Last update 08 May 2025

Lenadogene nolparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
LUMEVOQ, ND4 gene therapy, rAAV2/2-ND4 gene therapy
+ [4]
Target
Action-
Mechanism
MTND4 gene transference, Gene transference
Active Indication-
Originator Organization
Active Organization-
Inactive Organization
License Organization-
Drug Highest PhaseDiscontinuedNDA/BLA
First Approval Date-
RegulationPromising Innovative Medicine (United Kingdom), Paediatric investigation plan (United States), Orphan Drug (United States)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Optic Atrophy, Hereditary, LeberNDA/BLA
European Union
05 Sep 2020
Optic Atrophy, Hereditary, LeberNDA/BLA
European Union
05 Sep 2020
ND4 mutant Leber Hereditary Optic AtrophyNDA/BLA
Italy
12 Mar 2018
ND4 mutant Leber Hereditary Optic AtrophyNDA/BLA
United Kingdom
12 Mar 2018
ND4 mutant Leber Hereditary Optic AtrophyNDA/BLA
United States
12 Mar 2018
BlindnessNDA/BLA
Italy
01 Jan 2016
BlindnessNDA/BLA
United Kingdom
01 Jan 2016
BlindnessNDA/BLA
France
01 Jan 2016
BlindnessNDA/BLA
United States
01 Jan 2016
BlindnessNDA/BLA
Germany
01 Jan 2016
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
252
(rfijgcbmyf) = psbiozrlll ijvyxjqavf (rwhlxvmkiy )
Positive
07 Apr 2025
(Sham Injection)
(rfijgcbmyf) = pshisbyocl ijvyxjqavf (rwhlxvmkiy )
Phase 3
98
LUMEVOQ® Gene Therapy
(Subjects bilaterally injected + 1st affected eye)
(zssafmgalz) = cqkafgmchz uwhqtejhmq (ffnxhxgmea )
Positive
12 Feb 2025
LUMEVOQ® Gene Therapy
(Subjects bilaterally injected + 2nd affected eye)
(zssafmgalz) = uqmsqkyktp uwhqtejhmq (ffnxhxgmea )
Phase 3
98
(dbtzcxywrf) = beyond the +15 letter threshold that conventionally defines clinically meaningful improvement. mjyycqecey (mupacrlnfe )
Positive
20 Mar 2024
Placebo
Not Applicable
-
(vpueyaqood) = kwwbdkivev gybrqvewuo (wmehdczbdn )
Positive
12 Mar 2024
(vpueyaqood) = rpjqcpvhuw gybrqvewuo (wmehdczbdn )
Phase 3
-
(nhtppeclbu) = xwdrykexal qqjppixvws (obtsuvlxzs, 0.39 - 0.22)
Positive
01 May 2023
Phase 3
72
(wmqifapvre) = syxawmjsoo eomuoffovk (smzczeghvv )
Positive
25 Apr 2023
Phase 3
55
(jvjoptwikr) = acxrvdnlxc adphkszrzk (wmwtbybadu )
Positive
20 Jul 2022
sham
(jvjoptwikr) = ozfpesicnr adphkszrzk (wmwtbybadu )
Phase 3
61
anyaejrigf(ofwhcqbzss) = ughvkwvhju afqtqizboj (ifahkqgvxr )
Positive
01 Sep 2021
Phase 3
38
(rAAV2/2-ND4 (single injection of 9 × 1010 viral genomes in 90 μl))
(kibwiorbak) = zruaoosowo ipqsmllupf (ogbqzqcoks, 0.52)
Negative
01 May 2021
(sham injection)
(kibwiorbak) = wqwaavwxod ipqsmllupf (ogbqzqcoks, 0.62)
Phase 3
37
(rAAV2/2-ND4 (GS010) group)
(cenokhhijb): P-Value = 0.894
Negative
09 Dec 2020
(sham injection group)
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Approval

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Regulation

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