full-length dystrophin DNA vector

SonoThera's full-length dystrophin DNA vector is a non-viral gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). The therapy utilizes a large, 14-kilobase DNA construct that encodes the complete, functional dystrophin protein, overcoming the packaging limitations of traditional adeno-associated virus (AAV) vectors which are restricted to smaller, truncated micro-dystrophin variants. The vector is delivered using SonoThera's proprietary RIPPLE (Remote Induction of Pulsed Pressure Lateral to Energy) technology, an ultrasound-mediated system that facilitates targeted, non-invasive, and potentially re-dosable delivery to skeletal, cardiac, and diaphragm muscles. Preclinical studies in rodent and non-human primate (NHP) models have demonstrated robust expression of full-length dystrophin and phenotypic improvement without the significant immunogenicity or liver toxicity often associated with viral delivery methods.

Other names

full-length dystrophin vectorSonoThera full-length dystrophin DNA vector