University of Massachusetts

Matthew Hassler

Anastasia Khvorova

Julia Alterman

Bruno Miguel Da Cruz Godinho

Neil Aronin

Anton A Turanov

Andrew Coles

Mehran Nikan

Maire Osborn

Reka Haraszti

Annabelle Biscans

Reka Agnes Haraszti

Therapeutic oligonucleotides comprising pharmacokinetic (PK)-modifying anchors are provided. Methods for treating diseases or disorders comprising administering to a subject a therapeutic oligonucleotide comprising one or more PK-modifying anchors are provided.

Dynamic pharmacokinetic-modifying anchors

Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

Fully stabilized asymmetric siRNA

Bioactive conjugates for oligonucleotide delivery

Provided are compositions and methods comprising two-tailed siRNAs (tt-siRNAs) that exhibit unprecedented cellular uptake and silencing. Also provided are methods of treating neurological and other diseases with the two-tailed siRNAs of the invention.

Two-tailed self-delivering siRNA

Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.

Branched oligonucleotides

Provided herein are conjugated oligonucleotides that are characterized by efficient and specific tissue distribution.

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Worcester, MA, United States of America

Matthew Hassler