by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. We believe this mechanism will ...

Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced that the company will present ...

DM1 is caused by a trinucleotide (CUG) repeat expansion of the RNA encoding myotonic dystrophy protein kinase (DMPK ), resulting in the formation of nuclear aggregates that bind and sequester splicing ...

– The Company today reported positive initial results from the ongoing FREEDOM-DM1 trial in patients with DM1; mean splicing correction of 12.3% and 29.1% in 5 and 10 mg/kg cohorts, respectively ...

PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular ...

BOSTON, February 24, 2025--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of ...

Sarepta's Q4 2024 EPS exceeded forecasts by 23.4%. Revenue grew by 75% year-over-year, driven by Alevitus sales. The stock fell 3.14% in aftermarket trading despite positive earnings. Guidance for ...

PepGen Inc. , a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological ...

BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment ...

The first issue to complicate the interpretation of RNAi datasets in recent years has been the targeting specificity inherent to the various types of dsRNA reagents used to direct the RNAi pathway ...