Alnylam Pharmaceuticals is an American biopharmaceutical company focused on discovering, developing, and commercializing RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
Regeneron announced FDA and EMA review acceptance for cemdisiran in generalized myasthenia gravis and FDA priority review acceptance for garetosmab in fibrodysplasia ossificans progressiva. The garetosmab BLA is supported by Phase 3 OPTIMA trial data showing 94% and 90% reductions in new bone lesions.
Alnylam Pharmaceuticals entered a $1.23 billion discovery collaboration with Tenaya Therapeutics to identify up to 15 novel genetic targets for heart disease. The agreement includes $10 million upfront and up to $1.13 billion in milestone payments.
Alnylam reported full-year 2025 profitability and $2.987 billion in global net product revenues, driven by U.S. approval and growth of AMVUTTRA. The company also outlined 2026 pipeline goals and additional HELIOS-B analyses for vutrisiran.
The cardiovascular biologics market is projected to reach $4.23 billion by 2033, while the cell therapy market is expected to reach $5.38 billion by 2032, driven by RNA-based therapies and regulatory approvals.
Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.
A review in The Lancet highlights emerging long-acting injectable therapies for hypertension that could reduce treatment to two doses annually instead of daily pills, with several candidates now in late-stage clinical trials.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07575308 |
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB |
NOT_YET_RECRUITING | PHASE2 |
| NCT07553442 |
A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran |
RECRUITING | PHASE2 |
| NCT07535606 |
A Study to Evaluate ALN-4915 in Adult Healthy Volunteers |
RECRUITING | PHASE1 |
| NCT07465224 |
A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus |
RECRUITING | PHASE2 |
| NCT07463846 |
A Study to Evaluate ALN-2232 in Participants With Obesity |
RECRUITING | PHASE1/PHASE2 |
| NCT07358078 |
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting |
RECRUITING | |
| NCT07295717 |
A Study to Evaluate ALN-4285 in Adult Healthy Volunteers |
RECRUITING | PHASE1 |
| NCT07223203 |
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy |
RECRUITING | PHASE3 |
| NCT07214727 |
A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease |
RECRUITING | PHASE1 |
| NCT07181109 |
Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease |
RECRUITING | PHASE3 |
