ALS
Voyager Therapeutics Exploring Alternate Payload in SOD1-ALS Gene Therapy Program
New three-month data from a study of nonhuman primates underpins the biotech firm's decision to amend the small interfering RNA program.
UniQure Testing Higher Gene Therapy Dose in Phase I/II SOD1-ALS Trial
With permission from a data safety monitoring committee, the firm will begin treating a second cohort with AMT-162 in the EPISOD1 trial in early 2025.
In a first-of-its-kind trial, physicians will review neurofilament test results with patients and decide whether to continue giving them an experimental drug.
Trace Neuroscience Launches With $101M Series A Funding
The company is developing an antisense oligonucleotide to restore UNC13A protein expression, which it says could treat up to 97 percent of ALS patients.
UniQure Doses First Patient in Phase I/II Trial of SOD1-ALS Gene Therapy
Investigators will evaluate the safety, tolerability, and exploratory signs of efficacy, including neurofilament light chain and SOD1 protein.