290 patents
Page 10 of 15
Utility
Polynucleotide Agents Targeting SERPINC1 (AT3) and Methods of Use Thereof
29 Apr 21
The invention relates to polynucleotide agents targeting the Serpinc1 (AT3) gene, and methods of using such polynucleotide agents to inhibit expression of Serpinc1 and to treat subjects having a bleeding disorder, e.g., a hemophilia.
Gregory Hinkle
Filed: 10 Jul 20
Utility
In-line Product Monitoring In Integrated Continuous Bio-manufacturing
22 Apr 21
Methods for controlling a biological manufacturing system include directing a light beam to pass through a wall of a vessel containing a first fluid generated by the biological manufacturing system, measuring an angle of refraction of the light beam in the first fluid, the angle of refraction corresponding to an angle between a propagation direction of the light beam in the first fluid and a normal to an interface between the vessel wall and the first fluid, determining information about the first fluid based on the measured angle of refraction, and adjusting a parameter of the biological manufacturing system based on the information about the first fluid.
Robert Snow, Joseph P. Kutzko, Xuezhen Kang
Filed: 21 Oct 20
Utility
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13 Apr 21
Methods to introduce highly phosphorylated mannopyranosyl oligosaccharide derivatives containing mannose-6-phosphate (M6P), or other oligosaccharides bearing other terminal hexoses, to carbonyl groups on oxidized glycans of glycoproteins while retaining their biological activity are described.
Yunxiang Zhu
Filed: 12 Jun 19
Utility
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8 Apr 21
The present invention provides methods for improved label-free absolute quantification of relatively low abundant polypeptides by liquid chromatography/mass spectrometry analysis of peptide products obtained from simple or complex polypeptide mixtures.
Martha STAPELS, Michelle BUSCH
Filed: 17 Dec 20
Utility
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6 Apr 21
Provided herein are methods of processing a fluid that include the use of one or both of a circuit system including a tangential flow filtration (TFF) unit and a circuit system including a tangential flow virus filtration (TFVF) unit.
Tarl Vetter, Michael Coolbaugh, Veena Warikoo, Konstantin Konstantinov
Filed: 15 Aug 17
Utility
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25 Mar 21
Systems for measuring a product quality attribute of an analyte of a biological sample include a first flow control device, a sample purification device, a second flow control device in fluid communication with first and second sample analyzers, where the first sample analyzer includes a first chromatography column, and a control unit configured so that during operation of the system, the control unit adjusts a configuration of the second flow control device to direct a portion of the biological sample to one of the first and second sample analyzers, and determines a product quality attribute of an analyte of the biological sample based on an analysis of the portion of the biological sample by the one of the first and second sample analyzers.
Marina Hincapie, Victoria Berger
Filed: 22 Sep 20
Utility
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23 Mar 21
This invention relates to a method of preparing inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
Craig S. Siegel, Rayomand Gimi, Michael Reardon, Jin Zhao
Filed: 14 Feb 20
Utility
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23 Mar 21
The present invention relates to methods of treating patients with advanced forms of cancer, such as clear cell renal cell carcinoma, in which X4P-001 is administered in order to reduce angiogenic escape that typically occurs with TKI therapy.
Paula Marie Ragan, Robert D. Arbeit, Kieran James Crowley
Filed: 14 Dec 16
Utility
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18 Mar 21
The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.
Edward H. Schuchman, Robert J. Desnick, Gerald F. Cox, Laura P. Andrews, James M. Murray
Filed: 26 May 20
Utility
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4 Mar 21
The present invention relates to antibody molecules, in particular antibody molecules that bind Transforming Growth Factor beta (TGFβ) and uses thereof.
Steven R. Ledbetter, Celia Patricia Hart, Robert G. Holgate, Lutz U. Jermutus, Catriona L. Buchanan, Alexander R. Duncan, Donna K. Finch
Filed: 19 Aug 20
Utility
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2 Mar 21
The present disclosure provides anti-complement factor Bb antibodies, and compositions comprising the antibodies.
Sandip Panicker, Graham Parry, Karen Sue Christopherson, Tony SangYoung Byun
Filed: 3 Apr 17
Utility
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18 Feb 21
Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence.
Lisa M. STANEK, Adam PALERMO, Brenda RICHARDS, Sergio Pablo SARDI, Catherine O'RIORDAN, Antonius SONG
Filed: 28 Jul 20
Utility
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16 Feb 21
Provided herein are methods of reducing bioburden of (e.g., sterilizing) a chromatography resin that include exposing a container including a composition including a chromatography resin and at least one antioxidant agent and/or chelator to a dose of gamma-irradiation sufficient to reduce the bioburden of the container and the chromatography resin, where the at least one antioxidant agent and/or chelator are present in an amount sufficient to ameliorate the loss of binding capacity of the chromatography resin after/upon exposure to the dose of gamma-irradiation.
Rahul Godawat, Veena Warikoo, Rohan Patil, Konstantin Konstantinov, Venkat Kishore Ryakala
Filed: 21 Aug 18
Utility
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16 Feb 21
The invention as disclosed herein provides a method for purifying a non-antibody protein from solution, comprising a chromatography step wherein the solution is passed over an affinity construct containing an affinity ligand-coupled solid support, wherein the affinity construct is associated with a bioprocess unit operation, and isolating the non-antibody protein from solution.
Veena Warikoo, Kevin Brower
Filed: 4 Dec 17
Utility
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16 Feb 21
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject.
Catherine O'Riordan, Samuel Wadsworth
Filed: 29 Dec 17
Utility
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11 Feb 21
Compositions and methods for treating macular degeneration are disclosed.
Samuel WADSWORTH, Abraham SCARIA, Chi-Chao CHAN
Filed: 31 Mar 20
Utility
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11 Feb 21
Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle).
Xiaoying JIN, Catherine O'RIORDAN, Lin LIU, Kate ZHANG
Filed: 14 Aug 17
Utility
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9 Feb 21
The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS).
Marco A. Passini, Greg Stewart, James C. Dodge
Filed: 1 Jul 16
Utility
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4 Feb 21
The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase.
Carol A. NELSON, Bruce M. WENTWORTH, Ronald K. SCHEULE, Timothy E. WEEDEN, Nicholas P. CLAYTON
Filed: 5 May 20
Utility
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2 Feb 21
The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group.
Yunxiang Zhu, Seng H. Cheng, Canwen Jiang, Luis Z. Avila
Filed: 6 Feb 19