290 patents
Page 13 of 15
Utility
SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
8 Jul 20
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
Akin Akinc, Alfica Sehgal, Ivanka Toudjarska, Donald Foster, Stuart Milstein, Brian Bettencourt, Martin A. Maier, Klaus Charisse, Satyanarayana Kuchimanchi, Kallanthottathil G. Rajeev, Muthiah Manoharan
Filed: 13 Dec 18
Utility
VARIANT RNAi
8 Jul 20
Provided herein are RNAi molecules for treating Huntington's disease.
Catherine R. O'RIORDAN, Adam PALERMO, Brenda RICHARDS, Lisa M. STANEK
Filed: 20 Sep 18
Utility
Methods for purification of recombinant AAV vectors
29 Jun 20
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: 11 Jun 18
Utility
Methods for Treating Proteinopathies
24 Jun 20
This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound.
Seng H. CHENG, Lamya SHIHABUDDIN, Sergio Pablo SARDI
Filed: 1 Mar 20
Utility
Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
15 Jun 20
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: 24 May 18
Utility
Method of Preparing Glucosylceramide Synthase Inhibitors
10 Jun 20
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: 13 Feb 20
Utility
Methods and Compositions for Treating a Bleeding Event In a Subject Having Hemophilia
27 May 20
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to treat a bleeding event in a subject having a hemophilia (e.g., with or without inhibitors).
Akin AKINC
Filed: 9 Jul 18
Utility
Methods for Producing Preparations of Recombinant Aav Virions Substantially Free of Empty Capsids
20 May 20
Methods for separating AAV empty capsids from mixtures of AAV vector particles and AAV empty capsids are described.
Guang QU, John Fraser WRIGHT
Filed: 11 Aug 19
Utility
Methods for Introducing Mannose 6-PHOSPHATE and Other Oligosaccharides Onto Glycoproteins and Its Application Thereof
13 May 20
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: 11 Jun 19
Utility
Methods of controlling the formation of disulfide bonds in protein solutions
11 May 20
Disclosed herein are methods that have been developed to control the formation of disulfide bonds between polypeptides of a multimeric protein produced by a bioprocess.
Kevin P. Brower, Chris Hwang, Rao Koduri, Konstantin B. Konstantinov, Veena Warikoo, Marcella Yu, Jin Yin
Filed: 24 Mar 15
Utility
Method of identifying risk for thyroid disorder
11 May 20
A method for identifying a patient that is at risk for developing a thyroid disorder that occurs subsequent to treatment with a regimen that depletes lymphocytes, comprising determining whether antibodies directed against thyroid peroxidase or thyroid microsomes are present in the patient, wherein if the antibodies are present in the patient then the patient is at increased risk for developing a thyroid disorder.
David H. Margolin
Filed: 9 May 17
Utility
Hyperglycosylated Binding Polypeptides
6 May 20
Provided are binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof, comprising a CH1 domain (e.g., a human IgG1 CH1 domain), wherein the CH1 domain has an engineered N-linked glycosylation site at amino acid position 114, according to Kabat numbering.
Clark PAN, Huawei QIU, Pradeep DHAL, Bo CHEN, Diego GIANOLIO
Filed: 16 Oct 19
Utility
Compositions and methods for treating and preventing macular degeneration
4 May 20
Compositions and methods for treating macular degeneration are disclosed.
Samuel Wadsworth, Abraham Scaria, Chi-Chao Chan
Filed: 16 Apr 14
Utility
SCFV-FC Dimers That Bind Transforming Growth FACTOR-BETA1 with High Affinity, Avidity and Specificity
29 Apr 20
An scFv-Fc dimer binds and neutralizes TGFβ1 selectively and with high affinity and avidity.
Huawei Qiu, Clark Pan, Julie Bird
Filed: 29 Oct 19
Utility
Elution of Biomolecules from Multi-modal Resins Using Mes and Mops As Mobile Phase Modifiers
29 Apr 20
The present invention relates to methods for purifying or enriching a biomolecule using multimodal resins and an elution buffer containing a Good's buffer.
Rahul Godawat, Daniel Cummings, Veena Warikoo
Filed: 22 Dec 19
Utility
Treatment of myocardial infarction using TGF-β antagonists
27 Apr 20
Disclosed herein is a method of treating a patient suffering a myocardial infarction, particularly an acute myocardial infarction, or of reducing an adverse consequence of a myocardial infarction in a patient comprising administering an antagonist of TGF-β to the patient during the acute stage of the myocardial infarction.
Geoffrey Y. Akita, Scott Lonning, Richard C Gregory, Jr., Amelia B Kudej
Filed: 11 Dec 16
Utility
Gene therapy for neurometabolic disorders
27 Apr 20
The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS).
Marco A. Passini, James Dodge
Filed: 1 Nov 07
Utility
Biomarkers of Proteopathies and Uses Thereof
22 Apr 20
Provided herein are methods of determining the efficacy of a treatment for a proteopathy, diagnosing a proteopathy in a subject, determining a subject's risk of developing a proteopathy, determining the stage of a proteopathy in a subject, monitoring a proteopathy in a subject, selecting a treatment for a proteopathy for a subject, and selecting a subject for a clinical trial that include determining a level of at least one sphingolipid in a sample including a biological fluid from the subject.
Sergio Pablo Sardi, Clemens Scherzer
Filed: 23 Mar 17
Utility
VARIANT RNAi
8 Apr 20
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: 9 Sep 19
Utility
Gene Therapy for Amyotrophic Lateral Sclerosis and Other Spinal Cord Disorders
1 Apr 20
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject.
James DODGE, Lamya SHIHABUDDIN, Catherine O'RIORDAN
Filed: 19 Aug 19