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Capella

Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi toward innovative medicines.

Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

September 16, 2014

Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

We presented pre-clinical data with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 25th International Complement Workshop held September 14 – 18, 2014, in Rio de Janeiro, Brazil. Data demonstrated potent and clamped C5 knockdown as well as robust inhibition of complement activity in non-human primates for up to 100 days with a subcutaneous, monthly dosing regimen. Further, in a rat model of membranous nephropathy, ALN-CC5 administration resulted in a significant reduction in proteinuria due to complement-mediated disease activity in the kidney.



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Pre-Clinical Data with ALN-AGT for Treatment of Hypertensive Disorders of Pregnancy, Including Preeclampsia

September 12, 2014

Pre-Clinical Data with ALN-AGT for Treatment of Hypertensive Disorders of Pregnancy, Including Preeclampsia

We announced a new program, ALN-AGT, an RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertensive disorders of pregnancy (HDP), including preeclampsia. Pre-clinical data were presented at the American Heart Association’s High Blood Pressure Research 2014 Scientific Sessions, held September 9 – 12, 2014, in San Francisco. Data from experiments in an established rat model of preeclampsia demonstrated that an RNAi therapeutic targeting AGT ameliorates the clinical sequelae of preeclampsia and improves outcomes for the fetus.  This treatment approach has the potential for selective delivery to the pregnant mother without fetal drug exposure, as our study confirmed undetectable siRNA levels in the fetus.




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August 21, 2014

Summer RNAi Roundtable Series

We hosted a series of online “RNAi Roundtables” during July and August, at which Alnylam scientists and key clinical collaborators reviewed recent progress from several “Alnylam 5×15” programs and discussed the related disease areas.

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RNAi Roundtable: ALN-AS1 for the treatment of Hepatic Porphyrias

August 21, 2014

RNAi Roundtable: ALN-AS1 for the treatment of Hepatic Porphyrias

On August 21, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-AS1 in development for the treatment of hepatic porphyrias.




RNAi Roundtable: ALN-AAT for the treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

August 20, 2014

RNAi Roundtable: ALN-AAT for the treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

On August 20, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-AAT in development for the treatment of alpha-1 antitrypsin deficiency-associated liver disease.




RNAi Roundtable: ALN-PCSsc for the Treatment of Hypercholesterolemia

August 14, 2014

RNAi Roundtable: ALN-PCSsc for the Treatment of Hypercholesterolemia

On August 14, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-PCSsc in development for the treatment of hypercholesterolemia.




RNAi Roundtable: ALN-CC5 for the treatment of Complement-Mediated Diseases

August 13, 2014

RNAi Roundtable: ALN-CC5 for the treatment of Complement-Mediated Diseases

On August 13, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-CC5 in development for the treatment of Complement-Mediated Diseases.




RNAi Roundtable: ALN-AT3 for the treatment of Hemophilia and Rare Bleeding Disorders

August 7, 2014

RNAi Roundtable: ALN-AT3 for the treatment of Hemophilia and Rare Bleeding Disorders

On August 7, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-AT3 in development for the treatment of Hemophilia and Rare Bleeding Disorders.




RNAi Roundtable: ALN-HBV for the treatment of Hepatitis B Virus (HBV) Infection

July 31, 2014

RNAi Roundtable: ALN-HBV for the treatment of Hepatitis B Virus (HBV) Infection

On July 29, 2014, we hosted an online RNAi Roundtable to review the progress with ALN-HBV in development for the treatment of Hepatitis B Virus (HBV) infection.




RNAi Roundtable: Advances in Delivery of RNAi Therapeutics with Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA Conjugates

July 22, 2014

RNAi Roundtable: Advances in Delivery of RNAi Therapeutics with Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA Conjugates

On July 22, 2014, we hosted an online RNAi Roundtable to discuss advances in delivery of RNAi therapeutics with Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugates.