Patents Issued in January 25, 2024
  • Publication number: 20240026320
    Abstract: Described herein is a method for editing the MHY7 gene in a cell by genome editing comprising introducing into the cell one or more deoxyribonucleic acid (DNA) endonucleases to effect one or more double stranded breaks (DSBs) within or near enhancer regions of the MYH7 gene or MYH6 gene that results in deletion of one or more enhancer regions of the MYH7 gene.
    Type: Application
    Filed: December 3, 2021
    Publication date: January 25, 2024
    Inventors: Elizabeth M. McNally, Anthony Martin Gacita
  • Publication number: 20240026321
    Abstract: The present disclosure provides RNA-guided CRISPR-Cas effector proteins, nucleic acids encoding same, and compositions comprising same. The present disclosure provides ribonucleoprotein complexes comprising: an RNA-guided CRISPR-Cas effector protein of the present disclosure; and a guide RNA. The present disclosure provides methods of modifying a target nucleic acid, using an RNA-guided CRISPR-Cas effector protein of the present disclosure and a guide RNA. The present disclosure provides methods of modulating transcription of a target nucleic acid.
    Type: Application
    Filed: May 8, 2023
    Publication date: January 25, 2024
    Inventors: Jennifer A. Doudna, Basem Al-Shayeb, Jillian F. Banfield, Patrick Pausch
  • Publication number: 20240026322
    Abstract: The present disclosure provides novel nucleic acid-guided nucleases and methods of using the nucleases for genome editing. The present disclosure further provides a system for editing a target region in a genome comprising a nucleic acid-guided nuclease, a heterologous guide nucleic acid for complexing with the nucleic acid-guided nuclease, and an editing polynucleotide configured to bind to the target region.
    Type: Application
    Filed: June 16, 2023
    Publication date: January 25, 2024
    Inventors: David Scott JOHNSON, Jan Fredrik SIMONS, Yoong Wearn LIM, Matthew James SPINDLER, Kyle Pierce CARTER, Savreet Kaur SANDHU, Ellen Kathleen WAGNER, Garry Coles, Robert EDGAR
  • Publication number: 20240026323
    Abstract: The present disclosure provides methods of modifying a target RNA in a eukaryotic cell. The present disclosure provides methods detecting a target RNA in a eukaryotic cell.
    Type: Application
    Filed: June 20, 2023
    Publication date: January 25, 2024
    Inventors: Jennifer A. Doudna, David Colognori
  • Publication number: 20240026324
    Abstract: The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject.
    Type: Application
    Filed: August 10, 2023
    Publication date: January 25, 2024
    Inventors: Robert Charles Altshuler, Anne Helen Bothmer, Cecilia Giovanna Silvia Cotta-Ramusino, Randi Michelle Kotlar, Ananya Ray, Nathaniel Roquet, Carlos Sanchez, Barrett Ethan Steinberg
  • Publication number: 20240026325
    Abstract: The present disclosure provides engineered eukaryotic cells comprising a surface displayed catalytic domain of an endoglycosidase and methods of use.
    Type: Application
    Filed: June 30, 2023
    Publication date: January 25, 2024
    Inventor: Weixi ZHONG
  • Publication number: 20240026326
    Abstract: The present invention provides engineered amylase polypeptides and compositions thereof. The engineered amylase polypeptides have been optimized to provide improved thermostability, protease stability, and stability under a range of pH conditions, including acidic (pH <7) conditions. The invention also relates to the use of the compositions comprising the engineered amylase polypeptides for therapeutic and/or nutritional purposes. The present invention also provides polynucleotides encoding the engineered amylase polypeptides, as well as methods for making the engineered polynucleotides and amylase polypeptides.
    Type: Application
    Filed: September 6, 2023
    Publication date: January 25, 2024
    Inventors: Chinping Chng, Nikki Dellas, Da Duan, Ravi David Garcia, Harvinder Chagger Maniar
  • Publication number: 20240026327
    Abstract: Modified PH20 hyaluronidase polypeptides, including modified polypeptides that exhibit increased stability and/or increased activity, are provided. Also provided are compositions and formulations and uses thereof.
    Type: Application
    Filed: June 22, 2023
    Publication date: January 25, 2024
    Inventors: Ge WEI, H. Michael Shepard, Qiping Zhao, Robert James Connor
  • Publication number: 20240026328
    Abstract: Modified PH20 hyaluronidase polypeptides, including modified polypeptides that exhibit increased stability and/or increased activity, are provided. Also provided are compositions and formulations and uses thereof.
    Type: Application
    Filed: June 23, 2023
    Publication date: January 25, 2024
    Inventors: Ge WEI, H. Michael Shepard, Qiping Zhao, Robert James Connor
  • Publication number: 20240026329
    Abstract: Provided herein are fusion protein comprising: an effector domain comprising a catalytic domain of a deubiquitinase, or a functional fragment or functional variant thereof; and a targeting domain comprising a moiety that specifically binds a nuclear protein. Also provided herein are methods of using the fusion proteins to treat a disease, including genetic diseases.
    Type: Application
    Filed: November 5, 2021
    Publication date: January 25, 2024
    Inventors: Andreas LOEW, Samuel W. HALL
  • Publication number: 20240026330
    Abstract: Provided herein are fusion protein comprising: an effector domain comprising a catalytic domain of a deubiquitinase, or a functional fragment or functional variant thereof; and a targeting domain comprising a moiety that specifically binds a mitochondrial protein. Also provided herein are methods of using the fusion proteins to treat a disease, including genetic diseases.
    Type: Application
    Filed: November 5, 2021
    Publication date: January 25, 2024
    Inventors: Andreas LOEW, Samuel W. HALL
  • Publication number: 20240026331
    Abstract: The present disclosure relates to methods of expressing recombinant neprosin polypeptide, host cells and expression vectors for expressing the recombinant neprosin polypeptide, and methods of using the expressed neprosin polypeptide for therapeutic and other purposes.
    Type: Application
    Filed: May 10, 2023
    Publication date: January 25, 2024
    Inventors: Arianna I. Celis Luna, Da Duan, Paul Campion Jordan, Kathleen Susan Molnar, Adam P. Silverman, Tricia Windgassen
  • Publication number: 20240026332
    Abstract: The present invention provides a liquid enzyme preparation of a protein deamidase having excellent activity stability. This liquid enzyme preparation that contains a protein deamidase and at least 30 w/v % of sorbitol and that has a pH of 5.5 or more can enhance the stability of protein deamidase activity.
    Type: Application
    Filed: December 2, 2021
    Publication date: January 25, 2024
    Inventors: Saki NORIFUNE, Yusuke TANAKA
  • Publication number: 20240026333
    Abstract: A polymer brush composed of statistical copolymers of hydrophilic and hydrophobic monomers for enzyme immobilization. The heterogeneous polymer brushes stabilized four different lipases against high temperatures. Notably, the statistical copolymers stabilized the four lipases to a greater extent than a homopolymer brush. Additionally, in the case of Rhizomucor miehei lipase, statistical copolymers stabilized the enzyme to a greater extent than homopolymers of either hydrophilic or hydrophobic monomers. The resulting increase in high-temperature stability enabled significant improvements in catalytic rates by operating reactions at elevated temperatures, which is desirable for enzyme catalysis and sensing applications. Additionally, stabilization against elevated temperatures implies stabilization against non-aqueous solvents, which is of critical importance to numerous applications of biocatalysts.
    Type: Application
    Filed: November 15, 2021
    Publication date: January 25, 2024
    Applicant: The Regents of the University of Colorado, a body corporate
    Inventors: JAMES S. WELTZ, JOEL L. KAAR, DANIEL K. SCHWARTZ, HECTOR SANCHEZ-MORAN
  • Publication number: 20240026334
    Abstract: Disclosed herein are compositions comprising magnetoelectric composite materials and collagen, along with uses and kits thereof. Described herein is the use of DNA aptamer assemblies of varying DNA length, structure, and sequence to both bind to collagen and other proteins, to then act as a biocompatible, degradable, reversible, or permanent 3D crosslinkers between proteins, and to service as a biologically functional material when using the appropriate aptamer sequence. Therefore, disclosed herein are compositions comprising collagen fibers crosslinked with DNA aptamers. Also disclosed are devices and implants made from or coated with collagen fibers crosslinked with DNA aptamers. Also disclosed are methods of making collagen fibers. Also disclosed are kits for producing collagen fibers. Also disclosed herein are compositions DNA aptamers in a collagen fiber matrix that stabilizes the DNA aptamer.
    Type: Application
    Filed: September 10, 2021
    Publication date: January 25, 2024
    Inventors: Josephine ALLEN, Jennifer S. ANDREW, Noah D. FERSON, Bryan D. JAMES
  • Publication number: 20240026335
    Abstract: A method of fixating a tissue sample including: treating the tissue sample with a nano-tissue fixative solution to form a fixated tissue sample. A composition of nucleic acids in the fixated tissue sample is not altered. The nano-tissue fixative solution includes acetic acid, at least one alcohol, chloroform, titanium dioxide nanoparticles, zinc oxide nanoparticles, and silver nanoparticles.
    Type: Application
    Filed: July 20, 2022
    Publication date: January 25, 2024
    Applicant: Imam Abdulrahman Bin Faisal University
    Inventors: Khaled Fikry SALAMA, Amal Abdullah ALODAINI
  • Publication number: 20240026336
    Abstract: A nucleic acid extraction device for molecular testing comprises a film-flipping mechanism, a cap mechanism, a dispensing mechanism, an extraction plate transfer mechanism, and a nucleic acid extraction module. A sealing film is adhered onto top of an extraction plate and folded along an outer edge of the extraction plate. A film-flipping head of the film-flipping mechanism abuts against a bottom surface of the extraction plate and pushes an edge of the sealing film to unfold the sealing film. A film gripper clamps and removes the unfolded sealing film. The cap mechanism opens the specimen containers. The dispensing mechanism transfers the specimens from the specimen containers to the extraction plate. The extraction plate transfer mechanism moves the extraction plate with the specimens to the nucleic acid extraction module. As a result, molecular testing is automated to reduce labor and improve quality.
    Type: Application
    Filed: July 21, 2022
    Publication date: January 25, 2024
    Inventors: Hsin-Wu MI, Hsin-Fei HUANG
  • Publication number: 20240026337
    Abstract: A treatment apparatus includes a heating block (11) configured to heat a container (30) accommodating a sample (100) including a liquid within a first range (11A) in a gravity direction, and a cooling block (12) configured to cool the container (30) heated by the heating block (11) within a second range (12A) narrower than the first range (11A) in the gravity direction.
    Type: Application
    Filed: November 4, 2021
    Publication date: January 25, 2024
    Inventor: Masahiro KUWATA
  • Publication number: 20240026338
    Abstract: A system, methods, and apparatus are described to collect and prepare single cells and groups of cells from microsamples of specimens and encode spatial information of the physical position of the cells in the specimen. In some embodiment, beads or surfaces with oligonucleotides containing spatial barcodes are used to analyze DNA or RNA. The spatial barcodes allow the position of the cell to be defined and the nucleic acid sequencing information, such as target sequencing, whole genome, gene expression, used to analyze the cells in a microsample for cell type, expression pattern, DNA sequence, and other information, in the context of the cell's physical position in the specimen. In other embodiment, markers such as isotopes are added to a microsample to encode spatial position with mass spectoscopy or other analysis. The spatial encoded information is then readout by analysis such as DNA sequencing, mass spectrometry, fluorescence, or other methods.
    Type: Application
    Filed: May 22, 2023
    Publication date: January 25, 2024
    Inventors: Stevan Bogdan Jovanovich, Peter Wagner
  • Publication number: 20240026339
    Abstract: The present invention relates to amphiphiles and specifically the amphiphiles that can be used as cell lysis reagents. The invention reveals the amphiphiles as well as their combinations for efficiently lysing the mammalian cells under relatively mild conditions that are compatible with a reverse transcription and polymerase chain reaction. The invention reveals the use of mixture of amphiphiles to improve the cell lysis and obtain increased yields of copy DNA during the reverse transcription reaction. The invention also provides a method for increasing the diversity of gene and transcript capture during single-cell RNA-sequencing using droplet microfluidics.
    Type: Application
    Filed: July 10, 2023
    Publication date: January 25, 2024
    Inventors: Linas Mazutis, Karolis Goda
  • Publication number: 20240026340
    Abstract: Methods and devices for stabilizing a biological sample for analysis, comprising the steps of obtaining in a sample collection device a biological sample from a subject, the biological sample including at least one circulating cell-free first nucleic acid from the subject. The methods may include a step of contacting the biological sample while within the sample collection device with a protective agent composition that includes a preservative agent, an optional anticoagulant, and a quenching agent to form a mixture that includes the protective agent composition and the sample.
    Type: Application
    Filed: September 27, 2023
    Publication date: January 25, 2024
    Inventors: Wayne L. Ryan, M. Rohan Femando, Kausik Das
  • Publication number: 20240026341
    Abstract: The present disclosure provides methods and systems for purification of chemically modified, specifically, sulfonated DNA. Additionally, the disclosure provides methods and systems for bisulfite conversion with improved purification of sulfonated DNA using silica supports, such as silica beads, and acidic conditions for binding sulfonated DNA to silica surfaces.
    Type: Application
    Filed: July 29, 2021
    Publication date: January 25, 2024
    Inventors: Evan M. Ragland, Zubin Gagrat, Michael J. Domanico
  • Publication number: 20240026342
    Abstract: Provided are fusion proteins and related molecules useful for conducting base editing with reduced or no off-target mutations. The fusion protein may include a first fragment comprising a nucleobase deaminase or a catalytic domain thereof, a second fragment comprising a nucleobase deaminase inhibitor, and a protease cleavage site between the first fragment and the second fragment. Also provided are improved prime editing systems, including prime editing guide RNA with improved stability.
    Type: Application
    Filed: September 29, 2023
    Publication date: January 25, 2024
    Inventors: Jia Chen, Bei Yang, Li Yang, Xingxu Huang, Lijie Wang
  • Publication number: 20240026343
    Abstract: Provided is a quick and simple method for screening dimerized cyclic peptides, a library therefor, and molecules including cyclic peptides that can be used in the method and the library. For example, the method for screening dimerized cyclic peptides includes (1) a step in which, in a cell-free expression system, a cyclic peptide, a dimerization domain including a cysteine residue and a leucine zipper region, and at least one polypeptide including a peptide linker existing between the cyclic peptide and the dimerization domain are synthesized, and a dimer of the polypeptide is prepared, and (2) a step in which activity based on binding between the dimer and a target molecule is measured.
    Type: Application
    Filed: July 21, 2023
    Publication date: January 25, 2024
    Applicant: AJINOMOTO CO., INC.
    Inventors: Kenichiro Ito, Kyohei Miyairi
  • Publication number: 20240026344
    Abstract: The present disclosure relates to the field of affinity chromatography, and more specifically to the provision of nucleic acid and polypeptide libraries encoding three-helix bundle protein domains suitable for selecting affinity ligands that specifically binds to a target molecule of interest. The disclosure also relates to methods of using those libraries to identify and isolate such affinity ligands to a target molecule.
    Type: Application
    Filed: October 13, 2021
    Publication date: January 25, 2024
    Inventors: Brandon Kier, Molly North, Sarmistha Bhattacharya, Thomas Scanlon, Warren Kett
  • Publication number: 20240026345
    Abstract: Among the various aspects of the present disclosure is the provision of compositions for single-cell reporter assays and methods of use thereof. Also provided are methods of determining individual activities of a plurality of nucleic acid regulatory elements, identifying a regulatory element having cell type-specific activity, or determining variance in activity of a plurality of nucleic acid regulatory elements.
    Type: Application
    Filed: November 10, 2022
    Publication date: January 25, 2024
    Applicant: Washington University
    Inventors: Barak Cohen, Siqi Zhao
  • Publication number: 20240026346
    Abstract: Provided herein is an antigen display library for detecting antibodies produced by an individual; and methods of using the antigen display library to generate an antibody signature, the method comprising contacting a biological sample containing antibodies from an individual with the antigen display library, isolating phage clones displaying antigenic epitopes recognized by antibody in the sample, and identifying the antigenic epitopes that were recognized by antibody in the sample. Also provided are kits for generating an antibody signature comprising the antigen display library, a substrate for isolating phage clones bound by antibody, and may further comprise reagents useful for generating the antibody signature.
    Type: Application
    Filed: September 27, 2023
    Publication date: January 25, 2024
    Inventors: Thomas F. TEDDER, Evgueni KOUNTIKOV
  • Publication number: 20240026347
    Abstract: The present disclosure discloses methods and systems for encoding digital information in nucleic acid (e.g., deoxyribonucleic acid) molecules without base-by-base synthesis, by encoding bit-value information in the presence or absence of unique nucleic acid sequences within a pool, comprising specifying each bit location in a bit-stream with a unique nucleic sequence and specifying the bit value at that location by the presence or absence of the corresponding unique nucleic acid sequence in the pool. Also disclosed are chemical methods for generating unique nucleic acid sequences using combinatorial genomic strategies (e.g., assembly of multiple nucleic acid sequences or enzymatic-based editing of nucleic acid sequences).
    Type: Application
    Filed: August 4, 2023
    Publication date: January 25, 2024
    Inventors: Devin Leake, Milena Lazova, Sarah Flickinger, Nathaniel Roquet, Hyunjun Park, Swapnil P. Bhatia
  • Publication number: 20240026348
    Abstract: Materials and methods for preparing nucleic acid libraries for next-generation sequencing are described herein. A variety of approaches are described relating to the use of unique molecular identifiers with transposon-based technology in the preparation of sequencing libraries. Also described herein are sequencing materials and methods for identifying and correcting amplification and sequencing errors.
    Type: Application
    Filed: September 28, 2023
    Publication date: January 25, 2024
    Applicants: Illumina, Inc., Illumina Cambridge Limited
    Inventors: Susan C. Verity, Robert Scott Kuersten, Niall Anthony Gormley, Andrew B. Kennedy, Sarah E. Shultzaberger, Andrew Slatter, Emma Bell, Sebastien Georg Gabriel Ricoult, Grace DeSantis, Fiona Kaper, Han-Yu Chuang, Oliver Jon Miller, Jason Richard Betley, Stephen M. Gross, Mats Ekstrand
  • Publication number: 20240026349
    Abstract: An improved method for Next Generation Sequencing which relies on the presence of the same distinct unique molecular identifier (UMI) located at each end of a linear nucleic acid molecule so that sequence reads of approximately 2 kb or longer are obtained, and which allows generation of a genomic map without the need of a reference sequence.
    Type: Application
    Filed: August 8, 2023
    Publication date: January 25, 2024
    Inventor: Mike Mkhitar MORADIAN
  • Publication number: 20240026350
    Abstract: Disclosed herein is a polynucleotide construct comprising one or more nuclease recognition sequences upstream and downstream of a Gene editing multi-site that comprises a plurality of recognition sequences for a site-specific recombinase or a nuclease. The plurality of recognition sequences facilitate insertion of one or more exogenous donor genes into the host cell.
    Type: Application
    Filed: April 7, 2021
    Publication date: January 25, 2024
    Inventors: Sicco Hans POPMA, Di ZHANG
  • Publication number: 20240026351
    Abstract: The present invention relates to compositions comprising RNA guides targeting TRAC, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
    Type: Application
    Filed: October 29, 2021
    Publication date: January 25, 2024
    Applicant: Arbor Biotechnologies, Inc.
    Inventor: Quinton Norman Wessells
  • Publication number: 20240026352
    Abstract: Disclosed herein are CRISPR/Cas systems comprising a fusion protein and at least one gRNA targeting a gene or a regulatory element thereof in a cell such as an immune cell, and vector compositions encoding the same. The systems and compositions may be used in methods of modulating expression of a gene in a cell such as an immune cell, as well as in methods of treating a disease such as cancer, autoimmune diseases, or viral infections.
    Type: Application
    Filed: November 12, 2021
    Publication date: January 25, 2024
    Inventors: Charles A. Gersbach, Sean McCutcheon
  • Publication number: 20240026353
    Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectrum disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.
    Type: Application
    Filed: August 6, 2021
    Publication date: January 25, 2024
    Inventors: Paymaan Jafar-Nejad, Huynh-Hoa Bui, Susan M. Freier, Frank Rigo
  • Publication number: 20240026354
    Abstract: Embodiments of the disclosure include methods for generating skeletal muscle by targeting the Hippo pathway. In particular embodiments, an individual with a need for skeletal muscle generation is provided an effective amount of a shRNA molecule that targets the SAV1 gene. Particular shRNA sequences are disclosed.
    Type: Application
    Filed: November 18, 2021
    Publication date: January 25, 2024
    Inventors: Richard A.F. Dixon, Qi Liu, James T. Willerson, James F. Martin
  • Publication number: 20240026355
    Abstract: Disclosed herein are methods are compositions and methods for enhancing regeneration in stressed damaged, and/or injured tissues.
    Type: Application
    Filed: November 16, 2021
    Publication date: January 25, 2024
    Applicant: Duke University
    Inventors: Kenneth Poss, Ruorong Yan, Junsu Kang, Joseph Goldman, Valentia Cigliola
  • Publication number: 20240026356
    Abstract: Disclosed herein are products, methods, and uses for treating, ameliorating, udaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a sarcoma. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides nucleic acids comprising U7 DUX4 antisense sequences for inhibiting or downregulating the expression of DUX4 and methods of using said antisense sequences to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer.
    Type: Application
    Filed: November 30, 2021
    Publication date: January 25, 2024
    Inventors: Scott Quenton Harper, Afrooz Rashnonejad, Nicolas Sebastien Wein
  • Publication number: 20240026357
    Abstract: A composition of matter comprising a synthetic miR-135 molecule comprising a nucleic acid sequence of a miR-135b as set forth in SEQ ID NO: 37, and a complementary strand as set forth in SEQ ID NO: 40 is disclosed. A conjugate comprising a composition of matter comprising a synthetic miR-135 molecule and a cell-targeting moiety is also disclosed.
    Type: Application
    Filed: July 14, 2023
    Publication date: January 25, 2024
    Applicants: Yeda Research and Development Co. Ltd., miCure Therapeutics Ltd.
    Inventors: Alon CHEN, Sharon MANASHIROV, Andres Pablo MONTEFELTRO
  • Publication number: 20240026358
    Abstract: Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
    Type: Application
    Filed: March 11, 2022
    Publication date: January 25, 2024
    Inventors: Prashant Monian, Chikdu Shakti Shivalila, Subramanian Marappan, Chandra Vargeese, Pachamuthu Kandasamy, Genliang Lu, Hui Yu, David Charles Donnell Butler, Luciano Henrique Apponi, Mamoru Shimizu, Stephany Michelle Standley, David John Boulay, Andrew Guzior Hoss, Jigar Desai, Jack David Godfrey, Hailin Yang, Naoki Iwamoto, Jayakanthan Kumarasamy, Anthony Lamattina, Tom Liantang Pu
  • Publication number: 20240026359
    Abstract: Provided herein are methods of treating ligament flavum hypertrophy and spinal stenosis including ligament flavum hypertrophy in a patient. The method comprises delivering to cells of hypertrophic ligament flavum in a patient a pharmaceutical composition comprising micro-RNA 29a or a precursor thereof, thereby decreasing type I and/or III collagen production by the cells. Alternatively, an antisense or RNAi reagent for knocking down type I collagen and/or type III collagen expression is delivered to cells of hypertrophic ligament flavum in a patient.
    Type: Application
    Filed: July 18, 2023
    Publication date: January 25, 2024
    Inventors: Joon Lee, Gwendolyn A. Sowa, Nam V. Vo, Richard Wawrose
  • Publication number: 20240026360
    Abstract: The present invention relates to antisense oligonucleotides that are capable of modulating expression of Tau in a target cell. The oligonucleotides hybridize to MAPT mRNA. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment of Tauopathies, Alzheimer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, Dravet syndrome, depression, seizure disorders and movement disorders.
    Type: Application
    Filed: July 21, 2023
    Publication date: January 25, 2024
    Inventors: Peter HAGEDORN, Anja Mølhart HØG, Richard E. OLSON, Marianne L. JENSEN
  • Publication number: 20240026361
    Abstract: Disclosed herein is a modified ribonucleotide comprising a nucleoside comprising N4-acetylcytidine and/or 5-hydroxymethyluridine, and polyribonucleotides comprising the same. Also provided herein are compositions comprising a polyribonucleotide of the present disclosure and methods of making and using the same.
    Type: Application
    Filed: August 17, 2023
    Publication date: January 25, 2024
    Inventors: Nikhil Dhar, Marianna Keaveney, Kyle Backman, Everett Webster, Nikolai Eroshenko, Justin Quinn
  • Publication number: 20240026362
    Abstract: Provided are interfering RNAs (e.g., siRNAs) targeting SARS-CoV (e.g., the POL, Spike, Helicase, or Envelop gene thereof) and therapeutic uses thereof for inhibiting SARS-CoV infection and/or treating diseases associated with the infection (e.g., COVID-19).
    Type: Application
    Filed: December 3, 2021
    Publication date: January 25, 2024
    Applicants: MICROBIO (SHANGHAI) CO. LTD., ONENESS BIOTECH CO. LTD.
    Inventors: Yi-Chung CHANG, Chi-Fan YANG, Yi-Fen CHEN, Chia-Chun YANG, Yuan-Lin CHOU
  • Publication number: 20240026363
    Abstract: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Superoxide Dismutase 1 (SOD1) gene. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an SOD1 gene. Pharmaceutical compositions that include one or more SOD1 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of SOD1 gene expression and a reduction in SOD1 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including amyotrophic lateral sclerosis (ALS.
    Type: Application
    Filed: June 14, 2023
    Publication date: January 25, 2024
    Inventors: Christine Esau, Ji Young Suk, Tao Pei, Anthony Nicholas, Xiaokai Li, Jeffrey Carlson
  • Publication number: 20240026364
    Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or ?-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.
    Type: Application
    Filed: July 25, 2023
    Publication date: January 25, 2024
    Applicant: Ionis Pharmaceuticals, Inc.
    Inventors: Shuling Guo, Mariam Aghajan, Eric E. Swayze
  • Publication number: 20240026365
    Abstract: Methods and compositions using dnayzme-based GATA3 inhibitors for the treatment of insulin resistance and Type II diabetes, for the stimulation of adipogenesis in vivo, and for the promotion of fat redistribution are disclosed.
    Type: Application
    Filed: May 19, 2023
    Publication date: January 25, 2024
    Inventors: Mohamed ELRAYESS, Layla AIMANSOORI
  • Publication number: 20240026366
    Abstract: A dual-specific aptamer comprising the nucleic acid sequence set forth in SEQ ID NO: 14. The dual-specific aptamer comprising an anti-HER2 segment capable of binding to a HER2 marker and an anti-CD16 segment capable of binding to a CD16 marker. The anti-HER2 segment comprises nucleotide 1 to nucleotide 42 of SEQ ID NO: 14, and the anti-CD16 segment comprises nucleotide 63 to nucleotide 103 of SEQ ID NO: 14. The dual-specific aptamer further comprises a nucleic acid linker comprising nucleotide 43 to nucleotide 62 of SEQ ID NO: 14 and is selected from the group consisting of a double-stranded nucleic acid linker or a single-stranded nucleic acid linker.
    Type: Application
    Filed: September 18, 2023
    Publication date: January 25, 2024
    Applicants: Aria Tina Gene Company
    Inventors: Majid Talkhabifard, Majid Shahbazi
  • Publication number: 20240026367
    Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides compositions for inducing targeted disruption of endogenous genes in eukaryotic cells. The composition may comprise a Cas9/guide RNA complex with a Cas9 protein to which a NLS is linked, and a guide RNA having a crRNA and a tracrRNA. The crRNA may comprise i) a portion to be hybridized with a portion of the tracrRNA, and ii) a portion complementary to a target DNA of the endogenous genes. In some embodiments, the Cas/guide RNA complex may be formed in vitro before being introduced into the eukaryotic cell.
    Type: Application
    Filed: September 15, 2023
    Publication date: January 25, 2024
    Applicant: TOOLGEN INCORPORATED
    Inventors: Jin-Soo Kim, Seung Woo Cho, Sojung Kim
  • Publication number: 20240026368
    Abstract: Disclosed herein, are methods of controlling expression of one or more genes of interest in one or more bacterial cells using a eukaryotic transcription factor, and inducible binary expression systems thereof.
    Type: Application
    Filed: September 2, 2021
    Publication date: January 25, 2024
    Inventors: Tara Deans, Ian Cody MacDonald
  • Publication number: 20240026369
    Abstract: The present invention generally relates to the technical field of targeted modification of a nucleotide sequence of interest in the genome of a plant by means of a site-specific nuclease, wherein the modification and precision is assisted by specifically enhancing DNA polymerase theta (Pol ?) activity to improve genome editing (GE) efficiencies, preferably for increasing targeted insertion of a DNA insert. The invention describes a method for increasing the efficiency of targeted transgene insertion in a plant or a plant cell. Further provided are methods to modulate endogenous repair pathways with the aim to favor Pol ? activity in the context of GE. Further disclosed are suitable sequences and GE tools suitable in the methods of the invention as well as cells, tissues, organs or materials obtainable by the methods. Finally, an expression construct assembly for conducting the methods of the invention is disclosed.
    Type: Application
    Filed: October 26, 2021
    Publication date: January 25, 2024
    Applicant: KWS SAAT SE & Co. KGaA
    Inventor: Behailu AKLILU