Alnylam Pharmaceuticals

This early-stage study tests a new drug called ALN-5288 in 50 adults with mild cognitive impairment or dementia caused by Alzheimer's disease. The drug is given via a spinal injection and aims to safely reduce tau protein levels in the brain. The main goal is to check safety and …

This Phase 3 study tests a drug called nucresiran in 1250 people with ATTR amyloidosis, a rare disease where abnormal proteins damage the heart. Participants receive either nucresiran or a placebo every 6 months. The goal is to see if the drug reduces deaths and heart-related hos…

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to …

This study tests a single dose of an experimental drug called ALN-AGT01 in adults with mild to moderate high blood pressure who have already been treated with another drug, zilebesiran. The goal is to see if it safely helps control blood pressure by measuring changes in blood pre…

This study tests an experimental drug called ALN-2232, given alone or together with the approved weight-loss drug tirzepatide, in 156 adults with obesity. The goal is to see if the combination is safe and leads to more weight loss than either drug alone. Participants will receive…

This study tests a single dose of a new drug, ALN-4324, to see if it improves how the body uses insulin in people with type 2 diabetes. About 40 adults who are on a stable dose of metformin will take part. The main goal is to measure changes in insulin sensitivity using a glucose…

This study tests a drug called mivelsiran in 58 adults with Down syndrome who have early-stage Alzheimer's. Participants receive either the drug or a placebo via injection into the spine every few months for two years. The goal is to see if the drug can reduce brain changes linke…

This early-stage trial tests a new drug called ALN-6222 in 88 adults with obesity (BMI 30-40). Participants receive a single injection of either the drug or a placebo. The main goal is to check safety and how the body processes the drug, not weight loss. It is the first time this…

This early-stage trial tests a drug called ALN-APP (mivelsiran) in 60 adults with mild cognitive impairment or mild dementia due to early-onset Alzheimer's disease. The drug is given via a spinal injection to see if it safely lowers certain proteins linked to Alzheimer's in the b…

This study tests an investigational drug called ALN-6400 in 24 women and teens with von Willebrand disease who have heavy menstrual bleeding. The goal is to see if the drug is safe and can reduce bleeding. Participants will receive multiple doses and be monitored for side effects…

This early-stage study tests an experimental drug called ALN-HTT02 in 66 adults with Huntington's disease. The drug is given as a single injection into the spine to see if it is safe and how it affects the body. The goal is to reduce levels of the harmful protein that causes the …

This study tests an experimental drug called ALN-APP in 200 adults with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. The drug is given via spinal injection and aims to slow disease progression and reduce new brain bleeds. Participants will be …

This phase 3 study is testing an injectable drug called zilebesiran in about 11,000 adults whose high blood pressure is not well controlled despite taking at least two standard medications. Participants also have heart disease or are at high risk for it. The goal is to see if add…

This early-stage study tests a new drug called ALN-BCAT, given alone or with the immunotherapy Keytruda, in people with advanced or metastatic liver cancer that has a specific genetic mutation (WNT pathway). The trial aims to find safe doses and see if the drugs shrink tumors. Ab…

This early-phase trial is testing the safety and tolerability of a single dose of ALN-4285 in 76 healthy adults. Participants receive either the drug or a placebo, and researchers monitor for side effects and how the drug moves through the body. The study does not aim to treat an…

This early-phase trial is testing the safety and tolerability of a new drug called ALN-4915 in 44 healthy adults. Participants receive a single injection of either the drug or a placebo, and researchers monitor for side effects and how the drug moves through the body. Since this …

This study monitors pregnancy outcomes in women who took patisiran-LNP for hereditary ATTR amyloidosis. Researchers will track birth defects, pregnancy complications, and infant health. The goal is to better understand the drug's safety during pregnancy, not to test a new treatme…

This observational study will follow 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, for many years. Researchers will track how the disease progresses, how patients are treated in real-world settings, and the safety of two approved drugs, pat…

This study is a global registry that will follow about 150 people with acute hepatic porphyria (AHP) over time. Researchers will collect information on how the disease progresses, how it is managed in real-world settings, and the safety and effectiveness of approved treatments li…

This study will follow about 2000 adults with a heart condition called ATTR-CM, where abnormal proteins build up and weaken the heart. Researchers want to see how patients do on a drug called vutrisiran in everyday medical care, not just in a controlled trial. They will measure s…