Capella

Capella—the Online Voice of Progress in RNAi

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 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.

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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.

Coelho et al. – “Impact of Prior TTR Stabilizer Use in Patients with Hereditary Transthyretin-Mediated Amyloidosis in the APOLLO Phase 3 Study of Patisiran”

Obici et al. – “Impact of Patisiran, an Investigational RNAi Therapeutic, on Nutritional Status in Patients with Hereditary Transthyretin-Mediated Amyloidosis”

Coelho et al. – “Outcomes of Patients with Hereditary Transthyretin-Mediated Amyloidosis with Early Onset V30M versus All Other Mutations in APOLLO, a Phase 3 Study of Patisiran”

Goel et al. – “Population Pharmacokinetic (PK)/Pharmacodynamic (PD) Model of Serum Transthyretin (TTR) Following Patisiran-LNP Administration in Healthy Volunteers and Patients with Hereditary TTR-Mediated (hATTR) Amyloidosis with Polyneuropathy”

Zhang et al. – “Patisiran-LNP Pharmacokinetics (PK), Pharmacodynamics (PD), and Exposure-Response (E-R) Relationship in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy”

Obici et al. – “Impact of Patisiran on Norfolk Quality of Life Questionnaire Diabetic Neuropathy (QOL-DN) in Patients with Hereditary Transthyretin-Mediated Amyloidosis: Results from the Phase 3 APOLLO Study”

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.

Slama et al. – “Analysis of NT-proBNP Baseline Levels in APOLLO as a Predictor of Survival in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis”

Merlini et al. – “Impact of Patisiran on Norfolk Quality of Life Questionairre Diabetic Neuropathy in Patients with Hereditary Transthyretin-Mediated Amyloidosis: Results from the Cardiac Subpopulation in the Phase 3 APOLLO Study”

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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.

Read article in Nature Biotech

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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.

Read our press release

Adams et al. – “Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis: Results from the Phase 3 APOLLO Study”

Adams et al. – “Evaluation of Quality of Life and Disability in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Following Treatment with Patisiran, an Investigational RNAi Therapeutic: Results from the Phase 3 APOLLO Study”

Mora et al. – “Utility of Genetic Testing to Identify Individuals Suspected of Having Hereditary ATTR (hATTR) Amyloidosis”

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We presented new positive results from the Phase 1, Phase 1/2, and EXPLORE natural history studies of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHPs), at the European Association for the Study of the Liver (EASL) 53rd Annual International Liver Congress, being held April 11-15, 2018 in Paris, France.

Read our press release

Sardh et al. – “Phase 1/2, Randomized, Placebo Controlled and Open Label Extension Studies of Givosiran, an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Acute Intermittent Porphyria”

Gouya et al. – “EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyrias (AHPs) with Recurrent Attacks”

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