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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
S00001 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 71 | M | 51.2 | White | CAN | SITE_015 | 3.03 | LDH_x_ULN | 2 | 0 | A*26:70 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-02-22T00:00:00Z | Dosed |
S00002 | D66 | Hemophilia A | AAV | AAV-FVIII | 25 | F | 38.2 | White | AUS | SITE_010 | 3.49 | FVIII_pct | 0 | 7.73 | A*30:94 | null | 30,000,000,000,000 | 2023-05-23T00:00:00Z | Dosed |
S00003 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 67 | M | 58.6 | Black or African American | ESP | SITE_011 | 72.64 | BM_plasma_pct | 3 | 0 | A*18:35 | Flu_Cy_lymphodepletion | 10,000,000 | 2024-04-05T00:00:00Z | Dosed |
S00004 | C91.00 | B-ALL | CAR_T | CART-CD19 | 8 | F | 18.1 | White | ITA | SITE_036 | 37.19 | BM_blast_pct | 5 | 0 | A*03:67 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-08-31T00:00:00Z | Dosed |
S00005 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 5 | F | 19.9 | White | ESP | SITE_022 | 24.37 | CHOP_INTEND | 0 | 0.66 | A*03:39 | null | 30,000,000,000,000 | 2024-05-09T00:00:00Z | Screen Failure - nAb+ |
S00006 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 7 | M | 33.2 | White | GBR | SITE_009 | 0.11 | GMFC_MLD | 0 | 0 | A*16:95 | Busulfan_reduced | 8,000,000 | 2023-07-05T00:00:00Z | Dosed |
S00007 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 58 | F | 55.5 | White | CAN | SITE_012 | 3.69 | LDH_x_ULN | 3 | 0 | A*08:55 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-04-26T00:00:00Z | Dosed |
S00008 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 55 | F | 59.8 | Black or African American | AUS | SITE_002 | 1.72 | LDH_x_ULN | 3 | 0 | A*11:32 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-03-25T00:00:00Z | Dosed |
S00009 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 8 | F | 23.7 | Asian | ITA | SITE_018 | 20.67 | NSAA | 2 | 3.56 | A*34:26 | null | 60,000,000,000,000 | 2023-12-29T00:00:00Z | Dosed |
S00010 | D66 | Hemophilia A | AAV | AAV-FVIII | 29 | M | 41.6 | White | AUS | SITE_018 | 4.82 | FVIII_pct | 1 | 11.47 | A*32:43 | null | 110,000,000,000,000 | 2023-01-20T00:00:00Z | Dosed |
S00011 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 12 | M | 17.1 | White | ITA | SITE_003 | 5.82 | Hb_g_dL | 3 | 0 | A*18:78 | Busulfan_full | 2,000,000 | 2023-01-28T00:00:00Z | Dosed |
S00012 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 49 | M | 57.7 | Black or African American | DEU | SITE_009 | 45.1 | BM_plasma_pct | 4 | 0 | A*14:23 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-03-23T00:00:00Z | Dosed |
S00013 | C91.00 | B-ALL | CAR_T | CART-CD19 | 17 | F | 26.8 | White | USA | SITE_011 | 46.65 | BM_blast_pct | 4 | 0 | A*14:43 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-02-26T00:00:00Z | Dosed |
S00014 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 49 | M | 58.3 | White | CAN | SITE_020 | 3.05 | LDH_x_ULN | 3 | 0 | A*08:73 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-05-14T00:00:00Z | Dosed |
S00015 | D66 | Hemophilia A | AAV | AAV-FVIII | 21 | M | 33.3 | White | CAN | SITE_025 | 1.55 | FVIII_pct | 0 | 10.91 | A*04:01 | null | 60,000,000,000,000 | 2023-04-12T00:00:00Z | Screen Failure - nAb+ |
S00016 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 29 | M | 17.3 | White | USA | SITE_023 | 8.36 | Hb_g_dL | 5 | 0 | A*29:49 | Busulfan_full | 2,000,000 | 2024-04-05T00:00:00Z | Dosed |
S00017 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 62 | M | 55.4 | Hispanic or Latino | GBR | SITE_011 | 42.16 | BCVA_letters | 1 | 130.09 | A*16:90 | null | 60,000,000,000,000 | 2023-02-12T00:00:00Z | Screen Failure - nAb+ |
S00018 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 2 | M | 21.5 | Black or African American | GBR | SITE_012 | 20.35 | CHOP_INTEND | 0 | 20.82 | A*28:08 | null | 60,000,000,000,000 | 2023-01-17T00:00:00Z | Dosed |
S00019 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 40 | M | 38.6 | Black or African American | AUS | SITE_029 | 3.22 | LDH_x_ULN | 6 | 0 | A*11:13 | Flu_Cy_lymphodepletion | 10,000,000 | 2023-03-21T00:00:00Z | Dosed |
S00020 | C91.00 | B-ALL | CAR_T | CART-CD19 | 12 | M | 14.7 | White | ITA | SITE_032 | 89.47 | BM_blast_pct | 4 | 0 | A*10:01 | Flu_Cy_lymphodepletion | 10,000,000 | 2023-01-09T00:00:00Z | Dosed |
S00021 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 83 | F | 73.2 | Hispanic or Latino | USA | SITE_036 | 2.61 | LDH_x_ULN | 2 | 0 | A*22:18 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-01-25T00:00:00Z | Dosed |
S00022 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 12 | M | 26.5 | Hispanic or Latino | USA | SITE_008 | 6.38 | Hb_g_dL | 2 | 0 | A*06:23 | Treosulfan | 5,000,000 | 2024-03-29T00:00:00Z | Dosed |
S00023 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 2 | F | 19.2 | Black or African American | USA | SITE_022 | 3.4 | GMFC_MLD | 2 | 0 | A*10:75 | Treosulfan | 8,000,000 | 2023-03-22T00:00:00Z | Dosed |
S00024 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 64 | M | 55.2 | White | DEU | SITE_018 | 34.7 | BM_plasma_pct | 4 | 0 | A*27:88 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-08-02T00:00:00Z | Dosed |
S00025 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 64 | M | 50.7 | White | ESP | SITE_008 | 3.58 | LDH_x_ULN | 6 | 0 | A*32:24 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-07-05T00:00:00Z | Dosed |
S00026 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 0 | F | 15.2 | White | FRA | SITE_004 | 24.24 | NSAA | 2 | 12.86 | A*33:56 | null | 30,000,000,000,000 | 2023-02-26T00:00:00Z | Dosed |
S00027 | D66 | Hemophilia A | AAV | AAV-FVIII | 30 | F | 28.8 | White | USA | SITE_009 | 3.97 | FVIII_pct | 1 | 106.39 | A*01:97 | null | 200,000,000,000,000 | 2024-03-10T00:00:00Z | Dosed |
S00028 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | M | 20 | Black or African American | ESP | SITE_023 | 20.23 | CHOP_INTEND | 1 | 28.69 | A*20:92 | null | 30,000,000,000,000 | 2023-09-10T00:00:00Z | Screen Failure - nAb+ |
S00029 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 5 | M | 20.7 | White | GBR | SITE_007 | 23.58 | NSAA | 0 | 18.79 | A*12:72 | null | 200,000,000,000,000 | 2023-04-18T00:00:00Z | Dosed |
S00030 | C91.00 | B-ALL | CAR_T | CART-CD19 | 11 | F | 17.2 | White | USA | SITE_040 | 58.93 | BM_blast_pct | 2 | 0 | A*02:46 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-05-21T00:00:00Z | Dosed |
S00031 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 65 | M | 66.3 | Black or African American | ESP | SITE_020 | 1.33 | LDH_x_ULN | 3 | 0 | A*09:14 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-06-18T00:00:00Z | Dosed |
S00032 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 3 | F | 24.7 | Not Reported | JPN | SITE_015 | 0.43 | GMFC_MLD | 2 | 0 | A*14:14 | Treosulfan | 3,000,000 | 2023-05-01T00:00:00Z | Dosed |
S00033 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | F | 16.7 | White | ITA | SITE_021 | 7.01 | Hb_g_dL | 2 | 0 | A*16:16 | Busulfan_full | 5,000,000 | 2023-07-22T00:00:00Z | Dosed |
S00034 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 14 | M | 12.5 | White | ITA | SITE_005 | 7.09 | Hb_g_dL | 5 | 0 | A*02:21 | Busulfan_reduced | 5,000,000 | 2023-08-09T00:00:00Z | Dosed |
S00035 | D66 | Hemophilia A | AAV | AAV-FVIII | 32 | M | 38 | Hispanic or Latino | USA | SITE_025 | 2.65 | FVIII_pct | 0 | 16.08 | A*07:25 | null | 60,000,000,000,000 | 2024-01-19T00:00:00Z | Dosed |
S00036 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 6 | F | 11.5 | White | ESP | SITE_010 | 17.43 | NSAA | 0 | 2.2 | A*06:08 | null | 60,000,000,000,000 | 2023-01-02T00:00:00Z | Dosed |
S00037 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 3 | M | 15.7 | White | JPN | SITE_034 | 15.84 | NSAA | 1 | 7.45 | A*07:94 | null | 110,000,000,000,000 | 2023-09-18T00:00:00Z | Dosed |
S00038 | D66 | Hemophilia A | AAV | AAV-FVIII | 54 | M | 57.5 | White | USA | SITE_011 | 1.12 | FVIII_pct | 1 | 9.94 | A*05:01 | null | 200,000,000,000,000 | 2024-05-09T00:00:00Z | Dosed |
S00039 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 32 | M | 34.6 | White | ITA | SITE_034 | 9.27 | Hb_g_dL | 6 | 0 | A*28:73 | Busulfan_reduced | 3,000,000 | 2024-03-29T00:00:00Z | Dosed |
S00040 | C91.00 | B-ALL | CAR_T | CART-CD19 | 15 | M | 19.8 | White | USA | SITE_003 | 65.48 | BM_blast_pct | 5 | 0 | A*02:74 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-03-04T00:00:00Z | Dosed |
S00041 | D66 | Hemophilia A | AAV | AAV-FVIII | 54 | F | 44.8 | Black or African American | JPN | SITE_001 | 4.97 | FVIII_pct | 0 | 2.61 | A*10:39 | null | 30,000,000,000,000 | 2024-03-06T00:00:00Z | Dosed |
S00042 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 58 | F | 57.4 | Hispanic or Latino | USA | SITE_026 | 3.18 | LDH_x_ULN | 3 | 0 | A*25:46 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-12-23T00:00:00Z | Dosed |
S00043 | C91.00 | B-ALL | CAR_T | CART-CD19 | 1 | M | 19.4 | White | ITA | SITE_008 | 69.08 | BM_blast_pct | 6 | 0 | A*29:58 | Flu_Cy_lymphodepletion | 1,000,000 | 2024-06-17T00:00:00Z | Dosed |
S00044 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 24 | F | 36.6 | White | DEU | SITE_031 | 7.21 | Hb_g_dL | 5 | 0 | A*13:93 | Treosulfan | 3,000,000 | 2024-02-01T00:00:00Z | Dosed |
S00045 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 60 | F | 63.1 | White | JPN | SITE_030 | 2.26 | LDH_x_ULN | 6 | 0 | A*07:64 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-07-04T00:00:00Z | Dosed |
S00046 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 57 | M | 54.9 | White | GBR | SITE_027 | 2.97 | LDH_x_ULN | 5 | 0 | A*23:30 | Flu_Cy_lymphodepletion | 1,000,000 | 2024-04-24T00:00:00Z | Dosed |
S00047 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 34 | F | 25.6 | White | GBR | SITE_037 | 4.52 | Hb_g_dL | 5 | 0 | A*30:27 | Treosulfan | 2,000,000 | 2023-06-01T00:00:00Z | Dosed |
S00048 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 25 | F | 35.8 | White | GBR | SITE_022 | 9.81 | Hb_g_dL | 5 | 0 | A*20:60 | Treosulfan | 2,000,000 | 2023-06-16T00:00:00Z | Dosed |
S00049 | C91.00 | B-ALL | CAR_T | CART-CD19 | 20 | F | 30.6 | Black or African American | ITA | SITE_022 | 51 | BM_blast_pct | 2 | 0 | A*24:86 | Flu_Cy_lymphodepletion | 10,000,000 | 2023-11-26T00:00:00Z | Dosed |
S00050 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 2 | F | 21.8 | White | AUS | SITE_022 | 20.86 | NSAA | 1 | 2.54 | A*22:80 | null | 200,000,000,000,000 | 2023-08-06T00:00:00Z | Dosed |
S00051 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 5 | F | 22 | Asian | CAN | SITE_040 | 24.68 | NSAA | 2 | 226.6 | A*22:45 | null | 15,000,000,000,000 | 2023-05-10T00:00:00Z | Dosed |
S00052 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 4 | M | 18.7 | Black or African American | GBR | SITE_017 | 15.47 | CHOP_INTEND | 1 | 36.06 | A*28:56 | null | 110,000,000,000,000 | 2023-07-17T00:00:00Z | Dosed |
S00053 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 49 | F | 60.9 | White | USA | SITE_028 | 1.35 | LDH_x_ULN | 5 | 0 | A*04:39 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-06-23T00:00:00Z | Dosed |
S00054 | C91.00 | B-ALL | CAR_T | CART-CD19 | 4 | M | 28.4 | Black or African American | AUS | SITE_038 | 88.75 | BM_blast_pct | 5 | 0 | A*03:92 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-11-14T00:00:00Z | Dosed |
S00055 | C91.00 | B-ALL | CAR_T | CART-CD19 | 12 | F | 20.1 | White | JPN | SITE_029 | 36.46 | BM_blast_pct | 6 | 0 | A*06:42 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-10-26T00:00:00Z | Dosed |
S00056 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 62 | F | 59.8 | Not Reported | USA | SITE_040 | 2.55 | LDH_x_ULN | 2 | 0 | A*04:67 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-03-05T00:00:00Z | Dosed |
S00057 | D66 | Hemophilia A | AAV | AAV-FVIII | 49 | M | 54.2 | White | CAN | SITE_015 | 2.41 | FVIII_pct | 2 | 19.71 | A*24:82 | null | 60,000,000,000,000 | 2024-03-19T00:00:00Z | Dosed |
S00058 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 53 | M | 64 | Asian | USA | SITE_020 | 41.26 | BCVA_letters | 0 | 8.25 | A*05:90 | null | 110,000,000,000,000 | 2023-06-25T00:00:00Z | Screen Failure - nAb+ |
S00059 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 4 | M | 21.4 | White | USA | SITE_004 | 24.84 | NSAA | 1 | 5.71 | A*12:64 | null | 15,000,000,000,000 | 2024-03-20T00:00:00Z | Dosed |
S00060 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | M | 4 | Asian | ITA | SITE_022 | 30.48 | CHOP_INTEND | 0 | 5.87 | A*04:54 | null | 15,000,000,000,000 | 2023-11-14T00:00:00Z | Screen Failure - nAb+ |
S00061 | C91.00 | B-ALL | CAR_T | CART-CD19 | 12 | M | 23.4 | White | USA | SITE_013 | 39.25 | BM_blast_pct | 3 | 0 | A*22:21 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-02-10T00:00:00Z | Dosed |
S00062 | D66 | Hemophilia A | AAV | AAV-FVIII | 43 | M | 42 | White | USA | SITE_033 | 2.59 | FVIII_pct | 2 | 10.82 | A*34:97 | null | 15,000,000,000,000 | 2023-04-07T00:00:00Z | Dosed |
S00063 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 31 | F | 48.5 | Hispanic or Latino | DEU | SITE_019 | 17.73 | BCVA_letters | 0 | 4.44 | A*33:29 | null | 200,000,000,000,000 | 2024-02-01T00:00:00Z | Dosed |
S00064 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 50 | F | 69.7 | Black or African American | FRA | SITE_002 | 1.15 | LDH_x_ULN | 6 | 0 | A*21:37 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-12-01T00:00:00Z | Dosed |
S00065 | C91.00 | B-ALL | CAR_T | CART-CD19 | 4 | F | 5 | Black or African American | USA | SITE_007 | 42.6 | BM_blast_pct | 3 | 0 | A*05:52 | Flu_Cy_lymphodepletion | 1,000,000 | 2023-06-19T00:00:00Z | Dosed |
S00066 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 45 | F | 51.6 | White | USA | SITE_009 | 41.31 | BCVA_letters | 2 | 17.43 | A*22:83 | null | 60,000,000,000,000 | 2023-02-21T00:00:00Z | Dosed |
S00067 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 48 | F | 54.6 | White | FRA | SITE_019 | 16.84 | BCVA_letters | 1 | 2.02 | A*01:21 | null | 60,000,000,000,000 | 2023-02-23T00:00:00Z | Dosed |
S00068 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | M | 26.8 | White | ESP | SITE_035 | 7.28 | Hb_g_dL | 3 | 0 | A*13:24 | Busulfan_full | 3,000,000 | 2023-07-12T00:00:00Z | Dosed |
S00069 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 4 | F | 13.9 | Hispanic or Latino | USA | SITE_038 | 12.5 | CHOP_INTEND | 2 | 0.59 | A*04:46 | null | 15,000,000,000,000 | 2023-08-08T00:00:00Z | Dosed |
S00070 | D66 | Hemophilia A | AAV | AAV-FVIII | 50 | F | 60.3 | White | USA | SITE_006 | 1.18 | FVIII_pct | 2 | 15.39 | A*03:32 | null | 30,000,000,000,000 | 2024-05-27T00:00:00Z | Dosed |
S00071 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 5 | F | 14.1 | Not Reported | GBR | SITE_020 | 20.91 | NSAA | 0 | 8.34 | A*34:79 | null | 200,000,000,000,000 | 2024-03-05T00:00:00Z | Dosed |
S00072 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 22 | M | 35.3 | White | ITA | SITE_032 | 6.36 | Hb_g_dL | 6 | 0 | A*20:36 | Treosulfan | 3,000,000 | 2023-08-26T00:00:00Z | Dosed |
S00073 | C90.00 | Multiple Myeloma | CAR_T | CART-BCMA | 62 | F | 43.9 | White | DEU | SITE_031 | 52.55 | BM_plasma_pct | 3 | 0 | A*33:56 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-09-07T00:00:00Z | Dosed |
S00074 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 24 | F | 26.8 | White | USA | SITE_016 | 6.38 | Hb_g_dL | 3 | 0 | A*06:50 | Treosulfan | 5,000,000 | 2023-07-30T00:00:00Z | Dosed |
S00075 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 5 | F | 18.1 | White | FRA | SITE_034 | 33.19 | CHOP_INTEND | 0 | 16.96 | A*21:98 | null | 110,000,000,000,000 | 2023-12-21T00:00:00Z | Screen Failure - nAb+ |
S00076 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 18 | F | 22.6 | White | CAN | SITE_025 | 8.72 | Hb_g_dL | 2 | 0 | A*32:56 | Busulfan_full | 2,000,000 | 2023-07-01T00:00:00Z | Dosed |
S00077 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 12 | M | 29.8 | White | JPN | SITE_023 | 7.32 | Hb_g_dL | 5 | 0 | A*30:87 | Treosulfan | 3,000,000 | 2024-04-04T00:00:00Z | Dosed |
S00078 | C91.00 | B-ALL | CAR_T | CART-CD19 | 13 | M | 20.9 | White | ITA | SITE_009 | 87.75 | BM_blast_pct | 4 | 0 | A*03:95 | Flu_Cy_lymphodepletion | 10,000,000 | 2024-04-07T00:00:00Z | Dosed |
S00079 | H35.50 | Leber Congenital Amaurosis | AAV | AAV-RPE65 | 9 | F | 21.7 | White | USA | SITE_005 | 38.38 | BCVA_letters | 1 | 5.2 | A*17:63 | null | 60,000,000,000,000 | 2024-01-29T00:00:00Z | Dosed |
S00080 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 76 | M | 65.4 | White | ITA | SITE_036 | 3.51 | LDH_x_ULN | 5 | 0 | A*03:28 | Flu_Cy_lymphodepletion | 5,000,000 | 2023-06-14T00:00:00Z | Dosed |
S00081 | C91.00 | B-ALL | CAR_T | CART-CD19 | 26 | M | 36.2 | White | DEU | SITE_028 | 35.62 | BM_blast_pct | 5 | 0 | A*23:12 | Flu_Cy_lymphodepletion | 2,000,000 | 2024-03-02T00:00:00Z | Dosed |
S00082 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 24 | M | 34.1 | White | ITA | SITE_018 | 4.75 | Hb_g_dL | 2 | 0 | A*17:34 | Busulfan_full | 2,000,000 | 2023-10-04T00:00:00Z | Dosed |
S00083 | C83.30 | Large B-Cell Lymphoma | CAR_T | CART-CD19-41BB | 65 | F | 75.4 | White | CAN | SITE_024 | 3.61 | LDH_x_ULN | 5 | 0 | A*31:63 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-08-20T00:00:00Z | Dosed |
S00084 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 4 | M | 13.6 | Not Reported | FRA | SITE_039 | 14.57 | NSAA | 0 | 26.64 | A*24:52 | null | 60,000,000,000,000 | 2023-07-29T00:00:00Z | Dosed |
S00085 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 5 | F | 21.3 | Hispanic or Latino | USA | SITE_038 | 23.37 | CHOP_INTEND | 0 | 3.63 | A*30:56 | null | 15,000,000,000,000 | 2023-08-03T00:00:00Z | Dosed |
S00086 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 3 | M | 4 | White | JPN | SITE_008 | 17.15 | CHOP_INTEND | 0 | 47.47 | A*33:67 | null | 60,000,000,000,000 | 2023-03-10T00:00:00Z | Dosed |
S00087 | C91.00 | B-ALL | CAR_T | CART-CD19 | 1 | F | 18.9 | Other | ESP | SITE_026 | 76.73 | BM_blast_pct | 2 | 0 | A*11:54 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-10-21T00:00:00Z | Dosed |
S00088 | D66 | Hemophilia A | AAV | AAV-FVIII | 38 | F | 51.4 | White | FRA | SITE_019 | 2.55 | FVIII_pct | 0 | 8.43 | A*13:12 | null | 110,000,000,000,000 | 2024-01-26T00:00:00Z | Screen Failure - nAb+ |
S00089 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 4 | M | 12.4 | Black or African American | USA | SITE_004 | 16.21 | NSAA | 0 | 0.54 | A*10:96 | null | 60,000,000,000,000 | 2023-04-29T00:00:00Z | Dosed |
S00090 | D67 | Hemophilia B | AAV | AAV-FIX-Padua | 32 | F | 26.1 | White | ITA | SITE_002 | 1.61 | FIX_pct | 2 | 3.28 | A*03:15 | null | 30,000,000,000,000 | 2023-07-16T00:00:00Z | Screen Failure - nAb+ |
S00091 | G71.01 | Duchenne Muscular Dystrophy | AAV | AAV-microdys | 5 | F | 22.6 | Asian | CAN | SITE_035 | 26.1 | NSAA | 1 | 43.18 | A*05:35 | null | 110,000,000,000,000 | 2023-02-18T00:00:00Z | Dosed |
S00092 | C91.00 | B-ALL | CAR_T | CART-CD19 | 29 | M | 43.1 | White | GBR | SITE_031 | 60.13 | BM_blast_pct | 4 | 0 | A*07:61 | Flu_Cy_lymphodepletion | 2,000,000 | 2023-04-11T00:00:00Z | Dosed |
S00093 | D66 | Hemophilia A | AAV | AAV-FVIII | 24 | M | 39.2 | Black or African American | ESP | SITE_037 | 4.75 | FVIII_pct | 0 | 2.01 | A*28:11 | null | 30,000,000,000,000 | 2023-09-26T00:00:00Z | Screen Failure - nAb+ |
S00094 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 24 | M | 28.3 | Not Reported | USA | SITE_020 | 5.91 | Hb_g_dL | 6 | 0 | A*15:96 | Busulfan_full | 5,000,000 | 2023-11-17T00:00:00Z | Dosed |
S00095 | D57.1 | Sickle Cell Disease | CRISPR | CRISPR-BCL11A | 34 | F | 41.1 | Not Reported | USA | SITE_019 | 9.59 | Hb_g_dL | 3 | 0 | A*17:71 | Busulfan_full | 5,000,000 | 2024-03-19T00:00:00Z | Dosed |
S00096 | C91.00 | B-ALL | CAR_T | CART-CD19 | 11 | F | 34.4 | Black or African American | USA | SITE_035 | 66.02 | BM_blast_pct | 3 | 0 | A*12:43 | Flu_Cy_lymphodepletion | 5,000,000 | 2024-02-25T00:00:00Z | Dosed |
S00097 | D56.1 | Beta-Thalassemia (TDT) | CRISPR | CRISPR-HbF | 15 | M | 29.2 | Asian | ESP | SITE_015 | 6.95 | Hb_g_dL | 4 | 0 | A*17:46 | Busulfan_reduced | 2,000,000 | 2023-11-19T00:00:00Z | Dosed |
S00098 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 4 | M | 17.7 | Asian | JPN | SITE_009 | 16.81 | CHOP_INTEND | 2 | 138.99 | A*04:94 | null | 60,000,000,000,000 | 2023-03-25T00:00:00Z | Dosed |
S00099 | G12.0 | SMA Type 1 | AAV | AAV-SMN1-rep | 7 | M | 24.4 | Hispanic or Latino | JPN | SITE_031 | 27.7 | CHOP_INTEND | 2 | 25.26 | A*06:78 | null | 30,000,000,000,000 | 2024-03-09T00:00:00Z | Dosed |
S00100 | E75.25 | Metachromatic Leukodystrophy | LENTIVIRAL | LV-ARSA | 4 | M | 17.6 | Black or African American | USA | SITE_034 | 0.42 | GMFC_MLD | 2 | 0 | A*15:48 | Busulfan_full | 8,000,000 | 2023-11-11T00:00:00Z | Dosed |
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