antisense
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.
Ionis Pharmaceuticals Prices $500.3M Stock Offering
The antisense oligonucleotide developer plans to use the funds raised to support its independent commercial launches, late-stage clinical programs, and more.
SynaptixBio Readying to Take First Antisense Oligonucleotide Therapy Into Clinical Development
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The UK startup hopes to quickly select a lead ASO therapy to advance to clinical studies for a rare brain disorder next year, as it eyes the UK and US markets.
Ionis Pharmaceuticals Prepares for Pivotal Study of Angelman Syndrome Gene Therapy
The company plans to finalize its Phase III study design with the FDA later this year and to launch that trial in the first half of next year.
FDA Grants Priority Review to Ionis Pharmaceuticals' RNA-Targeted Hereditary Metabolic Disorder Drug
The firm submitted data showing olezarsen can lower triglyceride levels in patients with familial chylomicronemia syndrome.
Apr 8, 2024
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Oct 30, 2023