The launch of the first clinical trial using exon skipping with an antisense oligonucleotide of the tricyclo-DNA class for the treatment of Duchenne Muscular Dystrophy
After receiving an approval from the French health regulatory authority ANSM for its "AVANCE 1" clinical trial, SQY Therapeutics, a French biotech, announces today the start of patient enrollment for the First-In-Human administration of the investigational medicinal product SQY51, a new generation of antisense oligonucleotide designed to restore semi-functional dystrophin by skipping exon 51 of …