Therapeutic Asset Ratings
3,848 results matching 1 filter
133,990 assets · 8,802 sponsors · 3,128 indications
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Phase
Rating
Axicabtagene Ciloleucel
Recurrent Transformed Follicular Lymphoma to Diffuse Large B-Cell Lymphoma
Ruijin Hospital
P4
BBB
(77)
Axicabtagene Ciloleucel
B-cell Lymphoma Recurrent
Lyell Immunopharma, Inc.
P4
BBB
(76)
Axicabtagene Ciloleucel
Diffuse Large B-Cell Lymphoma, Not Otherwise Specified
Ruijin Hospital
P4
BBB
(75)
Allogeneic Cd34+-Enriched And Cd45Ra-Depleted Pbscs
ALL
Fred Hutchinson Cancer Center
P2/3
BBB
(74)
Jwcar029
Relapsed or Refractory B-cell Lymphoma
Ruijin Hospital
P4
BBB
(72)
cd19-ucart
B-Cell Acute Lymphoblastic Leukemia, Adult
The General Hospital of Western Theater Command
P2
BBB
(72)
TIL
Melanoma (Skin)
City of Hope Medical Center
P2
BBB
(72)
cd19-ucart
Lymphoma, B-Cell
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
P4
BB
(71)
Axicabtagene Ciloleucel
Primary Mediastinal (Thymic) Large B-Cell Lymphoma
AIDS Malignancy Consortium
P2
BB
(71)
Axicabtagene Ciloleucel
Refractory High Grade B-Cell Lymphoma
AIDS Malignancy Consortium
P2
BB
(71)
Chimeric Antigen Receptor Modified T Cells Infusion
B Cell Lymphoma
The First Affiliated Hospital of Soochow University
P2/3
BB
(71)
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Hematologic Diseases
Vertex Pharmaceuticals Incorporated
P3
BB
(70)
Allogeneic Cd34+-Enriched And Cd45Ra-Depleted Pbscs
Acute Myelogenous Leukemia
Fred Hutchinson Cancer Center
P2/3
BB
(70)
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Genetic Diseases, Inborn
Vertex Pharmaceuticals Incorporated
P3
BB
(69)
Axicabtagene Ciloleucel
High Grade B-Cell Lymphoma
AIDS Malignancy Consortium
P2
BB
(69)
TIL
Metastatic Melanoma
East Metropolitan Health Service, Australia
P2
BB
(69)
Axicabtagene Ciloleucel
Large B-cell Lymphoma
Zhejiang Cancer Hospital
P4
BB
(68)
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Beta-Thalassemia
Daniel Bauer
P3
BB
(68)
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Hemoglobinopathies
Vertex Pharmaceuticals Incorporated
P3
BB
(68)
Cart-19 And 20 Cells
B Cell Lymphoma
Henan Cancer Hospital
P2
BB
(68)
cd22 car-transduced t cells
B-All
National Cancer Institute (NCI)
P2
BB
(68)
Sipuleucel-T
Metastatic Prostate Cancer
Yale University
P2
BB
(68)
Allogeneic Cd34+-Enriched And Cd45Ra-Depleted Pbscs
Myelodysplastic Syndromes
Fred Hutchinson Cancer Center
P2/3
BB
(68)
Axicabtagene Ciloleucel
Lymphoma, B-Cell
Lyell Immunopharma, Inc.
P4
BB
(67)
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Thalassemia
Vertex Pharmaceuticals Incorporated
P3
BB
(67)
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