Author Archives: Margarida Maia

AOC 1001 Earns Orphan Drug Status for DM1 in Europe

The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have the potential to treat rare, life-threatening, or very serious diseases that affect less than five in 10,000 patients…

PepGen Raises $112.5M to Move EDO Therapies to Human Trials

PepGen has raised funding totaling $112.5 million to advance toward the clinic its enhanced delivery oligonucleotide (EDO) lead programs in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The funding round keeps PepGen on track to start Phase 1 clinical trials in 2022 for DMD, and…