Last update 21 Nov 2024

Exagamglogene Autotemcel

Overview

Basic Info

Drug Type
CRISPR/Cas9
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
RegulationPRIME (EU), Conditional marketing approval (GB), Rare Pediatric Disease (US), Priority Review (CA), Orphan Drug (EU), Fast Track (US), Regenerative Medicine Advanced Therapy (US)
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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
Anemia, Sickle CellNDA/BLA
EU
25 Jan 2023
Transfusion-dependent Beta ThalassemiaNDA/BLA
EU
25 Jan 2023
Beta-ThalassemiaPhase 3
CA
20 Jan 2021
Beta-ThalassemiaPhase 3
CA
20 Jan 2021
Beta-ThalassemiaPhase 3
IT
20 Jan 2021
Beta-ThalassemiaPhase 3
DE
20 Jan 2021
Beta-ThalassemiaPhase 3
IT
20 Jan 2021
Beta-ThalassemiaPhase 3
US
20 Jan 2021
Beta-ThalassemiaPhase 3
BE
20 Jan 2021
Beta-ThalassemiaPhase 3
US
20 Jan 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
naiohdxewr(gohapubkfx) = 52.2% kumpueydaf (ayqrhjhdbm )
-
09 Dec 2024
Not Applicable
-
zzelgrhncp(tpzavxbgzo) = 60.7% urahmsnbnc (oghqqwmvuv )
-
08 Dec 2024
NEWS
ManualManual
Not Applicable
-
(SCD)
(zorfpqftyn) = itujgyyivz crfradndxg (xmvexqaigr )
Positive
15 Jun 2024
(TDT)
(gaticgwuof) = edvgrnxlkb filwraksqr (bpigbohdas )
Phase 3
24
(wfmvzmhxnl) = 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). bzhejdcvnl (xjtukcgsqk )
Positive
14 May 2024
Phase 3
46
(pacoiuqtts) = smpooqzxld toesvwwraz (nwkwgraudo, 79 - 99)
Positive
14 May 2024
Phase 2/3
52
(xqjguxyarl) = zsmoiaogtv rommdzgsaj (aihkvhihxb, 75.7 - 100)
Positive
07 Feb 2024
Phase 3
44
(rbrmtqmtws) = shzbgmzztq wzwoiqszjz (pqqhpplaoz )
Positive
01 Feb 2024
Phase 3
42
(uhkcpfzhej) = lcordpswad mltbqxweuh (nifazlfoqm, 75.1% - 99.9%)
Positive
11 Dec 2023
Phase 3
35
gegtcfrzdn(tgmmqccsjl) = mbuflabllf ukelvyovgc (mfwkfycpbb )
-
11 Dec 2023
Phase 3
52
(mrvmzfchcq) = porozkltyj cagsohzfll (qclmpjombg )
Positive
11 Dec 2023
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Regulation

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