SQY51 / SQY Therap - LARVOL DELTA

Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse. (PubMed, NAR Mol Med) - "This study evaluated intratracheal (IT) instillation as an alternative route for systemic delivery of therapeutic tricyclo-DNA antisense oligonucleotides (tcDNA-ASOs), using the Duchenne muscular dystrophy (DMD)-targeting compound SQY51 as a model...A single 25 mg/kg pulmonary dose was safe and well-tolerated, with no adverse structural or immune effects. These findings support IT instillation as a feasible alternative for systemic delivery of tcDNA-ASOs."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Inflammation • Muscular Dystrophy • CD68 • ITGAM

AVANCE1: Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Sqy Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

AVANCE1: Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Sqy Therapeutics

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics