Oligonucleotide
Tofersen
Own facility · Oligonucleotide
Tofersen is an antisense oligonucleotide targeting SOD1 mRNA, developed for SOD1-associated amyotrophic lateral sclerosis (ALS) and delivered intrathecally.
Supply offerings
| Business line | Business model | Regulatory status |
|---|---|---|
| APIOligonucleotide | Own facility supply | n/a |
Supplied for pharmaceutical manufacturing and R&D by licensed and qualified entities. Not for human use as supplied. Availability is subject to regulatory approval in the buyer's jurisdiction.