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Inhalable mRNA offers hope for patients with lung disease

Scientists have reported the success of an inhalable mRNA therapy that can induce the production of a target protein in the lungs of mice.

4 Jan 2019

Genetically modified pigs could limit replication of classical swine fever virus, study shows

Researchers have developed genetically modified pigs that are protected from classical swine fever virus, according to a study published December 13 in the open-access journal PLOS Pathogens by Hongsheng Ouyang of Jilin University, and colleagues.

14 Dec 2018

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

RNAi therapy shown to alleviate preeclampsia in animal model

Researcher have used a small interfering RNA (siRNA) to reduce preeclampsia symptoms in an animal model. The approach could provide a new way of treating preeclampsia in humans.

19 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

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