https://en.bioecon.ru/companies/sarepta-therapeutics/Recent content in Sarepta-Therapeutics on BioeconHugoen-USThu, 23 Jul 2026 23:05:33 +0700https://en.bioecon.ru/technology/gene-therapy-in-vivo-aav-lentiviral/Thu, 23 Jul 2026 00:00:00 +0000https://en.bioecon.ru/technology/gene-therapy-in-vivo-aav-lentiviral/One-time, potentially curative genetic medicines delivered by engineered adeno-associated and lentiviral vectors — eight FDA-approved in-vivo products since 2017, from Luxturna to Beqvez, now a multi-billion-dollar clinical reality alongside honest commercial retrenchments.https://en.bioecon.ru/technology/nucleic-acid-therapeutics/Sat, 11 Jul 2026 00:00:00 +0000https://en.bioecon.ru/technology/nucleic-acid-therapeutics/Short synthetic oligonucleotides — siRNA and antisense — bind a target messenger RNA to silence or reprogram a disease gene, enabled by GalNAc liver targeting and sugar/backbone chemistries, with multiple approved drugs across rare, neurological and cardiometabolic disease.https://en.bioecon.ru/technology/personalized-regenerative-medicine/Fri, 19 Jun 2026 00:00:00 +0000https://en.bioecon.ru/technology/personalized-regenerative-medicine/Gene and cell therapy platforms for rare monogenic and oncological diseases: AAV vectors, lentiviral HSC editing, ex vivo CRISPR (Casgevy) and in vivo CRISPR (LNP delivery) now deliver durable one-time treatments approved by FDA and EMA.