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aln-ttr01
- Development stage
- Phase 1
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ALN-TTR01 is a first-generation, systemically delivered small interfering RNA (siRNA) therapeutic developed for the treatment of transthyretin-mediated amyloidosis (ATTR). It targets the transthyretin (TTR) gene using RNA interference to reduce both wild-type and mutant TTR protein levels in patients. ALN-TTR01 employs lipid nanoparticle (LNP) technology for delivery, specifically utilizing Tekmira’s SNALP platform. The drug demonstrated rapid, dose-dependent, and durable reductions in serum TTR protein levels in Phase 1 clinical trials but was discontinued in favor of next-generation candidates. ALN-TTR01 was developed by Alnylam Pharmaceuticals and received orphan drug designation for ATTR[1][2][3][4][5][6][7].
02
Targets
TTR (Transthyretin)
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