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Ocular

Targeting recurrent ABCA4 variant with antisense oligonucleotides as new strategy for Stargardt disease

Feb. 3, 2025
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud University developed a new antisense oligonucleotide (AON) therapy, designed to rescue the splicing defect caused by this variant.
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Regulus heads to phase III after more positive kidney disease data

Jan. 29, 2025
By Jennifer Boggs
In an indication that has proved difficult for biopharma to conquer, Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGLS-8429 in autosomal dominant polycystic kidney disease and laid out its plans to move straight into a phase III trial later this year, with the potential for an accelerated U.S. approval.
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Dyne seeks accelerated path on positive dystrophy data; shares down

Jan. 10, 2025
By Lee Landenberger
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
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Neurology/psychiatric

Series A at Neumirna supports RNA therapies for neurological disorders

Jan. 8, 2025
A €20 million (US$20.6 million) series A financing round at Neumirna Therapeutics ApS is set to support the company’s development of RNA therapies for epilepsy, Parkinson’s disease and other neurological disorders, and enable the company to advance its lead asset into the clinic.
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FDA Approved stamp

Ionis notches Arrowhead win; Tryngolza cleared for FCS

Dec. 20, 2024
By Randy Osborne
Ionis Pharmaceuticals Inc. CEO Brett Monia predicted that diagnoses of familial chylomicronemia syndrome (FCS) will “accelerate fairly quickly” now that the firm has gained U.S. FDA clearance for Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with the rare form of severe hypertriglyceridemia (sHTG) that can lead to life-threatening acute pancreatitis (AP).
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