- In June 2025, Amylyx Pharmaceuticals received FDA Fast Track designation for AMX0114, an intrathecal antisense oligonucleotide (ASO) targeting calpain‑2 in ALS. The Phase 1 LUMINA trial is underway, with dosing initiated and early cohort data expected later this year
- In April 2025, Rutgers University researchers published pharmacokinetic data in mice showing that intrathecally administered U1 adaptor oligonucleotides effectively concentrate in key CNS regions (lumbar/cervical spinal cord, cerebellum) with minimal systemic leakage — supporting safer intrathecal delivery platforms
- In February–March 2025, Novartis reported positive late-stage data for intrathecal Zolgensma (onasemnogene abeparvovec‑IT) in older SMA patients, showing statistically significant motor improvements (HFMSE scale) in the Phase III STEER and Phase IIIb STRENGTH trials
