Expressing:Article Title: Advances in Gene Therapy for Age-Related Macular Degeneration: A Narrative Review
Article Snippet: AdGVPEDF.11D , GenVec, Gaithersberg, MD, USA , Expression of PEDF that binds to and inhibits VEGF receptors , Adenovirus , Intravitreal , Phase 1 , NCT00109499 , 2002–2005 , Completed , Safety and tolerability , - , Safe and well-tolerated. However, further development was halted due to insufficient efficacy.. .. AAV2-sFLT01 , Genzyme, a Sanofi Company, Cambridge, MA, USA , Expression of sFLT-1 that binds to and inhibits VEGF , AAV , Intravitreal , Phase 1 , NCT01024998 , 2010–2018 , Completed , Maximum tolerated dose, adverse events through week 52 and up to 4 years , Decreased retinal thickness through week 52 and up to 4 years , Safe and well-tolerated. However, further development was halted due to insufficient efficacy.. .. rAAV.sFLT-1 , Lions Eye Institute, Perth, Western Australia , Expression of sFLT-1 that binds to and inhibits VEGF , AAV , Subretinal , Phase 1/2 , NCT01494805 , 2011–2017 , Completed , Ophthalmic complications, toxicity or systemic complications at 1-month post-injection , Maintenance or improvement of vision without the necessity of ranibizumab re-injections up to 3 years , Safe and well-tolerated. However, further development was halted due to insufficient efficacy..
Bioprocessing:Article Title: Advances in Gene Therapy for Age-Related Macular Degeneration: A Narrative Review
Article Snippet: AdGVPEDF.11D , GenVec, Gaithersberg, MD, USA , Expression of PEDF that binds to and inhibits VEGF receptors , Adenovirus , Intravitreal , Phase 1 , NCT00109499 , 2002–2005 , Completed , Safety and tolerability , - , Safe and well-tolerated. However, further development was halted due to insufficient efficacy.. .. AAV2-sFLT01 , Genzyme, a Sanofi Company, Cambridge, MA, USA , Expression of sFLT-1 that binds to and inhibits VEGF , AAV , Intravitreal , Phase 1 , NCT01024998 , 2010–2018 , Completed , Maximum tolerated dose, adverse events through week 52 and up to 4 years , Decreased retinal thickness through week 52 and up to 4 years , Safe and well-tolerated. However, further development was halted due to insufficient efficacy.. .. rAAV.sFLT-1 , Lions Eye Institute, Perth, Western Australia , Expression of sFLT-1 that binds to and inhibits VEGF , AAV , Subretinal , Phase 1/2 , NCT01494805 , 2011–2017 , Completed , Ophthalmic complications, toxicity or systemic complications at 1-month post-injection , Maintenance or improvement of vision without the necessity of ranibizumab re-injections up to 3 years , Safe and well-tolerated. However, further development was halted due to insufficient efficacy..
other:Article Title: Age-Related Macular Degeneration: Ocular Genetic Studies
Article Snippet: AAV2-sFLT01 (Genzyme, a Sanofi Company), VEGFA’yı inhibe eden sFLT-1 genini taşıyan bir vektörün tek doz olarak göziçine enjekte edilmesidir.44 Başka bir şirket ise, daha önce tanımlanmış olan üründen yalnızca sFLT-1 füzyon sekansının IgG1’in Fc bölgesine bağlanması yönüyle farklılık gösteren rAAV.sFlt-1 (NCT01024998) adlı rekombinant adeno-ilişkili virüs (rAAV) gen tedavisi geliştirmiştir.41 Bu tedavinin, ıslak YBMD’de Eylea’nın indüklenebilir aşırı ekspresyonu sayesinde KNV oluşumunu engelleyeceği düşünülmektedir.45 Ranibizumaba benzer çözünür bir antiVEGF monoklonal antikor proteinini kodlayan genetik materyali içeren bir rAAV vektörü olan RGX-314 (Regenxbio, Inc.) de, subretinal enjeksiyon yoluyla uygulanmasının güvenli ve tolere edilebilir olduğu gösterilmiş ve devam eden faz I/IIa çalışması ile etkinliği araştırılmaktadır.46 Yaş tip YBMD’de potansiyel bir tek uygulamalı tedavi olarak etkinliğini belirlemek amacıyla şu anda birçok klinik çalışma yürütülmektedir (NCT04514653, NCT04832724, NCT03999801, NCT05210803 ve NCT04704921).
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