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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

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

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

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

New eye drop may be potentially effective in treating seasonal eye allergies

A recent study shows that a new eye drop may be a potentially effective treatment for seasonal eye allergies, a condition affecting millions of people worldwide.

4 May 2018

Sylentis presents clinical study results of new treatment for dry eye syndrome

Sylentis, Pharmaceutical Company belonging to the PharmaMar Group, has presented results from the clinical studies carried out with tivanisiran for the treatment of dry eye syndrome and that has enabled the start-up of the Phase III "Helix" clinical trial.

3 May 2018

Discovery of new ALS gene points to cytoskeleton as potential target for drug development

An international team of researchers led by John Landers, PhD, at UMass Medical School, and Bryan Traynor, MD, PhD, at the National Institute on Aging at the National Institutes of Health, has identified KIF5A as a new gene associated with the development of amyotrophic lateral sclerosis.

21 Mar 2018

Researchers find link between increased proteostasis and immortality of stem cells

Stem cells are considered to be immortal in culture and, therefore, of great interest for aging research. This immortality is regulated by increased proteostasis, which controls the quality of proteins.

8 Mar 2018

Mosquitoes can rapidly learn and remember the smell of hosts, study shows

Your grandmother's insistence that you receive more bug bites because you're 'sweeter' may not be that far-fetched after all, according to pioneering research from Virginia Tech scientists.

25 Jan 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

KU Leuven researchers find that humans have lost ancient defense mechanism to combat viruses

Insects and plants have an important ancient defense mechanism that helps them to fight viruses. This is encoded in their DNA. Scientists have long assumed that vertebrates - including humans - also had this same mechanism. But researchers at KU Leuven, Belgium, have found that vertebrates lost this particular asset in the course of their evolution.

15 Sep 2017

RNAi News and Research

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

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

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

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

Genes associated with autism interact to modulate variable symptoms of disease

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

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

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

New eye drop may be potentially effective in treating seasonal eye allergies

Sylentis presents clinical study results of new treatment for dry eye syndrome

Discovery of new ALS gene points to cytoskeleton as potential target for drug development

Researchers find link between increased proteostasis and immortality of stem cells

Mosquitoes can rapidly learn and remember the smell of hosts, study shows

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

KU Leuven researchers find that humans have lost ancient defense mechanism to combat viruses

Could the mTOR pathway be used to protect against cancer?

Genetic manipulation of exosomes may offer new therapeutic approach to treating pancreatic cancer

Leti's New Deal project could revolutionize IBD therapies for great benefit of patients in the European Union

New research could lead to improved therapies for global parasitic disease

Scientists reveal novel drug delivery approach to fight swine flu at gene level

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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