Title: Conductance of Improperly Folded Proteins Through the Secretory Pathway and Related Methods for Treating Disease. Number: 20030236300. Filed: Jan. 14, 2003. Lead Inventor: Michael Caplan, Yale University.
“This invention,” states the patent application’s abstract, “provides the methodology and agents for treating [a] disease or clinical condition which is at least partly the result of endoplasmic reticulum-associated retention of proteins.” As a result, methods and agents of the invention “provide for the release of normally retained proteins from the endoplasmic reticulum,” the abstract adds.
The patent application states that the invention “provides siRNA compositions targeted to UDP glucose:glycoprotein glycosyl transferase, calnexin, endoplasmic reticulum Ca++ ATPase, or any chaperone involved in retention of a misfolded or misassembled protein in the ER, [for] which retention is associated with a disease or clinical condition.”
Title: Charge Reversal of Polyion Complexes and Treatment of Peripheral Occlusive Disease. Number: 20030236214. Filed: April 15, 2003. Lead Inventor: Jon Wolff, Mirus.
The patent application’s abstract states that the invention is a process “for the delivery of a therapeutic polynucleotide to a tissue suffering from or potentially suffering from ischemia.”
The process involves an ionic polymer used to recharge “a condensed polynucleotide complex for purposes of nucleic acid delivery to a cell,” the abstract states. “The resulting recharged complex can be formed with an appropriate amount of positive or negative charge such that the resulting complex has the desired net charge.”
The application specifically covers a polynucleotide that consists of “an RNA function inhibitor,” including an siRNA that blocks expression of an angiogenesis inhibitor.
Title: Hybridization Signal Amplification Method (HSAM) Nanostructures for Diagnostic and Therapeutic Uses. Number: 20030236205. Filed: June 21, 2002. Lead Inventor: David Zhang, Mount Sinai School of Medicine.
According to the patent application’s abstract, “the present invention relates to a hybridization signal amplification method (HSAM) that can be used to form nanostructures for use in drug delivery and diagnostics and may comprise molecules aimed at a specific target cell of interest.”
The nanostructures, which may be used to treat infectious diseases and physiological disorders, “comprise nucleic acid molecules having affinity pairs incorporated into their structure,” the abstract states. “These affinity pairs are formed from ligand and ligand-binding moieties that bind to nucleic acid molecules.”
The bound entity, the abstract notes, is “a complex, web-like structure that serves as a matrix or framework for delivery of therapeutic or diagnostic agents.”
The patent application states that the agents composing the nanostructures include polynucleotides “selected from the group consisting of a RNAi molecule, an antisense molecule, and a ribozyme molecule.”
Title: Novel Methods for the Delivery of Polynucleotides to Cells. Number: 20030235916. Filed: June 16, 2003. Lead Inventor: Sean Monahan, Mirus.
The patent application’s abstract states that the invention involves delivering a polynucleotide to a cell, either in vivo or in vitro, by “forming a salt stable complex between the polynucleotide and a cationic surfactant.
“Ternary complexes are also made by associating an amphipathic compound with the binary complex,” the abstract adds.
Title: Methods and Compositions for Preventing and Treating Microbial Infections. Number: 20030235577. Filed: April 8, 2003. Lead Inventor: Steven Shapiro, Brigham and Women’s Hospital.
The patent application’s abstract states that the invention “involves administration of MMPAP-12 polypeptides and nucleic acids for the treatment or prevention of infectious disease associated with microorganisms in subjects.”
The invention also relates to “kits and compositions relating to the MMPAP-12 molecules,” the abstract states.
The patent application specifically claims a “method for preparing an animal model of a disorder characterized by aberrant expression of an MMPAP-12 molecule, comprising: administering to a non-human subject an effective amount of an antisense, siRNA, or RNAi molecule to an MMPAP-12 nucleic acid molecule to reduce expression of the MMPAP-12 nucleic acid molecule in the non-human subject.”
Title: Prostate Specific Genes and Uses Thereof in Design or Therapeutics. Number: 20030235533. Filed: Feb. 19, 2003. Inventor: Dennis Gately, Idec Pharmaceuticals.
This patent application, states its abstract, covers “genes that are upregulated in human prostate tumor tissues and the corresponding proteins [that] are identified.” These genes and their corresponding antigens, the abstract adds, “are suitable targets for the treatment, diagnosis or prophylaxis of prostate cancer.”
The patent application specifically claims a method of treating prostate cancer by inhibiting the expression of an Kv3.2a or Kv3.2b gene with an antisense oligo or “interfering RNA.”
Title: Antisense Modulation of PAZ/PIWI Domain-Containing Protein Expression. Number: 20030232442. Filed: June 17, 2002. Inventor: Kenneth Dobie, Isis Pharmaceuticals.
According to the patent application’s abstract, the invention involves “compositions and methods … for modulating the expression of PAZ/PIWI domain-containing protein.” The compositions “comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding PAZ/PIWI domain-containing protein,” the abstract states.
Specifically, the patent application claims a compound “8 to 80 nucleobases in length targeted to a nucl- eic acid molecule encoding PAZ/PIWI domain-containing protein, wherein said compound specifically hybridizes with said nucleic acid molecule encoding PAZ/PIWI domain-containing protein and inhibits the expression of PAZ/PIWI domain-containing protein.”
The application also claims the use of this compound to modulate RNAi in an organism.
Title: Methods of Screening Subjects for Expression of Soluble Receptors of Vascular Endothelial Growth Factor (VEGF) for Use in Managing Treatment and Determining Prognostic Outcome. Number: 20030232400. Filed: May 15, 2003. Lead Inventor: Susan Radka, Sirna Therapeutics.
The patent application’s abstract states that the invention “relates generally to compounds and methods relating to detection of soluble VEGFR expression for generating prognostic criteria useful in establishing methods of treatment in cancer patients.”
The method described by the abstract involves using levels of soluble VEGF to detect cancer, evaluate efficacy of treatments, predict the prognosis of a subject, and discover new therapies.
The application specifically cites colorectal cancer, breast cancer, and non-small cell lung cancer. It also claims a method of determining the efficacy of a small interfering nucleic acid-based cancer therapy and predicting the prognosis of a patient being treated with a small interfering nucleic acid-based cancer therapy.