Alnylam Pharmaceuticals
Alnylam is a pioneering biopharmaceutical company specializing in RNA interference (RNAi) therapeutics, a revolutionary approach to treating diseases by silencing the genes that cause or contribute to them. Founded in 2002, Alnylam has developed a robust pipeline of investigational medicines focused on rare and prevalent diseases, with several approved therapies. The company is committed to scientific innovation, patient-centric development, and making a positive impact on global health.
Industries
Nr. of Employees
Very Large (1000+)
Alnylam Pharmaceuticals
Cambridge, Massachusetts, United States, North America
Products
AMVUTTRA (vutrisiran)
An siRNA-based therapeutic approved for transthyretin-mediated amyloidosis indications.
ONPATTRO (patisiran)
An siRNA therapeutic approved for hereditary transthyretin-mediated amyloidosis with polyneuropathy.
GIVLAARI (givosiran)
An siRNA therapeutic approved for the treatment of acute hepatic porphyria.
OXLUMO (lumasiran)
An siRNA therapeutic approved for primary hyperoxaluria type 1.
Qfitlia (fitusiran)
An RNA-directed therapeutic indicated for hemophilia A or B, commercialized via partnership/licensing arrangements.
Leqvio (inclisiran) — licensed program
A cholesterol-lowering RNAi therapeutic program referenced as out-licensed and commercialized by a partner.
AMVUTTRA (vutrisiran)
An siRNA-based therapeutic approved for transthyretin-mediated amyloidosis indications.
ONPATTRO (patisiran)
An siRNA therapeutic approved for hereditary transthyretin-mediated amyloidosis with polyneuropathy.
GIVLAARI (givosiran)
An siRNA therapeutic approved for the treatment of acute hepatic porphyria.
OXLUMO (lumasiran)
An siRNA therapeutic approved for primary hyperoxaluria type 1.
Qfitlia (fitusiran)
An RNA-directed therapeutic indicated for hemophilia A or B, commercialized via partnership/licensing arrangements.
Leqvio (inclisiran) — licensed program
A cholesterol-lowering RNAi therapeutic program referenced as out-licensed and commercialized by a partner.
Services
Patient support programs
Personalized assistance and support services for individuals receiving therapies, including counseling, treatment navigation and a U.S.-specific support platform.
Medical affairs and medical information
Provision of clinical and scientific information, investigator support and facilitation of genetic testing requests for select indications.
Medical publication support
Review and support process for unsolicited publication requests via a submission portal, with compliance tracking for transfers of value.
Expanded access consideration
Case-by-case review process to evaluate pre-approval access to investigational medicines for individual patients when trial enrollment is not possible.
Patient and HCP educational resources (media kit)
Provision of downloadable disease fact sheets, educational booklets, media kit assets and patient access reports for clinicians, payers and the public.
Patient support programs
Personalized assistance and support services for individuals receiving therapies, including counseling, treatment navigation and a U.S.-specific support platform.
Medical affairs and medical information
Provision of clinical and scientific information, investigator support and facilitation of genetic testing requests for select indications.
Medical publication support
Review and support process for unsolicited publication requests via a submission portal, with compliance tracking for transfers of value.
Expanded access consideration
Case-by-case review process to evaluate pre-approval access to investigational medicines for individual patients when trial enrollment is not possible.
Patient and HCP educational resources (media kit)
Provision of downloadable disease fact sheets, educational booklets, media kit assets and patient access reports for clinicians, payers and the public.
Expertise Areas
- RNAi and siRNA therapeutic development
- Ligand-conjugate oligonucleotide delivery strategies
- CNS-targeted oligonucleotide approaches
- Clinical trial design and operations (Phase 1–3)
Key Technologies
- RNA interference (RNAi)
- Small interfering RNA (siRNA) therapeutics
- Ligand-conjugate oligonucleotide delivery (e.g., GalNAc)
- CNS-directed oligonucleotide delivery methods