The collaboration is based on new data that the University of California, San Francisco (UCSF) scientists, along with collaborators, published in the journal Nature suggesting that GNAQ plays an important role in the development of uveal melanoma.
GNAQ is involved in mediating intracellular signals which are capable of promoting cell proliferation and survival. Normally, GNAQ is only activated by its associated receptor; however, mutated forms of GNAQ confer a persistently activated state for the protein, which may lead to inappropriate signaling.
Jared Gollob, senior director of clinical research at Alnylam Pharmaceuticals, said: “The emerging data on cancer genomes and the molecular basis for malignant cell transformation form a very compelling opportunity for the advancement of RNAi therapeutics targeting genes derived from somatic mutations. In this case, we are excited to be collaborating with UCSF on this program, as there is a clear need for novel therapeutics to effectively treat patients with advanced uveal melanoma.
“We believe that an RNAi therapeutic targeting GNAQ in uveal melanoma that has spread to the liver may represent an ideal treatment option for this devastating disease. Uveal melanoma represents another example of an orphan indication that Alnylam intends to pursue where there is a very high unmet need and a clear opportunity for a significant therapeutic impact for patients.”