121 patents
Page 3 of 7
Utility
Compositions for Treating Muscular Dystrophy
3 Feb 22
Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.
Edward M. KAYE
Filed: 21 Oct 21
Utility
Adeno-associated Virus Antibodies and Fragments Thereof
3 Feb 22
The present disclosure relates to an isolated anti-AAV (adeno-associated virus) antibody or an antigen-binding fragment thereof capable of specifically binding an epitope of AAVrh74 capsid protein and uses thereof.
Sohrab KHAN, Danielle GRIFFIN, Louise RODINO-KLAPAC
Filed: 17 Sep 21
Utility
Antisense-induced exon2 inclusion in acid alpha-glucosidase
1 Feb 22
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.
Stephen Donald Wilton, Sue Fletcher, Gunnar James Hanson, Richard Keith Bestwick
Filed: 5 Sep 14
Utility
Compound and method for treating myotonic dystrophy
1 Feb 22
An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed.
Hong M. Moulton, Ryszard Kole
Filed: 24 Apr 14
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
20 Jan 22
Frederick Joseph Schnell, Baozhong Cai, Ankur Desai, Richard K. Bestwick
Filed: 11 Jun 21
Utility
Peptide oligonucleotide conjugates
18 Jan 22
Oligonucleotide analogues conjugated to carrier peptides are provided.
Gunnar J. Hanson
Filed: 4 Oct 19
Utility
Compound and Method for Treating Myotonic Dystrophy
6 Jan 22
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.
Gunnar J. Hanson, Ryszard Kole
Filed: 5 Feb 21
Utility
Functionally-modified oligonucleotides and subunits thereof
28 Dec 21
Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided.
Gunnar J. Hanson, Dwight D. Weller, Bao Zhong Cai, Ming Zhou
Filed: 20 May 19
Utility
Adeno-Associated Virus Vector Delivery for Muscular Dystrophies
23 Dec 21
The disclosure provides method of treating muscular dystrophy in a subject in need comprising administering a gene therapy vector, such as adeno-associated virus (AAV) vector, expressing a miniaturized human micro-dystrophin gene in combination with a step of suppressing the subject's immune system.
Louise Rodino-Klapac, Jerry R. Mendell, Ellyn Peterson, Rachael Potter, Danielle Griffin
Filed: 15 Jun 21
Utility
Adeno-associated Virus Antibodies and Fragments Thereof
16 Dec 21
The present disclosure relates to an isolated anti-AAV (adeno-associated virus) antibody or an antigen-binding fragment thereof capable of specifically binding an epitope of AAVrh74 capsid protein and uses thereof.
Sohrab KHAN, Danielle GRIFFIN, Louise RODINO-KLAPAC
Filed: 15 Jun 21
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
4 Nov 21
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: 30 Apr 21
Utility
Trimeric Peptides for Antisense Delivery
23 Sep 21
Provided herein are oligonucleotides, trimeric peptides, and peptide-oligonucleotide-conjugates.
Justin Wolfe, Colin M. Fadzen, Bradley L. Pentelute, Gunnar J. Hanson
Filed: 29 Jul 19
Utility
Methods and Compounds for Treatment of Lymphocyte-related Diseases and Conditions
16 Sep 21
Dan V. Mourich, Gunnar J. Hanson, Frederick Joseph Schnell, Johannes Christian Dworzak
Filed: 30 Apr 21
Utility
Chimeric Peptides for Antisense Deliver
26 Aug 21
Provided herein are oligonucleotides, chimeric peptides, and peptide-oligonucleotide-conjugates.
Bradley L. PENTELUTE, Colin M. FADZEN, Rebecca L. HOLDEN, Justin M. WOLFE, Zi-Ning CHOO, Monica YAO, Gunnar J. HANSON
Filed: 15 Mar 19
Utility
Peptide oligonucleotide conjugates
24 Aug 21
Provided herein are oligonucleotides, peptides, and peptide-oligonucleotide-conjugates.
Gunnar J. Hanson, Ming Zhou
Filed: 28 Apr 20
Utility
DsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
27 Jul 21
Morpholino oligomers containing modified intersubunit linkages and/or terminal groups are provided for use within dsRNA molecules.
Patrick L. Iversen
Filed: 6 Jul 18
Utility
Oligonucleotide analogues targeting human LMNA
27 Jul 21
Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
Ryszard Kole, Richard Keith Bestwick
Filed: 17 Apr 19
Utility
Exon Skipping Oligomers and Oligomer Conjugates for Muscular Dystrophy
22 Jul 21
Antisense oligomers and antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Marco A. PASSINI, Frederick Joseph SCHNELL, Chia-Ling WU
Filed: 13 Jun 19
Utility
Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
13 Jul 21
The present disclosure relates to modified antisense oligonucleotides.
Frederick Joseph Schnell
Filed: 17 Apr 17
Utility
Processes for Preparing Oligomers
1 Jul 21
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: 16 Nov 20