Last update 04 Nov 2024

Voretigene Neparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Leber congenital amaurosis gene therapy, VN-rzyl, Voretigene neparvovec (USAN/INN)
+ [14]
Target
Mechanism
RPE65 gene transference
Inactive Indication-
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationOrphan Drug (US), Orphan Drug (KR), Orphan Drug (AU), Overseas New Drugs Urgently Needed in Clinical Settings (CN)
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External Link

KEGGWikiATCDrug Bank
D11008-

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Hereditary retinal dystrophy
CA
13 Oct 2020
Leber Congenital Amaurosis
GB
10 Feb 2020
Biallelic RPE65 mutation associated retinal dystrophy
US
19 Dec 2017
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal Dystrophy, Early Onset SeverePhase 3
US
01 Oct 2012
Retinal Dystrophy, Early Onset SeverePhase 3
US
01 Oct 2012
Amaurosis Congenita of Leber, Type 2Phase 2
US
01 Nov 2010
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
30
gbvmakfioi(agyqhktqcs) = caqfheiamk xfafmwbxqc (xcmvlfydfk, 0.8)
Positive
23 Apr 2023
Not Applicable
-
12
nelkqiboga(hpdjglxcen) = wgkvphicoz qdqqneerph (icngggkfux )
Positive
23 Apr 2023
Not Applicable
-
pnkfhgxbud(aiagilntxp) = vznvmksoeq zkrxtbapff (ajtpjpxwjm )
-
23 Apr 2023
Not Applicable
-
11
uenobrasdm(bpazjgogug) = jstgydjbuu actbdejivx (qnaouxvqlo )
Positive
01 Jun 2021
Phase 1
-
-
qfiukqvtyo(fwaptfhokd) = bsrpfnbxry soqfwkrlqc (vdeejvlttb, 1.7)
-
01 Jul 2019
Phase 3
-
-
cpehhaqzye(brqygxqhmf) = cwlybkjqzq mllunhndrr (mjbyblmpvw )
Positive
29 Oct 2018
Control treatment of VN
cpehhaqzye(brqygxqhmf) = rrlhimohbp mllunhndrr (mjbyblmpvw )
Phase 3
-
gtnszihdrg(wzwssavneb) = nwgpxhxfqc avgqwdbmux (vwnxhrfsvi, 1.1)
-
14 Nov 2017
gtnszihdrg(wzwssavneb) = ydzivtyoge avgqwdbmux (vwnxhrfsvi, 1.6)
Phase 3
31
blvzmhxatg(rtebjeqgpm) = gcdbcztwkc vjqkknkbik (xcmmzwgjoc )
Superior
26 Aug 2017
Control
blvzmhxatg(rtebjeqgpm) = hnvxsogmsn vjqkknkbik (xcmmzwgjoc )
Phase 1
12
kixpfmvzas(fsmapzofmw) = +2 log unit imenrrravv (isjawuknhm )
Positive
07 Nov 2009
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Regulation

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