Bonza! 3 more patents filed yesterday and on Monday, in addition...

Bonza! 3 more patents filed yesterday and on Monday, in addition to the 2 patents filled last week.

Now a total of 5 patents for Improved compositions and method for treatment of monogenic neurodevelopmental disorders.

While all eyes are on the PKD drug and the Phalen McDermid Syndrome drug might be pushed back or even put on the backburner, it is alive and well. The impetus might well be the conversation they have had with the Mount Sinai Medical Research Centre in New York. As reported in my Post #: 71116375, on the 2023 AGM, Rohan Hockings said:

"With the Phelan McDermid Syndrome program, we had a terrific conversation with Mount Sinai Medical Centre in New York and the clinicians there want to scale up behind the drug which targets the SHANK-3 gene. They are waiting for one additional piece of data. They want to see if it can you do what we’ve done in the reporter cell in a human neuron. That readout is coming soon in the early parts of next year. This is the drug that can change the lives of children who are unable to communicate with their parents with very severe phenotype."

The SHANK3 drug for treating Phelan McDermid Syndrome might also be used for Autistic Spectrum Disorder ASD and Schizophrenia.

Reporter cell lines are stable cell lines that have been labeled with reporter genes, allowing the visualization and tracking of protein expression. The reporter gene is inserted next to the gene promoter of interest within a vector and transfected to a cell line. If the promoter is expressed in the cell line, the reporter gene will also be expressed, allowing monitoring of cellular metabolic processes, transcription factors, and protein localization. Common reporter genes include luciferase and fluorescent proteins like GFP and RFP.



GFP was first isolated from the jellyfish Aequorea Victoria in1960 and has rapidly become one of the most widely studied and exploited fluorescent proteins in biochemistry and cell biology. This popularity is due to its ability to generate a highly visible, efficiently emitting internal fluorophore. GFP has become well established as a reporter of gene expression and protein targeting in intact cells and organisms. See a sample application of this technique in the research of PKD drug below.


In case you don't know what a child with Phelan McDermid Syndrome is like, watch the YouTube video below.

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