subject_id stringlengths 6 6 | indication_icd10 stringclasses 12
values | indication stringclasses 12
values | modality stringclasses 5
values | product_name stringclasses 12
values | age_at_dosing int64 0 84 | sex stringclasses 2
values | weight_kg float64 4 78.9 | race_ethnicity stringclasses 6
values | country stringclasses 9
values | site_id stringclasses 40
values | baseline_severity_score float64 0.1 109 | baseline_severity_scale stringclasses 11
values | prior_therapy_count int64 0 6 | nab_titer_baseline float64 0 404 | hla_type stringlengths 7 7 | conditioning_regimen stringclasses 4
values | dose_vg_per_kg float64 0.1 200,000B | infusion_date stringdate 2023-01-01 00:00:00 2024-06-23 00:00:00 | enrollment_status stringclasses 2
values |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
S00101 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 70 | M | 59.5 | White | JPN | SITE_014 | 38.82 | BM_plasma_pct | 3 | 0 | A*08:46 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-07-31T00:00:00Z | Dosed |
S00102 | C91.00 | B-ALL | CAR_T | CART-CD19 | 1 | F | 31.7 | White | DEU | SITE_006 | 53.52 | BM_blast_pct | 5 | 0 | A*11:85 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-05-01T00:00:00Z | Dosed |
S00103 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 23 | F | 41.6 | Black or African American | USA | SITE_001 | 10 | Hb_g_dL | 6 | 0 | A*17:61 | Busulfan_reduced | 5,000,000 | 2024-03-25T00:00:00Z | Dosed |
S00104 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 3 | M | 26.7 | White | AUS | SITE_024 | 2.97 | GMFC_MLD | 1 | 0 | A*17:40 | Busulfan_reduced | 5,000,000 | 2024-02-17T00:00:00Z | Dosed |
S00105 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 48 | M | 40.9 | Asian | ITA | SITE_003 | 3.98 | LDH_x_ULN | 6 | 0 | A*23:86 | Flu_Cy_lymphodepletion | 1,000,000 | 2024-04-25T00:00:00Z | Dosed |
S00106 | C91.00 | B-ALL | CAR_T | CART-CD19 | 18 | M | 26.8 | Black or African American | ITA | SITE_038 | 47.4 | BM_blast_pct | 6 | 0 | A*19:61 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-04-27T00:00:00Z | Dosed |
S00107 | D66 | Hemophilia A | AAV | AAV-FVIII | 18 | M | 22.9 | White | USA | SITE_003 | 3.73 | FVIII_pct | 2 | 2.66 | A*26:18 | null | 200,000,000,000,000 | 2024-03-14T00:00:00Z | Dosed |
S00108 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 20 | F | 30.1 | Black or African American | CAN | SITE_033 | 6.94 | Hb_g_dL | 6 | 0 | A*10:34 | Treosulfan | 3,000,000 | 2023-05-23T00:00:00Z | Dosed |
S00109 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 5 | M | 18.7 | Asian | ITA | SITE_031 | 19.46 | CHOP_INTEND | 2 | 1.26 | A*29:67 | null | 110,000,000,000,000 | 2023-11-05T00:00:00Z | Dosed |
S00110 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 35 | M | 49 | White | ITA | SITE_030 | 34.45 | BM_plasma_pct | 6 | 0 | A*05:39 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-02-08T00:00:00Z | Dosed |
S00111 | D66 | Hemophilia A | AAV | AAV-FVIII | 18 | M | 19.7 | White | DEU | SITE_020 | 4.45 | FVIII_pct | 1 | 2.66 | A*22:14 | null | 60,000,000,000,000 | 2023-03-30T00:00:00Z | Dosed |
S00112 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 4 | F | 30.4 | Black or African American | DEU | SITE_004 | 15.37 | NSAA | 2 | 43.4 | A*05:20 | null | 110,000,000,000,000 | 2023-04-11T00:00:00Z | Dosed |
S00113 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 34 | M | 40.4 | Hispanic or Latino | FRA | SITE_033 | 7.06 | Hb_g_dL | 5 | 0 | A*10:12 | Busulfan_full | 2,000,000 | 2023-03-14T00:00:00Z | Dosed |
S00114 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 58 | M | 78.9 | Hispanic or Latino | ITA | SITE_016 | 32.81 | mNIS_plus_7 | 0 | 0 | A*06:78 | null | 0.7 | 2023-08-23T00:00:00Z | Dosed |
S00115 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 6 | F | 28.3 | Hispanic or Latino | JPN | SITE_003 | 14.71 | NSAA | 0 | 33.41 | A*19:30 | null | 110,000,000,000,000 | 2023-12-01T00:00:00Z | Dosed |
S00116 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 63 | M | 61.4 | Other | USA | SITE_030 | 52.06 | BM_plasma_pct | 2 | 0 | A*30:19 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-06-15T00:00:00Z | Dosed |
S00117 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 68 | F | 58.9 | White | JPN | SITE_040 | 2.51 | LDH_x_ULN | 3 | 0 | A*23:50 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-11-11T00:00:00Z | Dosed |
S00118 | C91.00 | B-ALL | CAR_T | CART-CD19 | 10 | F | 17.9 | White | JPN | SITE_030 | 69.07 | BM_blast_pct | 3 | 0 | A*18:79 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-07-01T00:00:00Z | Dosed |
S00119 | D66 | Hemophilia A | AAV | AAV-FVIII | 18 | F | 28.6 | Not Reported | JPN | SITE_016 | 3.55 | FVIII_pct | 1 | 91.62 | A*03:97 | null | 200,000,000,000,000 | 2023-02-20T00:00:00Z | Dosed |
S00120 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 65 | F | 62.8 | Asian | ITA | SITE_016 | 108.4 | mNIS_plus_7 | 1 | 0 | A*31:77 | null | 0.3 | 2023-05-15T00:00:00Z | Dosed |
S00121 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 51 | F | 53.6 | Asian | JPN | SITE_038 | 47.61 | mNIS_plus_7 | 0 | 0 | A*32:70 | null | 0.3 | 2023-11-01T00:00:00Z | Dosed |
S00122 | C91.00 | B-ALL | CAR_T | CART-CD19 | 21 | M | 34.8 | Hispanic or Latino | JPN | SITE_002 | 50.77 | BM_blast_pct | 4 | 0 | A*33:85 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-07-14T00:00:00Z | Dosed |
S00123 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | F | 12.8 | White | JPN | SITE_022 | 29.48 | CHOP_INTEND | 1 | 205.3 | A*28:23 | null | 30,000,000,000,000 | 2023-01-15T00:00:00Z | Dosed |
S00124 | D66 | Hemophilia A | AAV | AAV-FVIII | 21 | F | 30.8 | Asian | CAN | SITE_022 | 2.9 | FVIII_pct | 0 | 10.63 | A*16:31 | null | 200,000,000,000,000 | 2023-01-10T00:00:00Z | Dosed |
S00125 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 2 | M | 21.5 | White | AUS | SITE_011 | 32.57 | CHOP_INTEND | 2 | 3.26 | A*29:33 | null | 60,000,000,000,000 | 2023-04-02T00:00:00Z | Dosed |
S00126 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 18 | M | 16.4 | White | USA | SITE_040 | 1.3 | FIX_pct | 0 | 1.98 | A*05:73 | null | 30,000,000,000,000 | 2023-02-15T00:00:00Z | Dosed |
S00127 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 22 | F | 30.2 | White | JPN | SITE_038 | 7.79 | Hb_g_dL | 5 | 0 | A*24:34 | Busulfan_full | 3,000,000 | 2023-02-06T00:00:00Z | Dosed |
S00128 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 2 | M | 13.3 | White | USA | SITE_032 | 18.62 | NSAA | 2 | 18.91 | A*22:84 | null | 60,000,000,000,000 | 2023-02-11T00:00:00Z | Dosed |
S00129 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 4 | M | 24.9 | Black or African American | ESP | SITE_037 | 17.38 | CHOP_INTEND | 2 | 34.43 | A*20:34 | null | 30,000,000,000,000 | 2023-06-03T00:00:00Z | Screen Failure - nAb+ |
S00130 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 82 | F | 73 | Black or African American | ITA | SITE_025 | 84.61 | mNIS_plus_7 | 2 | 0 | A*21:08 | null | 1 | 2023-08-30T00:00:00Z | Dosed |
S00131 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 3 | M | 23.8 | White | AUS | SITE_030 | 17.32 | NSAA | 2 | 3.93 | A*29:04 | null | 15,000,000,000,000 | 2024-06-02T00:00:00Z | Dosed |
S00132 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 82 | F | 74.8 | White | FRA | SITE_039 | 60.1 | BM_plasma_pct | 4 | 0 | A*34:73 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-02-08T00:00:00Z | Dosed |
S00133 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 64 | M | 51.9 | White | ITA | SITE_019 | 106.98 | mNIS_plus_7 | 1 | 0 | A*16:58 | null | 0.1 | 2023-08-19T00:00:00Z | Dosed |
S00134 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | M | 25.6 | Asian | USA | SITE_022 | 6.13 | Hb_g_dL | 5 | 0 | A*08:56 | Treosulfan | 2,000,000 | 2023-02-27T00:00:00Z | Dosed |
S00135 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 60 | F | 60.5 | Black or African American | CAN | SITE_013 | 90.77 | mNIS_plus_7 | 1 | 0 | A*06:23 | null | 0.1 | 2024-05-04T00:00:00Z | Dosed |
S00136 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | F | 24.8 | Not Reported | JPN | SITE_009 | 17.17 | CHOP_INTEND | 1 | 9.38 | A*09:63 | null | 30,000,000,000,000 | 2024-02-10T00:00:00Z | Screen Failure - nAb+ |
S00137 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 5 | M | 16.4 | Hispanic or Latino | CAN | SITE_017 | 1.22 | GMFC_MLD | 2 | 0 | A*13:52 | Treosulfan | 8,000,000 | 2023-05-02T00:00:00Z | Dosed |
S00138 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 45 | F | 39.5 | Hispanic or Latino | GBR | SITE_024 | 2.08 | FIX_pct | 2 | 1.55 | A*22:54 | null | 110,000,000,000,000 | 2023-10-30T00:00:00Z | Dosed |
S00139 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 63 | F | 57 | Asian | JPN | SITE_014 | 1.99 | LDH_x_ULN | 4 | 0 | A*05:43 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-10-15T00:00:00Z | Dosed |
S00140 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 1 | M | 16 | Black or African American | AUS | SITE_008 | 17.91 | CHOP_INTEND | 0 | 3.01 | A*09:65 | null | 60,000,000,000,000 | 2023-05-29T00:00:00Z | Screen Failure - nAb+ |
S00141 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 43 | M | 39.6 | White | USA | SITE_034 | 1.62 | FIX_pct | 1 | 18.5 | A*21:67 | null | 30,000,000,000,000 | 2023-07-26T00:00:00Z | Dosed |
S00142 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 47 | F | 49.2 | White | USA | SITE_024 | 3.2 | FIX_pct | 2 | 10.28 | A*31:20 | null | 60,000,000,000,000 | 2023-12-23T00:00:00Z | Dosed |
S00143 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 54 | F | 44.3 | White | ITA | SITE_013 | 53.86 | BM_plasma_pct | 5 | 0 | A*13:73 | Flu_Cy_lymphodepletion | 1,000,000 | 2024-02-01T00:00:00Z | Dosed |
S00144 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 75 | F | 68.3 | White | USA | SITE_022 | 78.83 | mNIS_plus_7 | 1 | 0 | A*22:77 | null | 0.7 | 2024-03-13T00:00:00Z | Dosed |
S00145 | D66 | Hemophilia A | AAV | AAV-FVIII | 41 | M | 37.4 | Black or African American | ESP | SITE_010 | 1.17 | FVIII_pct | 2 | 0.84 | A*08:16 | null | 30,000,000,000,000 | 2023-02-14T00:00:00Z | Screen Failure - nAb+ |
S00146 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | F | 32 | Hispanic or Latino | USA | SITE_022 | 9.7 | Hb_g_dL | 2 | 0 | A*18:94 | Busulfan_reduced | 2,000,000 | 2024-04-16T00:00:00Z | Dosed |
S00147 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 17 | F | 20.7 | Black or African American | JPN | SITE_032 | 9.4 | Hb_g_dL | 2 | 0 | A*03:61 | Treosulfan | 3,000,000 | 2023-09-17T00:00:00Z | Dosed |
S00148 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 47 | M | 36.2 | White | DEU | SITE_029 | 4.4 | FIX_pct | 2 | 35.89 | A*25:50 | null | 30,000,000,000,000 | 2023-06-18T00:00:00Z | Screen Failure - nAb+ |
S00149 | D66 | Hemophilia A | AAV | AAV-FVIII | 28 | F | 41.5 | White | DEU | SITE_009 | 2.86 | FVIII_pct | 1 | 45.19 | A*33:10 | null | 200,000,000,000,000 | 2024-05-26T00:00:00Z | Dosed |
S00150 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | F | 11.7 | Black or African American | AUS | SITE_020 | 16.45 | CHOP_INTEND | 2 | 1.32 | A*22:97 | null | 30,000,000,000,000 | 2023-03-23T00:00:00Z | Dosed |
S00151 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 40 | F | 35.7 | White | DEU | SITE_020 | 3.26 | LDH_x_ULN | 2 | 0 | A*10:51 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-01-14T00:00:00Z | Dosed |
S00152 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 64 | F | 67.1 | Other | AUS | SITE_011 | 35.79 | BM_plasma_pct | 5 | 0 | A*13:87 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-09-22T00:00:00Z | Dosed |
S00153 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 3 | M | 11.2 | White | FRA | SITE_012 | 15.96 | NSAA | 0 | 22.54 | A*15:54 | null | 30,000,000,000,000 | 2023-05-26T00:00:00Z | Screen Failure - nAb+ |
S00154 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 31 | F | 26 | Black or African American | GBR | SITE_031 | 10.48 | BCVA_letters | 0 | 67.03 | A*12:45 | null | 200,000,000,000,000 | 2023-08-01T00:00:00Z | Screen Failure - nAb+ |
S00155 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 2 | M | 14.4 | Black or African American | USA | SITE_001 | 22.88 | CHOP_INTEND | 2 | 2.39 | A*12:73 | null | 110,000,000,000,000 | 2023-02-22T00:00:00Z | Screen Failure - nAb+ |
S00156 | C91.00 | B-ALL | CAR_T | CART-CD19 | 16 | M | 33.9 | Asian | JPN | SITE_037 | 76.19 | BM_blast_pct | 3 | 0 | A*18:62 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-05-16T00:00:00Z | Dosed |
S00157 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 41 | M | 38.4 | White | FRA | SITE_006 | 7.35 | Hb_g_dL | 2 | 0 | A*34:19 | Busulfan_full | 2,000,000 | 2023-09-02T00:00:00Z | Dosed |
S00158 | C91.00 | B-ALL | CAR_T | CART-CD19 | 21 | M | 19.6 | Not Reported | GBR | SITE_019 | 33.51 | BM_blast_pct | 3 | 0 | A*02:72 | Flu_Cy_lymphodepletion | 10,000,000 | 2024-06-16T00:00:00Z | Dosed |
S00159 | C91.00 | B-ALL | CAR_T | CART-CD19 | 11 | M | 27.4 | Other | USA | SITE_030 | 88.12 | BM_blast_pct | 4 | 0 | A*34:26 | Flu_Cy_lymphodepletion | 10,000,000 | 2023-09-30T00:00:00Z | Dosed |
S00160 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 45 | M | 51 | Hispanic or Latino | AUS | SITE_016 | 2.59 | LDH_x_ULN | 6 | 0 | A*34:23 | Flu_Cy_lymphodepletion | 1,000,000 | 2024-01-17T00:00:00Z | Dosed |
S00161 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | M | 23 | Black or African American | GBR | SITE_019 | 26.87 | CHOP_INTEND | 0 | 11.16 | A*18:35 | null | 30,000,000,000,000 | 2023-07-08T00:00:00Z | Dosed |
S00162 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 84 | M | 62.8 | Hispanic or Latino | USA | SITE_023 | 40.69 | BM_plasma_pct | 2 | 0 | A*31:43 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-02-05T00:00:00Z | Dosed |
S00163 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | M | 46.3 | Asian | USA | SITE_014 | 9.23 | Hb_g_dL | 2 | 0 | A*31:92 | Busulfan_full | 3,000,000 | 2023-03-23T00:00:00Z | Dosed |
S00164 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 1 | F | 29.5 | White | ESP | SITE_005 | 23.33 | CHOP_INTEND | 1 | 5.8 | A*16:61 | null | 200,000,000,000,000 | 2023-01-08T00:00:00Z | Dosed |
S00165 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 39 | M | 39.6 | White | JPN | SITE_010 | 19.59 | BCVA_letters | 2 | 3.31 | A*02:27 | null | 110,000,000,000,000 | 2024-05-07T00:00:00Z | Screen Failure - nAb+ |
S00166 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 22 | M | 23.1 | White | DEU | SITE_025 | 4.81 | Hb_g_dL | 2 | 0 | A*10:75 | Busulfan_reduced | 5,000,000 | 2023-07-09T00:00:00Z | Dosed |
S00167 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 60 | M | 52 | Asian | CAN | SITE_003 | 3.53 | LDH_x_ULN | 5 | 0 | A*17:88 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-08-23T00:00:00Z | Dosed |
S00168 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 63 | F | 66.1 | Hispanic or Latino | FRA | SITE_004 | 3.98 | LDH_x_ULN | 4 | 0 | A*27:04 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-05-30T00:00:00Z | Dosed |
S00169 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 28 | F | 30.5 | Black or African American | ESP | SITE_021 | 6.39 | Hb_g_dL | 6 | 0 | A*26:28 | Treosulfan | 3,000,000 | 2024-04-04T00:00:00Z | Dosed |
S00170 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 5 | M | 22.4 | White | USA | SITE_002 | 1.62 | GMFC_MLD | 2 | 0 | A*30:88 | Treosulfan | 5,000,000 | 2024-02-13T00:00:00Z | Dosed |
S00171 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 22 | M | 30.9 | White | GBR | SITE_002 | 7.41 | Hb_g_dL | 2 | 0 | A*21:56 | Busulfan_reduced | 3,000,000 | 2023-09-18T00:00:00Z | Dosed |
S00172 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 36 | F | 37.2 | Other | AUS | SITE_024 | 1.57 | FIX_pct | 2 | 10.47 | A*19:17 | null | 200,000,000,000,000 | 2024-03-17T00:00:00Z | Dosed |
S00173 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 33 | M | 35.4 | White | GBR | SITE_023 | 8.88 | Hb_g_dL | 3 | 0 | A*03:34 | Busulfan_reduced | 2,000,000 | 2024-03-16T00:00:00Z | Dosed |
S00174 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 57 | M | 54.4 | Black or African American | DEU | SITE_005 | 76.85 | BM_plasma_pct | 3 | 0 | A*31:26 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-05-21T00:00:00Z | Dosed |
S00175 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 2 | F | 10.4 | White | GBR | SITE_029 | 28 | NSAA | 0 | 16.62 | A*11:86 | null | 200,000,000,000,000 | 2023-09-18T00:00:00Z | Screen Failure - nAb+ |
S00176 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 5 | M | 41 | Hispanic or Latino | ESP | SITE_038 | 17.87 | CHOP_INTEND | 0 | 1.63 | A*13:73 | null | 30,000,000,000,000 | 2023-06-16T00:00:00Z | Dosed |
S00177 | D66 | Hemophilia A | AAV | AAV-FVIII | 39 | M | 46.1 | White | USA | SITE_024 | 3.61 | FVIII_pct | 0 | 143.97 | A*21:65 | null | 30,000,000,000,000 | 2023-02-20T00:00:00Z | Dosed |
S00178 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 1 | F | 15.8 | White | AUS | SITE_013 | 2.21 | GMFC_MLD | 2 | 0 | A*19:46 | Busulfan_full | 8,000,000 | 2023-02-02T00:00:00Z | Dosed |
S00179 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 50 | M | 51.4 | White | ESP | SITE_011 | 61.35 | BM_plasma_pct | 3 | 0 | A*14:24 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-01-10T00:00:00Z | Dosed |
S00180 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 59 | M | 61.1 | Other | FRA | SITE_012 | 3.73 | LDH_x_ULN | 4 | 0 | A*32:78 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-02-01T00:00:00Z | Dosed |
S00181 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 56 | F | 62 | White | USA | SITE_004 | 58.1 | BM_plasma_pct | 2 | 0 | A*05:70 | Flu_Cy_lymphodepletion | 10,000,000 | 2023-01-17T00:00:00Z | Dosed |
S00182 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 54 | F | 53.5 | Black or African American | FRA | SITE_018 | 72.12 | BM_plasma_pct | 5 | 0 | A*18:86 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-01-20T00:00:00Z | Dosed |
S00183 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 36 | M | 57.7 | Black or African American | FRA | SITE_017 | 1.75 | FIX_pct | 2 | 67.76 | A*12:31 | null | 200,000,000,000,000 | 2024-01-09T00:00:00Z | Dosed |
S00184 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 21 | F | 22.5 | White | USA | SITE_008 | 8.83 | Hb_g_dL | 6 | 0 | A*20:60 | Busulfan_full | 2,000,000 | 2023-11-24T00:00:00Z | Dosed |
S00185 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 54 | F | 46.8 | White | GBR | SITE_039 | 3.85 | LDH_x_ULN | 5 | 0 | A*11:75 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-09-12T00:00:00Z | Dosed |
S00186 | C91.00 | B-ALL | CAR_T | CART-CD19 | 1 | M | 6.2 | White | ITA | SITE_018 | 63.88 | BM_blast_pct | 5 | 0 | A*31:61 | Flu_Cy_lymphodepletion | 10,000,000 | 2024-03-22T00:00:00Z | Dosed |
S00187 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 23 | M | 34.9 | White | USA | SITE_011 | 7.06 | Hb_g_dL | 2 | 0 | A*29:49 | Busulfan_reduced | 5,000,000 | 2024-03-31T00:00:00Z | Dosed |
S00188 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 1 | M | 23 | White | USA | SITE_016 | 15.22 | CHOP_INTEND | 2 | 103.61 | A*05:57 | null | 30,000,000,000,000 | 2023-09-26T00:00:00Z | Dosed |
S00189 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 76 | M | 77 | White | JPN | SITE_018 | 3.07 | LDH_x_ULN | 3 | 0 | A*30:82 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-10-28T00:00:00Z | Dosed |
S00190 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | M | 32.5 | Black or African American | ITA | SITE_038 | 8.89 | Hb_g_dL | 3 | 0 | A*01:07 | Treosulfan | 5,000,000 | 2024-01-18T00:00:00Z | Dosed |
S00191 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 55 | M | 49.6 | Hispanic or Latino | AUS | SITE_030 | 57.41 | BM_plasma_pct | 3 | 0 | A*27:42 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-03-05T00:00:00Z | Dosed |
S00192 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 39 | M | 52.6 | White | ITA | SITE_034 | 6.21 | Hb_g_dL | 3 | 0 | A*17:10 | Treosulfan | 2,000,000 | 2023-12-26T00:00:00Z | Dosed |
S00193 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 4 | M | 21 | White | GBR | SITE_021 | 20.47 | NSAA | 1 | 36.47 | A*12:76 | null | 200,000,000,000,000 | 2024-04-12T00:00:00Z | Dosed |
S00194 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 68 | F | 74.4 | White | DEU | SITE_019 | 3.54 | LDH_x_ULN | 5 | 0 | A*04:24 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-03-23T00:00:00Z | Dosed |
S00195 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 3 | M | 17.8 | Not Reported | USA | SITE_002 | 24.47 | NSAA | 0 | 58.63 | A*03:34 | null | 15,000,000,000,000 | 2024-03-31T00:00:00Z | Screen Failure - nAb+ |
S00196 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 3 | F | 9.3 | White | GBR | SITE_016 | 15.73 | NSAA | 0 | 21.1 | A*21:59 | null | 200,000,000,000,000 | 2024-03-03T00:00:00Z | Screen Failure - nAb+ |
S00197 | E85.4 | ATTR Amyloidosis | LNP_mRNA | LNP-TTR-edit | 63 | F | 49.6 | White | FRA | SITE_023 | 53.79 | mNIS_plus_7 | 2 | 0 | A*12:53 | null | 1 | 2023-10-19T00:00:00Z | Dosed |
S00198 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 50 | M | 53.8 | White | ESP | SITE_014 | 41.84 | BM_plasma_pct | 2 | 0 | A*34:52 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-02-06T00:00:00Z | Dosed |
S00199 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 5 | M | 16.6 | Asian | AUS | SITE_004 | 26.6 | NSAA | 2 | 14.97 | A*25:06 | null | 15,000,000,000,000 | 2024-04-01T00:00:00Z | Dosed |
S00200 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 21 | F | 36 | Black or African American | ITA | SITE_019 | 8.79 | Hb_g_dL | 3 | 0 | A*07:94 | Busulfan_full | 3,000,000 | 2023-04-13T00:00:00Z | Dosed |
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