New gapmer antisense oligonucleotide (ASO) candidates have been designed at Eisai Co. Ltd. to reduce microtubule-associated protein tau.

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3).

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.

Recent work proposes a promising therapeutic approach for dysferlinopathies.

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.

The dengue virus (DENV) is a single-stranded RNA virus with increasing worldwide prevalence causing severe mosquito-borne viral infections. Therefore, effective strategies to prevent and treat DENV infections are urgently needed.

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.