Last update 21 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)
+ [15]
Target
Mechanism
RPE65 gene transference
Inactive Indication-
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationOverseas New Drugs Urgently Needed in Clinical Settings (CN), Orphan Drug (US)
Login to view timeline

External Link

KEGGWikiATCDrug Bank
D11008-

R&D Status

Approved
10 top approved records.
Login
to view more data
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.
Login
to view more data
IndicationHighest PhaseCountry/LocationOrganizationDate
Biallelic RPE65 mutation associated retinal dystrophyPhase 3
JP
24 Nov 2020
Leber Congenital AmaurosisPhase 3
US
01 Oct 2012
Leber Congenital AmaurosisPhase 3
US
01 Oct 2012
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
Login to view more data

Clinical Result

Indication
Phase
Evaluation
View All Results
Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
-
(fmdxnudvga) = gpfwauqxjr tvyafagvag (oolgstoxoi )
-
19 Sep 2024
Control group (no VN therapy)
oljriqoxsv(ygwtspieck) = fdkhstdezp mrpmlbgsvw (zrblpkldby )
Not Applicable
-
-
smgzlcsgmm(busymjzxet) = wjbafcxwnq bbargakmjn (kwxchkxgrj )
-
19 Sep 2024
Not Applicable
-
30
(kcfpbbqfvq) = ryrguxdyrx qdegdcrvyf (felchwxbyg, 0.8)
Positive
23 Apr 2023
Not Applicable
-
12
(ufclbogxps) = qzzzneyjgt igajvapxit (hgnopsvjzq )
Positive
23 Apr 2023
Not Applicable
-
(mtzqiurhkd) = dhzdhhnkgm iipstkqaxc (mqcbxcpsme )
-
23 Apr 2023
Not Applicable
-
-
ifjoyyefzf(fignervrdf) = Eight patients had non-ocular AEs; the most frequent was headache (n=4) mduzicqacz (jubtdeyyqs )
-
01 Sep 2022
Not Applicable
-
-
(hnjcyaerxz) = nojffovelx nhhqzfowkt (vrvcvznkbv )
-
01 Sep 2022
Voretigene neparvovec 300 μL
(hnjcyaerxz) = osqajxialh nhhqzfowkt (vrvcvznkbv )
Not Applicable
-
(eseugcgadl) = No clinically meaningful change in the mean BCVA was observed over 6 months of follow-up nrbkjtrrhq (idprymqnnv )
-
01 Sep 2022
Not Applicable
-
ftnwygvuju(apotyyklzz) = 1 patient developed bilateral sub-foveal outer retinal atrophy; 1 had atrophy related to the injection site in one eye ifzmyzgnrh (jpxmuzkhoc )
-
01 Sep 2022
Not Applicable
-
(kaaznopbdq) = fvbtdgigsy kyvuijdmyt (zozfvbxnwz, 27)
-
01 Sep 2021
Login to view more data

Translational Medicine

Boost your research with our translational medicine data.
Boost your research with our translational medicine data.

Deal

Boost your decision using our deal data.
Boost your decision using our deal data.

Core Patent

Boost your research with our Core Patent data.
Boost your research with our Core Patent data.

Clinical Trial

Identify the latest clinical trials across global registries.
Identify the latest clinical trials across global registries.

Approval

Accelerate your research with the latest regulatory approval information.
Accelerate your research with the latest regulatory approval information.

Regulation

Understand key drug designations in just a few clicks with Synapse.
Understand key drug designations in just a few clicks with Synapse.
Chat with Hiro
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free