RNA Interference News and Research

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Novel DNA nanoplatform delivers anticancer agents to multidrug-resistant tumors

One of the most successful techniques to combat multidrug resistance in cancer cells is the downregulation of those genes responsible for drug resistance.

22 Nov 2018

New initiative may benefit entrepreneurs working to transform patient care

Entrepreneurs working to improve the lives and promote the independence of patients may soon benefit from an initiative run by the Massachusetts Medical Device Development Center.

9 Nov 2018

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

Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

Arrowhead Pharmaceuticals Inc. today announced that it has completed dosing of a Phase 1 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency.

31 Aug 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

Scientists publish study results of AAV therapy product for treatment of RHO-adRP

Ophthotech Corporation announced today that scientists at the University of Pennsylvania and University of Florida published proof-of-concept study results on an adeno-associated virus gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa licensed by Ophthotech.

22 Aug 2018

Scientists pinpoint key gene that controls body segmentation in spiders and insects

Scientists have pinpointed a key gene that controls segmentation during spider development, which reveals a further similarity to the control of segmentation in insects, a study in eLife reports.

21 Aug 2018

Scientists report novel gene therapy that halts vision loss in canine model of blinding disease

Scientists funded by the National Eye Institute report a novel gene therapy that halts vision loss in a canine model of a blinding condition called autosomal dominant retinitis pigmentosa (adRP). The strategy could one day be used to slow or prevent vision loss in people with the disease. NEI is part of the National Institutes of Health.

21 Aug 2018

Researchers describe link between poor oocyte development and oxidative stress in obese mice

Excessive body fat is associated with negative effects on female fertility and pregnancy. In mice, maternal obesity impairs proper development of egg precursor cells called oocytes.

17 Aug 2018

FDA approves first targeted RNA-based treatment for rare, fatal genetic disease

The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

10 Aug 2018

Researchers use DNA editing tool to reveal mechanisms behind autoimmune diseases

A group of researchers at the University of São Paulo in Brazil used a DNA editing tool called CRISPR/Cas9 to manipulate on of the genes associated to the autoaggressive T lymphocytes responsible for inducing autoimmune diseases such as autoimmune polyglandular syndrome type 1 and type 1 diabetes.

2 Aug 2018

Researchers describe strategy used by Oropouche virus to replicate in human cells

The strategy used by the Oropouche virus to replicate in human cells has been described for the first time by researchers at the University of São Paulo in Brazil and international collaborators in an article published in the journal PLOS Pathogens.

31 Jul 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

Genes associated with autism interact to modulate variable symptoms of disease

Genes located in a large chromosomal aberration associated with autism interact with each other to modulate the variable symptoms of the disease, according to new research.

29 Jun 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

Researchers identify protein essential for eye lens development and clear vision

If you want to take clear photographs, you don't use sandpaper to clear a smudge from your camera's lens. Similarly, if you want to see clearly, the lens of your eye has to be free of obstruction.

21 May 2018

Phase 1/2 study evaluates safety, tolerability of multiple-ascending doses of ARO-HBV in hepatitis B patients

Arrowhead Pharmaceuticals Inc. today announced that it has dosed the first 3 hepatitis B virus patients in the multiple-ascending dose portion of its ongoing Phase 1/2 study of ARO-HBV, the company's third generation subcutaneously administered RNA interference therapeutic being developed as a potentially curative therapy for patients with chronic HBV infection.

14 May 2018