Capella

Capella—the Online Voice of Progress in RNAi

Arian Ebank welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Arian Ebank, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.

To view our corporate presentation, click here.

We presented additional pre-clinical data further elucidating fitusiran’s novel mechanism of action at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany.

Presentation on the development of a PK-PD model of fitusiran

Presentation on measurement of thrombin generation in conditions of low antithrombin

View the presentation on the use of bypassing agents in the context of antithrombin reduction

 

We presented pre-clinical data with ALN-F12 at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany. Data showed that reduction of hepatic factor XII (FXII) expression in mice by ALN-F12 inhibits thrombosis without increasing bleeding risk. We believe that reduction of plasma FXII by ALN-F12 represents a promising approach for the prophylactic treatment of thrombosis.

View the presentation

(more…)

We reported new positive results from our ongoing Phase 2 open-label extension (OLE) study with fitusiran in patients with hemophilia A and B, with or without inhibitors, at the International Society of Thrombosis and Haemostasis (ISTH) Congress, held July 8 – 13, 2017 in Berlin, Germany.

View the complete Phase 2 OLE data presentation

(more…)

We reported interim results from our ongoing Phase 1 study with givosiran at the 2017 International Congress on Porphyrins and Porphyrias (ICPP), held June 25 – 28, 2017 in Bordeaux, France. Data presented were from the first three unblinded cohorts from Part C, in patients with acute intermittent porphyria (AIP) that experience recurrent attacks and initial data from the open-label extension (OLE) study.


View the complete Phase 1 interim and OLE data presentation
View the results from the EXPLORE natural history study
View the poster on healthcare utilization and costs
View the poster on disease burden in patients with AIP and recurrent attacks
(more…)

We presented data evaluating the measurement properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with symptomatic hereditary ATTR (hATTR) amyloidosis with polyneuropathy. R-ODS is a 24-item patient-reported outcome instrument that assesses activity and social participation limitation. R-ODS assessments were collected from two trials investigating patisiran. These data were presented at the International Society for Pharmacoeconomics and Outcomes Research annual meeting in Boston, Massachusetts.


(more…)

We reported final 24-month results from our Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis. These data were presented at the American Academy of Neurology (AAN) 2017 Annual Meeting in Boston, Massachusetts.



(more…)

SIGN UP FOR PATIENT CONNECT

Receive updates on our investigational therapies and clinical trials.