PTC Therapeutics. has filed a patent for a recognition element for splicing modifier (REMS) that can be recognized by a compound. The patent also includes methods for modulating the amount of a gene’s product using the compound, as well as artificial gene constructs and methods for altering endogenous genes to include REMS. The claim details have been canceled. GlobalData’s report on PTC Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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Recognition element for splicing modifier compound
A recently filed patent (Publication Number: US20230310651A1) describes a method for modulating the amount of an RNA transcript produced from a precursor RNA of a gene. The method involves contacting a compound or a form thereof with the precursor RNA in a cell. The precursor RNA contains a recognition element for splicing modifier (REMS) with a specific sequence. The compound or form thereof has a specific structure.
The patent claims also specify that the precursor RNA sequence includes exons, one or more introns, a 5' splice site, a branch point, and a 3' splice site. The REMS, which is either endogenous or non-endogenous, is located at the 5' splice site and is upstream of the branch point and the 3' splice site. The method can be performed in a cell culture or cell lysate.
The REMS sequence mentioned in the patent claims is GAgurngn (SEQ ID NO: 1), where r can be either A or G, and n can be any nucleotide. Another REMS sequence mentioned is ANGAguragu (SEQ ID NO: 4), where r can be either A or G, and N can be any nucleotide.
The method described in the patent claims can be used to modulate the amount of a functional protein produced from the precursor RNA of a gene. The compound or form thereof is contacted with the precursor RNA in a cell, and the precursor RNA contains the REMS sequence at the 5' splice site. The method can be performed in a cell culture or cell lysate.
The patent claims also mention that the precursor RNA may result from aberrant expression of the gene. Aberrant expression can refer to abnormal down regulation of an RNA transcript in the cell compared to a corresponding healthy cell. The method can be performed using cells from a cell line known to have aberrant RNA transcript levels for the gene or cells from a subject with a disease associated with an aberrant amount of an RNA transcript for the gene.
The patent claims list several genes for which the method can be applied, including ABHD10, APLP2, ARMCX6, COL1A1, COL1A2, COL3A1, COL5A2, DIAPH3, ERGIC3, FADS2, FN1, FOXM1, GALC, GGCT, LAMA2, LARP7, MADD, PARP1, PRKDC, RCC1, STRN3, and VPS29.
In summary, the filed patent describes a method for modulating the amount of an RNA transcript or a functional protein produced from a precursor RNA of a gene. The method involves contacting a compound or a form thereof with the precursor RNA in a cell, and the precursor RNA contains a specific REMS sequence. The method can be used in cell cultures or cell lysates and may be applicable to genes associated with aberrant expression in certain diseases.
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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
