Ionis Pharmaceuticals has been granted a patent for compounds and methods to modulate the expression of huntingtin, potentially slowing or preventing the progression of Huntington’s Disease. The patent also covers delaying or preventing the onset of the disease in susceptible individuals, as well as the use of the compounds in the manufacture of a medicament for treating diseases and disorders. The patent includes an antisense oligonucleotide with specific nucleotide sequences and modifications. GlobalData’s report on Ionis Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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Data Insights Ionis Pharmaceuticals Inc - Company Profile

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According to GlobalData’s company profile on Ionis Pharmaceuticals, Nucleic acid active pharmaceutical ingredient was a key innovation area identified from patents. Ionis Pharmaceuticals's grant share as of September 2023 was 38%. Grant share is based on the ratio of number of grants to total number of patents.

A recently granted patent (Publication Number: US11761000B2) discloses an antisense oligonucleotide with specific characteristics and its use in pharmaceutical compositions. The antisense oligonucleotide described in claim 1 is 12 to 35 nucleotides long and contains at least 12 consecutive nucleotides from a specific nucleotide sequence. This sequence can be selected from a group of sequences identified as SEQ ID NOs: 106-107. Additionally, the antisense oligonucleotide must have at least one modified internucleoside linkage, sugar moiety, or nucleobase.

Claim 2 states that the antisense oligonucleotide should have at least 95% complementarity to a specific sequence identified as SEQ ID NO: 4. In claim 3, it is mentioned that the antisense oligonucleotide can have at least 100% complementarity to SEQ ID NO: 4.

The patent also describes a chimeric oligonucleotide in claim 4, which consists of a gap segment positioned between 5' and 3' wing segments. Claim 5 specifies that the gap segment is made up of 2'-deoxynucleotides, while the wing segments contain nucleotides with modified sugar moieties. Examples of modified sugar moieties mentioned are 2'-OMe or a bicyclic nucleic acid.

Claim 7 provides further details on the chimeric oligonucleotide, stating that the gap segment consists of ten 2'-deoxynucleotides, and each wing segment consists of five 2'-O-methoxyethyl-modified nucleotides. The length of the antisense oligonucleotide in this case is 20 nucleotides, as mentioned in claim 8.

Other claims in the patent describe additional features of the antisense oligonucleotide, such as the use of phosphorothioate internucleoside linkages (claim 9) and the presence of 5-methylcytosine in each cytosine (claim 10). The length of the oligonucleotide can vary between 17 to 25 nucleotides (claim 11), 19 to 23 nucleotides (claim 12), or specifically 20 nucleotides (claim 13).

Finally, claim 14 discloses a pharmaceutical composition comprising the antisense oligonucleotide described in claim 1 and a pharmaceutically acceptable diluent.

Overall, this patent provides detailed information on the design and characteristics of an antisense oligonucleotide and its potential use in pharmaceutical compositions. The specific sequences, modifications, and lengths described in the claims highlight the novelty and potential applications of this technology in the field of molecular therapeutics.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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