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.
Initial Results from Ongoing Phase 1/2 Study with ALN-GO1, in Development for the Treatment of Primary Hyperoxaluria Type 1
We presented initial clinical data from ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the 17th Congress of the International Pediatric Nephrology Association (IPNA), held September 20 – 24, 2016 in Iguaçu, Brazil.
We reported new pre-clinical results in animal models of myasthenia gravis (MG) from ALN-CC5, an investigational RNAi therapeutic in development for the treatment of complement-mediated diseases, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held September 14-17 in New Orleans, Louisiana.
Positive Interim Results from Ongoing Phase 1 Study with ALN-AS1, in Development for the Treatment of Acute Hepatic Porphyrias
We presented interim results from our ongoing Phase 1 clinical trial with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP), at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, held from September 6 – 9, 2016 in Rome, Italy.