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21 October 2024

The Nobel Prize Winners 2024: a snapshot of their patent footprints | Physiology or Medicine

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Nobel Committees announced the much-anticipated Nobel Prize winners of 2024.
Australia Intellectual Property

Last week, the Nobel Committees announced the much-anticipated Nobel Prize winners of 2024, honouring the contributions that, as per Alfred Nobel's will of 1895, "have conferred the greatest benefit to humankind".

In this series of articles, we present selected patents of these winners that, at least to some extent, result from or lead to their celebrated works. For those with an interest in the most commemorated scientific and technological achievements in 2024 and intellectual property, this series makes for interesting reading.

On 7 October 2024, the Nobel Assembly at Karolinska Institute announced that the 2024 Nobel Prize in Physiology or Medicine was rewarded to Victor Ambros and Gary Ruvkun "for the discovery of microRNA and its role in post-transcriptional gene regulation".

The Committee commented that:

Victor Ambros and Gary Ruvkun were interested in how different cell types develop. They discovered microRNA, a new class of tiny RNA molecules that play a crucial role in gene regulation. Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans. It is now known that the human genome codes for over one thousand microRNAs. Their surprising discovery revealed an entirely new dimension to gene regulation. MicroRNAs are proving to be fundamentally important for how organisms develop and function.

Highlighted below are two patent families which list Ambros or Ruvkun as co-inventors. These patent families relate to applications of microRNAs for potential treatment or diagnosis of human diseases and/or conditions.

Victor Ambros | Isolating circulating microRNA (miRNA)

This patent family claims priority from US Provisional Application No. 62/030,773 and has an earliest priority date of 30 July 2014. Granted US Patent No. 9,896,683 has two independent claims:

  1. A method for isolating RNA from a sample, the method comprising:
    • digesting the sample with Proteinase K in the presence of chaotropic salts and detergent
    • extracting RNA from the sample using an alkaline phenol:chloroform extraction, and
    • isolating the extracted RNA from the sample.
  1. A method of detecting a level of a circulating miRNA in a subject, the method comprising:
    • providing a sample comprising plasma or serum from a subject
    • lysing the sample
    • extracting RNA from the sample using ph 8.0 phenol:chloroform extraction
    • isolating the extracted RNA from the sample, and
    • determining a level of the miRNA in the extracted RNA.

Recognising that the levels of specific microRNAs in normal and diseased tissue, or in body fluids, can provide information about the disease status of a patient, this patent family discloses methods of isolating RNAs from a sample. An example; methods of detecting a level of circulating miRNA from plasma or serum sample of humanblood using alkaline phenol:chloroform extraction. These methods may be able to significantly increase the yield of many assayable small RNAs such as miRNA, some by tenfold or more compared to standard or previous methods.

The cycle threshold values obtained using the invention were compared with those obtained by standard commercial methods at the time (miRNeasy and acid phenol). It was found that most of the cycle threshold values are lower, meaning that the invention provided better detection outcomes than existing standard methods.

The invention provides a convenient, inexpensive, and non-invasive way to diagnose and/or track the progress of treatment of diseases that would be otherwise difficult to diagnose or treat.

This patent family has only been filed and subsequently granted in the US.

Gary Ruvkun | Methods and compositions for inhibiting detoxification response

This patent family claims priority from US Provisional Application No. 62/210,685 and has an earliest priority date of 27 August 2015. Granted US Patent No. 10,988,765 has three independent claims:

  1. A method of attenuating a detoxification response and/or treating related symptoms in a subject in need of such treatment, the method comprising administering an inhibitor of expression of a daf-22 gene or its human homolog, SCPx.
  1. A method of reducing toxicity of a pharmaceutical compound in a subject, the method comprising:
    co-administering to the subject, (1) said pharmaceutical compound, and (2) an effective amount of an inhibitor of expression of a daf-22 gene or its human homolog, SCPx, wherein the toxicity of the pharmaceutical compound is reduced in the presence of the inhibitor compared to the toxicity of the pharmaceutical compound administered in the absence of the inhibitor.
  1. A method of increasing efficacy of a pharmaceutical compound in a subject, the method comprising:
    co-administering to the subject, (1) said pharmaceutical compound, and (2) an effective amount of an inhibitor of expression of a daf-22 gene or its homolog SCPx thereof, wherein the efficacy of said pharmaceutical compound is increased in the presence of the inhibitor compared to the efficacy of said pharmaceutical compound in the absence of said inhibitor.

This invention relates to the induction detoxification response and immune response in distinct somatic cells by germline mutations in translation components.

The inventors identified genes of the activation pathways that are responsible for the induction of these responses, making the identified genes targets for attenuating translation defect induced detoxification and immune response.

It discloses methods and compositions for attenuating detoxification response and immune response, and/or treat related symptoms, by using inhibitors, particularly microRNA inhibitors, to inhibit the expression of identified genes.

These methods may be used to treat subjects with translation defect to reduce toxicity or side effects of a pharmaceutical compound by inhibiting expression of identified genes or activation of identified pathways. They may also be used to increase efficacy of a pharmaceutical compound, which may induce translation defects and exhibit poor pharmacokinetics.

This patent family entered national phase into Europe and the US. The European application is currently pending and the US application has been granted.

If you are interested in these patents of Ambros and Ruvkun, they are well worth a more detailed read.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

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