information: Submission of a clinical trial application
Announcement: submission of a clinical trial application
Company: Alnylam Therapeutics (USA - MA)
- RNAi. ALN-AAT02 is an investigational, subcutaneously administered RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease). ALN-AAT02 is Alnylam’s first investigational RNAi therapeutic utilizing the company’s enhanced stabilization chemistry plus (ESC+) GalNAc-conjugate technology. This technology enables subcutaneous dosing with increased selectivity and a wide therapeutic index.
Disease: AAT deficiency-associated liver disease (alpha-1 liver disease)
area: Rare diseases - Genetic diseases - Hepatic diseases - Liver diseases
- • On October 3, 2018, Alnylam Pharmaceuticals announced that it has submitted an Clinical Trial Authorization (CTA) application to the Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study of ALN-AAT02 for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).