04 Oct, 2018 Updated Results from Phase 1/2 Study of Lumasiran at 2018 ESPN Annual Meeting

We presented updated positive results from the Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting, held October 3-6, 2018 in Antalya, Turkey.

Read our press release

View the presentation

(more…)

02 Oct, 2018 New Platform Innovations for RNAi Therapeutics at OTS 2018 Annual Meeting

We presented further progress on our platform efforts in extrahepatic delivery of novel siRNA conjugates, including CNS and ocular delivery in rat and non-human primates, at the Oligonucleotide Therapeutics Society (OTS) 2018 Annual Meeting, held September 30 to October 3 in Seattle, WA.

Read our press release

Maier et al. – “ESC+ Design Minimizes the Off-Target Potential and Further Maximizes the Therapeutic Index of GalNAc-siRNA Conjugates”

Janas et al. – “Safety Evaluation of 2′-Deoxy-2′-Fluoro-Modified Nucleotides in GalNAc-siRNA Conjugates”

Milstein et al. – “Robust and Durable Target Silencing in the CNS with siRNA Conjugates”

Agarwal et al. – “Mechanisms of Rat Hepatotoxicity of GalNAc-siRNA Conjugates”

Theile et al. – “Bis-RNAi™ Conjugates for Simultaneous Silencing of Two Different Gene Transcripts”

Nair et al. – “Efficient and Durable Ocular Gene Silencing of TTR After Single Intravitreal Administration of siRNA Conjugates”

Rocca et al. – “The Influence of GalNAc Valency on the Pharmacokinetic and Pharmacodynamic Parameters of siRNA in Rats”

14 Sep, 2018 Exploratory Cardiac Endpoint Data from APOLLO Published in Circulation

Data from exploratory cardiac assessments and post-hoc analyses of safety data in the APOLLO Phase 3 study of patisiran, an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, were published in the journal Circulation.

Read the paper in Circulation

23 Jul, 2018 New Analyses of Clinical Results from APOLLO Phase 3 Study of Patisiran at PNS Annual Meeting

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.

Read our press release

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…)

08 Jul, 2018 New Clinical Results from APOLLO Phase 3 Study of Patisiran at ICNMD

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”

04 Jul, 2018 APOLLO Phase 3 Results for Patisiran Published in “The New England Journal of Medicine”

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.”

Read the paper in The New England Journal of Medicine

(more…)