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

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16 Jun, 2018 New Clinical Results for Patisiran at EAN

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 4^th 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”

08 Jun, 2018 New Positive Clinical Results for Lumasiran at OxalEurope

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.

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29 May, 2018 New Clinical Results from APOLLO Phase 3 Study of Patisiran at ESC-HF

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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17 May, 2018 Nonclinical Research Published in Nature Biotechnology Describing REVERSIR Platform for Potent and Rapid Reversal of siRNA Activity for Tailored Control of RNAi Pharmacology

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. REVERSIR^TM 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.

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09 May, 2018 Nonclinical Safety Evaluation of GalNAc-siRNA Conjugates

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.

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08 May, 2018 New Preclinical Data Demonstrating CNS Delivery of RNAi Therapeutics

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.

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