Ginkgo Bioworks has been granted a patent for gene therapy compositions using Adeno-associated virus (AAV) vectors to treat NPC1. The patent includes a specific AAV vector design with a sequence of capsid protein subunit and a transfer cassette encoding NPC1 protein. GlobalData’s report on Ginkgo Bioworks gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Ginkgo Bioworks, was a key innovation area identified from patents. Ginkgo Bioworks's grant share as of April 2024 was 25%. Grant share is based on the ratio of number of grants to total number of patents.
Gene therapy composition for treating npc1 using aav vectors
A recently granted patent (Publication Number: US11905523B2) discloses an adeno-associated virus (AAV) vector designed for gene therapy applications. The vector includes a protein capsid with a specific capsid protein subunit sequence and a nucleic acid containing a transfer cassette. The transfer cassette consists of various elements, including inverted terminal repeats, a promoter, a transgene encoding a Niemann-Pick intracellular cholesterol transporter 1 (NPC1) protein, a polyadenylation signal, and more. Additionally, the vector may contain an intronic sequence and an enhancer to enhance its therapeutic efficacy.
Furthermore, the patent covers the use of this AAV vector in compositions, cells, and methods for treating individuals with Neimann-Pick Disease Type C. By administering an effective amount of the AAV vector to a subject in need, particularly human subjects, the method aims to address the genetic disorder by delivering the NPC1 protein-encoding transgene. The inclusion of specific sequences and elements in the vector, as outlined in the claims, ensures the targeted and efficient delivery of the therapeutic gene for potential clinical applications in treating this rare genetic condition.
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