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Six New RNAi-Related Patent Applications Published By US Patent Office: Apr 23, 2004

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Title: Modulation of Biological Signal Transduction by RNA Interference. Number: 20040077574. Filed: May 23, 2003. Lead Inventor: Richard Klinghoffer, Ceptyr.

The patent application, its abstract states, covers “compositions and methods relating to small interfering RNA polynucleotides [used for the] modulation of biological signal transduction.”

The abstract states that the invention relates to “siRNA polynucleotides that interfere with expression of members of the protein tyrosine phosphatase class of enzymes that mediate signal transduction, and with certain MAP kinase kinases. The invention also provides siRNA polynucleotides that interfere with expression of chemotherapeutic target polypeptides,” it adds.


Title: Intravascular Delivery of Non-Viral Nucleic Acid. Number: 20040072785. Filed: July 28, 2003. Lead Inventor: Jon Wolff, Mirus.

According to the patent application’s abstract, the invention is a process for the “delivery of a therapeutic polynucleotide to a tissue suffering from or potentially suffering from ischemia.”

This process involves inserting a polynucleotide “into a mammalian vessel such as an artery or a vein,” the abstract states. “Prior to insertion, subsequent to insertion, or concurrent with insertion, the volume of the tissue is increased such that the genetic material is delivered to the parenchymal cell.”

The application specifically claims the delivery to muscle tissue of an RNA function inhibitor, such as an siRNA, that prevents expression of an angiogenesis inhibitor in order to enhance blood flow.


Title: Methods for Design and Selection of Short Double-Stranded Oligonucleotides, and Compounds of Gene Drugs. Number: 20040072769. Filed: Sept. 16, 2002. Inventor: James Qinwei Yin, Harvard-MIT Division of Health Sciences and Technology.

The patent application, its abstract states, covers “methods for designing and selecting efficacious [short double-stranded oligonucleotides] as a gene drug that can specifically inactivate a group of corresponding genes. In particular, this invention relates to a process including the recruitment of target genes causing a disease, the identification of an endogenous siRNA sequence, the prediction of an efficacious [short double-stranded oligonucleotide], and [its] assembly … into related carriers with the ability [to target a diseased cell or tissue],” the abstract adds.


Title: Methods for Genetic Modification of Hematopoietic Progenitor Cells and Uses of the Modified Cells. Number: 20040072771. Filed: July 10, 2002. Lead Inventor: Geoffrey Symonds, School of Women’s and Children’s Health.

The patent application describes “compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor cells, to transduced cells, and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects,” its abstract states. “The invention particularly relates to ex vivo gene therapy of [hematopoietic progenitor] cells for treatment or prevention of HIV infection.”

The application claims a composition that expressed an anti-HIV agent, namely an RNAi molecule.


Title: Methods of Identifying Compounds That Inhibit Nonstop Degradation of mRNA. Number: 20040072201. Filed: April 16, 2003. Lead Inventor: Harry Dietz, Johns Hopkins University School of Medicine.

According to the patent application’s abstract, the invention “provides screening methods for the identification of compounds that inhibit nonstop degradation of mRNA, including compounds that inhibit the exosome. The invention further provides methods of treatment for genetic disorders caused by premature termination codons,” the abstract adds.

Specifically claimed by the application is a method “of treating a genetic disorder in a subject caused by a premature termination codon” involving the administration of an siRNA capable of inhibiting the nonstop degradation of mRNA and a compound that “induces readthrough of premature termination codons.”


Title: GPR 39 Modulators That Control Cancerous Cell Growth. Number: 20040071708. Filed: Sept. 26, 2002. Lead Inventor: Gisela Claassen, Immusol.

The patent application, its abstract states, covers “compositions, systems, and methods for identifying anti-cancer agents using both in vitro and in vivo techniques. Embodiments include the screening of combinatorial libraries of peptides, antibodies, and small organic molecules, as well as siRNAs, ribozymes, and antisense nucleotides directed against nucleic acids encoding the GPR 39 protein.”

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