Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a biopharmaceutical company focusing on the discovery, development, and commercialization of RNAi therapeutics for treating rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS) diseases that is headquartered in Boston, Massachusetts and was founded in 2002 by David Bartel, John Clarke, Paul Schimmel, Phillip D. Zamore, Phillip Sharp, Robert Langer, and Thomas Tuschl.

RNAi Therapeutics - How this New Class of Medicines Works.

Products

ONPATTRO

ONPATTRO (patisiran) is a prescription medicine for treating polyneuropathy in adults caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). The most common side effects of patients taking ONPATTRO are respitory infections (colds, sinus infections, nasal congestion, and infusion-related reactions). ONPATTRO is known to lower vitamin A levels circulating in the blood. It is made to decrease the amount of TTR protein created in the liver. TTR protein levels were shown to decrease by 84% on average in patients taking ONPATTRO for 18 months.

GIVLAARI

GIVLAARI (givosiran) prescription medicine for treating adult acute hepatic porphyria (AHP). The most common side effects of adults taking GIVLAARI include injection site reactions and nausea. GIVLAARI reduces the amount of ALAs1 present in the liver which leads to the lowering of ALA and PBG neurotoxins associated with the AHP symptoms and attacks. A six month study showed that patients treated with GIVLAARI hadreduced days of hemin and a 70% reduction in AHP attacks (average of 1.9 AHP attacks) compared to the placebo group (average of 6.5 AHP attacks) not receiving GIVLAARI.

Product Pipeline

Fitusiran

Fitusiran (ALN-AT3) is a RNAi therapeutic for treating hemophilia and rare bleeding disorders (RBDs). It uses the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform and is delivered subcutaneously to target antithrombin (AT). Sanofi Genzyme is helping Alnylam Pharmaceuticals is the development of Fitusiran.

Inclisiran

Inclisiran (ALN-PCSsc) is a RNAi therapeutic for treating hypercholesterolemia. It uses the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform and is delivered subcutaneously to target proprotein convertase subtilisin kexin type 9 (PCSK9). Novartis is working with Alnylam Pharmaceuticals to develop Inclisiran.

Lumasiran

Lumasiran (ALN-GO1) is a RNAi therapeutic for treating Primary Hyperoxaluria Type 1 (PH1). It uses the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform and is delivered subcutaneously to target glycolate oxidase (GO).

Vutrisiran

Vutrisiran (SLN-TTRsc02) is a RNAi therapeutic for treating transthyretin-mediated (ATTR) amyloidosis. It uses the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform and is delivered subcutaneously to target transthyretin (TTR).

Cemdisiran

Cemdisiran (ALN-CC5) is a RNAi therapeutic for treating complement-mediated diseases. It uses the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform and is administered subcutaneously for targeting the C5 component of the complement pathway.

ALN-AAT02

ALN-AAT02 is a RNAi therapeutic for treating AAT deficiency-associated liver disease (alpha-1 liver disease). It uses the company's Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform and is delivered subcutaneously to target alpha-1 antitrypsin (AAT).

ALN-HBV02

ALN-HBV02 is a RNAi therapeutic for treating chronic hepatitis B virus infection. It uses the company's Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform and is delivered subcutaneously for targeting the hepatitis B virus (HBV) genome. ALN-HBV02 is made to inhibit hepatitis B surface antigen (HBsAg) and the expression of all other HBV proteins. Vir Biotechnology is working with Alnylam Pharmaceuticals to develop ALN-HBV02.

ALN-AGT

ALN-AGT is a RNAi therapeutic for treating hypertension. Is uses the company's Enhanced Stabilization Chemistry Plus (ESC+)-GalNAc delivery platform and is delivered subcutaneously to target angiotensinogen (AGT).

ALN-HSD

ALN-HSD is a RNAi therapeutic for treating non-alcoholic steatohepatitis (NASH). It uses the company's Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform and is delivered subcutaneously to target HSD17B13. Regeneron is working with Alnylam Pharmaceuticals in the development of ALN-HSD.

ALN-COV

ALN-COV is a RNAi therapeutic for treating and preventing COVID-19. It uses a novel conjugate lung delivery system in combination with the company's Enhanced Stabilization Chemistry (ESC) delivery platform and it delivered through inhalation to target the SARS-COV-2 viral genome. Vir Biotechnology is working with Alnylam Pharmaceuticals in the development of ALN-COV.

Awards

Innovative Growth Companies 2016

Alnylam Pharmaceuticals was named #100 on the Forbes Innovative Growth Companies 2016 list. The company did not make the Forbes Innovative Growth Companies 2017 list.