Alnylam Pharmaceuticals

Alnylam Pharmaceuticals(ALNY)

CAMBRIDGE, MA
Pharmaceutical3 H-1B visas (FY2023)
WebsiteLinkedInCareers Page

Focus: RNAi

Alnylam Pharmaceuticals is a life sciences company focused on RNAi.

Gene TherapyNeurology
Funding Stage
PUBLIC
Total Funding
$250M
Open Jobs
0

Products & Portfolio (4)

AMVUTTRA
vutrisiran
Peak
RNASUBCUTANEOUS · SOLUTION
double-stranded siRNA-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adultsthe cardiomyopathy of wild-typehereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality+3 more
2022
0
GIVLAARI
givosiran sodium
Peak
RNASUBCUTANEOUS · SOLUTION
double-stranded small interfering RNA that causes degradation of aminolevulinate synthase 1 ( ALAS1 ) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP.
2019
0
ONPATTRO
patisiran
Peak
RNAINTRAVENOUS · SOLUTION
double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
2018
0
OXLUMO
lumasiran
Peak
RNASUBCUTANEOUS · SOLUTION
(GO) enzyme by targeting the hydroxyacid oxidase 1 ( HAO1 ) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. As the GO enzyme is upstream of the deficient alanine: glyoxylate aminotransferase (AGT) enzyme that causes PH1, the mechanism of action of lumasiran is independent of the underlying AGXT gene mutation. OXLUMO is not expected to be effective in primary hyperoxaluria type 2 (PH2) or type 3 (PH3) because its mechanism of action does not affect the metabolic pathways causing hyperoxaluria in PH2 and PH3.
primary hyperoxaluria type 1 (PH1) to lower urinaryplasma oxalate levels in pediatric
2020
0

Pipeline & Clinical Trials

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
Acute Hepatic Porphyria
N/A
Clinical Trials (1)
NCT02240784EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
N/A
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Re
Transthyretin Amyloidosis With Cardiomyopathy
N/A
Clinical Trials (1)
NCT07358078DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
N/A
A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
Amyloidosis
N/A
Clinical Trials (1)
NCT03774784A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
N/A
N/A
Clinical Trials (1)
NCT06360289Observational 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
N/A
DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac
Familial Amyloidotic Cardiomyopathy (FAC)
N/A
Clinical Trials (1)
NCT02252653DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis
N/A
N/A
Clinical Trials (1)
NCT02939820Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
N/A
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients Wit
Acute Hepatic Porphyria
N/A
Clinical Trials (1)
NCT03547297INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
N/A
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
Hereditary Transthyretin-mediated (hATTR) Amyloidosis
N/A
Clinical Trials (1)
NCT05040373Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
N/A
99mTc-pyrophosphate Scintigraphy
Transthyretin Amyloidosis
N/A
N/A
Clinical Trials (1)
NCT04201418A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
N/A
N/A
Clinical Trials (1)
NCT05505838Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
N/A
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis
Transthyretin-Mediated Amyloidosis
N/A
Clinical Trials (1)
NCT04561518ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
N/A
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 1
N/A
Clinical Trials (1)
NCT04982393BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
N/A
No intervention. Retrospective observation only.
Chronic Inflammatory Demyelinating Polyneuropathy
N/A
N/A
Clinical Trials (1)
NCT04125472Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
N/A
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positiv
Transthyretin Amyloidosis
N/A
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Acute Hepatic Porphyria
N/A
Clinical Trials (1)
NCT04883905ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
N/A
N/A
Clinical Trials (1)
NCT04056481Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
N/A
Phase 1
Phase 1
Clinical Trials (1)
NCT03505853A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
Phase 1
Phase 1
Clinical Trials (1)
NCT02503683A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
Phase 1
Phase 1
Clinical Trials (1)
NCT06600321A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
Phase 1
Phase 1
Clinical Trials (1)
NCT01437059Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
Phase 1
Phase 1
Clinical Trials (1)
NCT02797847A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
Phase 1
Phase 1
Clinical Trials (1)
NCT01814839A 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
Phase 1
Phase 1
Clinical Trials (1)
NCT06585449A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
Phase 1
ALN-AGT01
Hypertension
Phase 1
Clinical Trials (1)
NCT03934307A Study to Evaluate ALN-AGT01 in Patients With Hypertension
Phase 1
Phase 1
Clinical Trials (1)
NCT02314442A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)
Phase 1
ALN-VSP02
Solid Tumors
Phase 1
Clinical Trials (1)
NCT01158079Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment
Phase 1
Phase 1
Clinical Trials (1)
NCT02826018A Study of ALN-HBV in Healthy Adult Volunteers and Non-cirrhotic Patients With Chronic Hepatitis B Virus (HBV) Infection
Phase 1
ALN-VSP02
Solid Tumors
Phase 1
Clinical Trials (1)
NCT00882180Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver Involvement
Phase 1
Pozelimab
Healthy
Phase 1
Phase 1
Clinical Trials (1)
NCT01148953Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis
Phase 1
Phase 1
Clinical Trials (1)
NCT05661916A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
Phase 1
Phase 1
Clinical Trials (1)
NCT02053454A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
Phase 1
ALN-AGT01 RVR
Healthy Volunteers
Phase 1
Clinical Trials (1)
NCT06675565A Study to Evaluate ALN-AGT01 RVR in Adult Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT05231785A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
Phase 1
Phase 1
Clinical Trials (1)
NCT01559077Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
Phase 1
Computer algorithm for ATTR
Transthyretin Amyloidosis
Phase 1
Clinical Trials (1)
NCT04569903Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records
Phase 1
Phase 1
Clinical Trials (1)
NCT04565717A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)
Phase 1
Phase 1
Clinical Trials (1)
NCT05023889Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
Phase 1
Phase 1
Clinical Trials (1)
NCT07295717A Study to Evaluate ALN-4285 in Adult Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT02452372A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
Phase 1
Phase 1/2
Clinical Trials (1)
NCT02706886Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT05761301A Phase 1/2 Study to Evaluate ALN-KHK in Overweight to Obese Healthy Volunteers and Obese Patients With T2DM
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT06659640A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT02949830A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT02352493A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT06423352A Study to Evaluate Zilebesiran in Japanese Patients With Mild to Moderate Hypertension
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT03672188Study of VIR-2218 in Healthy Subjects and Patients With Chronic Hepatitis B
Phase 1/2

Open Jobs (0)

No open positions listed yet. Check their careers page directly.

Interview Prep Quick Facts
Portfolio: 4 approved products, 86 clinical trials
Top TAs: Cardiovascular, Neurology, Gastroenterology
H-1B (2023): 3 approvals
SEC Filings: 2 available
Portfolio Health
Peak4 (100%)
4 total products
Therapeutic Area Focus
Cardiovascular
1 marketed13 pipeline
Neurology
2 marketed11 pipeline
Respiratory
3 pipeline
Nephrology
3 pipeline
Oncology
3 pipeline
Marketed
Pipeline

Financials (FY2025)

Revenue
$1.8B76%
R&D Spend
$1.0B(55%)14%
Net Income
-$440M
Cash
$813M
Source: SEC 10-K Filing

Visa Sponsorship

Sponsors Work Visas
H-1B Petitions (FY2023)
3
Approved
0
Denied
100%
Rate

Source: USCIS H-1B Employer Data Hub