121 patents
Page 5 of 7
Utility
Compound and method for treating myotonic dystrophy
23 Feb 21
Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
Ryszard Kole, Gunnar J. Hanson
Filed: 18 Oct 18
Utility
Compositions for enhancing transport of molecules into cells
2 Feb 21
Compositions and methods for enhancing delivery of molecules, e.g. biological agents, into cells are described.
Patrick L. Iversen, Hong M. Moulton, Michelle H. Nelson, David A. Stein, Andrew D. Kroeker
Filed: 9 Apr 19
Utility
Modified antisense oligomers for exon inclusion in spinal muscular atrophy
2 Feb 21
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.
Marco A. Passini, Gunnar J. Hanson
Filed: 26 Aug 16
Utility
Exon skipping compositions for treating muscular dystrophy
2 Feb 21
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
Richard K. Bestwick, Diane Elizabeth Frank
Filed: 14 Dec 18
Utility
Methods for Treating Muscular Dystrophy
14 Jan 21
The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy.
Edward M. KAYE
Filed: 20 Jul 20
Utility
Oligonucleotide Analogues Targeting Human Lmna
14 Jan 21
Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
Filed: 17 Sep 20
Utility
Exon skipping oligomer conjugates for muscular dystrophy
12 Jan 21
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
Marco A. Passini, Gunnar J. Hanson
Filed: 13 Dec 17
Utility
Processes for preparing oligomers
28 Dec 20
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer).
Baozhong Cai, Mitchell Martini, Ross Shimabuku, Katie Thomas, Diane Elizabeth Frank, Richard K. Bestwick
Filed: 23 May 17
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
2 Dec 20
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Marco A. PASSINI, Gunnar J. HANSON
Filed: 31 May 18
Utility
Antisense-induced exon exclusion in type VII collagen
30 Nov 20
The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
Dan V. Mourich
Filed: 31 May 16
Utility
Processes for Preparing Phosphorodiamidate Morpholino Oligomers Via Fast-flow Synthesis
18 Nov 20
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer).
Kyle A. Totaro, Mark D. Simon, Ming Zhou, Hong Zong, Gunnar J. Hanson, Bradley L. Pentelute
Filed: 24 Sep 18
Utility
Oligonucleotide analogues targeting human LMNA
2 Nov 20
Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
Michael R. Erdos, Francis S. Collins, Kan Cao, Ryszard Kole, Richard Keith Bestwick, Leslie B. Gordon
Filed: 27 Apr 17
Utility
Exon Skipping Compositions for Treating Muscular Dystrophy
28 Oct 20
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Richard K. BESTWICK, Diane Elizabeth FRANK
Filed: 3 Dec 19
Utility
Cell-Penetrating Peptides For Antisense Delivery
7 Oct 20
Provided herein are oligonucleotides, cell penetrating peptides, and peptide-oligonucleotide-conjugates.
Justin M. Wolfe, Colin M. Fadzen, Zi-Ning Choo, Rebecca L. Holden, Monica Yao, Gunnar J. Hanson, Bradley L. Pentelute
Filed: 16 Oct 18
Utility
Antisense molecules and methods for treating pathologies
21 Sep 20
An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 59.
Stephen Donald Wilton, Sue Fletcher, Abbie Adams, Penny Meloni
Filed: 18 Mar 19
Utility
Exon skipping oligomer conjugates for muscular dystrophy
7 Sep 20
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.
Gunnar J. Hanson, Marco A. Passini, Frederick Joseph Schnell
Filed: 29 May 18
Utility
Peptide Oligonucleotide Conjugates
2 Sep 20
Oligonucleotide analogues conjugated to carrier peptides are provided.
Gunnar J. Hanson
Filed: 3 Oct 19
Utility
Exon skipping oligomer conjugates for muscular dystrophy
31 Aug 20
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.
Richard K. Bestwick, Frederick Joseph Schnell
Filed: 29 May 18
Utility
Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
31 Aug 20
Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided.
Gunnar J. Hanson, Alexander Charles Rudolph, Bao Zhong Cai, Ming Zhou, Dwight D. Weller
Filed: 18 Dec 18
Utility
Combination Therapies for Treating Muscular Dystrophy
12 Aug 20
The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal anti-inflammatory compound.
Marco A. PASSINI, Jill C. MILNE, Andrew J. NICHOLS
Filed: 27 Sep 18