RNAi Therapeutics News and Research

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RNA interference (RNAi) was discovered less than a decade ago and already there are human clinical trials in progress or planned. A major advantage of RNAi versus other anti-sense based approaches for therapeutic applications is that it utilizes cellular machinery that efficiently allows targeting of complementary transcripts, often resulting in highly potent down-regulation of gene expression.

Exploring gene silencing mechanisms for improved RNAi therapeutics

RNA interference (RNAi) therapeutics have garnered significant attention in clinical research due to their potential for treating various diseases, including genetic disorders, viral infections and cancer.

20 Aug 2024

Orbit Discovery and SanegeneBio collaborate to identify targeting peptides for RNAi drugs

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From Orbit Discovery 5 Jan 2023

Researchers discover how a workhorse protein keeps protein production on track

Cells produce proteins like little factories. But if they make too much at the wrong times it can lead to diseases like cancer, so they control production with a process called RNA interference (RNAi).

31 May 2022

Application of various nanoparticle-capsulated siRNA as therapeutics for SARS-CoV-2 infection

In a review published in the International Journal of Molecular Sciences, researchers have been reviewing the use of siRNAs to target coronaviruses.

25 Feb 2022

Plant-derived siRNAs could bring a 'green revolution' in RNAi tools and therapeutics

"Green revolution" in the early 1950s, the extensive cultivation of Dwarf Rice solves the food problem in developing countries. At present, chronic infection with hepatitis B virus (HBV) has been a major public health problem.

11 Jun 2019

RBSC announces partnership with Mitacs to develop and test yeast-based RNAi technology

Renaissance BioScience Corp., a leading global microorganism bioengineering company, is pleased to announce a new partnership with Mitacs, the University of British Columbia and the University of Manitoba for a three-year, Cdn$975,000 multi-investigator research and development project.

24 Jan 2019

Alnylam announces positive results from Phase 1/2 study of lumasiran for treating primary hyperoxaluria-1

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today updated positive results from its Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1.

4 Oct 2018

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that it has submitted a Clinical Trial Authorization application to the Medicines and Healthcare products Regulatory Agency to initiate a Phase 1/2 study of ALN-AAT02, an investigational RNAi therapeutic targeting alpha-1 antitrypsin for the treatment of AAT deficiency-associated liver disease.

3 Oct 2018

Alnylam reports further progress on broadening the therapeutic potential of RNAi

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today further progress on the Company's platform efforts in extrahepatic delivery of novel siRNA conjugates, including central nervous system (CNS) and ocular delivery in rat and non-human primates (NHPs).

3 Oct 2018

Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today positive topline results from the interim analysis of the ENVISION Phase 3 Study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria.

27 Sep 2018

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization for ONPATTRO (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

30 Aug 2018

Alnylam’s patisiran receives positive opinion from CHMP for treating hATTR amyloidosis

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Committee for Medicinal Products for Human Use has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

27 Jul 2018

Alnylam announces new positive results from Phase 1/2 study of lumasiran in patients with PH1

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1.

8 Jun 2018

Alnylam achieves delivery of novel siRNAs conjugates to the central nervous system

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Company has achieved delivery of novel small interfering RNA conjugates to the central nervous system and is planning to advance a pipeline of investigational RNAi therapeutics into clinical development.

8 May 2018

Regeneron forms consortium to accelerate largest widely-available human sequencing resource

By the end of 2019, Regeneron plans to sequence the exomes of all 500,000 people within the UK Biobank resource, all with associated health records, creating an unprecedented resource linking human genetic variations to human biology and disease.

9 Jan 2018

Alnylam reports new results from investigational RNAi therapeutic programs

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today preliminary results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, both investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis).

6 Jul 2016

Alnylam presents positive ongoing phase 2 open-label extension data for Patisiran and Revusiran

Alnylam Pharmaceuticals, Inc. has announced new results from its ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).

3 Nov 2015

Trends, R&D progress, and predicted revenues in RNAi therapies

When will RNAi therapies reach the market? Visiongain's brand new report shows you potential revenues to 2025, assessing data, trends, opportunities and prospects there.

25 Jul 2015

Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen.

24 Jun 2015

Alnylam files clinical trial application for alpha-1 liver disease, presents data at DDW

Alnylam Pharmaceuticals, Inc. has announced that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

19 May 2015