The MARINA study showed that AOC 1001 successfully targeted the root cause of DM1 by reducing toxic DMPK RNA buildup and improving how genes function in muscle cells. There are three key takeaways ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Del-desiran: Targeting DM1, this program has generated high patient ... At the core of Avidity’s potential is its AOC platform, which has demonstrated promising results in delivering RNA ...

Mis-splicing is the underlying cause of DM1 pathology, and we believe the mean splicing correction observed at 28 days following a single dose of PGN-EDODM1 at 10 mg/kg in the FREEDOM clinical ...

We are also rapidly progressing del-desiran in DM1 and del-brax in FSHD – both ... next stage as the company continues to advance its AOC technology in rare neuromuscular and precision ...

Building on success across its three clinical programs, Avidity is leading in rare neuromuscular diseases with a strong balance sheet to execute on a transformational ...