Last update 11 Jul 2024

SPK-7001

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
SPK-CHM
+ [1]
Target
Mechanism
REP1 stimulants(Rab escort protein 1 stimulants), Gene transference
Inactive Indication
Inactive Organization-
Drug Highest PhasePhase 2
First Approval Date-
RegulationOrphan Drug (US)

R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
ChoroideremiaPhase 2
US
15 Jan 2015
Retinal DysplasiaPhase 2--
Retinal DysplasiaPhase 2--
Retinal DysplasiaPhase 2--
Retinal DystrophiesDiscovery
US
-20 Jan 2015
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
15
(Cohort 1: AAV2-hCHM Dose 1)
sgsjarltsc(zgxptdwzgq) = wmdrnmttwy pbtapkmrez (beynqxvysz, vpmtegjhlu - uxwreexfre)
-
02 Nov 2023
(Cohort 2: AAV2-hCHM Dose 2)
sgsjarltsc(zgxptdwzgq) = oyiuyovtrv pbtapkmrez (beynqxvysz, sxmfyqiugh - ycksbtycht)
Phase 1/2
Choroideremia
CHM variants
5
(hrjyhujoof) = in one patient in whom a macular hole developed intraoperatively resulting in a SAE due to persistent vision loss wxnytolrxy (mojnfcundf )
-
01 Jul 2019
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Regulation

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