Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.
We have published results from the APOLLO Phase 3 trial of patisiran in The New England Journal of Medicine in a paper titled, “Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.”
On June 26, 2018, we hosted an online RNAi Roundtable to review advancements in our RNAi therapeutics platform technology.
We are hosting a series of online “RNAi Roundtables” from June through September, at which Alnylam scientists, clinical collaborators, and patients or patient advocates review recent progress in many of the Company’s late-stage pipeline programs and discuss the related disease areas.
We presented additional results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 4th Congress of the European Academy of Neurology (EAN), held June 16-19, 2018 in Lisbon, Portugal.
We presented new positive results from the Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the OxalEurope, European Hyperoxaluria Consortium, held June 8, 2018 in Naples, Italy.
We presented new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the European Society of Cardiology Heart Failure 2018 Congress, held May 26-29, 2018 in Vienna, Austria.
One of the main features of our ESC GalNAc-siRNA conjugates platform is potent and durable target knockdown in the liver sustained for several months in humans. In some cases, RNAi therapeutics may benefit from a technology that enables reversal of target mRNA knockdown and provides finer control over pharmacology, a desired attribute for therapeutic entities. REVERSIRTM molecules are GalNAc-conjugated, short, single-stranded, high affinity oligonucleotide constructs designed to recognize and bind to the complementary, RISC-bound antisense strand of siRNAs, thereby leading to rapid and complete reversal of RNAi-mediated target gene silencing activity in vivo.
We presented a nonclinical safety evaluation of GalNAc-siRNA conjugates at the 8th Annual Biologics Symposium, being held May 8-9, 2018 in Plainsboro, NJ.
We presented initial data demonstrating delivery of nove small interfering RNA (siRNA) conjugates to the central nervous system (CNS) at the TIDES: Oligonucleotides and Peptide Therapeutics 2018 Annual Meeting, being held May 7-10, 2018 in Boston, MA.
We presented new positive results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the American Academy of Neurology (AAN) 2018 Annual Meeting, being held April 21-27, 2018 in Los Angeles.
