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Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report
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Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report
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Journal Article
Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report
2021
Overview
Leber congenital amaurosis due to
CEP290
ciliopathy is being explored by treatment with the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger cohort (ClinicalTrials.gov
NCT03140969
) was studied for 15 months after a single intravitreal sepofarsen injection. Concordant measures of visual function and retinal structure reached a substantial efficacy peak near 3 months after injection. At 15 months, there was sustained efficacy, even though there was evidence of reduction from peak response. Efficacy kinetics can be explained by the balance of AON-driven new CEP290 protein synthesis and a slow natural rate of CEP290 protein degradation in human foveal cone photoreceptors.
Post hoc analyses and follow-up of a patient with Leber congenital amaurosis treated with the antisense oligonucleotide sepofarsen as part of a clinical trial indicates sustained improvement of vision 15 months after receiving a single dose of the drug.
Publisher
Nature Publishing Group US,Nature Publishing Group
Subject
/ Antigens, Neoplasm - genetics
/ Antigens, Neoplasm - metabolism
/ Biomedical and Life Sciences
/ Cell Cycle Proteins - genetics
/ Cell Cycle Proteins - metabolism
/ Cytoskeletal Proteins - genetics
/ Cytoskeletal Proteins - metabolism
/ Humans
/ Leber Congenital Amaurosis - genetics
/ Leber Congenital Amaurosis - physiopathology
/ Leber Congenital Amaurosis - therapy
/ Leber's congenital amaurosis
/ Mutation
/ Oligonucleotides, Antisense - therapeutic use
/ Patients
/ Photoreceptor Cells - metabolism
/ Proteins
/ Structure-function relationships
/ Vision
