Pipeline

Click on bar or (i) in the in pipeline for more information
Alnylam 5x15 Programs
Licensed programs ABOUT OUR PARTNERS
Partner Programs
Development Programs
Programs Discovery Development Phase I Phase II Phase III
TTR Amyloidosis (ALN-TTR02)
 
TTR Amyloidosis (ALN-TTRsc)
 
Hemophilia
 
Acute Intermittent Porphyria
 
Hypercholesterolemia
 
Beta-thalassemia and Iron-overload disorders
 
Alpha-1-Antitrypsin Deficiency
 
 
Liver Cancers
 
 
RSV Infection
 
Licensed Programs
Programs Discovery Development Phase I Phase II Phase III
 
Diabetic Macular Edema
 
 
Acute Kidney Injury
 
 
Solid Tumors
 
 
Hypercholesterolemia
 
 
Solid Tumors
 
 
Hepatitis C
 
 
JC Virus / PML
 
 
Novartis Programs
 
 
Oncology, Metabolic
 
 
Immuno-Inflammatory
 
 
Heart Failure/Fibrosis
 
 
Solid Tumors
 
 
Solid Tumors
 
 
Malaria
 
Alnylam 5x15 Programs
Licensed programs ABOUT OUR PARTNERS
Partner Programs
TTR Amyloidosis (ALN-TTR02)
The ALN-TTR02 Phase II study is an open-label, multi-center, multi-dose, dose-escalation trial. Primary objectives are to evaluate safety and tolerability of multiple doses of ALN-TTR02 and to measure clinical activity.
TTR Amyloidosis (ALN-TTRsc)
ALN-TTRsc is a subcutaneously administered RNAi therapeutic for the treatment of ATTR,
Hemophilia
ALN-AT3 is an RNAi therapeutic for the treatment of hemophilia, an inherited, orphan, bleeding disorder.
Acute Intermittent Porphyria
ALN-AS1 is a subcutaneous RNAi therapeutic targeting aminolevulinate synthase 1 (ALAS-1) for the treatment of acute intermittent porphyria (AIP), an ultra-rare genetic disease.
Hypercholesterolemia
ALN-PCS is an RNAi therapeutic, being developed to treat severe hypercholesterolemia, or high levels of cholesterol in the blood.
Beta-thalassemia and Iron-overload disorders
ALN-TMP, an RNAi therapeutic targeting Tmprss6 for the for the treatment of beta-thalassemia and iron-overload disorders.
Alpha-1-Antitrypsin Deficiency
ALN-AAT is an RNAi therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency.
Liver Cancers
We have advanced our RNAi therapeutic, ALN-VSP, into the clinic for the treatment of liver cancers and potentially other solid tumors with liver involvement. The Phase I study showed that ALN-VSP was generally well tolerated.
RSV Infection
The Phase IIb trial is a multi-center, global, randomized, double-blind, placebo-controlled study in RSV-infected lung transplant patients with a primary endpoint of a reduction in incidence of new or progressive BOS.
Diabetic Macular Edema
Quark received an InterfeRX license to develop RNAi therapeutics targeting RTP801, a gene implicated in ocular diseases. The RTP801 program is a partnership between Quark and Pfizer.
Acute Kidney Injury
Quark received an InterfeRx license to develop RNAi therapeutics targeting the p53 gene for renal failure.
Solid Tumors
Calando received an InterfeRx license to develop RNAi therapeutics for RRM2, a cancer target.
Hypercholesterolemia
Tekmira received an InterfeRx license to develop RNAi therapetics targeting the apoB gene for the treatment of hypercholesterolemia.
Solid Tumors
Tekmira received an InterfeRx license to develop RNAi therapetics targeting the PLK1 gene, a cancer target.
Hepatitis C
Regulus is developing a microRNA therapeutic targeting miR-122 for the treatment of HCV infection.
JC Virus / PML
PML is caused by infection of the central nervous system with a virus called "JC virus" and can occur in certain immune-suppressed patients, including those receiving immunomodulatory therapies.
Novartis Programs
Alnylam and Novartis Institute for BioMedical Research (NIBR) will discover, develop and commercialize RNAi therapeutics across multiple therapeutic areas in Novartis' research portfolio.
Oncology, Metabolic
Takeda is developing RNAi therapeutics for oncology and metabolic disease targets.
Immuno-Inflammatory
GSK and Regulus Therapeutics formed the first ever microRNA-focused strategic alliance to develop microRNA-targeted therapeutics.
Heart Failure/Fibrosis
Regulus is researching the role of microRNA, known as miR-21, in heart failure and fibrosis.
Solid Tumors
Calando received an InterfeRx license for the HIF-2α cancer gene target.
Solid Tumors
GeneCare received an InterfeRx license to develop RNAi therapeutics directed against two DNA helicase genes associated with cancer.
Malaria
Cenix is researching potential RNAi therapeutics for the silencing of multiple disease targets for the treatment of malaria infection.
The ALN-VSP program is partnered with Ascletis for HCC in China (including Hong Kong, Macau, and Taiwan)
The ALN-RSV program is partnered in Asia with Kyowa Hakko Kirin. We plan to seek a partner to continue to advance the program in other regions.
Quark received an InterfeRX license to develop RNAi therapeutics targeting RTP801, a gene implicated in ocular diseases. The RTP801 program is a partnership between Quark and Pfizer.
Quark received an InterfeRx license to develop RNAi therapeutics targeting the p53 gene for renal failure.
Calando received an InterfeRx license to develop RNAi therapeutics for RRM2, a cancer target.
Tekmira received an InterfeRx license to develop RNAi therapetics targeting the apoB gene for the treatment of hypercholesterolemia.
Tekmira received an InterfeRx license to develop RNAi therapetics targeting the PLK1 gene, a cancer target.
Regulus is developing a microRNA therapeutic targeting miR-122 for the treatment of HCV infection.
Alnylam and Biogen Idec will initially conduct investigative research into the potential of using RNAi technology to develop therapeutics to treat PML.
Alnylam and Novartis Institute for BioMedical Research (NIBR) will discover, develop and commercialize RNAi therapeutics across multiple therapeutic areas in Novartis' research portfolio.
Takeda is developing RNAi therapeutics for oncology and metabolic disease targets.
GSK and Regulus Therapeutics formed the first ever microRNA-focused strategic alliance to develop microRNA-targeted therapeutics.
Regulus is researching the role of microRNA, known as miR-21, in heart failure and fibrosis.
Calando received an InterfeRx license for the HIF-2α cancer gene target.
GeneCare received an InterfeRx license to develop RNAi therapeutics directed against two DNA helicase genes associated with cancer.
Cenix is researching potential RNAi therapeutics for the silencing of multiple disease targets for the treatment of malaria infection.