Capella

Capella—the Online Voice of Progress in RNAi

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.

On September 11, 2018, we hosted an online RNAi Roundtable to review the progress with ONPATTRO™ (patisiran) and ALN-TTRsc02, RNAi therapeutics for the treatment of transthyretin-mediated amyloidosis*.

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* ONPATTRO is approved in the U.S. for the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults, and in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. **ALN-TTRsc02 is an investigational RNAi therapeutic, in development for the treatment of ATTR amyloidosis. 

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We presented new analyses from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the 2018 Peripheral Nerve Society (PNS) Annual Meeting, held July 22-25, 2018 in Baltimore, MD.

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Ajroud-Driss et al. – “Impact of Patisiran on Overall Health Status in hATTR Amyloidosis: Results from the APOLLO Trial”

Polydefkis et al. – “Infusion Related Reactions in Patients with hATTR Amyloidosis Treated with Patisiran”

Dyck et al. – “Neuropathy Progression in Patients with hATTR Amyloidosis: Analysis of the APOLLO Placebo Arm”

Coelho et al. – “Longitudinal Changes in mNIS+7 are Associated with Changes in Ambulatory Status in Hereditary Transthyretin-Mediated Amyloidosis”

Coelho et al. – “Indirect Comparison of Patisiran and Tafamidis for Treatment of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy”

Gonzalez-Duarte et al. – “Changes in Neuropathy Stage in Patients with Hereditary Transthyretin-Mediated Amyloidosis Following Treatment with Patisiran, an Investigational RNAi Therapeutic: An Analysis from the Phase 3 APOLLO Study” (more…)

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 International Congress on Neuromuscular Diseases (ICNMD), held July 6-10, 2018 in Vienna, Austria.

Obici et al. – “APOLLO Phase 3 Study: Impact of Baseline Neuropathy Severity on Response to Patisiran”

Coelho et al. – “Transthyretin Reduction with Patisiran in the APOLLO Phase 3 Study”

Mauermann et al. – “Impact of Patisiran on Autonomic Neuropathy in Hereditary Transthyretin-Mediated Amyloidosis Patients”

Goel et al. – “Population Pharmacokinetics (PK) of Patisiran in Healthy Volunteers and hATTR Patients”

Zhang et al. – “Pharmacokinetics of Patisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis”

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”