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Alnylam

Capella

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

August 20, 2014

Summer RNAi Roundtable Series

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

Next RNAi Roundtable:

ALN-AS1 for the treatment of Hepatic Porphyrias
Thursday, August 21: 4:00 – 5:00 p.m. ET

  • Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development
  • Moderator: Barry Greene, President and Chief Operating Officer
  • Guest Speaker: Karl Anderson, M.D., FACP, Professor, Departments of Preventive Medicine and Community Health (Division of Human Nutrition) and Internal Medicine (Division of Gastroenterology), and Director, Porphyria Laboratory & Center at the University of Texas Medical Branch

To register and access the webcast for this event, please click here.

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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.




RNAi Roundtable: Patisiran and ALN-TTRsc for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR)

July 15, 2014

RNAi Roundtable: Patisiran and ALN-TTRsc for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR)

On July 15, 2014, we hosted an online RNAi Roundtable to review the progress with patisiran and ALN-TTRsc in development for the treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR).




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

June 9, 2014

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

We presented pre-clinical data with our Development Candidate for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 7th International Conference on Complement Therapeutics, held June 6 – 11, 2014, in Olympia, Greece.  These data demonstrate that ALN-CC5 led to an up to 98.7% knockdown of serum C5 and an up to 96.8% inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration.



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Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

May 21, 2014

Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

We have published pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias in the Proceedings of the National Academy of Sciences (PNAS). The new paper highlights work that was done in collaboration with researchers at the Icahn School of Medicine at Mount Sinai in New York City. Data from studies performed in a mouse model of acute intermittent porphyria (AIP) demonstrate that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid, and durable suppression of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP.



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