Alnylam Pharmaceuticals
Biopharmaceutical company focused on discovery, development, manufacturing and commercialization of RNA interference (RNAi) therapeutics based on small interfering RNA (siRNA). Core activities include siRNA design and delivery platform development, oligonucleotide manufacturing and technical operations, clinical development and trial support, genetics-driven target identification, and patient access and support programs.
Industries
Nr. of Employees
Very Large (1000+)
Alnylam Pharmaceuticals
Patents
Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
US-12723247-B2
View DetailsMannan binding lectin serine peptidase 2 (MASP2) IRNA compositions and methods of use thereof
US-12709751-B2
View DetailsSerpin family F member 2 (SERPINF2) iRNA compositions and methods of use thereof
US-12692494-B2
View DetailsComplement component C3 iRNA compositions and methods of use thereof for treating or preventing complement component C3-associated diseases
US-12680099-B2
View Details
Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
US-12723247-B2
View DetailsMannan binding lectin serine peptidase 2 (MASP2) IRNA compositions and methods of use thereof
US-12709751-B2
View DetailsSerpin family F member 2 (SERPINF2) iRNA compositions and methods of use thereof
US-12692494-B2
View DetailsComplement component C3 iRNA compositions and methods of use thereof for treating or preventing complement component C3-associated diseases
US-12680099-B2
View DetailsProducts
Commercial RNAi therapeutics (approved siRNA medicines)
Marketed oligonucleotide therapies developed using RNAi mechanisms, targeting diseases such as transthyretin-mediated amyloidosis, acute hepatic porphyria, primary hyperoxaluria and certain cardiometabolic conditions.
OXLUMO
OXLUMO (lumasiran) is a HAO1-directed small interfering ribonucleic acid (siRNA) injection indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels.
GIVLAARI
GalNAc-conjugated siRNA therapeutic (givosiran) administered subcutaneously for targeted delivery to the liver.
AMVUTTRA
GalNAc-conjugated siRNA therapeutic (vutrisiran) administered subcutaneously for targeted delivery to the liver.
Vutrisiran
An investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, administered subcutaneously every three months.
ONPATTRO
Our first approved RNAi therapeutic, patisiran, utilizes LNP-based delivery and is administered intravenously (IV).
Commercial RNAi therapeutics (approved siRNA medicines)
Marketed oligonucleotide therapies developed using RNAi mechanisms, targeting diseases such as transthyretin-mediated amyloidosis, acute hepatic porphyria, primary hyperoxaluria and certain cardiometabolic conditions.
OXLUMO
OXLUMO (lumasiran) is a HAO1-directed small interfering ribonucleic acid (siRNA) injection indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels.
GIVLAARI
GalNAc-conjugated siRNA therapeutic (givosiran) administered subcutaneously for targeted delivery to the liver.
AMVUTTRA
GalNAc-conjugated siRNA therapeutic (vutrisiran) administered subcutaneously for targeted delivery to the liver.
Vutrisiran
An investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, administered subcutaneously every three months.
ONPATTRO
Our first approved RNAi therapeutic, patisiran, utilizes LNP-based delivery and is administered intravenously (IV).
Services
Personalized patient support covering insurance navigation, financial assistance options, education resources, and care navigation.
Provision of no-charge genetic testing and counseling through independent third-party laboratories and counseling providers for eligible patients in select geographies.
Consideration of support for unsolicited publication requests from healthcare professionals via a submission and review platform, aligned with local laws and good publication practice.
In-house manufacturing of oligonucleotide drug substance and coordination of finish/fill and distribution for clinical trials and commercial supply, with external partnerships for scaling.
Evaluation and administration of requests for access to investigational medicines outside clinical trials in accordance with published compassionate use policy.
Personalized patient support covering insurance navigation, financial assistance options, education resources, and care navigation.
Provision of no-charge genetic testing and counseling through independent third-party laboratories and counseling providers for eligible patients in select geographies.
Consideration of support for unsolicited publication requests from healthcare professionals via a submission and review platform, aligned with local laws and good publication practice.
In-house manufacturing of oligonucleotide drug substance and coordination of finish/fill and distribution for clinical trials and commercial supply, with external partnerships for scaling.
Evaluation and administration of requests for access to investigational medicines outside clinical trials in accordance with published compassionate use policy.
Expertise Areas
- RNAi and siRNA therapeutics development
- Oligonucleotide delivery platform engineering
- Oligonucleotide chemical manufacturing and scale-up
- Clinical development and trial operations
Key Technologies
- RNA interference (RNAi)
- Small interfering RNA (siRNA) therapeutics
- Receptor-targeted conjugate delivery
- Lipid nanoparticle delivery systems
News & Updates
$250 million investment to expand manufacturing capacity and add enzymatic ligation capabilities at the U.S. site.
Annual report reflecting company progress and strategic outlook for 2025.
Report detailing progress against corporate responsibility goals and data aligned to GRI and SASB frameworks.
Recognition by Fast Company for innovation (noted as third time).
Regulatory acceptance of an enzymatic-ligation-based oligonucleotide manufacturing platform into an emerging technology program to facilitate engagement.
$250 million investment to expand manufacturing capacity and add enzymatic ligation capabilities at the U.S. site.
Annual report reflecting company progress and strategic outlook for 2025.
Report detailing progress against corporate responsibility goals and data aligned to GRI and SASB frameworks.
Recognition by Fast Company for innovation (noted as third time).
Regulatory acceptance of an enzymatic-ligation-based oligonucleotide manufacturing platform into an emerging technology program to facilitate engagement.