Abstract: The present disclosure relates generally to bacterial delivery vehicles for use in efficient transfer of a desired payload into a target bacterial cell. More specifically, the present disclosure relates to bacterial delivery vehicles with desired host ranges based on the presence of a chimeric receptor binding protein (RBP) composed of a fusion between the N-terminal region of a RBP derived from a lambda-like bacteriophage and the C-terminal region of a different RBP.
Abstract: A nerve in a mammal is optogenetically transduced, wherein the nerve is susceptible to stimulus by selective application of transdermal light, and a light source is applied to dermis of the mammal at or proximate to the optogenetically transduced nerve, to thereby stimulate the nerve. A wearable device for optogenetic motor control and sensation restoration of a mammal includes a wearable support, a power source at the wearable support, a controller at the wearable support and in electrical communication with a power source, and a transdermal light source coupled to the controller.
Type:
Grant
Filed:
October 31, 2017
Date of Patent:
November 22, 2022
Assignee:
Massachusetts Institute of Technology
Inventors:
Hugh M. Herr, Benjamin Maimon, Anthony Zorzos
Abstract: Disclosed herein are methods of treatment for an intraocular pressure (IOP)-associated condition in a subject, that include administering to the subject an effective amount of a tissue plasminogen activator (tPA) therapeutic agent. In one embodiment, the IOP-associated condition is glaucoma. The administration of a tPA therapeutic agent can be an extended administration intended to cause a reduction in IOP in the subject for a period of at least one day to a year or more, relative to IOP levels in the subject prior to administration of the tPA therapeutic agent. The tPA therapeutic agent can be, for example, tPA, a tPA derivative, a small molecule direct or indirect tPA agonist, or a gene therapy vector.
Type:
Grant
Filed:
May 8, 2020
Date of Patent:
October 25, 2022
Assignees:
The Research Foundation for The State University of New York, Icahn School of Medicine at Mount Sinai
Inventors:
Ioannis Danias, Oscar A. Candia, Rosana Gerometta
Abstract: Methods of treating a tumor in a subject include identifying a subject having, at risk for, or suspected of having a tumor, and administering to the subject an effective amount of an SRPX.
Type:
Grant
Filed:
March 11, 2020
Date of Patent:
October 25, 2022
Assignee:
University of Massachusetts
Inventors:
Michael R. Green, Guangping Gao, Manas Kumar Santra, Sanchita Bhatnagar
Abstract: Methods, tip assemblies and kits are provided for introducing material into cells. The tip assemblies include an attachment portion, a channel portion, and a constriction that function to reduce fluid pressure as a fluid passes through the constriction portion from the channel portion, whereby the tip assemblies form pores in the membranes of cells and introduce material into the cells. The material includes for example one selected from the group of: an inorganic compound, a drug, a genetic material, a protein, a carbohydrate, a synthetic polymer, and a pharmaceutical composition.
Abstract: Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B.
Type:
Grant
Filed:
December 29, 2017
Date of Patent:
October 18, 2022
Assignee:
The Trustees of the University of
Pennsylvania
Inventors:
James M. Wilson, Jenny Agnes Sidrane, Lakshmanan Govindasamy
Abstract: The present disclosure provides methods and compositions related to the modification of immune effector cells to increase therapeutic efficacy. In some embodiments, immune effector cells modified to reduce expression of one or more endogenous target genes, or to reduce one or more functions of an endogenous protein to enhance effector functions of the immune cells are provided. In some embodiments, immune effector cells further modified by introduction of transgenes conferring antigen specificity, such as exogenous T cell receptors (TCRs) or chimeric antigen receptors (CARs) are provided. Methods of treating a cell proliferative disorder, such as a cancer, using the modified immune effector cells described herein are also provided.
Type:
Grant
Filed:
March 29, 2022
Date of Patent:
October 4, 2022
Assignee:
KSQ Therapeutics, Inc.
Inventors:
Micah Benson, Jason J. Merkin, Gregory V. Kryukov, Solomon Martin Shenker, Michael R. Schlabach, Noah Jacob Tubo, James Martin Kaberna, II
Abstract: The present disclosure relates to recombinant viral vectors for the treatment and prevention of cancer. Oncolytic viral vectors incorporate one or more of the following features: viral replication restriction by insertion of tumor-suppressive microRNA (miRNA) target sequences into the viral genome; disruption of oncogenic miRNA function; cancer microenvironment remodeling; and cancer cell targeting by incorporation of protease-activated antibodies into the viral particle.
Type:
Grant
Filed:
July 10, 2019
Date of Patent:
September 27, 2022
Assignee:
Oncorus, Inc.
Inventors:
Kenneth P. Greenberg, Mitchell H. Finer
Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR-Cas systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.
Type:
Grant
Filed:
October 20, 2021
Date of Patent:
September 20, 2022
Assignee:
ARBOR BIOTECHNOLOGIES, Inc.
Inventors:
David R. Cheng, David A. Scott, Winston X. Yan
Abstract: The present disclosure provides methods that combine RNA fluorescent in situ sequencing (FISSEQ) with other molecular detection modalities, forming an integrated panomic detection platform. In various embodiments, the present disclosure provides systems and methods to prepare a biological sample to preserve the spatial relationships of biomolecules of interest within the biological sample for FISSEQ detection.
Type:
Grant
Filed:
February 28, 2019
Date of Patent:
September 20, 2022
Assignee:
President and Fellows of Harvard College
Inventors:
George M. Church, Evan R. Daugharthy, Richard C. Terry
Abstract: Described is an “artificial virus” (AV) programmed with biomolecules that can enter human cells and carry out precise human genome modification. The AVs comprise: at least one viral vector, such as bacteriophage T4; at least one therapeutic molecule, such as DNA, RNA, protein and their complex; and a lipid coating. Also described is a method of human genome modification, using such an AV, and a method of program such an AV.
Abstract: rAAV compositions useful for preventing the toxicity associated with organophosphate exposure are provided herein. The rAAV co-express a human butyrylcholinesterase and a proline-rich peptide. Each the esterase and the peptide may have an exogenous signal sequence. Also provided are compositions, e.g., aqueous liquid suspensions containing the rAAV.
Type:
Grant
Filed:
April 15, 2017
Date of Patent:
August 2, 2022
Assignee:
The Trustees of the University of Pennsylvania
Abstract: Aspects of the disclosure relate to compositions and methods of treating certain genetic disease (e.g., Neurofibromatosis type I) by delivering functional neurofibromin 1 (NF1) protein (e.g., mini-NF1 protein and/or full-length NF1 protein) to target cell (e.g., cells and/or tissue of a subject). The disclosure is based, in part, on isolated nucleic acids (e.g., rAAV vectors) and rAAVs engineered to express a functional NF1 protein (e.g., mini-NF1 protein and/or full-length NF1 protein) or variants thereof.
Abstract: Methods and compositions for treating diseased or damaged tissue, such as Inflammatory Bowel Disease, e.g., Ulcerative Colitis, include tissue regeneration using stem cells or tissue grafts which stimulate stem cell migration to the damaged tissue. The tissue grafts can be extracellular matrix (ECM) material, such as tissue-specific extracellular matrix (TS-ECM). The methods can also include mucosal resection of the damaged or diseased tissue prior to placement of the graft.
Type:
Grant
Filed:
March 14, 2017
Date of Patent:
July 19, 2022
Assignees:
Regentys Corporation, University of Pittsburgh—of the Commonwealth System of Higher Education
Inventors:
Marc Ramer, Stephen F. Badylak, Timothy Keane
Abstract: The present disclosure relates to methods for increasing telomere length in one or more human cells and/or increasing genome stability of one or more human cells, for example by contacting one or more human cells with an agent that increases expression of Zscan4 in the one or more human cells. Methods of treating a subject in need of telomere lengthening, treating a disease or condition associated with a genomic and/or chromosome abnormality, of rejuvenating one or more human cells, of rejuvenating tissues or organs, and of rejuvenating a subject in need thereof, for example by contacting one or more human cells in the subject with an agent that increases expression of Zscan4, or by administering to a subject in need thereof, an agent that increases expression of Zscan4 are also provided.
Abstract: The present invention provides a novel method of treatment for treating brain disorders that manifest oxidative stress by providing targeted populations of neurons with the ability to catabolize the acetylated amino acid derivative, N-acetylaspatic acid (NAA) and further supply extraphysiological levels of ATP to neurons via the targeted expression of the NAA catabolic enzyme aspartoacylase (ASPA) in neurons and astrocytes.
Abstract: Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
Type:
Grant
Filed:
December 29, 2017
Date of Patent:
July 12, 2022
Inventors:
James M. Wilson, Jenny Agnes Sidrane, Scott Ashley
Abstract: The present invention relates to the production of cell cultures and tissues from undifferentiated pluripotent stem cells using three-dimensional biomimetic materials. The resultant cell cultures or tissues can be used in any of a number of protocols including testing chemicals, compounds, and drugs. Further, the methods and compositions of the present invention further provide viable cell sources and novel cell delivery platforms that allow for replacement of diseased tissue and engraftment of new cardiomyocytes from a readily available in vitro source. The present invention includes novel methods required for the successful production of cell cultures and tissues, systems and components used for the same, and methods of using the resultant cell and tissue compositions.
Type:
Grant
Filed:
May 23, 2019
Date of Patent:
June 28, 2022
Assignee:
Auburn University
Inventors:
Elizabeth A. Lipke, Petra Kerscher, Alexander J. Hodge
Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.
Abstract: The present disclosure provides methods for re-stimulating TIL populations that lead to improved phenotype and increased metabolic health of the TILs and provides methods of assaying for TIL populations to determine suitability for more efficacious infusion after re-stimulation.