Alnylam Pharmaceuticals Inc. (A1LN34)

Overview

Alnylam Pharmaceuticals, Inc. is a leading biopharmaceutical company that specializes in discovering, developing, and commercializing innovative therapeutics based on ribonucleic acid interference (RNAi). Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam is at the forefront of RNAi technology, employing this revolutionary science to tackle complex diseases at the genetic level. The company's commitment to innovation is underscored by its dynamic pipeline of products and its strategic collaborations with major players in the pharmaceutical industry, such as Regeneron Pharmaceuticals, Inc., Roche, Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. These partnerships aim to expand the potential of RNAi therapy across various diseases, including those affecting the eye, central nervous system (CNS), and more.

Products and Services

• ONPATTRO (patisiran)

  Approved for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults, this product exemplifies Alnylam's commitment to addressing rare diseases with significant unmet needs.

• AMVUTTRA

  Also targeting hATTR amyloidosis, AMVUTTRA is specifically approved for treating adults with polyneuropathy, showcasing the company's focused approach on this genetic disorder.

• GIVLAARI (givosiran)

  For adults with acute hepatic porphyria (AHP), a rare metabolic disorder, GIVLAARI represents a significant advancement in treatment options, highlighting Alnylam's dedication to tackling challenging hepatic conditions.

• OXLUMO (lumasiran)

  This product is designed for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder, further demonstrating Alnylam's expertise in developing treatments for rare conditions.

• Development Pipeline
  • Patisiran - in development for ATTR amyloidosis with cardiomyopathy.
  • Cemdisiran - targeted to treat complement-mediated diseases.
  • Belcesiran - for the treatment of alpha-1 liver disease.
  • Elebsiran - aimed at chronic HBV infection treatment.
  • Zilebesiran - under development to treat hypertension, in collaboration with Roche.
  • ALN-APP - for Alzheimer's disease and cerebral amyloid angiopathy.
  • ALN-HSD - targeted to treat NASH.
  • Fitusiran - for the treatment of hemophilia, showcasing Alnylam's expansion into blood disorders.
  • Inclisiran - aimed at treating hypercholesterolemia, highlighting Alnylam's foray into cardiovascular diseases.
  • Vutrisiran - in phase 3 clinical trial for ATTR amyloidosis, underscoring ongoing commitment to this disease area.