Pipeline
Click on bar or (i) in the in pipeline for more information
| Our Proprietary Programs | Co-Developed Programs | |||
| Partnered Programs | Joint Ventures |
- Development Pipeline
-
Programs Discovery Development Phase I Phase II Phase III RSV InfectionLiver Cancers PCSK9/Hypercholesterolemia TTR Amyloidosis Huntington's Disease - Partnered Programs
-
Programs Discovery Development Phase I Phase II Phase III Diabetic Macular EdemaAcute Kidney InjurySolid TumorsHypercholesterolemiaSolid TumorsHepatitis COncology, Metabolic, Respiratory, LiverPandemic InfluenzaJC Virus / PMLOncology, MetabolicImmuno-InflammatoryHeart Failure/FibrosisSolid TumorsSolid TumorsMalaria - Collaborations
-
Programs Discovery Development Phase I Phase II Phase III Parkinson's DiseaseHepatic SteatosisDiabetes/ObesityMultiple Therapeutic AreasUveal MelanomaEbolaSolid TumorsDystoniaAsthmaHerpesOvarian Cancer
| Our Proprietary Programs | Co-Developed Programs | |||
| Partnered Programs | Geographically Partnered Programs |
RSV Infection
In April 2008, Phase II clinical trial was initiated to assess the safety and tolerability of aerosolized ALN-RSV01 versus placebo in adult lung transplant patients naturally infected with RSV.
Liver Cancers
ALN-VSP is an RNAi therapeutic being developed for the treatment of liver cancers and potentially other solid tumors. This program is an IND candidate for 2008.
PCSK9/Hypercholesterolemia
ALN-PCS is an RNAi therapeutic, being developed to treat hypercholesterolemia, or high levels of cholesterol in the blood. ALN-PCS is a 2009 IND candidate.
TTR Amyloidosis
ALN-TTR, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR amyloidosis. ALN-TTR is a 2009 IND candidate.
Huntington's Disease
ALN-HTT is designed to silence the huntingtin gene, which is responsible for the expression of a toxic mutated protein, ultimately causing the disease. ALN-HTT is a 2009 IND candidate.
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.
Oncology, Metabolic, Respiratory, Liver
Roche is developing RNAi therapeutics for multiple targets implicated in oncology, metabolic, respiratory, and liver diseases.
Pandemic Influenza
Novartis is working to advance RNAi therapeutics targeting the influenza genome for the treatment of pandemic flu.
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.
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.
Parkinson's Disease
The Michael J. Fox Foundation awarded Alnylam and scientists at the Mayo Clinic a grant to research potential RNAi therapeutics targeting α-Synuclein, a protein implicated in the development of Parkinson's disease.
Hepatic Steatosis
Scientists at the UTSW are researching new approaches for reducing LDL-cholesterol levels using RNAi therapeutics targeting the PCSK9 gene, a gene involved in the metabolism of LDL.
Diabetes/Obesity
The UTSW was awarded a "Roadmap Grant" from the National Institutes of Health (NIH) to research disease targets involved in the cause or pathway of obesity.
Multiple Therapeutic Areas
Scientists at the UTSW are researching RNAa, a biological discovery that may have applications in many diseases, including certain genetic disorders and cancer.
Uveal Melanoma
Scientists at the UCSF are researching RNAi therapeutics targeting GNAQ for the treatment of metastatic uveal melanoma.
Ebola
The NIAID, a division of the National Institutes of Health (NIH) awarded Alnylam a $23M contract to develop an RNAi anti-viral therapeutic against the Ebola virus.
Solid Tumors
Scientists at the Universitat Bonn are researching "3p-siRNAs," a new class of RNAi therapeutics that could potentially be highly effective in treating cancer.
Dystonia
Scientists from Massachusetts General Hospital (MGH) are researching potential RNAi therapeutics for the silencing of a key gene implicated in early onset torsion dystonia, known as mutant torsinA.
Asthma
Scientists at Yale University are researching RNAi therapeutics for angiopoietin 2, a disease target associated with acute lung injury.
Herpes
The Immune Disease Institute (IDI) is researching RNAi therapeutics targeting the HSV-2 gene and the cell surface receptor nectin-1, both implicated in the transmission of Herpes Simplex Virus-2 (HSV-2).
Ovarian Cancer
Scientists at MIT are researching RNAi therapeutics for silencing claudin-3, a tight junction protein that is highly over-expressed in approximately 90% of ovarian tumors.
The ALN-RSV program is partnered in Asia with Kyowa Hakko Kirin and in the rest of the world with Cubist Pharmaceuticals
Being developed in collaboration with Medtronic, where we aim to develop a drug-device combination product.
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.
Roche is developing RNAi therapeutics for multiple targets implicated in oncology, metabolic, respiratory, and liver diseases.
Novartis is working to advance RNAi therapeutics targeting the influenza genome for the treatment of pandemic flu.
Alnylam and Biogen Idec will initially conduct investigative research into the potential of using RNAi technology to develop therapeutics to treat PML.
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.
The Michael J. Fox Foundation awarded Alnylam and scientists at the Mayo Clinic a grant to research potential RNAi therapeutics targeting α-Synuclein, a protein implicated in the development of Parkinson's disease.
Scientists at the UTSW are researching new approaches for reducing LDL-cholesterol levels using RNAi therapeutics targeting the PCSK9 gene, a gene involved in the metabolism of LDL.
The UTSW was awarded a "Roadmap Grant" from the National Institutes of Health (NIH) to research disease targets involved in the cause or pathway of obesity.
Scientists at the UTSW are researching RNAa, a biological discovery that may have applications in many diseases, including certain genetic disorders and cancer.
Scientists at the UCSF are researching RNAi therapeutics targeting GNAQ for the treatment of metastatic uveal melanoma.
The NIAID, a division of the National Institutes of Health (NIH) awarded Alnylam a $23M contract to develop an RNAi anti-viral therapeutic against the Ebola virus.
Scientists at the Universitat Bonn are researching "3p-siRNAs," a new class of RNAi therapeutics that could potentially be highly effective in treating cancer.
Scientists from Massachusetts General Hospital (MGH) are researching potential RNAi therapeutics for the silencing of a key gene implicated in early onset torsion dystonia, known as mutant torsinA.
Scientists at Yale University are researching RNAi therapeutics for angiopoietin 2, a disease target associated with acute lung injury.
The Immune Disease Institute (IDI) is researching RNAi therapeutics targeting the HSV-2 gene and the cell surface receptor nectin-1, both implicated in the transmission of Herpes Simplex Virus-2 (HSV-2).
Scientists at MIT are researching RNAi therapeutics for silencing claudin-3, a tight junction protein that is highly over-expressed in approximately 90% of ovarian tumors.

