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RARE Ultragenyx Pharmaceutical

APP
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Methods and Compositions for Treating Glycogen Storage Diseases
20 Jan 22
This invention provides a variety of novel adeno-associated vims (AAV) vectors for gene therapy applications in the treatment of glycogen storage disease type 1a (GSD-Ia).
Christopher Tipper, Kelly Reed Clark, Samuel Wadsworth
Filed: 18 Dec 19
GRANT
Utility
Methods for treating hypophosphatemic disorders
21 Dec 21
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: 26 Mar 20
APP
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Gene Therapy for Treating Propionic Acidemia
16 Sep 21
This present disclosure provides adeno-associated viral vectors, 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: 1 Oct 19
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Methods and Compositions for the Treatment of Amyloidosis
29 Jul 21
Methods and compositions for the treatment or prevention of amyloidosis are provided.
Emil D. Kakkis, Michel Claude Vellard, Andrzej Swistowski
Filed: 8 Oct 20
GRANT
Utility
Methods of heat inactivation of adenovirus
25 May 21
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: 28 Mar 17
GRANT
Utility
Sialic acid analogs
12 Jan 21
The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
Emil Kakkis, Steven Jungles, He Zhao
Filed: 16 Sep 19
APP
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Creatine Prodrugs, Compositions and Methods of Use Thereof
6 Jan 21
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: 2 Dec 18
APP
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Effective and Efficient Control of Serum Phosphate for Optimal Bone Formation
21 Oct 20
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: 25 Mar 20
APP
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Engineered Producer Cell Lines and Methods of Making and Using the Same
14 Oct 20
This application relates to recombinant adeno-associated virus (rAAV) packaging and/or producer cell lines which have been engineered to reduce expression and/or activity of one or more genes and/or proteins to increase rAAV titers.
Aubrey R. Tiernan, Nicholas Richards
Filed: 8 Apr 20
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Sialylated Glycoprotein Compositons and Uses Thereof
9 Sep 20
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: 8 Jan 20
APP
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Therapeutics for Glycogen Storage Disease Type III
13 May 20
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 Prakash BHASKARAN, Christian W. COBAUGH, Sean Christopher DAUGHERTY
Filed: 30 May 18
GRANT
Utility
Methods for treating hypophosphatemic disorders
4 May 20
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: 28 May 15
APP
Utility
Sialic Acid Analogs
29 Apr 20
The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
Emil KAKKIS, Steven JUNGLES, He ZHAO
Filed: 15 Sep 19
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Scalable Method for Producing Transfection Reagents
22 Apr 20
The invention provides easy-to-scale methods of creating DNA/transfection reagent master mixes for transfecting cells in culture.
Jan Panteli, Ying Jing
Filed: 8 May 18
APP
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Solid Compositions of Triglycerides and Uses Thereof
4 Mar 20
The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof.
John KLOPP, Gabrielle MORRIS, Emil KAKKIS, Steven JUNGLES
Filed: 18 Jun 19
APP
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Methods for Enhancing Yield of Recombinant Adeno-associated Virus
12 Feb 20
The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), e.g., comprising the addition of a surfactant, e.g., Triton X-100, to a producer cell culture medium.
Ying Jing, Jan Panteli, Kelly Reed Clark
Filed: 21 Mar 18
APP
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Cell Culture Methods Involving Hdac Inhibitors or Rep Proteins
29 Jan 20
The invention relates to methods of culturing cells, generating cell lines, and delivering polynucleotides to cells involving the use of HDAC inhibitors and/or adeno-associated virus (AAV) rep proteins.
Aubrey R. Tiernan, Christopher Tipper
Filed: 21 Mar 18
GRANT
Utility
Sialic acid analogs
28 Oct 19
The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
Emil Kakkis, Steven Jungles, He Zhao
Filed: 30 Jul 18
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