Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques

Kovacs, Kyle D.; Ciulla, Thomas A.; Kiss, Szilárd

doi:10.1080/14712598.2022.2121646

Cited by 9 publications

(16 citation statements)

References 88 publications

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“…[48] AAV-based gene therapy has a good effect on monogenic IRDs, including LCA, choroideremia, achromatopsia, Stargardt disease, retinitis pigmentosa, and other diseases. [11] At the same time, research and clinical trials began focusing on acquired ocular diseases. [49][50][51][52] One important reason was that monogenic diseases had 1 single cause, while the pathogenesis of acquired ocular diseases was usually complicated.…”

Section: Discussionmentioning

confidence: 99%

“…8B). The US had the leading condition with the largest number of clinical trials (54), followed by the UK (20), France (11), and Germany (11) (Fig. 8C).…”

Section: Analysis Of References and Co-cited Referencesmentioning

confidence: 99%

“…[6–9] Some articles or reviews on ocular gene therapy have different emphases. [2,10,11] However, there is no comprehensive and visualized analysis of the evolution and trends of AAV-based ocular gene therapy. Web of Science Core Collection (WOSCC) is generally used as the data source for bibliometric study because it is comprehensive and influential.…”

Section: Introductionmentioning

confidence: 99%

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Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis

Chen

Nie

et al. 2023

Medicine

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Background: Adeno-associated virus (AAV) plays a vital role in ocular gene therapy and has been widely studied since 1996. This study summarizes and explores the publication outputs and future research trends of AAV-based ocular gene therapy. Methods: Publications and data about AAV-based ocular gene therapy were downloaded from the Web of Science Core Collection or ClinicalTrials.gov database. The publications and data were analyzed by Microsoft Excel, CiteSpace, VOS viewer, and a free online platform (http://bibliometric.com). Results: Totally 832 publications from the Web of Science Core Collection relevant to AAV-based ocular gene therapy were published from 1996 to 2022. These publications were contributed by research institutes from 42 countries or regions. The US contributed the most publications among these countries or regions, notably the University of Florida. Hauswirth WW was the most productive author. “Efficacy” and “safety” are the main focus areas for future research according to the references and keywords analysis. Eighty clinical trials examined AAV-based ocular gene therapy were registered on ClinicalTrials.Gov. Institutes from the US and European did the dominant number or the large proportion of the trials. Conclusions: The research focus of the AAV-based ocular gene therapy has transitioned from the study in biological theory to clinical trialing. The AAV-based gene therapy is not limited to inherited retinal diseases but various ocular diseases.

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Section: Discussionmentioning

confidence: 99%

“…8B). The US had the leading condition with the largest number of clinical trials (54), followed by the UK (20), France (11), and Germany (11) (Fig. 8C).…”

Section: Analysis Of References and Co-cited Referencesmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis

Chen

Nie

et al. 2023

Medicine

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“…1 In the United States, the era of gene therapy began in 2017 following Food and Drug Administration (FDA) approvals of ex-vivo gene therapy with chimeric antigen receptor (CAR)-T cells to treat B cell malignancies and adeno-associated virus (AAV) based in-vivo gene therapy to treat congenital blindness, specifically RPE65-associated Leber congenital amaurosis. 1 , 2 Due to its relative immune privilege, the eye represents an ideal environment to deliver viral vector-based in vivo gene therapy via intravitreal, subretinal, or suprachoroidal delivery. 2 , 3 Gene therapy for retinal disease is especially promising since most inherited retinal disorders (IRDs) are monogenetic, cells are post-mitotic, and the imaging modalities used for diagnosis and monitoring are well-described and non-invasive.…”

Section: Introductionmentioning

confidence: 99%

Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study

Khanani,

Boyer,

Wykoff

et al. 2024

eClinicalMedicine

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“…Intravitreal therapies are an established mode of treatment for patients with nAMD and other retinal diseases, typically demonstrating a rapid and substantial improvement in vision within weeks of commencing treatment. 8 , 9 Various modalities are under investigation for the management of GA including sustained delivery implants (Brimonidine), 10 and intravitreal and suprachoroidal gene therapy, 11 which would reduce the burden of injections. However, regular intravitreal therapies remain the frontrunners, with positive Phase 3 results recently reported.…”

Section: Introductionmentioning

confidence: 99%

Towards a Therapy for Geographic Atrophy: A Patient’s Experience

Enoch¹,

Ghulakhszian²,

Sekhon³

et al. 2023

PPA

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Purpose Geographic atrophy (GA) is the advanced form of the non-neovascular (dry) type of age-related macular degeneration. Presently, GA cannot be treated. However, new therapies administered by intravitreal injection are in late-stage development. These can slow down, but do not stop or reverse, GA progression. The acceptability of these emerging therapies to people with GA is currently unknown. The present case study explores the perspectives of a person living with GA who took part in the terminated Phase 3 clinical trial of Lampalizumab, a candidate intravitreal treatment for GA. We explored this patient’s perspective on the retrospective acceptability of regular Lampalizumab injections, and the prospective acceptability of future intravitreal therapies for GA. Patients and Methods A 78-year-old woman living in the UK was recruited as part of a mixed-methods pilot study and interviewed by telephone, regarding: her experience of the Lampalizumab trial injections; and her thoughts regarding emerging intravitreal therapies for GA. The Framework Method was used for initial inductive analysis of the interview transcript. Subsequently, deductive analysis was undertaken, informed by the Theoretical Framework of Acceptability (TFA). Results For this participant, intravitreal injections in the Lampalizumab trial were acceptable, although streamlining processes within the clinic would have improved the patient experience. Regarding prospective acceptability of new intravitreal therapies, the participant considered a delay in progression of GA a valuable goal. Potential discomfort, anxiety and inconvenience associated with regular intravitreal injections would be acceptable in the context of preserving her vision for as long as possible. Conclusion Analysis of one participant’s experience demonstrates the value of exploring GA patients’ unique views on the acceptability of new intravitreal treatments. Larger prospective studies will provide more insight that help to optimise treatment design and delivery, thereby maximising likelihood of adherence and persistence when these therapies eventually arrive in clinic.

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Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques

Cited by 9 publications

References 88 publications

Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis

Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis

Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study

Towards a Therapy for Geographic Atrophy: A Patient’s Experience

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