G.d. Searle & Company

Sarepta Therapeutics, Inc.

Joyant Pharmaceuticals, Inc.

Pharmacia Corporation

Serenex, Inc.

Massachusetts Institute of Technology

The University of Texas System

Other

C. D. Searle & Co.

Murdoch University

Pharmacia Corporation (of Pfizer, Inc.)

Esanex, Inc.

Gunnar J Hanson

John S Baran

Ming Zhou

Robert E Manning

Michael John Clare

Richard M Weier

Michael A Stealey

Lifeng Geng

Richard A Partis

Mark Andrew Russell

Dave Weissing

Shuyuan Liao

He Huang

Michael L Vazquez

Suzanne S Metz

Barbara B Chen

Marco A Passini

Joyce Zuowu Crich

Francis J Koszyk

Susan Harran

Dwight D Weller

Qi Wei

Xiangdong Xu

Bao Zhong Cai

John Jeffrey Talley

Daniel L Flynn

Matthew James Graneto

Paul Waddell Collins

Ish K Khanna

Thomas E Barta

Charles G Caldwell

Michael S South

Shaun Raj Selness

Rajesh Devraj

Shashidhar N Rao

Susan J Hartmann

Cathleen Elizabeth Hanau

Yi Yu

Ashok Anantanarayan

James Marvin Veal

Kenneth He Huang

Lindsay A Hinkley

Ashok S Naraian

Jeron Eaves

Frederick Joseph Schnell

Michael Hepperle

J Timothy Keane

Timothy J Hagen

Ryszard M Kole

Bradley Lether Pentelute

Patrick G Harran

Lai Wang

Michael E Hepperle

Stephen C Houdek

Win Naing

Nizal Chandrakumar

Donna M Iula

Theresa R Fletcher

Syaulan Yang

David Thomas

Justin M Wolfe

Colin M Fadzen

Alexander Charles Rudolph

Peter S Linsley

Michael B Tollefson

Patrick J Lennon

Bruce C Hamper

Arija A Bergmanis

Subo Liao

Dan V Mourich

Thomas Dale Owen

John Walker

Scott B Mohler

Rebecca Lynn Holden

Serge G Wolfson

Daniel P Getman

John Nicholas Freskos

Patrick L Iversen

Daniel Paul Becker

Deborah A Mischke

Joseph J McDonald

Stephen Donald Wilton

Clara I Villamil

Sue Fletcher

Joseph Gerace Rico

Richard Keith Bestwick

Susan Landis Hockerman

Terri L Boehm

Louis J Bedell

Roy Wendell Ware

Jeffery N Carroll

Xiaoming Xu

Mark David Simon

Brian James Leppert

Dominique Bonafoux

Nathan W Stehle

Stephen A Kolodziej

Madeleine H Li

Patrick B Mullins

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.

Exon skipping oligomer conjugates for muscular dystrophy

Provided herein are oligonucleotides, chimeric peptides, and peptide-oligonucleotide-conjugates, the chimeric peptides and peptide-oligonucleotide-conjugates comprising at least two cell-penetrating peptides, wherein at least one of the cell-penetrating peptides is an amphipathic peptide and at least one of the cell-penetrating peptides is an oligoarginine peptide. The oligoarginine peptide comprises the sequence [(RYR)] (SEQ ID NOs: 15-18), wherein R is arginine, Y is independently selected from aminohexanoic acid (X) or β-alanine (B). Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, chimeric peptides, and peptide-oligonucleotide-conjugates comprising at least two cell-penetrating peptides, wherein at least one of the cell-penetrating peptides is an amphipathic peptide and at least one of the cell-penetrating peptides is an oligoarginine peptide described herein.

Chimeric peptides for antisense delivery

Provided herein are oligonucleotides, peptides, and peptide-oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein.

Peptide oligonucleotide conjugates

Provided herein are oligonucleotides, trimeric peptides, and peptide-oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease in a subject in need thereof, comprising administering to the subject oligonucleotides, trimeric peptides, and peptide-oligonucleotide-conjugates described herein.

Trimeric peptides for antisense delivery

Provided herein are oligonucleotides, cell penetrating peptides, and peptide-oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein.

Cell-penetrating peptides for antisense delivery

The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.

Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.

The invention provides for a method for selectively reducing the expression of a mutant mRNA and/or protein having an expanded nucleotide repeat relative to a wild-type mRNA, comprising contacting a cell with an antisense oligonucleotide of sufficient length and complementarity to the expanded nucleotide repeat. More particularly it relates to selectively reducing the expression of mutant Huntington protein associated with Huntington's disease. The antisense oligonucleotide comprising either a nucleotide or a repeated three nucleotide sequence as defined in the claims.

Oligonucleotides for treating expanded repeat diseases

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

Antisense-induced exon2 inclusion in acid alpha-glucosidase

Growing community of inventors

Skokie, IL, United States of America

Gunnar J Hanson