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The report adds to the body of evidence the company and collaborators have generated suggesting the miRNA, as well as family member miR-195, may be targets for therapeutic intervention.

Mice lacking miR-133a developed adult-onset centronuclear myopathy, a congenital disease characterized by muscle weakness and abnormal centralization of nuclei in muscle myofibers, suggesting the miRNA may be a “potent modulator” of the disease.

Specifically, the intellectual property claims methods of use in modulating miR-143, which the company said plays a critical role in controlling smooth muscle cells at the site of vascular injury.

The new program, which received orphan drug status earlier this year, grew out of an effort to track down microRNAs associated with heart attack, Miragen's CEO said this week.

RXi and Miragen used the technology to create an miRNA mimic capable of down-regulating a reporter gene whose expression is controlled by the microRNA in cell culture model systems.

Despite advances, there remains much work to be done on the basic biology side of things if RNAi is ever to achieve its promise as a therapeutic modality, industry watchers said.

Despite the mounting data linking a variety of miRNAs to different diseases, in most cases the data are early-stage, and thus far only a handful of miRNAs have made it into the pipelines of companies in the field.

According to the company, miR-92 is "a key regulator of neo-angiogenesis as part of ischemic disease, which may be relevant to peripheral arterial disease and other cardiovascular disorders."

For instance, Tacere Therapeutics received $488,958 in two grants, the first of which supports development of a new hepatitis C treatment, as well as a previously undisclosed eye-disease program.

Under the sponsored-research portion of the deal, Miragen will fund the analysis of miRNA and gene-expression changes from a study conducted at the University of Colorado Cardiovascular Institute at the UC Denver School of Medicine.

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