Albert Einstein College of Medicine

University of Kansas

Vitrisa Therapeutics, Inc.

396419 B.c. Ltd.

Matthew Levy

Arijit Bhowmick

Christopher P Rusconi

Carl Erickson

Kevin G McLure

Brian Christopher Wengerter

Samantha E Wilner

Steven Craig Almo

Blake R Peterson

Deborah Palliser

Liang Xu

Amy Yan

Keith Everett Maier

Simon G Trevino

The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease. In some cases, stem-loop aptamers are provided for the inhibition of Factor D.

Stem-loop compositions and methods for inhibiting factor D

Provided are chemically modified ribonucleic acid (RNA) aptamers comprising one or more of 2'F guanylate, 2&#x2032;OMe cytidylate, 2&#x2032;OMe adenylate, and a deoxy pyrimidine nucleotide with a moiety on the 5 position of the pyrimidine; and methods of making the aptamers.

Chemically modified RNA aptamers and uses thereof

Methods and compositions for serum-stabilizing micelles for drug delivery or imaging agent delivery are provided, as well as methods and compositions to enhance micelle-mediated drug delivery. This invention provides a process for synthesizing a lipid micelle comprising one or more lipid-oligonucleotide conjugate molecules, the process comprising contacting an amount of lipid molecules with an amount of lipid-oligonucleotide conjugate molecules sufficient to create a lipid micelle comprising lipid-oligonucleotide conjugate molecules.

Programmed cargo release using nucleic acid-stabilized micelles

Methods and compositions are provided for rapidly identifying novel structure-switching aptamers.

Method of rapid isolation of aptamer beacons

A composition is provided comprising an oligonucleotide aptamer conjugated to an antigen, wherein the aptamer is directed against a cell-surface target of an antigen-presenting cell. Also provided are methods of delivering an antigen to a dendritic cell and of eliciting an immune response in a subject.

Aptamer-targetted antigen delivery

A method of delivering a cargo agent into cytosol of a cell can include: providing the delivery system of one of the embodiments described herein having the first and second delivery platforms; and administering the delivery system to a cell so as to cause targeting of two features on the cell so as to: cause endocytosis of the first and second delivery platforms of the delivery system into a common endosome, destabilize the endosome of the cell having the delivery system, release the cargo agent from the second linker; and release the cargo agent from the destabilized endosome into cytosol of the cell. A method of treating a disease can include: performing the method of method of delivering a cargo agent into cytosol of a cell in a subject having a disease, wherein the cargo agent is a therapeutic agent for the disease.

Targeting multiple receptors on a cell surface for specific cell targeting

The present invention provides methods of preparing an oligonucleotide, nucleoside or nucleoside analog for selective introduction into a subject's cells, the method comprising (1) selecting a targeted aptamer, internalizing nucleic acid or tumor-homing nucleic acid via iterative rounds of selection, and (i) hybridizing it to an oligonucleotide, (ii) replacing one or more nucleotide with a nucleoside or nucleoside analog, or (iii) synthesizing the it with one or more nucleoside or nucleoside analogs; or (2) preparing a naive combinatorial aptamer, internalizing nucleic acid or tumor-homing nucleic acid prodrug library, and running iterative rounds of selection for the cells. The present invention also provides the agent, the pharmaceutical composition, and methods of treating or preventing cancer and/or viral infection, the method comprising administration of the oligonucleotide, nucleoside or nucleoside analog for selective introduction into a subject's cells.

Methods of preparing targeted aptamer prodrugs

Aptamers targeted to a human transferrin receptor which do not compete with transferrin for binding are provided. Compositions and methods for aptamer-targeted liposomal drug delivery are also provided.

Growing community of inventors

New Rochelle, NY, United States of America

Matthew Levy