187 patents
Page 7 of 10
Utility
Methods for Purification of Recombinant Aav Vectors
13 May 21
Provided herein are methods for the purification of recombinant adeno-associated virus (rAAV) vectors that can be used for gene transfer and specifically for gene therapy or vaccination.
Paulene McLean Quigley SHELDON, Peter S. GAGNON, Gina NICHOLS, Barbara A. THORNE
Filed: 28 Sep 20
Utility
Compositions and Methods for Treating Spinal Muscular Atrophy
29 Apr 21
The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN.
Marco A. PASSINI, Lamya S. SHIHABUDDIN, Catherine R. O'RIORDAN, Seng H. CHENG
Filed: 25 Sep 20
Utility
ctbe7dyxxzoc090qw1xqnuhs32t5vt3sd8y5m4pf6mqgmjsd2dyf
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
epthwnom6uiqlxq1orhzqmfou8f
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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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
bnhh1ck4i7qj66dsc1m3cpi0z0j4q1ccuv3vk2
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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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
qaxtoe54ieq6laka2oms65m9dpainxbgfhqinueyj9rk9fb4vbdrc
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
s54b4r4a2o2chqnr0r0t6ig8h6tfotb
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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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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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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28 Jan 21
The present disclosure provides methods of treating an antibody-mediated disorder (e.g., an autoimmune disease) using an FcRn antagonist, e.g., a binding polypeptide (e.g., an antibody and/or an immunoadhesin), comprising a modified Fc domain.
Brian Mackness, Huawei Qiu
Filed: 24 Jul 20
Utility
k6165jgcvte4go6hts28qpbopol5esyfzkrl2w1fjysgekh7yprh8
14 Jan 21
The present application provides methods of treating a disease, such as Pompe disease, in a subject, comprising detecting an erythropoiesis biomarker in a sample of the subject after administration of methotrexate and a therapeutic agent to the subject, and administering further treatment with or without concurrently administering additional immune tolerance induction or immunosuppression therapy based on the level of the erythropoiesis biomarker.
Alexandra JOSEPH, Johnson TRAN, Susan M. RICHARDS
Filed: 21 Dec 18
Utility
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14 Jan 21
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject.
James DODGE, Lamya S. SHIHABUDDIN, Marco A. PASSINI, Seng H. CHENG, Catherine O'RIORDAN
Filed: 24 Jun 20
Utility
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6 Jan 21
Provided herein are isolation processes and the associated hardware to allow fluid streams to be isolated from a sterilized system (e.g., a sterile process vessel) that contains a sterile process.
Robert Snow
Filed: 20 Sep 20
Utility
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9 Dec 20
Methods for quantifying the amount of drug present in a prodrug composition are provided.
Kim Alving, Bing Wang
Filed: 5 May 20
Utility
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9 Dec 20
Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
Marco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
Filed: 17 Feb 20
Utility
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2 Dec 20
Provided herein are seed train processes and methods of producing a recombinant protein that include the use of these seed train processes.
Michael Bruninghaus, Konstantin Konstantinov, Benjamin Wright, Weichang Zhou
Filed: 23 Feb 20
Utility
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25 Nov 20
Provided herein are methods of performing chromatography with gamma-irradiated chromatography resin that include providing a chromatography column including a gamma-irradiated chromatography resin; performing a first cycle of chromatography through the column, where the cycle includes exposing the chromatography resin to a denaturing buffer; and performing at least one additional cycle of chromatography through the column.
Rahul Godawat, Veena Warikoo, Rohan Patil, Konstantin Konstantinov, Venkat Kishore Ryakala, Mahsa Rohani
Filed: 13 Aug 20