Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that it has submitted a Clinical Trial Authorization (CTA) application to the Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study of ALN-AAT02, an investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) 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.
"We are pleased to reignite our efforts to develop a treatment for alpha-1 liver disease, where there is high unmet need with liver transplantation as the only available treatment option," said Thomas Hoock, Ph.D., Vice President, Program Lead for ALN-AAT02 at Alnylam. "We are also excited for ALN-AAT02 to enter the clinic as the first investigational RNAi therapeutic that will leverage the significant enhancements we have made to our GalNAc-siRNA conjugate platform. Pending feedback from the MHRA, we look forward to evaluating the safety, pharmacodynamics, and clinical activity of this molecule in a Phase 1/2 study in healthy volunteers and adults with alpha-1 liver disease, which we expect to initiate by year-end 2018."
Exploring mouse models for studying liver fibrosis regression