Ginkgo BioWorks has been granted a patent for gene therapy compositions and methods to treat NPC1 using Adeno-associated virus (AAV) vectors. 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 February 2024 was 49%. Grant share is based on the ratio of number of grants to total number of patents.
Aav vector for treating npc1 with specific gene sequence
A recently granted patent (Publication Number: US11905523B2) discloses an adeno-associated virus (AAV) vector designed for gene therapy applications. The vector comprises a protein capsid with a specific capsid protein subunit sequence and encapsulates a nucleic acid containing a transfer cassette. This transfer cassette includes elements such as inverted terminal repeats, a promoter, a transgene encoding a Niemann-Pick intracellular cholesterol transporter 1 (NPC1) protein, a polyadenylation signal, and intronic sequences. The vector is further characterized by the presence of specific sequences for the ITRs, promoter, NPC1 protein, and polyadenylation signal.
The patent also covers compositions and methods utilizing this AAV vector for treating conditions such as Neimann-Pick Disease Type C in human subjects. The method involves administering the AAV vector to the subject to deliver the therapeutic transgene encoding the NPC1 protein. The inclusion of specific sequences and elements in the vector, such as the CBA promoter and SV40 polyadenylation signal, enhances its efficacy in gene therapy applications. Overall, this patented AAV vector presents a promising approach for treating genetic disorders like Neimann-Pick Disease Type C in human patients, highlighting the potential of gene therapy in addressing rare and debilitating conditions.
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