIDE (CD8.ALPHA. SP), A BCMA TARGETING DOMAIN CONSISTING OF TWO VARIABLE HEAVY CHAIN-ONLY ANTIBODIES (VHH1 AND VHH2) TARGETING TWO DISTINCT BCMA EPITOPES, A HUMAN CD8 ALPHA HINGE AND TRANSMEMBRANE DOMAIN (CD8.ALPHA. HINGE + TM), A HUMAN CD137 CYTOPLASMIC DOMAIN (4-1BB) AND A HUMAN CD3 ZETA CYTOPLASMIC DOMAIN (CD3.ZETA.). THE VECTOR GENOME CONTAINS A CHIMERIC ROUS SARCOMA VIRUS (RSV)/TRUNCATED 5'LTR AND A SELF-INACTIVATING 3'LTR-SIN (.DELTA.-U3) Extramedullary Plasmacytoma	Peter MacCallum Cancer Centre, Australia	P1	CC (43)
Autologous T cells obtained from peripheral blood mononuclear cells by leukapheresis, transduced with a self-inactivating, nonreplicating lentiviral vector, encoding a T cell chimeric antigen receptor targeting CD19. The expressed transgene comprises a CD8? leader sequence, a murine anti-CD19 single chain variable fragment (scFv) derived from the mouse hybridoma FMC63, a CD8? hinge and transmembrane region, and a 4-1BB (CD137) and CD3? (TCR?) signaling domain and is under control of the elongation factor 1 alpha (EF1?) promoter. The construct is flanked by 5' and 3' long terminal repeats (LTRs) and also contains a ? packaging signal, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and an optimized Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The leukapheresis material is enriched for CD4/CD8 T cells by positive immunoselection, activated by CD3 and CD28 agonists and transduced with the vector. Without further cell propagation, the T cells are washed, formulated for infusion, and cryopreserved. The substance is composed of ?80% T cells and ?1% B cells, with a mixture of transgene positive (>3.4%) and negative T cells. The CD4+ and CD8+ naïve T cell subsets (CD45RA+CCR7+) present in the leukapheresis material are largely retained Anaplastic Large Cell Lymphoma	Stanford University	P1	CC (43)
Autologous Tolerogenic Dendritic Cells Lynch Syndrome	Fundacion Clinic per a la Recerca Biomédica	P1	CC (43)
Axicabtagene Ciloleucel HIV Infectious Disease	AIDS Malignancy Consortium	P1	CC (43)
Axicabtagene Ciloleucel Primary Central Nervous System Lymphoma	Dana-Farber Cancer Institute	P1	CC (43)
azd0120 Idiopathic Inflammatory Myopathy	AstraZeneca	P1	CC (43)
azd0120 Rheumatoid Arthritis	AstraZeneca	P1	CC (43)
azd0120 Systemic Sclerosis	AstraZeneca	P1	CC (43)
b7-h3cart Nervous System Disorder	Stanford University	P1	CC (43)
b7-h3 car t cells Adrenal Cortex Carcinoma	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Clear Cell Sarcoma	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Desmoplastic Small Round Cell Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Malignant Germ Cell Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Hepatoblastoma	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Malignant Peripheral Nerve Sheath Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Rhabdoid Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3 car t cells Wilms Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3-car t cells Atypical Teratoid Rhabdoid Tumor	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3-car t cells Central Nervous System Neoplasm	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3-car t cells Ependymoma	St. Jude Children's Research Hospital	P1	CC (43)
b7-h3-car t cells Medulloblastoma	St. Jude Children's Research Hospital	P1	CC (43)
b7h3-il7ra car-t cells Childhood Malignant Neoplasm	Chulalongkorn University	P1	CC (43)
b7-h3 target, car gene modified gdt cell Acute Myeloid Leukemia	PersonGen BioTherapeutics (Suzhou) Co., Ltd.	P1	CC (43)
Baff Car-T B-Cell Non-Hodgkin Lymphoma	Luminary Therapeutics	P1	CC (43)
Baff Car-T B-Cell Chronic Lymphocytic Leukemia	Paolo Caimi, MD	P1	CC (43)

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