Patents Issued in January 25, 2024
  • Publication number: 20240024470
    Abstract: The disclosure provides a method of reducing the likelihood of forming a T cell-mediated allograft vasculopathy lesion in a mammalian transplant recipient comprising transplanting an allograft from a donor to a recipient and administering a therapeutically effective amount of an anti-C5 antibody, or antigen-binding fragment thereof, to the recipient, wherein the anti-C5 antibody, or antigen-binding fragment thereof reduces the likelihood of forming an allograft vasculopathy lesion in the allograft, compared to the absence of treatment with an anti-C5 antibody, or antigen-binding fragment thereof.
    Type: Application
    Filed: May 17, 2023
    Publication date: January 25, 2024
    Inventors: Jordan S. Pober, Daniel Jane-Wit, Lingfeng Qin, Yi Wang
  • Publication number: 20240024471
    Abstract: The disclosure provides pharmaceutical compositions comprising an anti-PD-1 antibody or an anti-PD-L1 antibody. In some aspects, the pharmaceutical compositions are formulated for subcutaneous delivery. In some aspects, the pharmaceutical compositions further comprise an endoglycosidase hydrolase enzyme. Other aspects of the present disclosure are directed to methods of subcutaneously delivering a pharmaceutical composition comprising an anti-PD-1 antibody or an anti-PD-L1 antibody.
    Type: Application
    Filed: August 21, 2023
    Publication date: January 25, 2024
    Applicants: Bristol-Myers Squibb Company, Halozyme, Inc.
    Inventors: Masano HUANG, Thomas Arthur HABY, Mehrnaz KHOSSRAVI, Scott Aaron HART, Rao Venkatramana MANTRI, Heather Elizabeth VEZINA, Amit ROY, Bindu Purnima MURTHY, Urvi Ashish ARAS, Kinjal SANGHAVI, Xiaochen ZHAO, Akintunde BELLO
  • Publication number: 20240024472
    Abstract: Pharmaceutical formulations comprising an antibody that specifically binds to human interleukin-4 receptor alpha (hIL-4R?) are provided. The formulations may contain, in addition to an anti-IL-4R? antibody, one or more buffers, at least one amino acid, at least one sugar, and a surfactant comprising a polyethylene glycol or a poloxamer. In one aspect, the pharmaceutical formulations do not have appreciable subvisible particle formation in the presence of lipase, and exhibit a substantial degree of antibody stability during storage and after being subjected to thermal and other physical stresses.
    Type: Application
    Filed: May 2, 2023
    Publication date: January 25, 2024
    Inventor: Xiaolin Tang
  • Publication number: 20240024473
    Abstract: The present disclosure relates to a stage-specific process for manufacturing a population of neutrophils, such as chimeric antigen receptor-expressing (CAR-expressing) neutrophils (e.g., T cells and natural killer (NK) cells), from human pluripotent stem cells (hPSCs) using defined media and related compositions, kits, and methods of use (e.g., targeted cancer immunotherapy). Stage-specific processes for generating neutrophils and chimeric antigen receptor (CAR) neutrophils from human pluripotent stem cells (hPSCs) using chemically defined, feeder-free platforms and stage-specific morphogens; cell lines; pharmaceutical compositions; a method of treating cancer; and a kit are within the scopes of this disclosure.
    Type: Application
    Filed: December 10, 2021
    Publication date: January 25, 2024
    Inventors: Xiaoping BAO, Qing DENG, Yun CHANG, Ramizah MOHD SABRI
  • Publication number: 20240024474
    Abstract: The present invention provides compositions and methods for treating cancer.
    Type: Application
    Filed: November 12, 2021
    Publication date: January 25, 2024
    Inventors: David Avigan, Jacalyn Rosenblatt, Donald Kufe
  • Publication number: 20240024475
    Abstract: An antigen-binding protein targeting CD70. In particular, the present invention relates to a chimeric antigen receptor containing the antigen binding protein and the use thereof, and cells containing or expressing the antigen binding protein. The antigen-binding protein binds to a CD70 protein at 1 nM of a KD value. The chimeric antigen receptor containing the antigen-binding protein has strong specific recognition and binding abilities to the CD70 protein. The cells containing or expressing the antigen-binding protein can secrete cytokines IL-2 and IFN-?.
    Type: Application
    Filed: November 30, 2021
    Publication date: January 25, 2024
    Inventors: Wenbo Wang, Yanni Lin
  • Publication number: 20240024476
    Abstract: The compositions and methods described herein are directed to treating solid tumor using CAR T therapy. For example, the compositions include CAR T cells comprising an extracellular domain that binds FCR1, MSLN, GPC-3, ALPP, CD70, CLDN6, ROR1, CD205, ACPP, ADAM12, or CLDN18.2.
    Type: Application
    Filed: January 6, 2022
    Publication date: January 25, 2024
    Applicants: Innovative Cellular Therapeutics Holdings, Ltd., Innovative Cellular Therapeutics, Inc.
    Inventors: Chengfei Pu, Zhiyuan Cao, Xiaogang Shen, Wensheng Wang, Beibei Jia, Dongqi Chen, Xiaoqiang Xu, Xudong Tang, Wei Ding, Xianyang Jiang, Yuzhe Peng, Guiting Han, Le Tian, Zhao Wu, Lei Xiao
  • Publication number: 20240024477
    Abstract: The invention relates to a T cell receptor (TCR) which is HIV-1 specific and HLA-E restricted. Particularly, the TCR is capable of binding to a peptide of RMYSPTSIL or a peptide of RMYSPTSIL in complex with HLA-E, or a peptide of ILVESPAVL or a peptide of ILVESPAVL in complex with HLA-E. The invention also relates to a nucleic acids and vector encoding the TCR.
    Type: Application
    Filed: December 2, 2021
    Publication date: January 25, 2024
    Inventors: Honbing YANG, Geraldine GILLESPIE, Margarida REI, Simon BRACKENRIDGE, Andrew MCMICHAEL, Hong SUN
  • Publication number: 20240024478
    Abstract: Compositions and methods for reducing HLA-A protein expression in a cell comprising genetically modifying HLA-A for use e.g., in adoptive cell transfer therapies.
    Type: Application
    Filed: June 22, 2023
    Publication date: January 25, 2024
    Applicant: Intellia Therapeutics, Inc.
    Inventors: Surbhi Goel, Yong Zhang, Reynald Michael Lescarbeau, Bradley Andrew Murray, Srijani Sridhar
  • Publication number: 20240024479
    Abstract: Modified natural killer (NK) or T cells expressing hematopoietic growth factor receptors are provided. In some aspects, the modified NK cells or T cells express a thrombopoietin receptor, an erythropoietin receptor, or a chimeric polypeptide including an extracellular domain of a thrombopoietin receptor, a transmembrane domain, and an intracellular domain including an interleukin-2 receptor beta intracellular signaling domain. Methods of treating a subject with cancer are also provided, including administering the modified NK cells or T cells to the subject in combination with a thrombopoietin receptor agonist or erythropoietin receptor agonist, and in some example, interleukin-2, particularly reduced or low-dose amounts of IL-2.
    Type: Application
    Filed: October 3, 2023
    Publication date: January 25, 2024
    Applicant: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
    Inventors: Richard W. Childs, David S.J. Allan
  • Publication number: 20240024480
    Abstract: An object is to provide a PRAM-binding molecule. This object is achieved by a PRAM-binding molecule comprising a heavy-chain variable region containing a heavy-chain CDR1 comprising the amino acid sequence represented by SEQ ID NO: 1, a heavy-chain CDR2 comprising the amino acid sequence represented by SEQ ID NO: 2, and a heavy-chain CDR3 comprising the amino acid sequence represented by SEQ ID NO: 3, and/or a light-chain variable region containing a light-chain CDR1 comprising the amino acid sequence represented by SEQ ID NO: 9, a light-chain CDR2 comprising the amino acid sequence represented by SEQ ID NO: 10, and a light-chain CDR3 comprising the amino acid sequence represented by SEQ ID NO: 11.
    Type: Application
    Filed: December 7, 2021
    Publication date: January 25, 2024
    Inventors: Hiroshi SHIKU, Yasushi AKAHORI, Hiroyuki HIRATSUKA
  • Publication number: 20240024481
    Abstract: The present invention provides a pharmaceutical composition comprising an aqueous solution of an ibuprofen solubilizing agent and ibuprofen, the ibuprofen solubilizing agent being in an effective amount such that the ibuprofen in the solution remains soluble at concentrations from 100 mg/mL to 5 mg/mL without undergoing a phase transition. The invention further provides a method of treating a condition chosen from pain, inflammation, fever, and/or patent ductus arteriosis, comprising administering to a patient in need thereof an effective amount of an aqueous solution a ibuprofen solubilizing agent and ibuprofen, the ibuprofen solubilizing agent being in an effective amount such that the ibuprofen in the solution remains soluble at concentrations from 100 mg/mL to 5 mg/mL without undergoing a phase transition, as well as a method for manufacturing the composition.
    Type: Application
    Filed: October 3, 2023
    Publication date: January 25, 2024
    Inventors: Leo PAVLIV, Andrew VILA
  • Publication number: 20240024482
    Abstract: The disclosure relates to the field of medicine, in particular to the field of veterinary medicine. The disclosure relates to a pharmaceutical (medicament) formulation of glucocorticoids, especially ciclesonide or a pharmaceutically acceptable derivative thereof.
    Type: Application
    Filed: October 5, 2023
    Publication date: January 25, 2024
    Inventors: Michael AVEN, Benjamin FRANZMANN, Matthias HAUSMANN
  • Publication number: 20240024483
    Abstract: The present disclosure discloses a homogeneous pharmaceutical excipient sodium stearyl fumarate with flaky structure and preparation method thereof, which belongs to the field of pharmaceutical excipient refining technology. The method of the present disclosure includes: heating and dissolving sodium stearyl fumarate to be treated in an ethanol solution and filtering to remove impurities, then adding crystal seeds for recrystallization. The crystallized sodium stearyl fumarate with flaky structure has narrow particle size distribution, average particle size of 5-15 ?m, intact flaky structure, good uniformity and purity up to 99.6% or more, and it is not easy-caking and can keep the powder state for a long time.
    Type: Application
    Filed: September 9, 2022
    Publication date: January 25, 2024
    Applicant: ANHUI SUNHERE PHARMACEUTICAL EXCIPIENTS CO., LTD.
    Inventors: Yong BI, Jiao WU, Liting REN, Huijuan BAO, Yunhe SHAO
  • Publication number: 20240024484
    Abstract: The purpose of the present invention is to provide a pharmaceutical composition that comprises a specific compound and exhibits a superior preservation efficacy, the specific compound being stable within the pharmaceutical composition, and to provide methods for improving the stability of the specific compound within the pharmaceutical composition and the preservation efficacy of the pharmaceutical composition. The pharmaceutical composition according to the present invention comprises isopropyl (6-{[4-(pyrazol-1-yl)benzyl](pyridin-3-ylsulfonyl)aminomethyl}pyridin-2-ylamino)acetate or a salt thereof, and further comprises edetic acid or a salt thereof.
    Type: Application
    Filed: October 2, 2023
    Publication date: January 25, 2024
    Inventor: Yoko ENDO
  • Publication number: 20240024485
    Abstract: Drug products adapted for nasal delivery comprising formulations with epinephrine and devices comprising such formulations are provided. Methods of treating anaphylaxis with epinephrine products are also provided.
    Type: Application
    Filed: June 7, 2023
    Publication date: January 25, 2024
    Inventors: Richard LOWENTHAL, Edward T. MAGGIO, Robert G. BELL, Pratik SHAH
  • Publication number: 20240024486
    Abstract: Disclosed is a pharmaceutical composition (F) for topical use, comprising at least one active pharmaceutical ingredient and, as thickening agent, a polymer (P) in the form of a pulverulent solid consisting of monomeric units derived from partially or totally salified glutamic acid (GA) and monomer units of at least one crosslinking agent (AR) having at least two glycidyl functions.
    Type: Application
    Filed: November 30, 2021
    Publication date: January 25, 2024
    Inventors: Miruna BODOC, Stéphane MONTEILLET
  • Publication number: 20240024487
    Abstract: Disclosed herein are self-repairing biomimetic lubricant compounds, compositions comprising the same, and methods of making and using the same.
    Type: Application
    Filed: November 5, 2021
    Publication date: January 25, 2024
    Inventors: Shyni Varghese, Anna Gilpin, Jiaul Hoque
  • Publication number: 20240024488
    Abstract: The present disclosure features compounds useful for the treatment of BAF complex-related disorders.
    Type: Application
    Filed: November 22, 2021
    Publication date: January 25, 2024
    Inventors: Matthew NETHERTON, Francois BRUCELLE, Jing DENG, Johannes H. VOIGT
  • Publication number: 20240024489
    Abstract: The present invention provides zwitterionic oligosaccharides, in particular fragments of the surface polysaccharides from Shigella sonnei and Shigella sonnei conjugates comprising them.
    Type: Application
    Filed: November 22, 2021
    Publication date: January 25, 2024
    Inventors: Laurence MULARD, Debashis DHARA, Helene PFISTER, Julie PAOLETTI, Armelle PHALIPON, Catherine GUERREIRO-INVERNO
  • Publication number: 20240024490
    Abstract: The present disclosure relates to bifunctional chemical conjugation molecules, which find utility as modifiers of target substrates. The present disclosure includes multifunctional compounds comprising an enzyme binding moiety, a chemical linker moiety, and a target binding moiety, which may further include an electrophilic reactive group. Molecules according to the present invention find use making substrate modifications such as post-translational modifications to proteins that are not the natural substrate of the enzyme. Diseases or disorders may be treated or prevented with molecules of the present disclosure.
    Type: Application
    Filed: April 8, 2022
    Publication date: January 25, 2024
    Inventors: Amit Choudhary, Veronika Shoba, Arghya Deb, Tuan Nguyen, Sophia Lai, Dhanushka Munkanatta Godage, Praveen Tiwari, Ashley Modell, Sachini Siriwardena
  • Publication number: 20240024491
    Abstract: Provided are compounds, compositions, and methods of repolarizing a tumor associated macrophage (TAM), reducing macrophage-mediated inflammation, and treating a disease. A compound or pharmaceutical composition may be administered to a subject in need thereof, where the compound comprises a polymeric carbohydrate backbone, one or more mannose-binding C-type lectin receptor targeting moieties, and a therapeutic agent comprises one or more reactive hydroxyl groups and coupled to the polymeric carbohydrate backbone via a degradable linker. The degradable linker may comprise one or more carbonate and/or disulfide moieties. The disease to be treated may include cancer, an autoimmune disease, an inflammatory disorder, Non-Alcoholic Steatohepatitis (NASH), acute respiratory distress syndrome (ARDS), sepsis, coronavirus infection, influenza infection, cytokine storms, and other macrophage involved diseases.
    Type: Application
    Filed: July 13, 2023
    Publication date: January 25, 2024
    Inventors: Jeffrey Scott Arnold, David A. Ralph
  • Publication number: 20240024492
    Abstract: The present application relates to an asymmetric RNAi-inducing nucleic acid molecule which inhibits the expression of retinoid-related orphan nuclear receptor-beta (ROR-beta) and use thereof, and more particularly to an asymmetric RNAi-inducing nucleic acid molecule comprising an antisense strand comprising a sequence complementary to mRNA encoding ROR-beta, and a sense strand forming complementary bonds with the antisense strand, and a pharmaceutical composition for ameliorating or treating a retinal disease, comprising the asymmetric RNAi-inducing nucleic acid molecule.
    Type: Application
    Filed: December 6, 2021
    Publication date: January 25, 2024
    Inventors: Hye Won CHUNG, June Hyun PARK, Seung Ya YANG
  • Publication number: 20240024493
    Abstract: Methods of purification for the large scale synthesis of CB[7]-PEG may include at least one of diafiltration or tangential flow filtration, column chromatography, affinity “pull-down” techniques, or selective precipitation methods. In one embodiment, the method includes providing a reaction mixture containing synthesized CB[7]-PEG, providing a membrane selected to be below the nominal molecular weight of CB[7]-PEG, and removing small molecular weight contaminant species from the reaction mixture using the membrane. In embodiments, regardless of which purification method is used, a copper catalyst component of the “click” chemistry reaction mixture may be removed using a commercially available metal-chelating resin.
    Type: Application
    Filed: June 22, 2023
    Publication date: January 25, 2024
    Inventor: Matthew J. Webber
  • Publication number: 20240024494
    Abstract: The present disclosure discloses a small-molecule conjugate or a pharmaceutical salt thereof, wherein a structure of the small-molecule conjugate is shown in a general formula I: A is a spacer linking group; B is a releasable linking group; and Y is a drug. The present disclosure further provides use of the small-molecule conjugate or the pharmaceutical salt thereof in the preparation of an antitumor drug.
    Type: Application
    Filed: August 30, 2021
    Publication date: January 25, 2024
    Inventor: Juan WANG
  • Publication number: 20240024495
    Abstract: Disclosed herein, are compositions comprising one or more a molecular guidance system (MGS) peptides and a cytotoxic agent. Also described herein, are methods of administering the compositions to patients with cancer.
    Type: Application
    Filed: July 14, 2023
    Publication date: January 25, 2024
    Applicant: SRI International
    Inventors: Kathlynn C. Brown, Curtis Allred, Michael McGuire
  • Publication number: 20240024496
    Abstract: The present invention pertains to the use of conjugated zearalenone (ZEA) in a method to protect an animal against ZEA induced mycotoxicosis, in particular to protect against decreased weight gain, kidney damage, liver damage and damage to a reproductive organ.
    Type: Application
    Filed: December 21, 2021
    Publication date: January 25, 2024
    Applicant: INTERVET INC.
    Inventors: Sietske Kooijman, Ruud Philip Antoon Maria Segers, Maarten Hendrik Witvliet
  • Publication number: 20240024497
    Abstract: The present invention relates to methods for the synthesis of conjugates useful for the treatment of viral infections, e.g., conjugates containing inhibitors of viral neuraminidase (e.g., zanamivir or an analog thereof) linked to an Fc domain monomer.
    Type: Application
    Filed: August 6, 2021
    Publication date: January 25, 2024
    Inventors: Allen BORCHARDT, Robert Michael HUGHES
  • Publication number: 20240024498
    Abstract: Provided are a pharmaceutical composition comprising an antibody-drug conjugate, and use of the pharmaceutical composition. Specifically provided is a pharmaceutical composition, comprising an anti-claudin antibody-drug conjugate.
    Type: Application
    Filed: September 30, 2021
    Publication date: January 25, 2024
    Inventors: Zhiwan WANG, Tingting WU, Xun LIU
  • Publication number: 20240024499
    Abstract: The present invention provides anti-GPC3 antibodies, antigen binding portions thereof and GPC3 conjugates thereof for use in the treatment of cancer.
    Type: Application
    Filed: November 19, 2021
    Publication date: January 25, 2024
    Inventors: Maria Leia SMITH, May Kung SUTHERLAND
  • Publication number: 20240024500
    Abstract: The present invention relates to a method of generating a nanoparticle comprising (c) contacting an antibody with a composition comprising a first conjugate (A), the first conjugate comprising a positively charged polypeptide conjugated to a bifunctional linker, characterized in that the composition is essentially free of unconjugated bifunctional linker, thereby obtaining a second conjugate (B), the second conjugate comprising the positively charged polypeptide, the bifunctional linker, and the antibody; and (d) contacting the second conjugate (B), a positively charged polypeptide, and a negatively charged molecule, thereby forming a nanoparticle. The present invention also relates to a nanoparticle obtainable by a method of the invention, as well as to a nanoparticle comprising (a) a positively charged polypeptide; (b) a second conjugate (B), the second conjugate comprising an antibody conjugated to a positively charged polypeptide; (c) one or more negatively charged molecules.
    Type: Application
    Filed: December 2, 2021
    Publication date: January 25, 2024
    Inventors: Sebastian BÄUMER, Nicole BÄUMER, Wolfgang BERDEL, Georg LENZ, Andreas FAUST, Lisa WITTMANN
  • Publication number: 20240024501
    Abstract: Provided herein are protein complexes comprising a sensor domain and a therapeutic domain linked by a linker, and methods of use thereof. In aspects of the present disclosure, activity of the therapeutic domain comprises a dependence on sensor domain binding to target markers.
    Type: Application
    Filed: February 10, 2023
    Publication date: January 25, 2024
    Inventors: John Thomas MULLIGAN, Shannon Lee OKADA, Justin Richard KILLEBREW, Diane Louise HOLLENBAUGH
  • Publication number: 20240024502
    Abstract: The present disclosure provides anti-MSLN antibodies, antigen binding portions thereof and MSLN conjugates thereof for use in the treatment of cancer.
    Type: Application
    Filed: October 15, 2021
    Publication date: January 25, 2024
    Inventors: May Kung SUTHERLAND, Maria Leia SMITH
  • Publication number: 20240024503
    Abstract: Methods and compositions for the production of phospholipid-ligand bioconjugates and uniform targeted microbubbles are provided. These methods and compositions find use in ultrasound and molecular imaging applications related to cancer and other diseases. The methods of the present disclosure comprise contacting a phospholipid comprising a maleimide containing functional group with a ligand comprising a C terminal cysteine residue. The methods disclosed herein solves the problems in producing ready-to-use and clinically translatable ultrasound molecular imaging agents by incorporating small protein ligands engineered to bind against biomarkers representing pathological angiogenesis or abnormal cells. The methods also overcome the current limitations in producing uniformly targeted microbubbles in a scalable, economical and reproducible manner.
    Type: Application
    Filed: July 13, 2023
    Publication date: January 25, 2024
    Inventors: Rakesh Bam, Jeremy Dahl
  • Publication number: 20240024504
    Abstract: The subject invention pertains to methods of acetylation of the Fc region of immunoglobulins, particularly Lys248 of the heavy chain of human Immunoglobulin G (IgG) using a novel Fc-III derived peptide. The acetylation reaction with the phenolic ester with an azide or alkyne enables site-selective functionalization of Lys248 with a bioorthogonal reactive group for further derivatization. Further methods are provided to synthesize an antibody-lipid conjugate that allows for the construction of an immunoliposome that can target cells expressing oncogenes, including HER2+ cells, and a bispecific antibody complex (bsAbC) linking two distinct antibodies. The bsAbC can induce an effector-cell mediated cytotoxicity at nanomolar concentrations.
    Type: Application
    Filed: October 21, 2022
    Publication date: January 25, 2024
    Inventors: Jiang XIA, Yu ZHANG, Dingdong YUAN
  • Publication number: 20240024505
    Abstract: The disclosure relates generally to methods and compositions for generation or enhancement of partial or complete cellular signaling pathways
    Type: Application
    Filed: July 12, 2023
    Publication date: January 25, 2024
    Applicant: Trustees of Boston University
    Inventors: Mark W. Grinstaff, Jack R. Kirsch, Amanda K. Williamson, Brett Tingley
  • Publication number: 20240024506
    Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.
    Type: Application
    Filed: June 1, 2023
    Publication date: January 25, 2024
    Inventors: Lei Jiang, Lin Tung Guey, Paolo G. V. Martini, Vladimir Presnyak
  • Publication number: 20240024507
    Abstract: Provided herein are compositions including targeting peptides linked thereto or inserted in a targeting protein of a recombinant vector having at least one exogenous peptide comprising an amino acid sequence of N-x-(T/I/V/A)-(K/R) (SEQ ID NO: 47). Compositions providing such conjugates, targeting peptides, or recombinant vectors having a mutant capsid or envelope protein are provided as are uses thereof.
    Type: Application
    Filed: December 1, 2021
    Publication date: January 25, 2024
    Applicant: The Trustees of the University of Pennsylvania
    Inventors: James M. Wilson, Joshua Joyner Sims, Yuan Yuan
  • Publication number: 20240024508
    Abstract: Provided herein are pharmaceutical compositions for administration to a suprachoroidal space of an eye of a subject. The pharmaceutical compositions can include a recombinant adeno-associated virus (AAV) encoding a transgene. Also provided herein are methods for treating or preventing a disease in a subject by administering a therapeutically effective amount of the pharmaceutical compositions to the subject in need.
    Type: Application
    Filed: October 6, 2021
    Publication date: January 25, 2024
    Inventors: Jared Bee, Tristan James Marshall, Sherri Van Everen, Stephen Joseph Pakola, Nicholas Alexander Piers Sascha Buss, Anthony Ray O'Berry, Jesse I. Yoo, Ewa Budzynski
  • Publication number: 20240024509
    Abstract: Provided are methods and compositions for treating cerebrospinal fluid disorders. In embodiments, the methods comprise administering an agent to a subject altering activity, expression, or level of NKCC1 in a cell.
    Type: Application
    Filed: July 27, 2023
    Publication date: January 25, 2024
    Applicant: The Children's Medical Center Corporation
    Inventors: Maria K. LEHTINEN, Ryann M. FAME, Cameron SADEGH, Huixin XU
  • Publication number: 20240024510
    Abstract: A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
    Type: Application
    Filed: September 27, 2023
    Publication date: January 25, 2024
    Applicant: The Trustees of the University of Pennsylania
    Inventors: Christian Hinderer, James M. Wilson
  • Publication number: 20240024511
    Abstract: The present application, in some embodiments, is directed to a polynucleotide including: (a) a first nucleic acid molecule including a sequence of a human endogenous Bruton's tyrosine kinase (BTK) promoter; and (b) a second nucleic acid molecule including a codon optimized sequence encoding a BTK or a functional analog thereof. Further provided are an expression vector, a cell, and a composition, all of which include the polynucleotide of the invention, and a method of using same, such as for treating X-linked Agammaglobulinemia (XLA) in a subject in need thereof.
    Type: Application
    Filed: December 7, 2021
    Publication date: January 25, 2024
    Inventors: Liron ELKOUBY, Noam DIAMANT, Liat SHACHNAI-PINKAS
  • Publication number: 20240024512
    Abstract: The present disclosure relates to compositions and methods for the treatment of TECPR2-associated diseases or disorders. Several embodiments provided for herein relate to virally-mediated transfer of a gene to target cells to induce expression of an encoded polypeptide, protein or other product in order to ameliorate one or more symptoms of a TECPR2-associated disease or disorder in a subject. In several embodiments, the disclosed methods and compositions relate to recombinant adeno-associated virus particles encoding human TECPR2 in order to treat TECPR2-associated diseases or disorders, including spastic paraplegic type 49 (SPG49) and/or hereditary sensory and autonomic neuropathy 9 (HSAN9).
    Type: Application
    Filed: July 8, 2023
    Publication date: January 25, 2024
    Applicant: University of Florida Research Foundation, Incorporated
    Inventors: Barry John Byrne, Manuela Corti, Shandra Trantham
  • Publication number: 20240024513
    Abstract: The invention relates to a nucleic acid encoding human FUS protein comprising a sequence having at least 69% sequence identity with the sequence SEQ ID NO 1 of intron 6 and/or a sequence having at least 60% sequence identity with the sequence SEQ ID NO 2 of intron 7, said sequences being located on either side of exon 7, to a recombinant vector comprising said nucleic acid encoding human FUS protein, and their use as medicament in the treatment and/or prevention of amyotrophic lateral sclerosis (ALS). The invention further relates to a pharmaceutical composition comprising said nucleic acid and/or vector for the prevention and/or treatment of amyotrophic lateral sclerosis (ALS). The present invention finds application in the therapeutic, veterinary and diagnostic medical technical fields.
    Type: Application
    Filed: April 9, 2021
    Publication date: January 25, 2024
    Inventors: Luc Dupuis, Immaculada Sanjuan Ruiz, Gina Picchiarelli
  • Publication number: 20240024514
    Abstract: Recombinant adenovirus-associated viruses are described. The recombinant adenovirus-associated viruses may include a capsid protein with enhanced tropism for liver cells. The recombinant adenovirus-associated viruses may also exhibit less immunogenicity in humans. The recombinant adenovirus-associated viruses may include expression cassettes including a polynucleotide sequence encoding a therapeutic agent useful in gene therapy treatment of a liver disease. Preparation systems for packaging the recombinant adenovirus-associated viruses, methods of producing the recombinant adenovirus-associated viruses, pharmaceutical compositions including the recombinant adenovirus-associated viruses, and uses of said compositions for treating liver diseases including Fabry disease and Hepatitis B, are also provided.
    Type: Application
    Filed: November 16, 2021
    Publication date: January 25, 2024
    Inventors: Tingting Zhang, Chao Wang
  • Publication number: 20240024515
    Abstract: The present invention relates to combination treatments for cystic fibrosis, particularly combinations of modulators of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and gene therapy.
    Type: Application
    Filed: April 11, 2023
    Publication date: January 25, 2024
    Inventors: Eric Alton, Jane Davies, Uta Grisenbach, Ana Sergijenko, Deborah R. Gill, Stephen C. Hyde, Christopher Boyd, Gerry McLachlan, Sebastian Kreuz, Alena Moiseenko
  • Publication number: 20240024516
    Abstract: Disclosed are compositions and methods for producing therapeutic fusion proteins in vivo. The compositions and methods disclosed herein are capable of ameliorating diseases by providing therapeutic protein delivery.
    Type: Application
    Filed: March 20, 2023
    Publication date: January 25, 2024
    Inventor: Michael Heartlein
  • Publication number: 20240024517
    Abstract: Disclosed, at least in part, are dosings of viral vectors concomitantly with synthetic nanocarriers attached to an immunosuppressant, in combination with dosings of the synthetic nanocarriers attached to an immunosuppressant without a viral vector or dosings of the synthetic nanocarriers attached to an immunosuppressant concomitantly with lower doses of the viral vector, and related compositions that provide reduced humoral immune responses and/or increased or durable transgene or nucleic acid material expression.
    Type: Application
    Filed: June 28, 2023
    Publication date: January 25, 2024
    Inventor: Takashi Kei Kishimoto
  • Publication number: 20240024518
    Abstract: The present invention provides a contrast agent which is efficiently and specifically absorbed by tumor cells, suitable for use in single photon emission computed tomography for the diagnosis, efficacy assessment and tumor tracking of neuroblastoma, pheochromocytoma or congestive heart failure.
    Type: Application
    Filed: January 19, 2023
    Publication date: January 25, 2024
    Applicant: INSTITUTE OF NUCLEAR ENERGY RESEARCH, ATOMIC ENERGY COUNCIL, EXECUTIVE YUAN
    Inventors: Shiu-Wen LIU, Yi-Jhih CHEN, Wei-Hsi CHEN, Yu CHANG, Cheng-Fang HSU
  • Publication number: 20240024519
    Abstract: The present disclosure provides radiopharmaceutical composition comprising copper-64 that performs as a targeted diagnostic PET-agent. The radiopharmaceutical composition disclosed herein has a short physical half-life of about 12.7 h.
    Type: Application
    Filed: July 18, 2023
    Publication date: January 25, 2024
    Inventors: Amanda Donovan, Lauren Radford, Allan Casciola