121 patents
Utility
Suspension Mode Seed Train Development for Adherent Cells
18 Jan 24
The disclosure is directed to a method for seed train expansion of adherent cells comprising culturing cells with a serum-supplemented growth medium in a N-2 vessel; removing the cells from the serum-supplemented medium; inoculating the cells from step into a serum-free growth medium in a N-1 vessel; culturing the cells in the N-1 vessel under suspension conditions; and inoculating a growth medium in a bioreactor with the suspension-cultured cells.
Maroof ALAM
Filed: 9 Dec 21
Utility
Antisense Oligomer Compounds
21 Dec 23
A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed.
Frederick J. SCHNELL, Baozhong CAI, Jason GATLIN, Patrick L. IVERSEN
Filed: 22 Dec 22
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
30 Nov 23
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: 9 Mar 23
Utility
Oligonucleotide analogues targeting human LMNA
31 Oct 23
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
Peptide Oligonucleotide Conjugates
5 Oct 23
Provided herein are oligonucleotides, peptides, and peptide-oligonucleotide-conjugates.
Gunnar J. Hanson, Ming Zhou
Filed: 6 Apr 23
Utility
Adeno-associated Virus Vectors for Treatment of Rett Syndrome
28 Sep 23
The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and/or delaying the onset of diseases associated with mutations in the mecp2 gene, such as Rett Syndrome.
Colin O'BANION
Filed: 19 Aug 21
Utility
Peptide oligonucleotide conjugates
22 Aug 23
Oligonucleotide analogues conjugated to carrier peptides are provided.
Gunnar J. Hanson
Filed: 7 May 20
Utility
Methods for Analyzing Aav Capsid Proteins
29 Jun 23
Provided are methods to characterize the VP1, VP2 and VPS capsid proteins in an adeno-associated virus (AAV) particle using liquid chromatography mass spectrometry, and/or ultraviolet (UV)-visible spectroscopy.
Kunal DAUD, Ju LI, Uditha DEALWIS
Filed: 30 Dec 20
Utility
Exon skipping oligomer conjugates for muscular dystrophy
9 May 23
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: 4 May 22
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
13 Apr 23
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: 27 Apr 22
Utility
Exon Skipping Compositions for Treating Muscular Dystrophy
6 Apr 23
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: 11 May 22
Utility
Processes for Preparing Phosphorodiamidate Morpholino Oligomers
16 Feb 23
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer).
Baozhong CAI, Mitchell MARTINI, Katie THOMAS, Ross SHIMABUKU
Filed: 15 Jun 22
Utility
Methods for Treating Muscular Dystrophy
9 Feb 23
The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy.
Edward M. KAYE
Filed: 31 Aug 18
Utility
Compositions and Methods for Restoring and Maintaining the Dystrophin-associated Protein Complex (Dapc)
9 Feb 23
Disclosed herein are methods of repairing or restoring a sarcoglycan complex or DAPC, stabilizing DAPC, restoring DAPC function, or increasing or enhancing expression of one or more components of a sarcoglycan complex or DAPC in a subject suffering from a muscular dystrophy.
Eric Pozsgai, Louise Rodino-Klapac
Filed: 15 Dec 20
Utility
Antisense oligomer compounds
17 Jan 23
A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed.
Frederick J. Schnell, Baozhong Cai, Jason Gatlin, Patrick L. Iversen
Filed: 18 Oct 18
Utility
Compositions for Treating Muscular Dystrophy
22 Dec 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: 3 Jan 22
Utility
Exon Skipping Oligomer Conjugates for Muscular Dystrophy
8 Dec 22
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.
Marco A. PASSINI, Gunnar J. HANSON
Filed: 26 May 22
Utility
Exon Skipping Oligomers for Muscular Dystrophy
17 Nov 22
Frederick Joseph SCHNELL, Anat SOMMELET
Filed: 25 Jul 19
Utility
Exon Skipping Compositions for Treating Muscular Dystrophy
17 Nov 22
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 Jun 22
Utility
Antisense Oligomers and Methods of Using the Same for Treating Diseases Associated with the Acid Alpha-glucosidase Gene
17 Nov 22
The present disclosure relates to modified antisense oligonucleotides.
Frederick Joseph SCHNELL
Filed: 9 Jun 21