ALN-TTR01: trials, targets and approvals

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Comparator or background therapy1 trial
Phase 1	NCT01148953 Comparator	Jun 2010 → Jan 2012	—	Alnylam Pharmaceuticals	Completed	No outcome recorded

Date	Issuer	Release
2012-05-10	Alnylam Pharmaceuticals	Alnylam Presents Final Results from a Phase I Clinical Trial of ALN-TTR01, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR) alnylam.com ↗
2011-11-21	Alnylam Pharmaceuticals	Alnylam Presents Positive Preliminary Clinical Results for ALN-TTR01, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis alnylam.com ↗
2011-01-25	Alnylam Pharmaceuticals	Alnylam Receives Positive Opinion for Orphan Drug Designation from European Medicines Agency for ALN-TTR01, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis alnylam.com ↗
2010-07-07	Alnylam Pharmaceuticals	Alnylam Initiates Dosing in Phase I Clinical Study of ALN-TTR01 in Patients with Transthyretin-Mediated Amyloidosis (ATTR) alnylam.com ↗
2010-06-29	Alnylam Pharmaceuticals	Alnylam Obtains Approvals to Initiate Phase I Study with ALN-TTR01 in Patients with TTR-Mediated Amyloidosis (ATTR) alnylam.com ↗

Every value below carries the sentence it was read from. 2 sources stand behind the page.

Field	Value	Cited text
Known as	ALN-TTR01	ClinicalTrials.gov intervention name — accepted as the source's own label NCT01148953 ↗
Modality	Small interfering RNA (siRNA)	“We identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct first- and second-generation formulations of lipid nanoparticles,...” PMID 23984729 ↗ Aug 2013
Route	Intravenous	“Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion” NCT01148953 ↗