Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
Type:
Grant
Filed:
July 10, 2023
Date of Patent:
May 21, 2024
Assignee:
Dyne Therapeutics, Inc.
Inventors:
Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
Abstract: This invention provides a fluid therapeutic placental product comprising placental cells and a placental dispersion comprising placental factors. The placental cells and the placental dispersion are derived from placental tissue. A placental tissue can optionally be an amnion, chorion, or a trophoblast-depleted chorion. The placental product of the present invention is useful in treating a patient with a tissue injury (e.g. wound or burn) by applying the placental product to the injury. Similar application is useful with ligament and tendon repair and for engraftment procedures such as bone engraftment.
Type:
Grant
Filed:
October 17, 2013
Date of Patent:
May 21, 2024
Assignee:
OSIRIS THERAPEUTICS, INC.
Inventors:
Timothy Jansen, Samson Tom, Alla Danilkovitch, Dana Yoo, Jaime Zerhusen
Abstract: Disclosed herein are compositions and methods of treating disclosure provides for compounds for use in treating Mitochondrial Neurogastrointestinal Encephalopathy Syndrome (MNGIE). In some embodiments, the compounds have cell penetrating activity and thymidine phosphorylase activity. In certain embodiments, the compounds disclosed herein comprise: a) at least one cell-penetrating peptide (CPP) moiety; and b) a thymidine phosphorylase, or an active fragment or analog thereof (TP), wherein the CPP is coupled, directly or indirectly, to TP.
Type:
Grant
Filed:
October 6, 2021
Date of Patent:
May 21, 2024
Assignee:
Entrada Therapeutics, Inc.
Inventors:
Natarajan Sethuraman, Jason Ruth, Louis A. Tartaglia, Dehua Pei, Ziqing Qian
Abstract: The methods and systems disclose enzymes that function to import malonic acid or malonates into a cell. The enzymes increase the output of precursor molecules by enriching certain pathways in the cell. The precursor molecules can be converted to cannabinoids. The enzymes are a family of proteins which have a majority of common alignments.
Abstract: The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4?) gene by targeting an HNF4? expression control region and methods of use thereof for treating an HNF4? associated disorder, e.g., cirrhosis.
Type:
Grant
Filed:
September 23, 2020
Date of Patent:
May 21, 2024
Assignee:
Omega Therapeutics, Inc.
Inventors:
Jesse Jerome Smith, Jodi Michelle Kennedy, Jeremiah D. Farelli, Kendrick Alan Goss, Adam Walter Scheidegger, Yoseph Kassa, Christian Wessel Cobaugh, Timsi Rao
Abstract: The invention provides methods of preparing 8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid and methods of making a pharmaceutical material comprising a purified amount of 8-hydroxy-2,2,14, 14-tetramethylpentadecanedioic acid. Also provided are compositions and pharmaceutical materials including a purified amount of 8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid as well as methods of treating various diseases and conditions using the compositions and pharmaceutical materials.
Type:
Grant
Filed:
July 19, 2022
Date of Patent:
May 21, 2024
Assignee:
Esperion Therapeutics, Inc.
Inventors:
Richard Copp, Mohamed Abdelnasser, Christopher M. Cimarusti, Jonathan Lane, Michael Barkman, Rasidul Amin, Arthur John Cooper, Damodaragounder Gopal, Philipp Selig
Abstract: Provided herein are methods of identifying genomic region(s) predictive of an outcome of treatment with a cell therapy and/or of a phenotype of function of the cells. In some embodiments, the methods include epigenetic and/or epigenomic analyses of the cells in connection with methods for preparing engineered cells for cell therapy and/or predicting response to a cell therapy, e.g., engineered cells for cell therapy. In some embodiments, the methods include steps to assess, characterize and analyze changes or modifications in an epigenetic property of gene region or regions, such as chromatin accessibility, nucleosome occupancy, histone modification, spatial chromosomal conformation, transcription factor occupancy and/or DNA methylation. In some embodiments, the epigenetic and/or epigenomic analysis includes determining the epigenetic properties of a cell, e.g., an engineered cell for cell therapy.
Type:
Application
Filed:
October 17, 2023
Publication date:
May 16, 2024
Applicant:
Juno Therapeutics, Inc.
Inventors:
Mark L. BONYHADI, David G. KUGLER, Timothy G. JOHNSTONE, Ronald James HAUSE, Jr., Lucas J. THOMPSON, Ryan P. LARSON
Abstract: The invention provides adenosine deaminase variants that are capable of deaminating adenine and/or cytosine in a target polynucleotide (e.g., DNA). The disclosure also provides multi-molecular complexes, fusion proteins, base editors, and base editor systems comprising the adenosine deaminase variants with adenine and cytosine deaminase activity and/or cytosine deaminase specificity, and methods of use thereof.
Type:
Application
Filed:
September 26, 2023
Publication date:
May 16, 2024
Applicant:
Beam Therapeutics Inc.
Inventors:
Nicole Gaudelli, Seung-Joo Lee, Patricia Rosa Feliciano, Dieter Ka Yeung Lam
Abstract: Provided herein are methods of increasing T-cell function and T-cells produced by these methods. Also provided herein are methods of treating a subject using T-cells produced by these methods.
Type:
Application
Filed:
January 19, 2024
Publication date:
May 16, 2024
Applicant:
Kyverna Therapeutics, Inc.
Inventors:
John Lee, Erin O’Brien, Jordan Tsai, Lih-Yun Hsu, Faye Wu
Abstract: The present disclosure relates to compounds which modulate the activity of Toll-like receptor (TLR) proteins, including agonists or activators, partial agonists, and antagonists. Of particular interest of compounds that modulate the activity of TLR2, as well as methods of using such compounds to treat cancer and other disorders associated with a TLR2 pathway.
Type:
Application
Filed:
November 17, 2023
Publication date:
May 16, 2024
Applicant:
Axial Therapeutics, Inc.
Inventors:
Christopher J. Oalmann, Dennis S. Yamashita, Patrick J. Stern
Abstract: Compositions and methods for the delivery of progestin hormones that have binding affinity to the Sex Hormone Binding Globulin (SHBG) are disclosed. The compositions combine such progestins with non-progestin SHBG ligands to displace at least part of the progestin from SHBG in the blood plasma, thereby increasing its bioavailability. Also disclosed are methods to modulate progestin and estrogen levels in the blood through the use of SHBG binding and displacement, to optimize the effectiveness of formulations for contraception and minimize the side effects and adverse events.
Type:
Application
Filed:
August 22, 2023
Publication date:
May 16, 2024
Applicant:
Agile Therapeutics, Inc.
Inventors:
Agis Kydonieus, Michael E. Kafrissen, Katie Macfarlane
Abstract: Provided herein are methods for assessing cell surface glycans, e.g., N-glycans, by assessing a sample of released surface glycans, and determining the presence, absence, or level of glycans present in the sample. Also provided are methods of assaying and/or evaluating a cell composition by assessing the cell surface glycan profile of the cell composition and comparing the profile to a reference sample. Methods for manufacturing and/or culturing a plurality of cell compositions having consistent surface glycan expression with low variability are also provided.
Type:
Application
Filed:
September 15, 2023
Publication date:
May 16, 2024
Applicant:
Juno Therapeutics, Inc.
Inventors:
Paul Ken KODAMA, Tom KOWSKI, Mirna MUJACIC, Kenneth Mayo PRENTICE
Abstract: Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.
Type:
Application
Filed:
November 3, 2023
Publication date:
May 16, 2024
Applicant:
Orna Therapeutics, Inc.
Inventors:
Allen T. HORHOTA, Junghoon YANG, Kevin J. KAUFFMAN, Thomas BARNES, Robert Alexander WESSELHOEFT, Amy M. BECKER, Gregory MOTZ
Abstract: The invention provides for the treatment of mutant epidermal growth factor receptor (EGFR) mediated cancer that has metastasized to the brain or other area of the central nervous system with a compound that degrades a mutant form of EGFR via the ubiquitination of the EGFR protein and subsequent proteasomal degradation. The invention also provides advantageous drug combinations for the treatment of such cancer that include a compound herein that degrades a mutant form of EGFR in combination with a second anti-cancer agent.
Type:
Application
Filed:
November 21, 2023
Publication date:
May 16, 2024
Applicant:
C4 Therapeutics, Inc.
Inventors:
Christopher G. Nasveschuk, Martin Duplessis, Jae Young Ahn, Alexander W. Hird, Ryan E. Michael, Kiel Lazarski, Yanke Liang, Georg Jaeschke, Antonio Ricci, Annick Goergler, Daniel Rueher
Abstract: Novel dry powder compositions comprising and methods relating thereto are provided. The dry powder compositions comprise PDE5 inhibitors, such as vardenafil, or pharmaceutically acceptable salts or esters thereof. The dry powder compositions may optionally include an carrier/excipient. The concentration of active agent may be at least about 2% by weight. Methods of aerosolizing the dry powder compositions and using them to treat various diseases are also disclosed.
Type:
Application
Filed:
January 22, 2024
Publication date:
May 16, 2024
Applicant:
Respira Therapeutics, Inc.
Inventors:
Zhen Xu, Hugh Smyth, Aileen Gibbons, Revati Shreeniwas, Pravin Soni, Dan Deaton, James Hannon, Stephen Lermer, Robert Curtis, Martin J. Donovan
Abstract: The present disclosure relates to methods of treating ulcerative colitis with anti-LIGHT antibodies. The disclosure also relates to assaying free LIGHT prior to, during, or after administration of an anti-LIGHT antibody to treat ulcerative colitis.
Abstract: Embodiments of the disclosure are drawn to an enteral feeding device for hydrolyzing triglycerides in a nutritional formula. The device may include a body housing a chamber, an inlet configured to fluidly couple with a source of nutritional formula, and an outlet configured to fluidly couple with an enteral feeding tube. The device may include a headspace and a plurality of particles contained within the chamber, wherein the lipase is covalently bonded to the plurality of particles. The device may include an inlet filter located between the inlet and the chamber, wherein the inlet filter contains a first plurality of openings, and an outlet filter located between the chamber and the outlet, wherein the outlet filter has a second plurality of openings smaller than the plurality of particles.
Type:
Application
Filed:
January 19, 2024
Publication date:
May 16, 2024
Applicant:
Alcresta Therapeutics, Inc.
Inventors:
Robert GALLOTTO, Greta L. LORING, Kenneth GARY, Edward S. PARK, David J. BROWN, Willem Robert Klaas SCHOEVAART, Michiel Christian Alexander van VLIET
Abstract: This invention provides for a method of treating secondary adrenal insufficiency by co-administrating therapeutically effective amounts of a glucocorticoid and a glucocorticoid receptor antagonist to the patient in need thereof. In some embodiments, the method includes the proviso that the patient not be otherwise in need of treatment with a glucocorticoid and a glucocorticoid receptor antagonist. The treatment method can increase the patient's morning or basal cortisol level to at least about 12 ?g/dL or a standard control level, and in turn, expedite significantly the recovery of the HPA axis. The method provided herein can improve health outcomes and life-threatening complications associated with secondary adrenal insufficiency.
Type:
Application
Filed:
December 22, 2023
Publication date:
May 16, 2024
Applicant:
Corcept Therapeutics, Inc.
Inventors:
Andreas G. Moraitis, Pejman Cohan, Joseph K. Belanoff