Alnylam Pharmaceuticals

This study tests a new medicine called ALN-AGT01 RVR 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 lowers blood pressure and reduces a protein linked to hypertension. About 93 part…

This study tests a new medicine, nucresiran, for people with a rare inherited disease that damages nerves (hATTR-PN). About 125 adults will receive either nucresiran or another drug (vutrisiran) to see which better slows nerve damage and improves quality of life. The goal is to c…

This study tests a new medicine called ALN-6400 for people with hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and frequent nosebleeds. The trial first checks safety in healthy volunteers, then tests how well the drug controls symptoms…

This early-stage study tests a new drug called ALN-5288 in 50 adults with mild Alzheimer's disease or related memory problems. The drug is given via a spinal injection to see if it is safe and can lower a harmful protein called tau in the brain. The goal is to slow disease progre…

This early-phase study tests an experimental drug called ALN-BCAT, alone or with an immunotherapy (pembrolizumab), in people with advanced liver cancer that has spread. The drug targets a specific genetic change (WNT pathway mutation) found in some liver cancers. The main goals a…

This study tests an experimental drug called zilebesiran in 11,000 adults whose high blood pressure is not well controlled despite taking at least two standard medications. Participants must have heart disease or be at high risk for it. The goal is to see if adding zilebesiran to…

This early-stage study is testing a new drug called ALN-APP for people with early-onset Alzheimer's disease. The drug is given as a shot into the spine to see if it is safe and how it affects the body. About 60 adults with mild memory problems or mild dementia will take part. The…

This study tests a new drug called ALN-HTT02 in 66 adults with early-stage 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. Researchers will also measure levels of the harmful huntingtin protein in spin…

This study tests a new medicine called ALN-4324 in two groups: first in healthy overweight volunteers, then in overweight people with type 2 diabetes. The goal is to see if it is safe and helps control weight and blood sugar. Participants receive either the drug or a placebo, and…

This study tests whether a single dose of the experimental drug ALN-4324 can improve how the body responds to insulin in people with type 2 diabetes. About 40 adults on stable metformin therapy will receive either the drug or a placebo. The main goal is to measure changes in insu…

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 by reducing new brain bleeds. Participants will be…

This study tests an investigational drug called nucresiran in about 1250 adults with a rare heart condition called ATTR amyloidosis with cardiomyopathy. The goal is to see if the drug can lower the risk of death and serious heart events compared to a placebo. Participants will re…

This study tests an experimental drug called ALN-2232, given alone or together with an existing weight-loss medication (tirzepatide), in adults with obesity. The goal is to see if the combination is safe and leads to greater weight loss. About 156 participants with a BMI between …

This study is following 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, to better understand how the condition progresses and how it is managed in everyday medical practice. Researchers will also track the safety and effectiveness of two appr…

This early-stage study is testing a new drug called ALN-4915 in 44 healthy adults to see if it is safe and how the body handles it. Participants receive either the drug or a placebo, and researchers monitor side effects and measure drug levels in blood and urine. This study does …

This study is a worldwide registry for people with acute hepatic porphyria (AHP), a rare liver condition. Researchers will collect information over time to better understand the disease and how current treatments work in everyday life. The goal is to improve care and knowledge fo…

This early-stage study tests the safety and tolerability of a new drug called ALN-4285 in 76 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. The goal is to g…

This study follows up to 10 women who were exposed to the drug patisiran-LNP during pregnancy to see how their pregnancies and babies turn out. It does not test a new treatment but collects real-world information on birth defects and pregnancy complications. The goal is to better…

This study looks at a protein called NfL in the blood of people who carry a TTR gene variant, both those without symptoms and those with hATTR amyloidosis affecting nerves. The goal is to see if NfL levels can help detect when the disease starts, how it progresses, and how well t…

This study will follow about 2000 adults with a rare heart condition called ATTR-CM, where abnormal proteins build up in the heart. Participants are starting treatment with the drug vutrisiran or another approved therapy. Researchers will track symptoms, quality of life, and heal…