Also known as: antisense oligonucleotides, Antisense oligonucleotides
Two experimental ALS therapies showed promise in recent clinical developments. Interim Phase 1/2 data suggest the antisense oligonucleotide QRL-201 restores a critical nerve protein and may slow disease decline, with a Phase 3 trial planned for 2027. Meanwhile, the oral therapy neflamapimod has been selected for testing in the UK's EXPERTS-ALS platform study, with first results expected this spring.
GSK's Phase III B-Well trials show bepirovirsen achieved 19% functional cure in chronic hepatitis B, rising to 26% in patients with lower viral activity, versus 0% for placebo. Regulatory decisions are expected in Q3 2026.
Huntington's disease provides neuroscience with a uniquely clear genetic model for studying brain disorders, featuring a single identifiable mutation and predictable disease progression. The condition serves as a testing ground for cutting-edge therapies and raises fundamental questions about brain regeneration. Its scientific clarity combined with a united patient and research community makes it an invaluable resource for advancing neurological understanding.
New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
The cardiovascular biologics market is projected to reach $4.23 billion by 2033, while the cell therapy market is expected to reach $5.38 billion by 2032, driven by RNA-based therapies and regulatory approvals.
Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.
The FDA issued draft guidance establishing a plausible mechanism framework for approving individualized therapies targeting ultra-rare diseases when randomized controlled trials are not feasible due to small patient populations.
