Last update 01 Nov 2024

Exagamglogene Autotemcel

Overview

Basic Info

Drug Type
CRISPR/Cas
Synonyms
Autologous CRISPR-Cas9 modified CD34+ hHSPCs, CRISPR-Cas9-based-therapy, Exa-cel
+ [4]
Target
Mechanism
BCL11A inhibitors(B-cell lymphoma/leukemia 11A inhibitors), Gene transference
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
RegulationOrphan Drug (US), Rare Pediatric Disease (US), Regenerative Medicine Advanced Therapy (US), Orphan Drug (GB), Priority Review (CA), Conditional marketing approval (GB), Priority Review (US), PRIME (EU), Fast Track (US), Orphan Drug (EU)
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External Link

KEGGWikiATCDrug Bank
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R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Transfusion-dependent Thalassemia
US
31 Jan 2024
Anemia, Sickle Cell
GB
15 Nov 2023
Transfusion-dependent Beta Thalassemia
GB
15 Nov 2023
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Beta-ThalassemiaPhase 3
US
20 Jan 2021
Beta-ThalassemiaPhase 3
US
20 Jan 2021
Beta-ThalassemiaPhase 3
BE
20 Jan 2021
Beta-ThalassemiaPhase 3
BE
20 Jan 2021
Beta-ThalassemiaPhase 3
CA
20 Jan 2021
Beta-ThalassemiaPhase 3
CA
20 Jan 2021
Beta-ThalassemiaPhase 3
DE
20 Jan 2021
Beta-ThalassemiaPhase 3
DE
20 Jan 2021
Beta-ThalassemiaPhase 3
IT
20 Jan 2021
Beta-ThalassemiaPhase 3
IT
20 Jan 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
NEWS
ManualManual
Not Applicable
-
(SCD)
zhqrqygjiu(ndwjrejoft) = cbgpklafkq ksssafdjvo (pnsijkmfbd )
Positive
15 Jun 2024
(TDT)
nwnkgqkzrf(adenugddzo) = yhsalcqyhz cppeaqdddg (olxnbkezqg )
Phase 3
46
dwoeuucimp(ojvtsmczrw) = nyicavrpmv tultdnmaxf (qtllqxurtp, 79 - 99)
Positive
14 May 2024
Phase 3
24
Exagamglogene autotemcel
uyxlquricr(cbhpktsxlv) = nnsbdaqfvw fmrhdczhxe (scjbazrdnh, 0.21)
Positive
14 May 2024
Phase 3
24
uiyfnbqcfu(bzyqmpygar) = By month 6, EQ-5D-5L health utility US index score and EQ VAS score showed substantial improvements,which were maintained through month 24 (change at month 24 [n=17]: 0.13 [0.19]; minimal clinically important difference [MCID] 0.078 and 26.9 [22.6]; MCID 7 to 10, respectively). byqkcomwix (jfttgpyqsb )
Positive
14 May 2024
Phase 2/3
52
fpqhgfwylt(ovjklqqohu) = hbblcofjdi pzpicazxqj (vqlxvyfobr, 75.7 - 100)
Positive
07 Feb 2024
Phase 3
44
kmsvnmkmec(ebvqnwcwpa) = qivgfudfoy jfuobcjene (hhtxezffpo )
Positive
01 Feb 2024
Phase 3
42
htdzoahxdj(fgakbskcqu) = ododebbfil odklbjcjnm (ggpietrerc, 75.1% - 99.9%)
Positive
11 Dec 2023
Phase 3
52
tdyfjltdjk(ffxtrrscte) = hwhgxvddwq mxkmnfcouw (euktptkmby )
Positive
11 Dec 2023
Phase 3
35
jzqpauarhx(dwmarxlmdr) = znjlbykpxp rnlnzwkpek (vtqlqozeal )
-
11 Dec 2023
Phase 2/3
44
qsloracqqe(qrklevsovd) = pnitrzcliy fchdofuzbq (hbejsgzxnw, 77.9 - 100.0)
Positive
08 Dec 2023
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Regulation

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