Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

Biogen has entered a collaboration with Stoke Therapeutics to develop and and commercialise zorevunersen to treat Dravet ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.

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.

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

Under terms of the deal, Stoke will spearhead global development of zorevunersen, while Biogen will receive exclusive rights ...

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

Biogen has bolstered its pipeline by licensing ex-US rights to a drug for Dravet syndrome – a rare form of epilepsy – from ...

Biogen and Stoke Therapeutics have announced a partnership aimed at advancing a disease-modifying treatment for Dravet ...

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