RegenxBio. has been granted a patent for a method of treating central nervous system disorders, specifically Spinal Muscular Atrophy, in children aged 3 to 12. The method involves administering scAAV9 vectors encoding the SMN1 protein via cerebrospinal fluid injection, targeting specific brain cells for effective treatment. GlobalData’s report on RegenxBio 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 RegenxBio, Adeno-associated virus vectors was a key innovation area identified from patents. RegenxBio's grant share as of July 2024 was 7%. Grant share is based on the ratio of number of grants to total number of patents.
Cns disorder treatment using scaav9 vector injection
The granted patent US12070510B2 outlines a method for treating Central Nervous System (CNS) disorders, specifically Spinal Muscular Atrophy (SMA), in human subjects aged between 3 to 12 years and 9 to 36 months. The treatment involves administering a flat dose of approximately 1.2×10^14 GC of scAAV9, which encodes the therapeutic protein SMN1, via injection into the cerebrospinal fluid through various routes, including intracerebroventricular, intrathecal cisternal, or intrathecal lumbar. The method aims to alleviate symptoms associated with SMA in the treated subjects.
Additionally, the patent claims include the administration of an immunosuppressive agent to enhance treatment efficacy. This agent may reduce the activity of cytotoxic T cells, mitigate the risk of encephalitis, or be a non-steroidal anti-inflammatory agent. The method is designed to ensure that the scAAV9 effectively transduces cells in critical brain regions, including the hippocampus, cerebellum, and cerebral cortex, thereby potentially improving the therapeutic outcomes for patients suffering from SMA.
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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
