In exchange for $165 million, Biogen now has access to zorevunersen, a potential first-of-its-kind treatment for Dravet ...

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

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 ...

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 ...

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 ...

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 ...

The deal, which features up to $385 million in milestones, gives Biogen exclusive rights to commercialize Stoke’s ...

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 ...

Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.