Pipeline

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.

ALN-TTRsc is a subcutaneously administered RNAi therapeutic for the treatment of ATTR,

ALN-AT3 is an RNAi therapeutic for the treatment of hemophilia, an inherited, orphan, bleeding disorder.

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.

ALN-PCS is an RNAi therapeutic, being developed to treat severe hypercholesterolemia, or high levels of cholesterol in the blood.

ALN-TMP, an RNAi therapeutic targeting Tmprss6 for the for the treatment of beta-thalassemia and iron-overload disorders.

ALN-AAT is an RNAi therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency.

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.

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.

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.

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.

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.