Pre-clinical Programs

Our strategy at Alnylam is to lead the translation of the science of RNAi into a robust drug discovery capability and to build a significant product pipeline of innovative medicines.

In order to prioritize our efforts, we focus on disease areas with significant unmet medical need, where the biology around the target is well characterized and understood, and where our delivery technologies can achieve efficient and specific effects.

Together with our collaborators, we are working towards developing novel therapeutics that have the potential to intervene in the disease processes of these major unmet medical needs.

Alnylam BioDefense^™: Alnylam BioDefense^™ was established to build a robust platform for developing RNAi therapeutics targeting threats of bioterrorism. Funding for the company's Ebola virus, pandemic flu, and viral hemorrhagic fever programs represents an example of broad public health and federal interest in the potential of RNAi technology to treat and prevent disease from these and other serious and life-threatening viruses.

In September 2006, Alnylam was awarded a contract to advance the development of a broad spectrum RNAi anti-viral therapeutic against hemorrhagic fever virus. With this grant, the company established Alnylam Biodefense^™ — an initiative to build a robust platform for developing RNAi therapeutics targeting biological threats.

As part of a public sector-private sector partnership with its Ebola program, Alnylam is working with the United States Army Medical Research Institute of Infectious Diseases (USAMRIID), an organization which is uniquely experienced in the handling, safety, and security requirements of specialized biological agents. Alnylam produces drug candidates which are sent to USAMRIID for in vitro and in vivo testing against the Ebola virus.

Additionally, in August 2007, was awarded a $38.6 million contract over 33 months from the United States Defense Threat Reduction Agency (DTRA) to develop a broad spectrum RNAi anti-viral therapeutic for the treatment of viral hemorrhagic fever. Viral hemorrhagic fevers are considered by federal agencies to be high priority agents that pose a risk to national security because they can be easily disseminated from person to person, result in high mortality rates, and require special action for public health preparedness.
Hepatitis C: Regulus Therapeutics' (a joint venture between Alnylam and Isis Pharmaceuticals) most advanced program is a microRNA therapeutic that targets miR-122 for the treatment of hepatitis C virus (HCV) infection, a significant disease worldwide where emerging therapies target viral genes and are therefore more prone to resistance. Regulus is targeting miR-122, an endogenous host gene required for viral infection by HCV.
Progressive Multifocal Leukoencephalopathy (PML): PML is caused by infection of the central nervous system with a virus called "JC virus" and can occur in certain immune-suppressed patients, including those receiving immunomodulatory therapies. Alnylam and Biogen Idec, Inc. have partnered to investigate the potential of using RNAi technology to discover and develop therapeutics to treat PML. The goal of the program is to design and optimize an RNAi therapeutic toward conserved regions of a viral genome to harness the cell's own capabilities to achieve an anti-viral therapeutic effect.
Pandemic Flu: The goal of Alnylam's influenza program is to develop an RNAi therapeutic effective against all flu strains including potentially highly virulent strains of flu, such as H5N1. By targeting key flu genes required for viral replication, Alnylam and its collaborator Novartis, aim to demonstrate potent anti-viral activity for the prevention and treatment of highly pathogenic flu strains. This program has previously received funding support from the Department of Defense's DARPA arm.
Novartis Programs: The 2005 partnership with Novartis and the Novartis Institute for BioMedical Research (NIBR) focuses on the joint discovery of new therapeutics using RNAi across multiple disease area gene targets emerging from Novartis' research portfolio.
Roche Programs: In July 2007, Alnylam and Roche formed a non-exclusive platform alliance which allows Roche to develop RNAi therapeutics in four therapeutic areas: oncology, respiratory diseases, metabolic diseases and certain liver diseases. Alnylam and Roche also will collaborate on RNAi drug discovery for one or more disease targets in these therapeutic areas.
Takeda Programs: In May 2008, Alnylam and Takeda formed a non-exclusive platform alliance which allows Takeda to develop RNAi therapeutics in the fields of oncology and metabolic disease with the option to expand to additional therapeutic areas. The agreement also includes the transfer of platform technology from Alnylam to Takeda, a collaboration and cross-license of delivery technologies between the two companies, and a drug discovery collaboration on certain RNAi therapeutic targets.