The new program will focus on a specific drug target proposed by Merck, and Alnylam will proceed with preclinical development of an RNAi therapeutic for this target. The drug target is in the Nogo pathway, which plays a key role in preventing regeneration of nerves after injury, such as spinal cord injuries. An RNAi therapeutic that inhibits this pathway could potentially reduce or prevent paralysis caused by such injuries.
Alnylam and Merck have entered into two separate, but complementary, strategic collaborations. The program to develop an RNAi therapeutic for spinal cord injury falls within the first of these collaborations, initiated in September 2003. The goals of this collaboration are to advance RNAi technology and to apply this technology to develop RNAi therapeutics that selectively target human diseases.
The second collaboration between Alnylam and Merck, initiated in June 2004, is focused on age-related macular degeneration (AMD) and other ocular diseases caused by abnormal growth or leakage of small blood vessels in the eye. This collaboration includes Alnylam’s most advanced program, to develop a direct RNAi therapeutic targeting vascular endothelial growth factor (VEGF) for the treatment of the “wet” form of AMD.