Aro Biotherapeutics a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, announced that it has received U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for ABX1100, a novel therapy currently being investigated in a phase 1 study in late-onset Pompe disease (LOPD) patients.
Aro presented data at the 2025 WORLDSymposium™ in San Diego, California, from a phase 1 study in normal healthy volunteers (NHVs) in which ABX1100 provided durable knockdown of glycogen synthase 1 (GYS1) messenger RNA (mRNA) and protein in the muscle. ABX1100 also was well-tolerated and exhibited predictable pharmacokinetics (PK) in the Phase 1 trial in NHVs.
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