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Alnylam Pharmaceuticals

Alnylam Pharmaceuticals

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.

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.

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.

Timeline

April 13, 2020
Alnylam Pharmaceuticals raises a $750,000,000 private equity from GSO Capital Partners.
April 13, 2020
Alnylam Pharmaceuticals raises a $100,000,000 private equity from Blackstone Life Sciences.
April 8, 2019
Alnylam Pharmaceuticals raises a $800,000 private equity from Regeneron Pharmaceuticals.
March 26, 2018
Alnylam Pharmaceuticals was founded by David Bartel, John Clarke, Paul Schimmel, Phillip D. Zamore, Phillip Sharp, Robert Langer and Thomas Tuschl.
January 13, 2014
Alnylam Pharmaceuticals raises a $700,000 private equity from Genzyme.
July 7, 2003
Alnylam Pharmaceuticals raises a $24,600,000 venture round from ARCH Venture Partners, Abingworth, Cardinal Partners and Merck Group.

Funding Rounds

SBIR/STTR Awards

Patents

Further Resources

Title
Author
Link
Type
Date

2020: Extraordinary Year, Extraordinary People

Web

January 25, 2021

Alnylam Challengers - A Community Impact Program to Improve Health Access in Underserved Communities

Web

October 13, 2021

News

Title
Author
Date
Publisher
Description
Kyle LaHucik
August 3, 2021
FierceBiotech
The pharma said it was ending ALN-COV, its RNAi antiviral being developed for the treatment of SARS-CoV-2, because of the number of effective vaccines and alternative treatments available. Alnylam and partner Vir Biotechnology had said they would start testing patients by the end of last year.
Alnylam Pharmaceuticals
June 7, 2021
www.prnewswire.com:443
/PRNewswire/ -- A Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), empresa líder em terapias de RNAi, anunciou hoje que a Agência Nacional de Vigilância Sanitária...
Alex Keown
May 14, 2021
BioSpace
Biopharma and life sciences companies strengthen their leadership teams and boards with Movers & Shakers.
May 3, 2021
BioSpace
Alnylam Announces Positive Early Results on Clinical Outcome Measures from ILLUMINATE-A Phase 3 Study of OXLUMO® (lumasiran) - read this article along with other careers information, tips and advice on BioSpace
BioSpace
April 27, 2021
BioSpace
Immuneering Corporation ("Immuneering") today announced the addition of Laurie Keating to its board of directors.
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References

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