Clinical trial pipeline
Currently open for enrollment
Dry age-related macular degeneration
Title: GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants with Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Purpose: The purpose of the study is to evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).
Leber Congenital amaurosis (Gene CEP290)
Title: Single Ascending Dose Study in Participants with LCA10
Purpose: The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused bya c.2991+1655A>G mutations in the CEP290 gene (“LCA10-IVS26”).X-linked retinitis pigmentosa (Gene: RPGR)
X-linked retinitis pigmentosa (Gene: RPGR)
Title: Natural History Study of Patients With X-linked Retinal Dystrophy Associated with Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
Purpose: Analysis of retinal structure and function to assess disease progression in X-linked retinitis pigmentosa
Institutional ethics board approved
(Likely to be enrolling in the next 3 months)
X-linked retinitis pigmentosa (Gene: RPGR)
Title: Analysis of retinal structure and function to assess disease progression
Purpose: A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
Site approved
(Likely to be enrolling in the next 6 months)
Achromatopsia (Genes: CNGA3, CNGB3)
Title: Natural History Study for Achromatopsia
Purpose: In preparation for human clinical trials there will be a detailed phenotypic study to help to identify patients who may be suitable for therapeutic intervention. In addition, with the recent availability of advanced imaging modalities, further detailed phenotypic investigations will also be valuable in helping to probe the relationship between structure and function and may shed light on disease mechanisms.