The investigational medication, ALN-GO1, may reduce or prevent the overproduction of oxalate in people with PH1, using a new approach called RNA interference (RNAi). Researchers at Alnylam Pharmaceuticals (a biopharmaceutical company) are also using this approach to research and develop other medicines for rare, inherited conditions.
In laboratory studies and early studies in healthy people, the investigational medication decreased the enzyme that produces oxalate. Alnylam’s program of clinical research studies will determine if it can also reduce the liver’s oxalate production in people with PH1.
For those participating in the study, a study nurse will administer the investigational medication as a monthly injection with a small needle under the skin.
Find out about taking part in our current
and future PH1 clinical research studies.
Alnylam is focused on using RNAi therapeutics to create treatments for rare and life-threatening genetic conditions where there are limited treatment options.
RNA interference is based on a natural process of gene silencing, and investigational RNAi therapeutics are being developed from this scientific discovery.