Ribapharm Aktie
WKN DE: 564808 / ISIN: US7625371080
23.07.2025 16:08:22
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Avidity Biosciences On Track To Seek Approval For Del-zota In DMD By Year-End
(RTTNews) - Avidity Biosciences Inc.'s (RNA) investigational ribonucleic acid therapy Del-zota has received Breakthrough Therapy designation from the FDA for the treatment of Duchenne muscular dystrophy in people living with mutations amenable to exon 44 skipping.
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
Duchenne muscular dystrophy (DMD) is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein, which often begins in early childhood.
Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44).
In a phase I/II trial of Del-zota in individuals with DMD44, dubbed EXPLORE44, that has been completed, the treatment demonstrated statistically significant increases in exon skipping, a substantial increase in dystrophin production, a significant and sustained reduction in creatine kinase levels to near normal, along with consistently favorable safety and tolerability.
The company is currently evaluating Del-zota in a phase II EXPLORE44 Open-Label Extension trial for people living with DMD44, dubbed EXPLORE44-OLE. The topline and functional data from the ongoing, fully enrolled phase II EXPLORE44-OLE trial are expected in the fourth quarter of 2025.
If all goes well as planned, the company intends to submit a Biologics License Application for Del-zota by the end of this year.
RNA has traded in a range of $21.51 to $56.00 in the last 1 year. As of this writing, the stock is at $33.69, up 0.46%.

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