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.
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.
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.
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.
Ausperbio Therapeutics Inc. raised $110 million from two financing rounds in 2024 to advance its lead antisense oligonucleotide candidate as a functional cure for chronic hepatitis B.
Ausperbio Therapeutics Inc. raised $110 million from two financing rounds in 2024 to advance its lead antisense oligonucleotide candidate as a functional cure for chronic hepatitis B.
Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded orphan drug designation by the FDA for the treatment of polycythemia vera.
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).
After missing out on the glucagon-like peptide 1 obesity market, Sanofi SA is prospecting for next-generation drugs and is making a strategic equity investment in Resalis Therapeutics Srl, providing the Italian biotech with funding to take its lead program RES-010 through to phase II.
RSS
