Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review
Abstract
:1. Introduction
2. Disease Overview
3. Function of Retinal Pigment Epithelium and Photoreceptor Cells
4. Novel Therapies in Stargardt Disease
4.1. Gene Therapy
4.1.1. Lentiviral Vectors
4.1.2. Adeno-Associated Viral Vectors
4.1.3. Other Genetic Approaches
Strategy/ Intervention | Sponsor/Name of Study | Therapy | Clinical Trial/Website | Timeline | Phase |
---|---|---|---|---|---|
Gene therapy | |||||
Gene supplementation | Ocugen/ GARDian West China Hospital | AAV (OCU410ST) AAV (JWK006) | NCT05956626 NCT06300476 | 2023/2025 2023/2026 | Phase 1/2 Phase 1/2 |
Gene supplementation. | Sanofi | Lentivirus (SAR422459) | NCT01367444 NCT01736592 | 2011/2019 2012/2033 | Phase 1/2 Phase 1/2 |
Gene supplementation | Splice Bio AAVantgarde Bio | Dual AAV | https://splice.bio/pipeline/; accessed on 26 June 2024 https://www.aavantgardebio.com/technology-pipeline/; accessed on 15 June 2024 | expected 2024/2025 | Phase 1/2 |
Gene modulation | - | AON | N/A | N/A | N/A |
Gene editing | Ascidian Therapeutics/ STELLAR | ACDN-01 | NCT06467344 | 2024/2030 | Phase 1/2 |
Gene editing | - | CRISPR | - | N/A | N/A |
Gene coding | Saliogen | SGT-1001 | https://www.saliogen.com/pipeline/; accessed on 20 May 2024 | expected 2024 | N/A |
Optogenetic therapy | Nanoscope Therapeutics/ STARLIGHT | vMCO-010 | NCT05417126 | 2022/2023 | Phase 2 |
Raytherapeutics | RTx-021 | https://raytherapeutics.com/pipeline/; accessed on 2 June 2024 | N/A | N/A |
4.2. Cell Replacement Therapy
4.3. Pharmacological Therapy
4.4. Dietary Supplements
4.5. Future Perspectives in Therapy
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Strategy/ Intervention | Sponsor/Name of Study | Therapy | Clinical Trial/Website | Timeline | Phase |
---|---|---|---|---|---|
Stem cell therapy | Federal University of São Paulo Astellas | hESC-RPE | NCT02903576 NCT02941991 | 2015/2019 2013/2019 | Phase I/II - |
MD Stem Cells/ SCOTS | BMSC | NCT01920867 | 2012/2020 | - | |
CHABiotech Astellas | MA09-hRPE | NCT01625559 NCT01345006 NCT01469832 NCT02445612 | 2012/2015 2011/2015 2011/2015 2012/2019 | Phase I Phase I/II Phase I/II N/A | |
BlueRock Therapeutics/FUJIFILM Cellular Dynamics/Opsis Therapeutics | iPSC (OpCT-001) | - | N/A | N/A |
Strategy/ Intervention | Sponsor/Name of Study | Therapy | Clinical Trial/Website | Timeline | Phase |
---|---|---|---|---|---|
Drug therapy | |||||
Deuterated vitamin A | Alkeus Pharmaceuticals TEASE | ALK-001 | NCT02402660 NCT02230228 NCT04239625 | 2015/2025 2014/2015 2019/2026 | Phase 2 Phase 1 Phase 2 |
RBP4 inhibition | Belitebio/ DRAGON DRAGON II PHOENIX | Tinlarebant | NCT05244304 NCT06388083 NCT05949593 | 2022/2025 2024/2027 2023/2027 | Phase 3 Phase 2/3 Phase 3 |
Stargazer Pharmaceuticals | STG-001 | NCT04489511 | 2020/2021 | Phase 2 | |
Sirion Therapeutics | Fenretinide | NCT00429936 | 2006/2010 | Phase 2 | |
Stargazer Pharmaceuticals | A1120 | - | N/A | N/A | |
Removal of lipofuscin | Katairo/ STARTT | Soraprazan (Remofuscin) | EudraCT No. 2018-001496-20 | 2019/2023 | Phase 2 |
C5 inhibition | Astellas Pharma | Avacincaptad pegol (Zimura) | NCT03364153 | 2018/2025 | Phase 2 |
Visual cycle modulator | Kubota Vision/seaSTAR | Emixustat | NCT03033108 NCT03772665 | 2017/2017 2019/2022 | Phase 2 Phase 3 |
Increase macroautophagy | National Eye Institute | Metformin | NCT04545736 | 2020/2027 | Phase 1/2 |
Strategy/ Intervention | Sponsor/Name of Study | Therapy | Clinical Trial/Website | Timeline | Phase |
---|---|---|---|---|---|
Diet | |||||
Ophthalmos Research and Education Institute/ MADEOS | Omega-3 fatty acids | NCT03297515 | 2019/2020 | Not applicable | |
Catholic University of the Sacred Heart/ STARSAF02 | Saffron | NCT01278277 | 2011/2017 | Phase 1/2 | |
National Eye Institute | DHA | NCT00060749 | 2003/2007 | Phase I |
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Ghenciu, L.A.; Hațegan, O.A.; Stoicescu, E.R.; Iacob, R.; Șișu, A.M. Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review. Int. J. Mol. Sci. 2024, 25, 8859. https://doi.org/10.3390/ijms25168859
Ghenciu LA, Hațegan OA, Stoicescu ER, Iacob R, Șișu AM. Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review. International Journal of Molecular Sciences. 2024; 25(16):8859. https://doi.org/10.3390/ijms25168859
Chicago/Turabian StyleGhenciu, Laura Andreea, Ovidiu Alin Hațegan, Emil Robert Stoicescu, Roxana Iacob, and Alina Maria Șișu. 2024. "Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review" International Journal of Molecular Sciences 25, no. 16: 8859. https://doi.org/10.3390/ijms25168859