Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Alnylam, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.
We presented new pre-clinical results for ALN-AGT, a subcutaneously administered RNAi therapeutic development candidate targeting angiotensinogen (AGT) for the treatment of hypertensive disorders of pregnancy (HDP), including preeclampsia, at the American Heart Association Scientific Sessions 2017 in Anaheim.
We presented positive preliminary data from the ongoing Phase 1/2 study with lumasiran (formerly known as ALN-GO1), an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1) at the American Society of Nephrology (ASN) Kidney Week 2017 Annual Meeting in New Orleans.
We presented positive complete results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, at the 1st European ATTR Amyloidosis Meeting for Patients and Doctors, held November 2-3, 2017 in Paris, France.
Berk et al. – “Long-Term, Open-Label Clinical Experience with Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy” (more…)
We and our collaborators presented new pre-clinical data highlighting our next generation “Enhanced Stabilization Chemistry Plus” (ESC+) GalNAc-siRNA conjugate platform, and additional data demonstrating our leadership in RNAi technologies, at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 24 – 27, 2017 in Bordeaux, France.
Knight et al. – “Investigational RNAi Mediated Oxalate Reduction Therapy”
Schlegel et al. – “Impact of Glycol Nucleic Acid (GNA) on siRNA Structure and Function”
A paper describing the rationale and design of the APOLLO Phase 3 study was published in BMC Neurology. APOLLO was a randomized, double-blind, placebo-controlled, global study evaluating the efficacy and safety of patisiran, an investigational RNAi therapeutic, in patients with hATTR amyloidosis with polyneuropathy.
We hosted a series of online “RNAi Roundtables” in August and September, at which Alnylam scientists, clinical collaborators, and patients or patient advocates reviewed recent progress in many of the company’s late-stage pipeline programs and discussed the related disease areas.
On September 7, 2017, we hosted an online RNAi Roundtable to review the progress with givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyrias.
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Alnylam and The Medicines Company reported new, one-year data from the ORION-1 Phase 2 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia, at the European Society of Cardiology (ESC) Congress 2017 in Barcelona, Spain.
On August 23, 2017, we hosted an online RNAi Roundtable to review advancements in our RNAi therapeutics platform technology.
On August 9, 2017, we hosted an online RNAi Roundtable to report the findings from the investigation into the ENDEAVOUR Phase 3 trial with revusiran.
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