@SonotheraI41798 oral presentation focuses on the safe delivery of DNA expression vectors to the liver in NHP using its RIPPLE delivery technology. Study results demonstrated durable protein expression of up to 65% of normal circulating FVIII
#ASH25
https://t.co/3CPx9Yeve5
Circular RNA with embedded aptmers. Went strait to human testing after positive results on rodent models. Chinese biotech!
https://t.co/5FlWxRvydg
#circRNA#aptamers#ChineseBiotech
Efficient EGFP reporter transgene delivery to NHP T cells by LNP. Next step is delivering the CAR in vivo
#ESGCT2025#InVivoCART
Cecilia Cotta-Ramusino @TesseraTx
Official press release for @SonotheraI41798 presentation @ #ESGCT2025.
Results show high levels of durable human dystrophin protein expression exceeding
50% of endogenous levels have been achieved in NHP.
Poster Session 2, Oct 9th. Poster P0266.
#Dmd#GeneTherapy#nonviral
The ability to deliver targeted, full-length human dystrophin to skeletal, heart and diaphragm muscles in a durable, safe, redosable and noninvasive manner shows how UMD can overcome all of the challenges of current gene therapy delivery options. Learn more: https://t.co/69afv3v7Ps #ESGCT2025 #newdata #geneticmedicine #ultrasound #microbubble #nonviral #duchennemusculardystrophy
Heading to #ESGCT2025 to share the @SonotheraI41798 teamโs latest advances in non-viral delivery of gene therapy vectors expressing full-length dystrophin gene.
#DMD#GeneTherapy#Nonviral