Drug Profile

Research programme: C16 conjugated small interfering RNA-based therapeutics - Alnylam Pharmaceuticals

Alternative Names: Research programme: C16-siRNA therapeutics - Alnylam Pharmaceuticals

Latest Information Update: 28 Oct 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

28 Oct 2025 No recent reports of development identified for preclinical development in CNS-disorders in USA (Intrathecal)
08 Jun 2022 Preclinical trials in Lung disorders in USA (Intranasal), before June 2022
02 Jun 2022 Preclinical trials in Eye disorders in USA (Intravitreous), before June 2022

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