Nonviral ultrasound-mediated platform delivering DNA and RNA payloads to targeted tissues using microbubble enhancing agents.
RIPPLE is SonoThera's proprietary nonviral gene delivery platform that pairs intravenously injected ultrasound enhancing agents with externally applied therapeutic ultrasound to transport DNA and RNA payloads into targeted tissues. The platform supports full-length transgenic proteins, gene editors, siRNAs, and antisense oligonucleotides without payload size restrictions and has been demonstrated preclinically in liver, heart, skeletal muscle, kidney, and brain.
The platform is designed to be redosable and to avoid the immunogenicity and toxicity associated with viral vectors and lipid nanoparticles. RIPPLE underpins SonoThera's pipeline programs in Duchenne muscular dystrophy, autosomal dominant polycystic kidney disease, X-linked Alport syndrome, and hemophilia A, with a first clinical trial planned in DMD.
