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0 Diseases |  |
2 Trials |
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2 Trials |  |
39 News |  |
- | rAAV2tYF-CB-hRS1 / AGTC
Preclinical, Journal, Gene therapy: The dose-response relationship of subretinal gene therapy with rAAV2tYF-CB-hRS1 in a mouse model of X-linked retinoschisis. (Pubmed Central) - Feb 28, 2024
Sham treatment resolves schisis cysts, but 8E8 vg/eye is needed for optimal retinal electrical function rescue. These findings offer a promising path for clinical translation to human trials.
- | rAAV2tYF-CB-hRS1 / AGTC, NN1213 / Novo Nordisk
Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Nov 18, 2023
P1/2, N=12, Active, not recruiting, Sponsor: National Eye Institute (NEI)
These findings offer a promising path for clinical translation to human trials. Trial primary completion date: Jun 2021 --> Jul 2025
- | rAAV2tYF-CB-hRS1 / AGTC
New P1 trial, Gene therapy: Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients (clinicaltrials.gov) - Oct 3, 2023
P1, N=9, Recruiting, Sponsor: Zhongmou Therapeutics
- ATSN-201 / Atsena Therap
IND?enabling studies to support the clinical development of ATSN?201, a subretinally delivered, laterally spreading gene replacement therapy for X?Linked Retinoschisis (XLRS) (Shed 2B) - Aug 29, 2023 - Abstract #ESGCT2023ESGCT_224;
A single, subretinal injection of ATSN?201 significantly improved retinal structure and function, supporting the clinical use of ATSN?201 for the treatment of XLRS. The ability of the novel AAV.SPR capsid to effectively deliver the RS1 to the central retina of primates following extrafoveal SRI represents a safer and potentially more effective treatment option for XLRS patients.
- || rAAV2tYF-CB-hRS1 / AGTC
Trial completion, Trial completion date: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Jun 12, 2023
P1/2, N=27, Completed, Sponsor: Applied Genetic Technologies Corp
The ability of the novel AAV.SPR capsid to effectively deliver the RS1 to the central retina of primates following extrafoveal SRI represents a safer and potentially more effective treatment option for XLRS patients. Active, not recruiting --> Completed | Trial completion date: Oct 2023 --> May 2023
- | rAAV2tYF-CB-hRS1 / AGTC
Journal: Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in X-linked Retinoschisis - 1 Year Clinical Results. (Pubmed Central) - Jul 6, 2022
Gene augmentation therapy with rAAV2tYF-CB-hRS1 for XLRS was generally safe and well-tolerated but failed to demonstrate a measurable treatment effect. The clinical trial is ongoing through 5-year follow-up to assess long-term safety.
- | rAAV2tYF-CB-hRS1 / AGTC, NN1213 / Novo Nordisk
Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - May 26, 2022
P1/2, N=12, Active, not recruiting, Sponsor: National Eye Institute (NEI)
The clinical trial is ongoing through 5-year follow-up to assess long-term safety. Trial primary completion date: Jul 2025 --> Jun 2021
- rAAV2tYF-CB-hRS1 / AGTC, human Rhodopsin / Kubota
Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS (Poster Board Number: M-177; Hall D) - May 6, 2022 - Abstract #ASGCT2022ASGCT_1556;
AAV.SPR-hGRK1-hRS1 leads to therapeutic levels of RS1 in subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function. In addition, AAV.SPR safely and efficiently mediated RS1 expression in the central retina of primate eyes following peripheral SRI.
- rAAV2tYF-CB-hRS1 / AGTC
Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS (A0341) - Apr 29, 2022 - Abstract #ARVO2022ARVO_1531;
AAV.SPR delivered therapeutic levels of hRS1 to subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function. In addition, AAV.SPR safely and efficiently mediated hRS1 expression in the central retina of primate eyes following peripheral subretinal injection.
- | rAAV2tYF-CB-hRS1 / AGTC, NN1213 / Novo Nordisk
Enrollment closed, Enrollment change: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Apr 22, 2022
P1/2, N=12, Active, not recruiting, Sponsor: National Eye Institute (NEI)
In addition, AAV.SPR safely and efficiently mediated hRS1 expression in the central retina of primate eyes following peripheral subretinal injection. N=24 --> 12 | Recruiting --> Active, not recruiting
- rAAV2tYF-CB-hRS1 / AGTC
AAV-RS1 Trials for X-linked Retinoschisis: Disease Condition, Trial Parameters, and Immune Status (Room 202) - Mar 12, 2022 - Abstract #ASGCT2022ASGCT_356;
- rAAV2tYF-CB-hRS1 / AGTC
[VIRTUAL] Retinal structural and functional rescue by subretinal delivery of RS1 gene in mouse models of X-linked juvenile retinoschisis () - May 13, 2021 - Abstract #ARVO2021ARVO_1306;
Gene supplementation shows promise for XLRS gene therapy. Less invasive delivery method and robust photoreceptor promoters for more efficient treatment are being explored.
- | rAAV2tYF-CB-hRS1 / AGTC, NN1213 / Novo Nordisk
Trial completion date, Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - May 14, 2020
P1/2, N=24, Recruiting, Sponsor: National Eye Institute (NEI)
Less invasive delivery method and robust photoreceptor promoters for more efficient treatment are being explored. Trial completion date: Jul 2023 --> Jul 2025 | Trial primary completion date: Jul 2023 --> Jul 2025
- | rAAV2tYF-CB-hRS1 / AGTC, NN1213 / Novo Nordisk
Trial completion date, Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Jun 12, 2019
P1/2, N=24, Recruiting, Sponsor: National Eye Institute (NEI)
Trial completion date: Jul 2023 --> Jul 2025 | Trial primary completion date: Jul 2023 --> Jul 2025 Trial completion date: Jul 2021 --> Jul 2023 | Trial primary completion date: Jul 2021 --> Jul 2023
- | rAAV2tYF-CB-hRS1 / AGTC
Enrollment closed, Trial completion date, Trial primary completion date: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Jan 11, 2019
P1/2, N=27, Active, not recruiting, Sponsor: Applied Genetic Technologies Corp
Trial completion date: Jul 2021 --> Jul 2023 | Trial primary completion date: Jul 2021 --> Jul 2023 Recruiting --> Active, not recruiting | Trial completion date: Oct 2022 --> Oct 2023 | Trial primary completion date: Oct 2018 --> Apr 2019
- | rAAV2tYF-CB-hRS1 / AGTC
Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Oct 30, 2017
P1/2, N=24, Recruiting, Sponsor: National Eye Institute (NEI)
Recruiting --> Active, not recruiting | Trial completion date: Oct 2022 --> Oct 2023 | Trial primary completion date: Oct 2018 --> Apr 2019 Trial primary completion date: Dec 2017 --> Jul 2021
- rAAV2tYF-CB-hRS1 / AGTC
Trial primary completion date: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Sep 1, 2017
P1/2, N=27, Recruiting, Sponsor: Applied Genetic Technologies Corp
Trial primary completion date: Dec 2017 --> Jul 2021 Trial primary completion date: Dec 2017 --> Oct 2018
- rAAV2tYF-CB-hRS1 / AGTC
Trial primary completion date: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Apr 22, 2016
P1/2, N=27, Recruiting, Sponsor: Applied Genetic Technologies Corp
Trial primary completion date: Dec 2017 --> Oct 2018 Trial primary completion date: Dec 2016 --> Dec 2017
- rAAV2tYF-CB-hRS1 / AGTC
Enrollment change: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Nov 5, 2015
P1/2, N=24, Recruiting, Sponsor: National Eye Institute (NEI)
Trial primary completion date: Dec 2016 --> Dec 2017 N=100 --> 24
- | rAAV2tYF-CB-hRS1 / AGTC
Enrollment open: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Jun 8, 2015
P1/2, N=27, Recruiting, Sponsor: Applied Genetic Technologies Corp
N=100 --> 24 Not yet recruiting --> Recruiting
- | rAAV2tYF-CB-hRS1 / AGTC
New P1/2 trial: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) - Apr 14, 2015
P1/2, N=27, Not yet recruiting, Sponsor: Applied Genetic Technologies Corp
- rAAV2tYF-CB-hRS1 / AGTC
Trial primary completion date: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Jan 28, 2015
P1/2, N=100, Recruiting, Sponsor: National Eye Institute (NEI)
Not yet recruiting --> Recruiting Trial primary completion date: Jun 2016 --> Dec 2017
- rAAV2tYF-CB-hRS1 / AGTC
Enrollment open: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Jan 16, 2015
P1/2, N=100, Recruiting, Sponsor: National Eye Institute (NEI)
Trial primary completion date: Jun 2016 --> Dec 2017 Not yet recruiting --> Recruiting
- | rAAV2tYF-CB-hRS1 / AGTC
New P1/2 trial: Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) - Dec 19, 2014
P1/2, N=100, Not yet recruiting, Sponsor: National Eye Institute (NEI)