Biogen and Stoke partner on zorevunersen for Dravet syndrome, with a Phase 3 trial starting in 2025. Stoke gets $165 million ...

With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...

Atherosclerotic cardiovascular disease is a major health concern worldwide and requires effective preventive measures. Lp(a) (lipoprotein [a]) has recently garnered attention as an independent risk ...

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 ...

Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...

In this study, we tested if locked nucleic acid (LNA) miR-181a-5p antisense oligonucleotides (ASO) could be used as a therapeutic to limit articular cartilage degeneration. Methods We used a variety ...

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 ...

PHILADELPHIA, PA — Imvax, Inc, recently closed a $29 million financing round from existing investors. This marks the latest milestone in the company’s fundraising efforts, which have totaled $86 ...

Imvax has received $29m in a financing round from current investors, aimed at advancing the Phase IIb trial of its autologous biologic-device combination product, IGV-001, in individuals with newly ...

Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...

worked to determine the quality antisense ONs (ASOs) attributes, such as purity, potency, and sequence for ONs, which can be challenging. This is due to the relatively large size of oligonucleotides, ...

Some genes might not be expressed in these organoids, making it impossible to target them effectively with ASO therapy. A precision approach to tumour treatment Another restriction is that only ...