Patents Issued in June 27, 2024
  • DIELS-ALDER CONJUGATION METHODS
    Publication number: 20240207412
    Abstract: Described herein are protein-payload conjugates and compositions thereof that are useful, for example, for target-specific delivery of therapeutic and/or imaging agent moieties. In certain embodiments, provided are specific and efficient methods for producing protein-payload constructs (e.g., antibody-drug conjugates) utilizing a combination of transglutaminase and Diels-Alder techniques. Antibody-drug conjugates and compositions which comprise glutaminyl-modified antibodies, Diels-Alder adducts, and reactive payloads and are provided.
    Type: Application
    Filed: May 12, 2023
    Publication date: June 27, 2024
    Applicant: Regeneron Pharmaceuticals, Inc.
    Inventor: Thomas NITTOLI
  • PROTEOLYSIS-TARGETING CHIMERIC MOLECULES (PROTACS) THAT INDUCE DEGRADATION OF C-MYC PROTEIN
    Publication number: 20240207413
    Abstract: Disclosed are proteolysis-targeting chimeric molecules (PROTACs) that induce degradation of c-MYC protein. The disclosed PROTACs typically include a first targeting moiety that binds to c-MYC (Mc-MYC) which may be derived from a substituted heterocycle that binds to c-MYC such as a substituted pyrazole. The first targeting moiety typically is linked via a bond or a linker (L) to a second targeting moiety that binds to an E3 ubiquitin ligase (ME3). As such, the disclosed PROTACS may be described as having a formula Mc-MYC-L-ME3 or ME3-L-Mc-MYC.
    Type: Application
    Filed: December 22, 2023
    Publication date: June 27, 2024
    Inventors: Gary E. Schiltz, Atul Jain, Huiying Han, Sarki A. Abdulkadir
  • DISULFIDE-BASED PRODRUG COMPOUND
    Publication number: 20240207414
    Abstract: The present application relates to a compound having the formula (I); A-L-B (I) wherein A is represented by; L is a bond or a self-immolative spacer; B is represented by; The compound is capable of releasing molecular cargo in the presence of a reductase and is thus suitable for treating, ameliorating, preventing or diagnosing a disorder selected from a neoplastic disorder; atherosclerosis; an autoimmune disorder; an inflammatory disease; a chronic inflammatory autoimmune disease; ischaemia; and reperfusion injury.
    Type: Application
    Filed: March 22, 2022
    Publication date: June 27, 2024
    Applicant: Ludwig-Maximilians-Universitat Munchen
    Inventors: Julia Thorn-Seshold, Jan Felber, Lukas Zeisel, Oliver Thorn-Seshold
  • BI-FUNCTIONAL COMPOUNDS AND METHODS FOR TARGETED UBIQUITINATION OF ANDROGEN RECEPTOR
    Publication number: 20240207415
    Abstract: The present invention relates to bi-functional compounds which function to recruit endogenous proteins to an E3 ubiquitin ligase for degradation, and methods for using same. More specifically, the present disclosure provides specific proteolysis targeting chimera (PROTAC) molecules which find utility as modulators of targeted ubiquitination of a variety of polypeptides and other proteins, in particular the androgen receptor of a slice variant of AR which lacks the LBD, labelled as AR-V7, which are then degraded and/or otherwise inhibited by the compounds as described herein.
    Type: Application
    Filed: November 8, 2023
    Publication date: June 27, 2024
    Inventors: Roy J. VAZ, Jenny DESANTIS, Michela ELEUTERI
  • STEREOCOMPLEX OF OLIGOLACTIC ACID CONJUGATES IN MICELLES FOR IMPROVED PHYSICAL STABILITY AND ENHANCED ANTITUMOR EFFICACY
    Publication number: 20240207416
    Abstract: The present technology relates generally to oligolactic acid conjugates and stereocomplexes of conjugates of gemcitabine and gemcitabine derivatives, micelle compositions containing such conjugates or stereocomplexes of conjugates, and methods of preparing and using such compositions to treat various cancers. The oligolactic acid conjugates and stereocomplexes of conjugates may include oligolactic acid comprising 2 to 20 lactic acid subunits and may be attached through an amide linkage to the nitrogen of the 4(N) of the gemcitabine or gemcitabine derivative. Compositions comprising water and a micelle comprising a polylactic acid-containing polymer and the oligolactic acid conjugate or stereocomplex of conjugates may be readily prepared. Methods of inhibiting or killing cancer cells and treating gemcitabine sensitive cancers are also provided.
    Type: Application
    Filed: January 26, 2024
    Publication date: June 27, 2024
    Applicant: Wisconsin Alumni Research Foundation
    Inventors: Glen Kwon, Yu Tong Tam
  • MODIFIED IL-2 POLYPEPTIDES AND USES THEREOF
    Publication number: 20240207417
    Abstract: The present disclosure relates to modified IL-2 polypeptides, compositions comprising modified IL-2 polypeptides, methods of making the same, and methods of using the modified IL-2 polypeptides for treatment of diseases. In one aspect, the disclosure relates to the treatment of cancer using the modified IL-2 polypeptides. In some embodiments, the disclosed IL-2 polypeptides exhibit preferential binding characteristics to IL-2 receptor ?? complex (IL-2R?) over IL-2 receptor ??? complex (IL-2R?). In some embodiments, the molecular weight distribution of the modified IL-2 polypeptides is monodispersed.
    Type: Application
    Filed: January 27, 2023
    Publication date: June 27, 2024
    Inventors: Vijaya Raghavan PATTABIRAMAN, Roberto IACONE, Jean-Philippe CARRALOT, Matilde ARÉVALO-RUIZ, Jeffrey William BODE, Alexander MAYWEG, Fränzi WEIBEL, Anna HAYDN, Régis BOEHRINGER, Görkem KURTULDU SAHIN, Claudia FETZ, Camille DELON, Eric ARMENTANI, Alexander FLOHR, Giorgio OTTAVIANI
  • LINKERS, DRUG LINKERS AND CONJUGATES THEREOF AND METHODS OF USING THE SAME
    Publication number: 20240207418
    Abstract: The present invention provides Polar units, Linker intermediates, Linkers, Drug-Linkers and Conjugates thereof.
    Type: Application
    Filed: July 28, 2023
    Publication date: June 27, 2024
    Inventors: Xiao SHANG, Haidong LIU, Julia GAVRILYUK, Baiteng ZHAO, Tae HAN
  • SOLID FORM OF 2-CHLORO-2'-DEOXY-ADENOSINE COMPLEX WITH HPßCD
    Publication number: 20240207419
    Abstract: A pure amorphous form of 2-chloro-2?-deoxy-adenosine (Cladribrine (1)) complex with HP?CD is provided, wherein the percentage of crystallinity is less than 0.5% (w/w), or alternatively less than 0.2% (w/w). Also provided is a process for the preparation of amorphous form of Cladribine (1) complex with pharmaceutically suitable excipient having percentage of crystallinity less than 0.5%, or alternatively less than 0.2%. (w/w).
    Type: Application
    Filed: April 9, 2022
    Publication date: June 27, 2024
    Inventors: Manik Reddy PULLAGURLA, Jagadeesh Babu RANGISETTY
  • ENZYMATICALLY-CLEAVABLE GLYCIDYL METHACRYLATE HYALURONIC ACID
    Publication number: 20240207420
    Abstract: Disclosed herein is a hydrogel comprising hyaluronic acid polymers that are at least partially crosslinked with fibrinogen molecules. Also disclosed is a composition comprising a glycidyl methacrylate hyaluronic acid monomer chemically conjugated to a fibrinogen molecule. Also disclosed is a method for producing a hydrogel that involves combining the disclosed composition containing GMHA-Fib precursors with an effective amount of a photoinitiator and light (e.g. UV light) to crosslink unconjugated methacrylate sites through free-radical polymerization. Also disclosed is a method for treating a subject with a peripheral nerve injury that involves implanting the disclosed hydrogel at the site of the injury to promote neural regeneration.
    Type: Application
    Filed: April 20, 2022
    Publication date: June 27, 2024
    Inventors: Christine E. Schmidt, Mary M. Kasper
  • NERVE TARGETING PEPTIDES AND METHODS OF USE
    Publication number: 20240207421
    Abstract: The present disclosure provides nerve targeting peptides, including those comprising the amino acid sequence of any one of SEQ ID NOS: 1-710; conjugates comprising such nerve targeting peptides and a cargo molecule; compositions comprising said peptides and conjugates; and methods of said peptides and conjugates in surgical procedures and other applications.
    Type: Application
    Filed: December 3, 2021
    Publication date: June 27, 2024
    Inventors: Michael WHITNEY, Quyen NGUYEN
  • NERVE TARGETING PEPTIDE CONJUGATES AND METHODS OF USE
    Publication number: 20240207422
    Abstract: Nerve targeting peptides, including those comprising the amino acid sequence of any one of SEQ ID NOS:1-557, Formulas (I)-(XLIV), and Subformulas (Ia)-(XLIVa); conjugates comprising such nerve targeting peptides and a cargo molecule; compositions comprising such peptides and conjugates; and their use in methods of fluorescent guided surgery and other applications.
    Type: Application
    Filed: December 6, 2023
    Publication date: June 27, 2024
    Inventors: Michael Whitney, Quyen Nguyen
  • PROGAMMABLE POLYMERIC DRUGS
    Publication number: 20240207423
    Abstract: Compounds useful as biologically active compounds are disclosed. The compounds have the following structure (I): or a stercoisomer, tautomer or salt thereof, wherein R1, R2, R3, L, L1, L2, L3, L4, M, m and n are as defined herein. Methods associated with preparation and use of such compounds is also provided.
    Type: Application
    Filed: February 28, 2024
    Publication date: June 27, 2024
    Inventor: Tracy MATRAY
  • USE OF HER2-TARGETING ANTIBODY-DRUG CONJUGATE IN TREATMENT OF HER2-LOW EXPRESSING BREAST CANCER
    Publication number: 20240207424
    Abstract: Provided is a method for treating a patient with Human Epidermal Growth Factor Receptor 2 (HER2)-low expressing breast cancer.
    Type: Application
    Filed: November 16, 2023
    Publication date: June 27, 2024
    Inventors: Jianmin FANG, Xiaohong SU
  • AURISTATIN LINKER-PAYLOADS, PHARMACEUTICAL COMPOSITIONS, AND USES THEREOF
    Publication number: 20240207425
    Abstract: The present disclosure is directed to linker-payloads, and pharmaceutically acceptable salts, solvates, or stereoisomer thereof, comprising a structure of formula I: The disclosure is also directed to pharmaceutical compositions comprising these compounds and the use of these compounds, intermediates thereof, and compositions in the prevention or treatment of cancers and/or tumors.
    Type: Application
    Filed: December 12, 2023
    Publication date: June 27, 2024
    Applicant: Merck Sharp & Dohme LLC
    Inventors: Manoj B. Charati, John A. Flygare, Rebecca Elizabeth Johnson, Simon B. Lang, W. Michael Seganish
  • COVALENTLY-MODIFIED STEROID ACID-PEPTIDES HAVING ENHANCED STABILITY AND/OR BIOLOGICAL ACTIVITY
    Publication number: 20240207426
    Abstract: Steroid acid-peptide conjugates covalently modified for improved stability and/or biological activity are described herein. Covalent modifications include the formation of multimeric compounds comprising at least two steroid acid-peptide monomers covalently bound to one another that behave as new chemical entities, as well as protecting one or more free thiol groups present in the steroid acid-peptide conjugates to improve their stability and/or biological activity.
    Type: Application
    Filed: May 16, 2023
    Publication date: June 27, 2024
    Inventor: Simon BEAUDOIN
  • SELECTIVE DRUG RELEASE FROM INTERNALIZED CONJUGATES OF BIOLOGICALLY ACTIVE COMPOUNDS
    Publication number: 20240207427
    Abstract: The invention relates to conjugates of biologically active compounds, wherein such a conjugate is comprised of a sequence of amino acids containing a tripeptide that confers selective cleavage by tumor tissue homogenate for release of free drug and/or improves biodistribution into the tumor tissue in comparison to normal tissue homogenate from the same species, wherein the normal tissue is the site of an adverse event associated with administration to a human subject in need thereof of a therapeutically effective amount of a comparator conjugate whose amino acid sequence is a dipeptide known to be selectively cleavable by Cathepsin B.
    Type: Application
    Filed: September 14, 2023
    Publication date: June 27, 2024
    Inventors: Scott JEFFREY, Ryan LYSKI, Philip MOQUIST, Nicole DUNCAN, Noah BINDMAN, Nicole OKELEY, Peter SENTER, Divya AWASTHI
  • COMPOSITIONS AND METHODS FOR THE DEPLETION OF CELLS
    Publication number: 20240207428
    Abstract: The invention provides compositions and methods useful for the depletion of cells, such as CD45+, CD135+, CD34+, CD90+, and/or CD110+ cells, and for the treatment of various hematopoietic diseases, metabolic disorders, cancers, and autoimmune diseases, among others. Described herein are antibodies, antigen-binding fragments, ligands, and conjugates thereof that can be applied to effect the treatment of these conditions, for instance, by depleting a population of CD45+, CD135+, CD34+, CD90+, or CD110+ cells in a patient, such as a human. The compositions and methods described herein can be used to treat a disorder directly, for instance, by depleting a population of CD45+, CD135+, CD34+, CD90+, or CD110+ cancer cells or autoimmune cells.
    Type: Application
    Filed: November 3, 2023
    Publication date: June 27, 2024
    Inventors: Andrew Nixon, Dwight Morrow, Adam Hartigan
  • LINKERS, DRUG LINKERS AND CONJUGATES THEREOF AND METHODS OF USING THE SAME
    Publication number: 20240207429
    Abstract: The present invention provides Polar units, Linker intermediates, Linkers, Drug-Linkers and Conjugates thereof.
    Type: Application
    Filed: July 28, 2023
    Publication date: June 27, 2024
    Inventors: Xiao SHANG, Haidong LIU, Julia GAVRILYUK, Baiteng ZHAO, Tae HAN
  • MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
    Publication number: 20240207430
    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: Application
    Filed: March 1, 2024
    Publication date: June 27, 2024
    Applicant: Dyne Therapeutics, Inc.
    Inventors: Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim
  • USE OF ANTIBODY-DRUG CONJUGATE IN COMBINATION WITH IMMUNE CHECKPOINT INHIBITOR IN TREATMENT OF UROTHELIAL CANCER
    Publication number: 20240207431
    Abstract: Provided is use of an antibody-drug conjugate targeting Her2 in combination with an immune checkpoint inhibitor such as PD-1 antibody or PD-L1 antibody, in the preparation of a medicine for treating patients with urothelial cancer, especially locally advanced or metastatic urothelial cancer. Compared to treatment with either of the two drugs alone, treatment with both in combination has a marked synergistic effect and significant therapeutic effect. In addition, the combination treatment had good efficacy for patients with low HER2 IHC expression (1+).
    Type: Application
    Filed: November 16, 2023
    Publication date: June 27, 2024
    Inventors: Jianmin FANG, Jing JIANG, Shenjun LI, Xiaohong SU
  • ANTIBODIES BINDING TROP2 AND USES THEREOF
    Publication number: 20240207432
    Abstract: Provided is an isolated monoclonal antibody that specifically binds human TROP2, or the antigen-binding portion thereof. A nucleic acid molecule encoding the antibody or the antigen-binding portion thereof, an expression vector, a host cell and a method for expressing the antibody or the antigen-binding portion thereof are also provided. Further provided are a bispecific molecule, an immunoconjugate, a chimeric antigen receptor, an oncolytic virus and a pharmaceutical composition comprising the antibody or the antigen-binding portion thereof, as well as a treatment method using an anti-TROP2 antibody or the antigen-binding portion thereof.
    Type: Application
    Filed: April 21, 2021
    Publication date: June 27, 2024
    Inventor: Mingjiu CHEN
  • DRUG CONJUGATES CONTAINING ALPHA-ENOLASE ANTIBODIES AND USES THEREOF
    Publication number: 20240207433
    Abstract: An immunoconjugate includes an anti-ENO-1 antibody, or a binding fragment thereof, and a therapeutic agent or a label, having the formula: Ab-(L-D)m, wherein Ab is the anti-ENO-1 antibody or the binding fragment thereof, L is a linker or a direct bond, D is the therapeutic agent or the label, and m is an integer from 1 to 12. The antibody may be a monoclonal antibody, which may be a humanized antibody or fully human antibody. A method for treating an inflammatory disease, immune disorder, or cancer includes administering to a subject in need of such treatment a pharmaceutically effective amount of an immunoconjugate containing an antibody against ENO-1, or a binding fragment thereof, and a therapeutic agent covalently conjugated with the antibody.
    Type: Application
    Filed: May 10, 2021
    Publication date: June 27, 2024
    Applicant: HUNILIFE BIOTECHNOLOGY, INC.
    Inventors: Ta-Tung Yuan, Chi-Kuan Chen, Mao-Lin Chen
  • METHODS FOR PREPARING CYCLOOCTENES ANDCONJUGATES THEREOF
    Publication number: 20240207434
    Abstract: The present invention pertains to improved methods for preparing cyclooctenes and conjugates thereof. In particular, methods for regioselective modification and/or enantiomerically enriching compounds are disclosed. In addition, compounds prepared by these methods are disclosed as well.
    Type: Application
    Filed: March 16, 2022
    Publication date: June 27, 2024
    Applicant: Tagworks Pharmaceuticals B.V.
    Inventors: Wolter TEN HOEVE, Marc Stefan ROBILLARD, Catalina FERRER, Laurens Henri Johan KLEIJN, Ronny Mathieu VERSTEEGEN
  • PROCESS FOR THE PREPARATION OF GLUCURONIDE DRUG-LINKERS AND INTERMEDIATES THEREOF
    Publication number: 20240207435
    Abstract: The present invention provides improved processes for the preparation of drug-linkers with a ?-glucuronide cleavable unit, as well as intermediates thereof.
    Type: Application
    Filed: June 23, 2023
    Publication date: June 27, 2024
    Inventors: Yunyu MAO, Philip MOQUIST, Anusuya CHOUDHURY, Wendel DOUBLEDAY
  • PREPARATION METHOD AND APPLICATION OF BIOMIMETIC NANO-PROTECTANT FOR DETOXIFYING DOX-INDUCED CARDIAC AND SYSTEMIC TOXICITY
    Publication number: 20240207436
    Abstract: A preparation method and application of a biomimetic nano-protectant for detoxifying doxorubicin (DOX)-induced cardiac and systemic toxicity are provided. The preparation method comprises the following steps: obtaining and mixing DNA and protamine (Pro) to prepare DNA&Pro nanoparticles; reacting heterofunctional polyethylene glycol DSPE-PEG2000-MAL with PCM/KALA to prepare DSPE-PEG2000-PCM/KALA; inserting DSPE-PEG2000-PCM/KALA into a surface of Red Blood Cell Membrane (RBCM) to prepare PCM/KALA-RBCM; and mixing the RBCM-PCM/KALA with the DNA&Pro nanoparticles to prepare a biomimetic nano-protectant DNA&Pro@RBCM-PCM/KALA. The biomimetic nano-protectant prepared is applied to detoxification of DOX.
    Type: Application
    Filed: June 27, 2022
    Publication date: June 27, 2024
    Applicant: The Second Affiliated Hospital of Wenzhou Medical University
    Inventors: Yijie CHEN, Mengchun CHEN, Weiliang XIA, Deli ZHUGE, Haiyan ZHU, Yingzheng ZHAO, Qi JIANG
  • MOLECULAR SELF-ASSEMBLED NANOPARTICLES AND PREPARATION METHOD AND APPLICATION THEREOF
    Publication number: 20240207437
    Abstract: Molecular self-assembled nanoparticles and a preparation method and application thereof are provided in the present application, relating to the technical field of precise treatment of arthritis. The molecular self-assembled nanoparticles include a neutrophil membrane and aspirin-functionalized polyethylene glycol (PEG) nanoparticles, where the neutrophil membrane is coated on a surface of the aspirin-functionalized PEG nanoparticles, and a mass ratio of the neutrophil membrane to the aspirin-functionalized PEG nanoparticles is 1:8.
    Type: Application
    Filed: August 3, 2023
    Publication date: June 27, 2024
    Inventors: Bolei CAI, Liang KONG, Feng CAO, Fuwei LIU, Le WANG, Yunpeng LI, Taiqiang DAI, Han BAO
  • LIQUID CRYSTAL-BASED MULTIFUNCTIONAL MICRO ROBOT
    Publication number: 20240207438
    Abstract: Disclosed is a liquid crystal-based multifunctional micro robot including a micro structure including at least one topological defect, an adherent micro particle that is bound to the topological defect and induces self-assembly of a target including a bacterium and a target material based on an antigen-antibody reaction, a physical binding reaction, or a chemical binding reaction, and the bacterium that is attached to the adherent micro particle and provides self-power so that the micro structure approaches the target material.
    Type: Application
    Filed: October 5, 2023
    Publication date: June 27, 2024
    Inventors: Young-Ki Kim, Sangmin Jeon, Chang Yun Son, Minjae Lee, Yena Choi, Jin-Kang Choi, Kwang-Suk Oh, Hyunsoo Han, Eunsu Cho
  • HIGH STEROL-CONTAINING LIPID NANOPARTICLES
    Publication number: 20240207439
    Abstract: The present disclosure provides a lipid nanoparticle comprising: a nucleic acid cargo molecule; sterol or a derivative thereof present at elevated content; neutral lipid; an ionizable lipid; and a hydrophilic polymer-lipid conjugate present at a content between 0.5 and 3 mol %, wherein each mol % content is relative to total lipid present in the lipid nanoparticle.
    Type: Application
    Filed: March 8, 2024
    Publication date: June 27, 2024
    Inventors: Kevin An, Daniel Kurek, Jayesh Kulkarni, Dominik Witzigmann
  • PHARMACEUTICAL COMPOSITION CONTAINING CELL-DERIVED NATURAL OR ARTIFICIAL NANOVESICLES LOADED WITH ANTISENSE OLIGONUCLEOTIDE-BASED DRUG
    Publication number: 20240207440
    Abstract: Pharmaceutical compositions and methods of making the composition are disclosed, wherein nucleic acid-based drugs can be introduced into cells by using cell-derived natural or artificial nanovesicles loaded with nucleic acid-based drugs. In the compositions, natural or artificial nanovesicles loaded with antisense oligonucleotides exhibit properties that inhibit proliferation and migration of cancer cells and increase drug sensitivity when co-administered with conventional chemotherapeutic agents.
    Type: Application
    Filed: December 29, 2023
    Publication date: June 27, 2024
    Applicant: PRIMORIS THERAPEUTICS Co., Ltd.
    Inventors: Jae-yong LEE, Hee-jin AHN, Dae-han LEE, Dong-yeol LEE, Kyu-heum NA
  • CAPSID VARIANTS AND USES THEREOF
    Publication number: 20240207441
    Abstract: Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., an anti-inflammatory transgene encoding one or more gene products) to a joint. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for joint tissues (e.g., cartilage, skin, muscle, and bone cells). Methods of treating arthritis in a joint by administering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.
    Type: Application
    Filed: April 26, 2022
    Publication date: June 27, 2024
    Applicant: University of Massachusetts
    Inventors: Yukiko Maeda, Phillip Tai, Guangping Gao
  • ALL-IN-ONE DENDRIMER-BASED LIPID NANOPARTICLES ENABLE PRECISE HDR-MEDIATED GENE EDITING IN VIVO
    Publication number: 20240207442
    Abstract: In some aspects, the present disclosure provides compositions comprising one or more of each of the following nucleic acids: (1) a mRNA; (2) a sgRNA; and (3) a DNA; and a lipid nanoparticle comprising at least one ionizable lipid; wherein the each of the nucleic acids are encapsulated within the lipid nanoparticle, and pharmaceutical compositions thereof. The present disclosure also provides methods employing said compositions and/or pharmaceutical compositions, such as methods of repairing genes, methods of performing homology directed repair on the genome, and methods of treating diseases or disorders.
    Type: Application
    Filed: April 22, 2022
    Publication date: June 27, 2024
    Applicant: The Board of Regents of The University of Texas System
    Inventors: Daniel J. SIEGWART, Lukas FARBIAK
  • GENE THERAPY FOR IMMUNO-ONCOLOGY APPLICATIONS
    Publication number: 20240207443
    Abstract: The invention provides compositions and methods for treatment of glioblastoma and other conditions. In particular, the invention provides a recombinant adeno-associated virus (AAV) vector comprising a transgene encoding one or more interferon polypeptides and a CAG promoter which directs the expression of the transgene. The compositions are particularly suitable for intratumoral administration in gene therapy applications.
    Type: Application
    Filed: June 30, 2021
    Publication date: June 27, 2024
    Applicants: CHAN ZUCKERBERG BIOHUB, INC., THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    Inventors: NICOLE K. PAULK, JOSEPH L DERISI, DAVID R. RALEIGH
  • LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING PHENYLALANINE HYDROXYLASE AND USES THEREOF
    Publication number: 20240207444
    Abstract: This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding phenylalanine hydroxylase.
    Type: Application
    Filed: March 24, 2022
    Publication date: June 27, 2024
    Inventors: Kerry Benenato, Mark Cornebise, Edward Hennessy
  • TUMOR-TARGETING SYNTHETIC ADENOVIRUSES AND USES THEREOF
    Publication number: 20240207445
    Abstract: Synthetic adenoviruses with liver detargeting mutations and expressing an adenovirus type 34 (Ad34) fiber protein, or a chimeric fiber protein with an Ad34 knob domain, are described. The synthetic adenoviruses traffic to sites of tumors. Use of the synthetic adenoviruses for delivering diagnostic or therapeutic transgenes to tumors are also described.
    Type: Application
    Filed: September 29, 2023
    Publication date: June 27, 2024
    Applicant: Salk Institute for Biological Studies
    Inventors: Clodagh O'Shea, Colin Powers, Lei Zhang
  • M6A-COUPLED EFFECTOR PROTEIN EXPRESSION SYSTEM AND METHODS OF MAKING AND USING SAME
    Publication number: 20240207446
    Abstract: The present disclosure provides systems and methods for m6A-dependent delivery and m6A-dependent delivery targeted of a polypeptide to a cell. In certain embodiments, compositions, systems, and methods are provided that provide for m6A-dependent delivery of effector proteins, for example, effector proteins, such as a tumor suppression proteins and m6a regulation systems, mediated by CRISPRi in embodiments.
    Type: Application
    Filed: October 12, 2023
    Publication date: June 27, 2024
    Applicant: Duke University
    Inventor: Kathryn Meyer
  • Recombinant Adeno-Associated Vectors For Targeted Treatment
    Publication number: 20240207447
    Abstract: Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.
    Type: Application
    Filed: October 13, 2023
    Publication date: June 27, 2024
    Inventors: Saswati CHATTERJEE, Laura SMITH, Kamehameha WONG
  • CRISPR/RNA-GUIDED NUCLEASE-RELATED METHODS AND COMPOSITIONS FOR TREATING RHO-ASSOCIATED AUTOSOMAL-DOMINANT RETINITIS PIGMENTOSA (ADRP)
    Publication number: 20240207448
    Abstract: CRISPR/RNA-guided nuclease-related compositions and methods for treatment of RHO-associated retinitis pigmentosa, e.g., autosomal-dominant retinitis pigmentosa (adRP).
    Type: Application
    Filed: April 15, 2022
    Publication date: June 27, 2024
    Applicant: EDITAS MEDICINE, INC.
    Inventors: Benjamin Aryeh DINER, Deepak REYON, Mariacarmela ALLOCCA
  • COMPOSITIONS AND METHODS FOR TREATMENT OF CRX-MEDIATED RETINOPATHIES
    Publication number: 20240207449
    Abstract: Among the various aspects of the present disclosure is the provision of compositions and methods of making regulatable, adeno-associated viral vectors for the therapeutic expression of CRX and methods of use thereof.
    Type: Application
    Filed: April 11, 2022
    Publication date: June 27, 2024
    Inventor: Shiming CHEN
  • GENE THERAPIES FOR STARGARDT DISEASE (ABCA4)
    Publication number: 20240207450
    Abstract: Aspects of the disclosure relate—compositions and methods useful for delivering minigenes—a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g., a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a ABCA4 protein (e.g., the gene product of ABCA4 gene) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the ABCA4 gene, for example, Stargardt disease.
    Type: Application
    Filed: April 25, 2022
    Publication date: June 27, 2024
    Applicant: University of Massachusetts
    Inventor: Hemant Khanna
  • G-PROTEIN-GATED-K+ CHANNEL-MEDIATED ENHANCEMENTS IN LIGHT SENSITIVITY IN ROD-CONE DYSTROPHY (RCD)
    Publication number: 20240207451
    Abstract: The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated inwardly rectifying potassium channel (GIRK), in particular GIRK4 S143T, activated by G proteins recruited by cone opsin expressed in degenerating cones.
    Type: Application
    Filed: October 20, 2023
    Publication date: June 27, 2024
    Inventor: Deniz DALKARA
  • NOVEL COMPOSITIONS WITH BRAIN-SPECIFIC TARGETING MOTIFS AND COMPOSITIONS CONTAINING SAME
    Publication number: 20240207452
    Abstract: Provided herein are compositions including brain-capillary binding and/or blood-brain barrier crossing (BBB) tissue-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 Y-G/A/R/K-Y/H-GNPA-T/R/H-RYFD-V/K. 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: April 22, 2022
    Publication date: June 27, 2024
    Applicant: The Trustees of the University of Pennsylvania
    Inventors: Joshua Joyner SIMS, James M. WILSON, Yuan YUAN
  • 13C METHACETIN GRANULE, AND PREPARATION METHOD THEREFOR AND USE THEREOF
    Publication number: 20240207453
    Abstract: The present invention relates to the field of pharmaceutical preparations, in particular to a 13C-methacetin granule, and a preparation method therefor and the use thereof. Provided is the 13C-methacetin granule, comprising 13C-methacetin, mannitol, a co-solvent and a wetting agent. The 13C-methacetin granule has good stability, is beneficial for storage and the production of subsequent preparations, etc. The 13C-methacetin granule has good water solubility, can be rapidly dissolved in water during clinical use, and is convenient to take. Further provided is the use of the 13C-methacetin granule in the preparation of drugs, reagents or kits for disease diagnosis, evaluation of the liver reserve function and preoperative and postoperative evaluation of the liver. The provided preparation method of the 13C-methacetin granule has mild conditions, simple operation and convenient production control.
    Type: Application
    Filed: April 24, 2022
    Publication date: June 27, 2024
    Inventor: Aihua Gong
  • NEURO-BEHAVIORAL ASSAY FOR PHYSIOLOGICAL EVALUATION OF WATER-SOLUBLE ANESTHETICS
    Publication number: 20240207454
    Abstract: Disclosed are a system and an in vivo assay for the physiological evaluation of water-soluble general anesthetics. The assay is based on the effects of anesthetics on the frequency of the pacemaker nucleus, an endogenous neural oscillator in the brainstem, of the weakly electric fish Apteronotus leptorhynchus.
    Type: Application
    Filed: December 13, 2023
    Publication date: June 27, 2024
    Inventors: Gunther Zupanc, David Lehotzky
  • COMBINATION COMPRISING A NEUROTENSIN RECEPTOR BINDING COMPOUND AND NAPOX
    Publication number: 20240207455
    Abstract: The present invention relates to a combination comprising a neurotensin receptor binding compound and NAPOX for use for the treatment of a neurotensin receptor overexpressing tumour in a subject.
    Type: Application
    Filed: December 15, 2023
    Publication date: June 27, 2024
    Inventors: Stephan Klinz, Diane-Charlotte Imbs, Elodie Lewkowicz, Sylvie Rolland, Thomas Rohban, John R. Forbes
  • PHOTOCURABLE COMPOSITIONS AND METHODS FOR DELIVERING RADIATION TO A SUBJECT
    Publication number: 20240207456
    Abstract: The disclosure is directed in part to polymerizable compositions that can incorporate a radioactive source and be used to treat subjects with certain conditions, and methods of producing and use thereof.
    Type: Application
    Filed: June 15, 2023
    Publication date: June 27, 2024
    Inventors: Richard Brauckman, Jacob Freeman
  • CANCER-SPECIFIC POLYPEPTIDE AND USE THEREOF
    Publication number: 20240207457
    Abstract: An aspect relates to a cancer-specific polypeptide consisting of an amino acid sequence of SEQ ID NO: 1. The polypeptide according to an aspect may bind specifically to LGR5 protein expressed in a tumor tissue, and when binding to a labeling material (for example, a fluorescent material), a cancer that expresses the LGR5 protein may be diagnosed. Furthermore, when the polypeptide binds to an isotope, a metastatic tumor may be also diagnosed. When the polypeptide according to an aspect binds to a photosensitizer and is then administered to a subject, the photosensitizer may be activated through light irradiation so that cancer cells may be killed, thereby enabling the prevention, amelioration, or treatment of cancer.
    Type: Application
    Filed: May 10, 2022
    Publication date: June 27, 2024
    Inventors: Jae Myung PARK, Moon Hwa KWAK
  • TARGETED ALPHA PARTICLE THERAPY FOR SOMATOSTATIN RECEPTOR 2 POSITIVE NEUROENDOCRINE TUMORS AND METASTASES
    Publication number: 20240207458
    Abstract: Disclosed are TATE derivatives having a linker between the TATE moiety and a macrocyclic radionuclide chelating moiety. Methods of synthesis and use are also disclosed. The compounds preferably exhibit a ratio of kidney: liver uptake by a subject within 24 hours of 5 or less, 3 or less, 1 or less, 0.5 or less, preferably from 2:1 to 1:2.
    Type: Application
    Filed: April 26, 2022
    Publication date: June 27, 2024
    Inventors: David MORSE, Haitao JI, Thaddeus WADAS, Darpan PANDYA
  • METHOD FOR STORING INTERMEDIATE FOR RADIOPHARMACEUTICAL COMPOSITION, METHOD FOR PREPARATION OR STORAGE OF RADIOPHARMACEUTICAL COMPOSITION, INTERMEDIATE COMPOSITION FOR RADIOPHARMACEUTICAL COMPOSITION, AND PHARMACEUTICAL FORMULATION
    Publication number: 20240207459
    Abstract: A storage method according to an aspect of the present invention includes storing an intermediate of a radiopharmaceutical composition in an acetate buffer. The intermediate is a complex of a 3,6,9,15-tetraazabicyclo[9.3.1]pentadeca-1(15), 11, 13-triene-3,6,9-triacetic acid (PCTA) and an anti-epidermal growth factor receptor antibody (anti-EGFR antibody). The intermediate is stored for a storage period of not less than 24 hours.
    Type: Application
    Filed: March 11, 2022
    Publication date: June 27, 2024
    Inventors: Yukie YOSHII, Hiroki MATSUMOTO, Chika IGARASHI
  • HER2 AFFIBODY 99MTC MARKER COMPOSITION AND APPLICATION THEREOF
    Publication number: 20240207460
    Abstract: An HER2 affibody 99mTc marker composition and application thereof are provided, which relates to the field of radiopharmaceuticals and nuclear medicine. The HER2 affibody 99mTc marker composition contains HEPES, sodium glucoheptonate, vitamin C, cysteine, stannous chloride and an HER2 affibody.
    Type: Application
    Filed: August 16, 2021
    Publication date: June 27, 2024
    Inventors: Jiong CAI, Yi LIU, Dalin GAO, Xu SONG, Kai XIAO, Chen LIANG, Guozhong DING, Mingxia LIU
  • RADIOACTIVE ANTITUMOR AGENT
    Publication number: 20240207461
    Abstract: The present invention aims to provide a Actinium-225-labeled anti-MUC5AC humanized antibody that is superior in the specificity for mucin subtype 5AC (MUC5AC) and accumulation in tumor, and shows reduced renal toxicity. The present invention relates to a conjugate of a chelating agent chelated with Actinium-225 and an antibody, wherein the antibody is a humanized antibody that specifically binds to mucin subtype 5AC and has a heavy chain variable region consisting of the amino acid sequence shown in any of SEQ ID NOs: 1 to 4, and a light chain variable region consisting of the amino acid sequence shown in any of SEQ ID NOs: 5 to 8.
    Type: Application
    Filed: April 21, 2022
    Publication date: June 27, 2024
    Applicants: NIHON MEDI-PHYSICS CO., LTD., Sumitomo Pharma Co., Ltd.
    Inventors: Shota KOMOTO, Naoaki YAMADA