Rocket Pharmaceuticals. has filed a patent for a gene therapy using a recombinant adeno-associated virus (rAAV) to treat GLUT1 Deficiency Syndrome and related disorders. The therapy involves expressing a GLUT1 protein or functional variant using an endothelial-specific promoter. The patent also includes methods of treatment and other compositions. GlobalData’s report on Rocket Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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According to GlobalData’s company profile on Rocket Pharmaceuticals, NSAID cancer drugs was a key innovation area identified from patents. Rocket Pharmaceuticals's grant share as of September 2023 was 28%. Grant share is based on the ratio of number of grants to total number of patents.
Gene therapy for glut1 deficiency syndrome using raav vector
A recently filed patent (Publication Number: US20230272422A1) describes an expression cassette that includes a polynucleotide sequence encoding GLUT1 or a functional variant thereof, which is linked to a promoter. The promoter can be an endothelial promoter, such as a Tie-1 promoter, Tie-2 (TEK) promoter, FLT-1 promoter, FLK-1 (KDR) promoter, ICAM-2 promoter, VE-Cadherin (CDH5) promoter, VWF promoter, ENG promoter, PDGFB promoter, ESM1 promoter, APLN promoter, or Claudin-5 (Ple261) promoter, as long as it is not a Glut1 promoter. Alternatively, the promoter can be a FLT-1 promoter, specifically a human FLT-1 (hFLT-1) promoter, which shares a certain level of identity with a specific sequence. Similarly, the expression cassette can also include a Tie-1 promoter, specifically a human Tie-1 (hTie-1) promoter, which shares a certain level of identity with a specific sequence. Another option is a vascular endothelial-cadherin (VE-cadherin) promoter, specifically a human VE-cadherin (hVE-cadherin) promoter, which also shares a certain level of identity with a specific sequence. The expression cassette can also have a ubiquitous promoter, such as a CMV promoter or a CAG promoter. Additionally, the expression cassette may include a polyA signal, a Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (WPRE), and a 3' untranslated region (3' UTR) with a specific level of identity to a sequence. The polynucleotide sequence encoding GLUT1 can be a SLC2A1 polynucleotide, specifically a human SLC2A1 polynucleotide, which shares a certain level of identity with a specific sequence. The expression cassette can be flanked by 5' and 3' inverted terminal repeats (ITRs), such as AAV2 ITRs or ITRs with a specific level of identity to a sequence. The expression cassette can also share a certain level of identity with specific sequences.
The patent also describes a gene therapy vector that includes the expression cassette mentioned above. Specifically, the gene therapy vector can be a recombinant adeno-associated virus (rAAV) vector, excluding AAV2 vector. The rAAV vector can be AAV6, AAV8, AAV9, AAVrh.74, or AAVrh.10 vector, or a functional variant thereof. The vector can have a capsid protein that shares a certain level of identity with specific sequences. The patent further discloses a method of treating and/or preventing a disease or disorder, particularly a neurological disorder, such as Glucose transporter 1 deficiency syndrome (GLUT1DS) or De Vivo Disease, by administering the vector to a subject, preferably by intracerebroventricular (ICV) injection. The administration of the vector can result in the expression of the polynucleotide sequence encoding GLUT1 in the brain, potentially at increased levels compared to a reference rAAV vector. This can lead to an increase in GLUT1 protein expression in the brain, as well as increased glucose and lactate levels in the cerebrospinal fluid (CSF). The vector can be administered at specific doses. Additionally, the method can cause increased glucose uptake by cerebral microvasculature endothelial cells compared to methods using an endogenous Glut1 promoter or a ubiquitous promoter. The patent also covers a method of expressing GLUT1 in cells, particularly endothelial cells and neurons, by contacting the cells with the vector. Furthermore, the patent includes a pharmaceutical composition and a kit comprising the vector or the pharmaceutical composition.
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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
