Alnylam Initiates Phase I Clinical Study of RNAi Therapeutic for the Treatment of Respiratory Syncytial Virus (RSV) Infection

Alnylam Becomes a Clinical-Stage Company with the Industry's First Trial of an RNAi Therapeutic for Viral Infectious Disease

09-Dec-2005

Alnylam Pharmaceuticals, Inc. announced that it has initiated a Phase I study in Europe to evaluate the human safety and pharmacology of ALN-RSV01, an RNAi therapeutic, in healthy volunteers. Additionally, the company's investigational new drug (IND) application to conduct a separate Phase I trial in the U.S. has cleared the 30-day review period by the food and Drug Administration (FDA), and the U.S. study is expected to begin by the end of the month.

"The initiation of this Phase I study represents significant progress in the field of RNAi as it is the first clinical trial of an RNAi therapeutic to treat an infectious disease," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam. "Importantly, this trial also marks Alnylam's transition to a clinical-stage company and is a major milestone in our continued effort to develop a pipeline of RNAi therapeutics to address major unmet medical needs."

ALN-RSV01 is a small interfering RNA (siRNA) that is designed to selectively and potently silence the RSV nucleocapsid 'N' gene. This gene is among the most highly conserved genes in the virus and is essential for RSV replication. Data presented to date show both in vitro and in vivo efficacy of ALN-RSV01 in pre-clinical studies. Intranasally delivered ALN-RSV01 specifically inhibits RSV replication in animals and is active in the prevention and treatment of RSV infection. Additionally, Alnylam scientists have reported completion of GLP toxicology studies for ALN-RSV01 without any significant toxicities being observed.

https://www.bionity.com/en/news/50771/alnylam-initiates-phase-i-clinical-study-of-rnai-therapeutic-for-the-treatment-of-respiratory-syncytial-virus-rsv-infection.html