The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.

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Caris Life Sciences, Inc., Chandler, AZ.

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

ORIC Pharmaceuticals is advancing ORIC-114 for 1st-line EGFR/HER2 exon 20 NSCLC, with comprehensive data expected in the 2nd ...

The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.

Among the major classes of RNAs in the cell, tRNAs remain the most difficult to characterize via deep sequencing approaches, as tRNA structure and nucleotide modifications can each interfere with cDNA ...

Sustained exon skipping, dystrophin restoration, and improved muscle indicators were observed, including reduced inflammation and fibrosis. A 3.8-second improvement in time to rise (TTR ...

Cambridge, USA-based RNS specialist Wave Life Sciences (Nasdaq: WVE) has announced positive data from the Phase II FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being ...

Despite so many other oligonucleotide exon skipping therapies out there, the chemistry modifications to WVE-N531 and beyond, brings about a possible best-in-class profile. Especially, as I will ...

Sarepta’s news came as several companies—including Dyne Therapeutics, REGENXBIO and Genethon—presented new data on their next-generation exon-skipping therapies and gene therapies for Duchenne ...

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.