Sarepta Therapeutics, Inc.

Frederick Joseph Schnell

Gunnar J Hanson

Richard Keith Bestwick

Patrick L Iversen

Marco A Passini

Dan V Mourich

Baozhong Cai

Jason Gatlin

Johannes Christian Dworzak

The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.

Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed. The oligonucleotide comprises a targeting sequence having a region complementary to at least one string of three or more identical contiguous nucleobases in a target sequence, wherein the target sequence comprises at least one additional nucleobase compared to the region of the targeting sequence and the at least one additional nucleobase has no complementary nucleobase in the region of the targeting sequence, and wherein the targeting region complementary to the at least one string of three or more identical contiguous nucleobases is internal to the targeting sequence.

Antisense oligomer compounds

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

Exon skipping oligomer conjugates for muscular dystrophy

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

Methods for treatment of lymphocyte-related diseases and conditions, such as cancer and automimmune diseases, are provided. The methods comprise administration of an effective amount of an oligomer to a patient in need thereof, wherein the oligomer comprises, inter alia, at least one intersubunit linkage having the following structure: wherein R, L, X, Y and Z are as defined herein. 

Methods and compounds for treatment of lymphocyte-related diseases and conditions

The present disclosure relates to antisense oligomers and related compositions and methods for decreasing the expression of functional human myostatin and methods for treating muscular dystrophy and related disorders and more specifically relates to inducing exclusion of myostatin exon 2 and thereby reducing the levels of myostatin protein.

Antisense-induced exon exclusion in myostatin

Provided are antisense oligonucleotides and other agents that target and modulate IL-17 and/or IL-23 signaling activity in a cell, compositions that comprise the same, and methods of use thereof. Also provided are animal models for identifying agents that modulate 17 and/or IL-23 signaling activity.

Growing community of inventors

Cambridge, MA, United States of America

Frederick Joseph Schnell