Pipeline — registered trials by phase
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
— · Transthyretin Amyloidosis With Cardiomyopathy · Recruiting · est. 2030-10-23
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
— · Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis · Recruiting · est. 2033-12-31
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
— · Acute Hepatic Porphyria · Completed · est. 2021-04-26
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
— · Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State · Active Not Recruiting · est. 2027-12-15
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
— · Acute Hepatic Porphyria · Recruiting · est. 2030-12-31
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
— · Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy · Recruiting · est. 2030-10-12
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
— · Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy · Completed · est. 2022-05-24
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
— · Primary Hyperoxaluria Type 1 · Active Not Recruiting · est. 2029-01-02
DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis
— · Familial Amyloidotic Cardiomyopathy (FAC) · Completed · est. 2016-01
Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment
— · Solid Tumors · Completed · est. 2012-08
Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver Involvement
— · Solid Tumors · Completed · est. 2011-07
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
— · Transthyretin (TTR)-Mediated Amyloidosis · Completed · est. 2014-03
A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease
— · Alzheimer's Disease · Recruiting · est. 2030-03-06
A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)
— · Hypercholesterolemia · Completed · est. 2015-05
A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers
— · TTR-mediated Amyloidosis · Completed · est. 2015-04
Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis
— · Transthyretin Mediated Amyloidosis (ATTR) · Completed · est. 2012-01
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
— · Acute Intermittent Porphyria · Completed · est. 2017-09-06
A Study to Evaluate ALN-6222 in Participants With Obesity
— · Obesity · Recruiting · est. 2027-12-14
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
— · Transthyretin-Mediated Amyloidosis · Completed · est. 2025-09-30
A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
— · Huntington's Disease · Recruiting · est. 2028-07-05
Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
— · TTR-mediated Amyloidosis · Completed · est. 2012-06
A Study to Evaluate ALN-AGT01 RVR in Adult Healthy Volunteers
— · Healthy Volunteers · Completed · est. 2025-09-08
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
— · Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) · Completed · est. 2018-01-12
A Study to Evaluate ALN-4285 in Adult Healthy Volunteers
— · Healthy Volunteers · Recruiting · est. 2026-12-29
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
— · Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma · Recruiting · est. 2027-09-30
A Study to Evaluate ALN-AGT01 in Patients With Hypertension
— · Hypertension · Completed · est. 2022-04-20
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
— · Early-Onset Alzheimer Disease · Recruiting · est. 2029-04-20
A Study to Evaluate ALN-4915 in Adult Healthy Volunteers
— · Healthy Volunteers · Recruiting · est. 2027-07-31
Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
— · Elevated LDL-Cholesterol (LDL-C) · Completed · est. 2012-03
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
— · Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Porphyria, Acute Intermittent · Completed · est. 2018-11-16
Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)
— · Respiratory Syncytial Virus Infections · Completed · est. 2012-04
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
— · TTR-mediated Amyloidosis · Completed · est. 2017-02-22
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
— · TTR-mediated Amyloidosis · Completed · est. 2014-10
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
— · TTR-mediated Amyloidosis · Completed · est. 2013-10
A Study to Evaluate Zilebesiran in Japanese Patients With Mild to Moderate Hypertension
— · Mild to Moderate Hypertension · Completed · est. 2025-07-17
Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial Virus
— · Respiratory Syncytial Virus Infections · Completed
A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate Hypertension
— · Hypertension · Completed · est. 2023-04-04
Zilebesiran as Add-on Therapy in Patients With High Cardiovascular Risk and Hypertension Not Adequately Controlled by Standard of Care Antihypertensive Medications (KARDIA-3)
— · High Cardiovascular Risk, Hypertension · Completed · est. 2025-06-19
Zilebesiran as Add-on Therapy in Patients With Hypertension Not Adequately Controlled by a Standard of Care Antihypertensive Medication (KARDIA-2)
— · Hypertension · Completed · est. 2023-12-11
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
— · TTR-mediated Amyloidosis · Completed · est. 2016-07
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
— · Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB) · Recruiting · est. 2027-06-30
A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM
— · Obese or Overweight Healthy Volunteers, Type 2 Diabetes Mellitus (T2DM) · Active Not Recruiting · est. 2027-08-31
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
— · Hereditary Hemorrhagic Telangiectasia · Active Not Recruiting · est. 2028-01-05
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
— · PH1, Primary Hyperoxaluria, RNAi Therapeutic · Completed · est. 2023-02-07
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
— · Acute Intermittent Porphyria · Completed · est. 2021-11-05
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
— · Down Syndrome-Associated Alzheimer's Disease (DS-AD) · Recruiting · est. 2030-05-18
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
— · Paroxysmal Nocturnal Hemoglobinuria (PNH) · Completed · est. 2016-04
Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)
— · Respiratory Syncytial Virus Infections · Completed · est. 2009-04
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
— · Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis · Completed · est. 2017-02-06
A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus
— · Type 2 Diabetes Mellitus (T2DM) · Recruiting · est. 2027-05-28
A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran
— · Hypertension · Recruiting · est. 2028-04-01
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
— · Cerebral Amyloid Angiopathy · Active Not Recruiting · est. 2027-08-09
A Study to Evaluate ALN-2232 in Participants With Obesity
— · Obesity · Recruiting · est. 2027-04-27
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
— · Primary Hyperoxaluria Type 1 (PH1) · Completed · est. 2019-01-23
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
— · TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial · Completed · est. 2017-08
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
— · Primary Hyperoxaluria Type 1 (PH1) · Completed · est. 2019-11-05
Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease
— · High Risk Cardiovascular Disease, Hypertension, High Cardiovascular Risk · Recruiting · est. 2030-09-23
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
— · Transthyretin Amyloidosis (ATTR) With Cardiomyopathy · Enrolling By Invitation · est. 2028-03-31
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
— · Primary Hyperoxaluria Type 1, Primary Hyperoxaluria · Completed · est. 2021-05-18
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
— · Amyloidosis, Hereditary, Transthyretin Amyloidosis · Completed · est. 2020-11-10
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
— · Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN · Recruiting · est. 2027-12-27
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
— · Amyloidosis · Completed · est. 2022-11-23
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
— · Transthyretin Amyloidosis (ATTR) With Cardiomyopathy · Completed · est. 2022-06-20
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
— · Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1) · Completed · est. 2020-06-29
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
— · Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial · Completed · est. 2017-03-30
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
— · Transthyretin Amyloidosis With Cardiomyopathy · Recruiting · est. 2030-05-28
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
— · Amyloidosis, Familial, Transthyretin Amyloidosis · Completed · est. 2020-10-06
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
— · Transthyretin Amyloidosis (ATTR) With Cardiomyopathy · Active Not Recruiting · est. 2024-05-08
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
— · Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent · Completed · est. 2019-01-31
No approved products listed under this sponsor in Drugs@FDA (partnered or subsidiary products may be listed elsewhere).
Source: ClinicalTrials.gov (trials where Alnylam is lead sponsor; 69 active or completed) and Drugs@FDA (0 approved products). Registry data can include older or partnered programs; the company's own pipeline page is the official list.
SEC filings — insider trading forms excluded
- 10-Q2026-07-30Quarterly report
- 8-K2026-07-30Current report (material event)
- 8-K2026-05-27Current report (material event)
- SCHEDULE 13G/A2026-05-14SCHEDULE 13G/A
- SCHEDULE 13G/A2026-05-14SCHEDULE 13G/A
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- 10-Q2026-04-30Quarterly report
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- SCHEDULE 13G2026-04-29SCHEDULE 13G
- ARS2026-04-06Annual report to holders
- DEFA14A2026-04-06DEFA14A
- DEF 14A2026-04-06Proxy statement
- SCHEDULE 13G/A2026-03-26SCHEDULE 13G/A
- 8-K2026-03-04Current report (material event)
- S-82026-02-12Employee stock plan
- 10-K2026-02-12Annual report
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- 8-K2026-01-12Current report (material event)
- 8-K2025-12-11Current report (material event)
- 8-K2025-12-03Current report (material event)
- SCHEDULE 13G/A2025-11-05SCHEDULE 13G/A
- 10-Q2025-10-30Quarterly report
- 8-K2025-10-30Current report (material event)
- 8-K2025-10-03Current report (material event)
- 8-K2025-09-12Current report (material event)
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- 10-Q2025-07-31Quarterly report
- 8-K2025-07-31Current report (material event)
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- UPLOAD2025-06-17UPLOAD
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- 10-Q2025-05-01Quarterly report
- 8-K2025-05-01Current report (material event)
- SCHEDULE 13G/A2025-04-07SCHEDULE 13G/A
- DEFA14A2025-04-04DEFA14A
- ARS2025-03-24Annual report to holders
- DEFA14A2025-03-24DEFA14A
- DEF 14A2025-03-24Proxy statement
Source: SEC EDGAR. Forms 3/4/5 and 144 (insider transactions) are intentionally left out.