We are hosting a series of online “RNAi Roundtables” in which Alnylam scientists and key clinical collaborators review recent progress in many of the company’s development-stage pipeline programs and discuss the related disease areas. Click here to access our current and previous presentations.
Interim Data from Ongoing Phase 1 Trial with Fitusiran for the Treatment of Hemophilia and Rare Bleeding Disorders
We reported new data from Parts C and D of our Phase 1 study with fitusiran, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders, at the World Federation of Hemophilia (WFH) World Congress, held July 24-28, 2016 in Orlando, Florida.
We reported new clinical data from our ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis). These data were presented at the XV International Symposium on Amyloidosis held July 3 – 7, 2016 in Uppsala, Sweden.
Initial Data in Paroxysmal Nocturnal Hemoglobinuria Patients from Ongoing Phase 1/2 Clinical Study with ALN-CC5 Support Potential for Eculizumab Sparing and Improved Disease Control for Eculizumab Inadequate Responders
We reported data from 6 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) enrolled in Part C of our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component 5 (C5) for the treatment of complement-mediated diseases. These data were presented at the 21st Congress of the European Hematology Association (EHA) Meeting in Copenhagen, Denmark, held June 9-12, 2016. In this part of the study, ALN-CC5 was evaluated as a monotherapy or as an adjunct to eculizumab, an approved anti-C5 monoclonal antibody indicated for the treatment of PNH.