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Six RNAi-Related Patent Applications Published by the US Patent Office: Sep 3, 2004


Title: Covalent Modification of RNA for In Vitro and In Vivo Delivery. Number: 20040167090. Filed: Feb. 19, 2004. Lead Inventor: Sean Monahan, Mirus.

According to the patent application’s abstract, the invention comprises “post-synthetic modification of RNA for the delivery of the RNA to a mammalian cell. The modifications enhance [resistance] of the RNA to nuclease digestion, and [enhance] delivery of the RNA to the cell whether the RNA is delivered alone or in combination with a transfection agent,” it states.

“Activity of the RNA in the cell is maintained,” the abstract adds.

Title: Vector Constructs. Number: 20040166580. Filed: May 18, 2001. Lead Inventor: Geert Plaetinck, Devgen.

The patent application, its abstract states, relates to “improved vector constructs useful in the expression of double-stranded RNA. The constructs are particularly useful for expression of double-stranded RNA in vitro and in vivo,” it notes.

Title: Treatment of Neurodegenerative Disease Through Intracranial Delivery of siRNA. Number: 20040162255. Filed: Nov. 25, 2003. Lead Inventor: William Kaemmerer, Medtronic.

“The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain; and discharging through the discharge portion of the catheter a predetermined dosage of a least one substance capable of inhibiting production of at least one neurodegenerative protein,” the patent application’s abstract states. “The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.

Title: Delivery of siRNA to Cells Using Polyampholytes. Number: 20040162235. Filed: Feb. 18, 2003. Lead Inventor: Vladimir Trubetskoy, Mirus.

“A polyampholyte is utilized in a complex with siRNA for purposes of siRNA delivery to a cell,” the patent application’s abstract states. “The complex can be formed with an appropriate amount of positive and/or negative charge such that the resulting complex can be delivered to a cell in vivo or in vitro.”

Title: Modified Oligonucleotides for Use in RNA Interference. Number: 20040161777. Filed: Nov. 4, 2003. Lead Inventor: Brenda Baker, Isis Pharmaceuticals.

“The present invention provides modified oligonucleotides for use in the RNA interference pathway of gene modulation,” according to the patent application’s abstract. “The modified oligonucleotides are also provided having a 3’ terminal cap group.”

The application specifically claims “a composition comprising a [complementary pair of siRNA oligomers], wherein: at least a portion of said first oligomer is capable of hybridizing with at least a portion of said second oligomer, at least a portion of said first oligomer is complementary to and capable of hybridizing with a selected target nucleic acid, and at least one of said first or said second oligomers includes a 3’ terminal cap.”

Title: Process for Delivering siRNA to Cardiac Muscle Tissue. Number: 20040157790. Filed: Feb. 3, 2004. Lead Inventor: Hans Herweijer, Mirus.

The patent application, its abstract states, covers “a process for delivering a polynucleotide to a cardiac tissue cell in a mammal [by] introducing a composition consisting of a polynucleotide into a blood vessel and increasing permeability of the blood vessel to the polynucleotide.

“The polynucleotide can be a small interfering RNA or microRNA and inhibit gene expression in the cell,” the abstract notes.

According to the application, the delivery process involves the following: making a polynucleotide containing a sequence that is substantially complementary to a nucleic acid sequence in the mammal heart cell; accessing an in vivo blood vessel; and inserting an injector into the blood vessel near or in the heart.” Additionally, the technique calls for “injecting a solution containing the polynucleotide into the blood vessel lumen and increasing hydrostatic pressure in the lumen thereby delivering the polynucleotide to the heart muscle cell outside of the blood vessel via the pressure, wherein invasiveness is limited to accessing the blood vessel, inserting the injector into the blood vessel, and injecting the solution.”


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