38 patents
Utility
DUX4 Inhibitors and Methods of Use Thereof
2 Nov 23
This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs.
Sean Christopher DAUGHERTY, Lishan CHEN, Francis Michael SVERDRUP
Filed: 1 Sep 21
Utility
Use of tonicifying agents to enhance recombinant adeno-associated virus yield
24 Oct 23
The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising culturing producer cells in media with increased osmolality.
Ying Jing, Jan Panteli
Filed: 13 Oct 17
Utility
Methods for treating tumor-induced osteomalacia
3 Oct 23
The present invention provides compositions and methods for treating tumor-induced osteomalacia.
Emil D. Kakkis, Javier San Martin, Tomohiro Sudo
Filed: 12 Nov 21
Utility
Creatine prodrugs, compositions and methods of use thereof
12 Sep 23
The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.
Yiumo Chan, Emil D. Kakkis, Alexandra Trotier-Faurion, William F. Brubaker, Arjun Natesan, Paul Lee, Sharyl Fyffe-Maricich, David Lapointe, Mike E. Lizarzaburu
Filed: 28 Mar 22
Utility
Compositions and Methods for Reducing Reverse Packaging of Cap and Rep Sequences In Recombinant Aav
27 Jul 23
The present disclosure provides compositions including recombinant nucleic acid constructs, vectors, and host cells, and methods of their use for reducing reverse packaging of cap and/or rep DMA sequences in the production of recombinant adeno-associated vims (rAAV).
Brady Camplin, Stewart Craig, Matthew Scott Fuller, Samuel Wadsworth
Filed: 19 Mar 21
Utility
Methods and Compositions for the Treatment of Amyloidosis
27 Apr 23
Methods and compositions for the treatment or prevention of amyloidosis are provided.
Emil D. KAKKIS, Michel Claude Vellard, Andrzej Swistowski
Filed: 6 Sep 22
Utility
Methods for Enhancing Recombinant Adeno-associated Virus Yield
27 Apr 23
The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof.
Jan Thomas Panteli, Cameron Tage Fulco, John Everett Allen, Mingyang Jiang, Rachael C. Hogan
Filed: 15 Mar 21
Utility
Gene Therapy for Treating Propionic Acidemia
27 Apr 23
This present disclosure provides recombinant adeno-associated virus (rAAV) and methods of their use in gene therapy for treating propionic acidemia (PA).
Matthew Scott Fuller, Samuel Wadsworth, Kelly Reed Clark, Sean Christopher Daugherty, Stewart Craig
Filed: 30 Mar 21
Utility
Chaotropic Agents for Reducing Formation of Double-stranded Rna
16 Mar 23
The present disclosure provides methods of reducing, minimizing, or inhibiting the formation of double-stranded ribonucleic acid (dsRNA) during the preparation of ribonucleic acid (RNA), such as messenger ribonucleic acid (mRNA), by adding at least one chaotropic agent to a starting reaction mixture.
Xijun PIAO, Wayne CHANG, Benjamin E. LINDENMUTH, Xiaowei WANG
Filed: 4 Feb 21
Utility
Gene Therapy for Treating CDKL5 Deficiency Disorder
23 Feb 23
This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated vims (rAAV), and methods of their use in gene therapy for treating CDKL5 deficiency disorder (CDD).
Sharyl Lynne Fyffe-Maricich, Matthew Scott Fuller, Margaret Caroline Wright, Lorelei Ioana Stoica, Stewart Craig, Sean Christopher Daugherty
Filed: 11 Feb 21
Utility
Methods for Improved Therapeutic Use of Recombinant Aav
3 Nov 22
Provided herein are methods for managing host immune responses to improve therapeutic outcomes in adeno-associated virus (AAV)-mediated gene therapy.
Barbara Anne Sullivan, Kathleen Phillips McKeever
Filed: 1 Oct 20
Utility
Therapeutics for Glycogen Storage Disease Type III
27 Oct 22
This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity.
Kiyoshi TACHIKAWA, Carlos Gustavo PEREZ-GARCIA, Padmanabh CHIVUKULA, Hari BHASKARAN, Christian W. COBAUGH, Sean Christopher DAUGHERTY
Filed: 4 May 22
Utility
Creatine Prodrugs, Compositions and Methods of Use Thereof
21 Jul 22
The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.
Yiumo CHAN, Emil D. KAKKIS, Alexandra TROTIER-FAURION, William F. BRUBAKER, Arjun NATESAN, Paul LEE, Sharyl FYFFE-MARICICH, David LAPOINTE, Mike E. LIZARZABURU
Filed: 28 Mar 22
Utility
Compositions and Methods for Treatment of Hemochromatosis
14 Jul 22
This application provides polynucleotides comprising a coding sequence for a functionally active hereditary hemochromatosis protein (HFE) or a functionally active fragment thereof.
Sean Christopher DAUGHERTY, Timothy Preston WONG, Rosaline Do CARSON, Jason Robert CATALDO
Filed: 22 May 20
Utility
Therapeutics for glycogen storage disease type III
5 Jul 22
This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity.
Kiyoshi Tachikawa, Carlos Gustavo Perez-Garcia, Padmanabh Chivukula, Hari Bhaskaran, Christian W. Cobaugh, Sean Christopher Daugherty
Filed: 31 May 18
Utility
Sialylated Glycoprotein Compositons and Uses Thereof
30 Jun 22
The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
Steven JUNGLES, Gabrielle MORRIS, Jeff GRUBB, Michael VELLARD, Emil D. KAKKIS
Filed: 11 Nov 21
Utility
Creatine prodrugs, compositions and methods of use thereof
17 May 22
The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.
Yiumo Chan, Emil D. Kakkis, Alexandra Trotier-Faurion, William F. Brubaker, Arjun Natesan, Paul Lee, Sharyl Fyffe-Maricich, David Lapointe, Mike E. Lizarzaburu
Filed: 3 Dec 18
Utility
Effective and Efficient Control of Serum Phosphate for Optimal Bone Formation
5 May 22
The present invention provides compositions and methods for treating a hypophosphatemic disorder, such as X-linked hypophosphatemia (XLH).
Emil D. KAKKIS, Javier SAN MARTIN, Tomohiro SUDO
Filed: 12 Nov 21
Utility
Gene Therapy Constructs for Treating Wilson Disease
24 Mar 22
This application relates to adeno-associated viral vectors encoding a truncated yet functional ATP7B for use in gene therapy for treating Wilson disease (WD).
Christine Livingston, Samuel Wadsworth
Filed: 3 Jan 20
Utility
Methods of Heat Inactivation of Adenovirus
10 Mar 22
The present disclosure generally relates to methods of protecting the genomic integrity and/or biological activity of AAV viral particles in a sample containing both AAV particles and helper virus particles during heat inactivation.
Christopher J. Morrison, James D. Maratt
Filed: 23 Apr 21