Patents Issued in June 8, 2023
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Publication number: 20230174945Abstract: A method for inducing differentiation into pancreatic ? cells includes: a step (a) of culturing endodermal cells, which have been induced to differentiate from pluripotent stem cells, in the presence of a bone morphogenetic protein (BMP) signaling inhibitor, and retinoic acid or a retinoic acid analog to induce differentiation into primitive gut tube (PGT) cells; a step (b) of culturing the primitive gut tube (PGT) cells to induce differentiation into pancreatic endocrine precursor (EP) cells; and a step (c) of culturing the pancreatic endocrine precursor (EP) cells to induce differentiation into pancreatic ? cells, in which the step (b) and the step (c) are performed in the absence of ascorbic acid.Type: ApplicationFiled: March 18, 2021Publication date: June 8, 2023Applicants: KANEKA CORPORATION, NATIONAL CENTER FOR GLOBAL HEALTH AND MEDICINEInventors: Keiichi KIMURA, Masato IBUKI, Hitoshi OKOCHI, Shigeharu YABE
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Publication number: 20230174946Abstract: Described herein are particular infection model systems, methods of studying infection, and method of screening compounds in various model systems. Particularly, SARS-CoV-2 is studied in these organ and infection models.Type: ApplicationFiled: April 30, 2021Publication date: June 8, 2023Applicants: CEDARS-SINAI MEDICAL CENTER, THE REGENTS OF THE UNIVERSITY OF CALIFORNIAInventors: Barry R. Stripp, Apoorva Mulay, Bindu Konda, Arun Sharma, Clive Svendsen, Vaithilingaraja Arumugaswami, Dhruv Sareen, Hanan Shaharuddin, Victoria Wang, Roberta S. Santos
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Publication number: 20230174947Abstract: Described herein are methods and compositions for use in improving a vascular barrier of endothelial cells. The methods may include the use of barrier agonist compounds and improved techniques for culturing endothelial cells. The improved methods and compositions for culturing endothelial cells may include at least one or more of the following: an adenylyl cyclase activator; an activator of Sphingosine-1-phosphate (S1P) receptor internalization; a direct or indirect inducer of tight junction protein expression, and a direct or indirect inducer of adherens junction protein expression.Type: ApplicationFiled: December 7, 2022Publication date: June 8, 2023Applicant: United Therapeutics CorporationInventors: Bryan Fioret, Thomas Petersen, Ryan Bonvillain, David Graham, Peter Vu, Lotte van den Goor
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Publication number: 20230174948Abstract: The present invention relates to endothelial cells and methods of generating endothelial cells from pluripotent stem cells.Type: ApplicationFiled: March 5, 2021Publication date: June 8, 2023Inventors: Gabor Foldes, Qasim A. Majid, Sian E. Harding
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Publication number: 20230174949Abstract: Provided are methods and compositions for tissue engineering including methods and compositions for the generation of vascularized organoids in vitro.Type: ApplicationFiled: December 2, 2022Publication date: June 8, 2023Inventors: Prashant Mali, Amir Dailamy, Udit Parekh
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Publication number: 20230174950Abstract: The present invention relates to a kidney organoid having a nephron-like structure and a production method therefor. A kidney organoid culture system using kidney dECM hydrogels according to the present invention induced the vascularization of the kidney organoid and the expression of podocytes, tubular transporters, and cilium genes, and has an effect of forming a more mature nephron-like structure. Therefore, the kidney organoid produced by the method of the present invention is an option for treating nephron loss through transplantation into humans, and is expected to be utilized as a kidney on a chip, which is an in vitro kidney model.Type: ApplicationFiled: November 24, 2020Publication date: June 8, 2023Inventors: Yong Kyun KIM, Dong-Woo CHO, Sun Ah NAM, Jin Won KIM, Jae Yeon LEE, Jae Yun KIM
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Publication number: 20230174951Abstract: The present invention relates to transgenic grape cells, compositions comprising such cells or extracts or fractions thereof, and methods of use thereof for inhibiting or reducing the incidence of cytokine release syndrome or cytokine storm in a subject. Disclosed herein are also methods for preventing, treating, reducing the incidence, suppressing or inhibiting a coronavirus infection or a symptom thereof.Type: ApplicationFiled: April 25, 2021Publication date: June 8, 2023Applicants: THE STATE OF ISRAEL, MINISTRY OF AGRICULTURE & RURAL DEVELOPMENT, AGRICULTURAL RESEARCH ORGANIZATION, B. G. Negev Technologies and Applications Ltd., at Ben-Gurion UniversityInventors: Avihai PERL, Michal OREN-SHAMIR, Miriam FRIEDMAN-EINAT, Ru WANG, Aaron FAIT
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Publication number: 20230174952Abstract: A method of producing plant virus-like particles includes freeze drying an aqueous solution of plant virus particles to produce a substantially RNA-free plant virus-like particles.Type: ApplicationFiled: July 19, 2022Publication date: June 8, 2023Inventors: Jonathan Pokorski, Nicole Steinmetz, Parker Lee
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Publication number: 20230174953Abstract: Method for producing a defective interfering viral genome (DVG), defective interfering particles comprising the DVG, and methods and uses thereof.Type: ApplicationFiled: March 26, 2021Publication date: June 8, 2023Inventors: MARCO VIGNUZZI, BJORN MEYER, VERONICA REZELJ, LAURA LEVI, VERONIKA BERNHAUEROVA, THOMAS VALLET, TANGUY PIEPLU, DJOSHKUN SHENGJULER, STEPHANIE BEAUCOURT, HERVE BLANC, NATHALIE PARDIGON, GIOVANNA BARBA-SPAETH, MARIA-CARLA SALEH
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Publication number: 20230174954Abstract: The present disclosure relates to bacteriophage and compositions capable of infecting and killing Pseudomonas, and use of the same for treating Pseudomonas, e.g. Pseudomonas aeruginosa, bacterial infections.Type: ApplicationFiled: April 27, 2021Publication date: June 8, 2023Inventors: Sébastien LEMIRE, Stacey Lynn KOLAR, Brian Clarke VARNUM
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Publication number: 20230174955Abstract: The disclosure herein provides a monomeric prolyl 4-hydroxylase. A microorganism including a monomeric prolyl 4-hydroxylase is provided. The disclosure provides a microorganism including a monomeric prolyl 4-hydroxylase; and another protein to be hydroxylated. A method for providing skincare benefits including applying the fusion protein of the present disclosure on skin is also taught.Type: ApplicationFiled: February 12, 2021Publication date: June 8, 2023Applicant: Modern Meadow, Inc.Inventors: Lixin DAI, Poonam SRIVASTAVA, Sumreet Singh JOHAR, Shashwat Mohan VAJPEYI, Mahadevan LAKSHMINARASIMHAN, James Egan WELCH
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Publication number: 20230174956Abstract: The present invention relates to a method of preparing a gene cluster construct including a plurality of genes encoding a multi-modular biosynthetic enzyme, the method including (A) a step of preparing a plurality of DNA fragments which are capable of reconstructing the gene cluster construct and have structures that can be ligated to each other and (B) a step of ligating the plurality of DNA fragments prepared in the step (A) to each other by mixing the plurality of DNA fragments in a solution to obtain the gene cluster construct.Type: ApplicationFiled: May 25, 2021Publication date: June 8, 2023Applicant: Spiber Inc.Inventors: David Lips, Gijs Verkleij
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Publication number: 20230174957Abstract: This application relates to mutant polymerases. This application discloses mutant phage-type RNA polymerases, such as a mutant T7, SP6, and T3 RNA polymerase, may use 2?-modified nucleoside triphosphates or deoxynucleotide triphosphates as substrates. Methods for producing nucleic acid molecules using these mutant polymerases are also disclosed.Type: ApplicationFiled: May 11, 2021Publication date: June 8, 2023Applicant: Thermo Fisher Scientific Baltics UABInventors: Arvydas Lubys, Zana Kapustina, Aiste Jaspone
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Publication number: 20230174958Abstract: The disclosure relates to methods and compositions for inhibiting the expression of the DUX4 gene in skeletal muscle cells. In some aspects, the invention includes a CRISPR interference platform that directs an epigenetic regulator to the DUX4 locus. In some aspects, methods described by the disclosure are useful in modulating DUX4 expression for the treatment of facioscapulohumeral muscular dystrophy (FSHD).Type: ApplicationFiled: April 6, 2021Publication date: June 8, 2023Inventors: Peter L. Jones, Charis L. Himeda
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Publication number: 20230174959Abstract: The present invention provides: a complex comprising a genome editing enzyme and a cell membrane-permeable peptide(CPP), wherein the CPP is fused to the genome editing enzyme; a complex comprising a genome editing enzyme, a target gene-specific nucleic acid, and a CPP, wherein the CPP is fused to the genome editing enzyme and/or the a target gene-specific nucleic acid; the complex comprising a polycationic moiety fused to the CPP, wherein the polycation moiety is statically bound to the target gene-specific nucleic acid; a genome editing method using the complex; and a kit for genome-editing, the kit including these complexes.Type: ApplicationFiled: December 17, 2021Publication date: June 8, 2023Applicants: NATIONAL UNIVERSITY CORPORATION TOTTORI UNIVERSITY, FOREST RESEARCH AND MANAGEMENT ORGANIZATIONInventors: Takashi Iwasaki, Kousei OMURA, Yoshino TANAKA, Yoshihiko NANASATO
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Publication number: 20230174960Abstract: The present invention relates to a fusion protein comprising a domain of a nuclease from the class II CRISPR system, and a Spo11 domain, wherein both domains of the fusion protein have deficient nuclease activity, as well as the use of this protein to induce targeted meiotic recombinations in a eukaryotic cell.Type: ApplicationFiled: May 20, 2021Publication date: June 8, 2023Inventors: ALEXANDRE SERERO, ALAIN NICOLAS
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Publication number: 20230174961Abstract: The present disclosure relates to automated multi-module instruments, compositions and methods for performing nucleic acid-guided nuclease editing; specifically, the disclosure provides nucleic acid cassettes, plasmids, vectors, and compositions comprising the same that employ homologous recombination for genome engineering by having a CRISPR nuclease cause a specific DSB while tethered to a repair nucleic acid.Type: ApplicationFiled: January 25, 2023Publication date: June 8, 2023Applicant: Inscripta, Inc.Inventors: Andrew Garst, Christian Siltanen
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Publication number: 20230174962Abstract: Disclosed are compositions and methods relating to variant ?-amylases. The variant ?-amylases are useful, for example, for starch liquefaction and saccharification, cleaning starchy stains, textile desizing, baking and brewing.Type: ApplicationFiled: July 30, 2019Publication date: June 8, 2023Inventors: Jonathan K. LASSILA, Patrica TRAN
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Publication number: 20230174963Abstract: Disclosed is a method for producing hyaluronidase or a variant thereof. Specifically, the method is capable of changing the N-glycan levels under culture conditions including a controlled concentration of glucose in the culture medium and a decreased culture temperature for a specific culture time period, thereby increasing the specific activity by 10% or more and improving the quality and production yield.Type: ApplicationFiled: August 6, 2021Publication date: June 8, 2023Inventors: Soon Jae PARK, Kyuwan KIM, Sang Hoon YUN, Jeong Soo CHO, Kibum PARK, Minsoo BYUN, Hyung Nam SONG, Ji-Sun KIM, Ki Seok NAM
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Publication number: 20230174964Abstract: The present disclosure relates to a novel serine protease variant.Type: ApplicationFiled: March 11, 2022Publication date: June 8, 2023Inventors: Joo Hee LEE, A-Ra CHO, Chang Jun JI
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Publication number: 20230174965Abstract: A method of producing a biofilm-like microorganism-polymer complex, the method comprising the step of exposing, in an aqueous medium, cells of a microorganism to a polymer comprising groups as defined by formula (la) or formula (lb), wherein: Y comprises an imine; and R2 comprises a CI-30 group. A biofilm-like microorganism-polymer complex comprising cells of a microorganism and a polymer comprising groups as defined by formula (la) and/or formula (lb). A method for producing a chemical and/or biological product using the microorganism-polymer complex, and the chemical and/or biological products obtained therefrom. A method of dispersing the microorganism-polymer complex. Polymers of formula (la) and of formula (lb).Type: ApplicationFiled: April 16, 2021Publication date: June 8, 2023Inventors: Francisco FERNANDEZ-TRILLO, Timothy William OVERTON, Pavan Rao ADONI, Omar Nicholas Edward HUNEIDI
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Publication number: 20230174966Abstract: The present invention in general relates to the field of RNA purification methods. In particular, it relates to a method or reducing the dsRNA content of RNA samples, and accordingly increasing the ssRNA concentration in such samples. Said method is based on a filtration step performed on RNA samples containing ssRNA, dsRNA and at least one salt in the absence of cellulose.Type: ApplicationFiled: June 21, 2021Publication date: June 8, 2023Inventors: Senne DILLEN, Michiel HOLTHOF, Lore DE BRUYNE, Isabelle DE NIJS
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Publication number: 20230174967Abstract: The present disclosure provides improved multiplex genome editing compositions and methods. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.Type: ApplicationFiled: October 21, 2022Publication date: June 8, 2023Applicant: 2seventy bio, Inc.Inventors: Baeckseung LEE, Alexander ASTRAKHAN
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Publication number: 20230174968Abstract: Provided herein are systems and methods for screening desirable biological variants using a high-throughput integrated system. The integrated system may be configured to input a plurality of parameters from functional studies of biological variants under applied conditions, in conjunction with integrated libraries of biological variants, and filter the inputs to produce desirable biological variants based on an input performance requirement. The system may output optimized strains, molecules, or novel molecules expected to have a desirable functional characteristic. Accordingly, the methods and systems disclosed herein enable multi-parametric studies of biological diversity and conditional diversity in systems biology.Type: ApplicationFiled: December 20, 2022Publication date: June 8, 2023Inventors: Jeremy AGRESTI, Andres ORNELAS VARGAS, Kevin Gregory HOFF
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Publication number: 20230174969Abstract: A barcoded transposase complex and an application thereof in high-throughput sequencing. Provided is a transposase recognition element, having the following structure: X(m)Y(f)N(n), in which X(m) represents a transposase recognition region of a double-stranded nucleic acid structure, Y(f) represents a spacer region of a single-stranded DNA structure, and N(n) represents a sample barcode of a single-stranded DNA structure. The high-molecular-weight DNA is processed using the barcoded transposase complex, to obtain a lot of barcoded DNA fragments. The barcoded DNA fragments obtained from each high-molecular-weight DNA are mixed to obtain a mixing sample. A carrier having a molecular barcode is adopted to capture. An exonuclease is adopted for processing, and then transposase is released. StLFR technology is adopted to construct a DNA library. The barcoded transposase complex can be applied to hybrid sequencing of a high-throughput sequencing platform.Type: ApplicationFiled: May 18, 2020Publication date: June 8, 2023Inventors: Xiaofang CHENG, Yan ZOU, Dan CHEN, Zhou LONG, Lei CAO, Lin XIE, Ping LIU
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Publication number: 20230174970Abstract: An imaging system having multiple cameras providing a large field of view with sufficient resolution can be used for tracking movements of cells from their positions in a tissue sample into multiple isolated areas such as into individual microwells in a well plate. By determining the beginning and the end of the movements of each cell, the imaging system can associate the microwell locations to the original cell positions in the sample. Together with an analysis of the cells in the microwells, either individually or together with barcode beads, the analysis can achieve the spatial information needed for constructing a map of the molecular information with respect to the positions of the cells in the sample.Type: ApplicationFiled: December 2, 2022Publication date: June 8, 2023Inventors: Natalie Alvarez, Aurelien Begue, Mark Harfouche, Gregor Horstmeyer, Robert Horstmeyer
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Publication number: 20230174971Abstract: Described herein are compositions and methods for identifying, selecting, or culturing cells comprising a subject nucleic acid sequence of interest. Generally, a nucleic acid comprising a subject nucleic acid and a sequence encoding an enzyme molecule involved in biosynthesis of an amino acid is introduced into a cell. The cell is then grown on media lacking the amino acid, such that cells comprising the introduced nucleic acid are capable of growth. In some instances, the cell further comprises an inhibitor of the enzyme molecule to increase the stringency of the selection.Type: ApplicationFiled: February 1, 2019Publication date: June 8, 2023Inventors: Robert YOUNG, Christopher Mark SMALES, Colin Mark JAQUES, Andrew J. RACHER, Gurdeep SINGH, James BUDGE, Joanne ROBOOL
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Publication number: 20230174972Abstract: The present application discloses methods useful for the multiplex measurement of enzymatic activities. Also disclosed are polynucleotide constructs, constructs libraries and compartments related suitable for use in the methods.Type: ApplicationFiled: October 15, 2020Publication date: June 8, 2023Inventors: Ting Xun Nicholas Ong, Wei Leong Chew
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Publication number: 20230174973Abstract: Methods of generating cDNAs from RNA involving cleavage of DNA/RNA complexes are provided.Type: ApplicationFiled: December 1, 2022Publication date: June 8, 2023Inventors: Rahul RATTAN, Jeffrey PERRY, Marta GONZALEZ-PLASKY, Stephanie SPOHN
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Publication number: 20230174974Abstract: RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.Type: ApplicationFiled: November 28, 2018Publication date: June 8, 2023Applicant: EmendoBio Inc.Inventors: Lior Izhar, David Baram, Joe Georgeson, Michal Golan-Mashiach, Asael Herman, Rafi Emmanuel
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Publication number: 20230174975Abstract: Described herein are systems for targeting viral genomes. Also described herein are methods for targeting viral genomes utilizing the systems described in the instant disclosure. In some cases, systems and methods disclosed herein can be used to treat viral infections. In preferred embodiments, the systems utilise a CRISPR/Cas13d complex to target the genome of an RNA virus such as coronavirus or influenza virus.Type: ApplicationFiled: March 12, 2021Publication date: June 8, 2023Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: Lei S. Qi, Marie F. LA RUSSA, Timothy R. ABBOTT, David B. LEWIS
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Publication number: 20230174976Abstract: Provided herein are compositions and methods relating to providing or engineering a structural target to attract ADAT editing to a desired site. Further provided herein are compositions and methods relating to recombinant adenosine deaminase acting on tRNA (ADAT) guide tRNAs (adat-gtRNA). In certain embodiments, such compositions and methods will be useful for modifying a coding sequence of a desired protein. Also provided are methods of treating a disease or disorder associated with loss of wild-type protein expression.Type: ApplicationFiled: April 15, 2021Publication date: June 8, 2023Inventors: Yiannis SAVVA, Richard Thomas Sullivan
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Publication number: 20230174977Abstract: Disclosed herein are engineered guide RNAs, engineered polynucleotides, precursor engineered polynucleotide, vectors omprising engineered polynucleotide, nucleic acids of engineered polynucleotide, pharmaceutical compositions thereof, methods of making the engineered polynucleotides and methods of treating or preventing a disease or condition by administering above described thereof.Type: ApplicationFiled: May 26, 2021Publication date: June 8, 2023Inventors: Thomas PACKARD, Yiannis SAVVA, Roberto PERALES, Richard SULLIVAN
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Publication number: 20230174978Abstract: The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.Type: ApplicationFiled: March 18, 2022Publication date: June 8, 2023Inventors: David Arthur Berry, Rahul Karnik, Laura Gabriela Lande
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Publication number: 20230174979Abstract: Provided herein are antisense oligonucleotides and prophylactic and therapeutic methods featuring such oligonucleotides. These oligonucleotides and methods are useful for treating or preventing ataxia telangiectasia in a subject. Specifically, the disclosure provides antisense nucleobase oligomers each comprising (8-40) nucleobases, wherein at least 90% of said nucleobases or more than (8) consecutive nucleobases of the oligomer are complementary to a nucleic acid sequence in an Ataxia-Telangiectasia Mutated (ATM) allele comprising a mutation associated with aberrant splicing.Type: ApplicationFiled: October 8, 2020Publication date: June 8, 2023Applicant: Children’s Medical Center CorporationInventors: Timothy W. YU, Jinkuk KIM
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Publication number: 20230174980Abstract: The inventions relate to compositions and articles of manufacture comprising connexin modulators, pannexin modulators, gap junction modulators, hemichannel modulators, and pannexin channel modulators and their use, alone or in combination, in treating ocular and other disorders.Type: ApplicationFiled: July 29, 2022Publication date: June 8, 2023Inventors: Colin Richard GREEN, Yeri KIM, Anthony PHILLIPS, Bradford James DUFT
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Publication number: 20230174981Abstract: A double-stranded nucleic acid complex that can have an excellent activity is provided. In one embodiment, the present invention relates to a double-stranded nucleic acid complex having a first nucleic acid strand and a second nucleic acid strand. The first nucleic acid strand is capable of hybridizing to at least part of a target gene or a transcription thereof, has an antisense effect on the target gene or transcription product thereof, and has at least two morpholino nucleic acids The second nucleic acid strand has a base sequence complementary to the first nucleic acid strand. The first nucleic acid strand is annealed to the second nucleic acid strand.Type: ApplicationFiled: March 12, 2021Publication date: June 8, 2023Applicant: NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITYInventors: Takanori YOKOTA, Tetsuya NAGATA
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Publication number: 20230174982Abstract: The present disclosure relates to engineered nucleic acids that target CAG repeat sequences or viral polynucleotides. The present disclosure also relates to the uses of the engineered nucleic acids for treating polyglutamine diseases or a viral infection.Type: ApplicationFiled: March 9, 2021Publication date: June 8, 2023Inventors: Nan ZHANG, Tetsuo ASHIZAWA
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Publication number: 20230174983Abstract: Provided is gRNA specifically targeting ?4GalNT2 gene. The gRNA specifically binds to the nucleotide sequence shown in any one of SEQ ID NOs. 1 and 2. Also provided are an animal model constructed using the gRNA, and an application thereof in the field of biomedicine.Type: ApplicationFiled: July 13, 2021Publication date: June 8, 2023Inventors: Yifan DAI, Haiyuan YANG
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Publication number: 20230174984Abstract: The present disclosure relates generally to compositions and methods suitable for treating a disorder associated with loss-of-function mutations in SYNGAP1. More specifically, the disclosure relates to methods for treating a disorder associated with heterozygous loss-of-function mutations of SYNGAP1, and to antisense oligonucleotides specific for SYNGAP1 and their use for treating a disorder associated with heterozygous loss-of-function mutations of SYNGAP1.Type: ApplicationFiled: May 11, 2021Publication date: June 8, 2023Inventor: Steven Petrou
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Publication number: 20230174985Abstract: The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.Type: ApplicationFiled: January 27, 2023Publication date: June 8, 2023Inventors: Danith H. LY, Shivaji A. THADKE, Ramesh U. BATWAL, Dietrich A. STEPHAN
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Publication number: 20230174986Abstract: The invention provides a medication and a diagnostic kit for inhibiting metastasis and invasion of breast cancer, an shRNA molecule for silencing expression of human LINC01614 and applications thereof. The shRNAs obtained by the invention can interfere with the expression of LINC01614, thereby reducing the migration and invasion ability of tumor cells, inhibiting the expression of EMT proteins, and inhibiting tumor formation and lung metastasis in an animal model in vivo. The invention provides a new solution for targeted therapy of breast cancer. Therefore, the kit for diagnosing metastasis and invasion of breast cancer and the medication for treating metastasis and invasion of breast cancer are developed. The invention provides a new way and strategy for diagnosing and treating metastasis and invasion of breast cancer.Type: ApplicationFiled: June 29, 2022Publication date: June 8, 2023Applicant: Zhejiang Chinese Medical UniversityInventors: Fangfang TAO, Zhiqian ZHANG, Wenhong LIU, Ye XU, Qingling LIU, Junfeng LI
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Publication number: 20230174987Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.Type: ApplicationFiled: July 21, 2022Publication date: June 8, 2023Applicants: Ionis Pharmaceuticals, Inc., University of RochesterInventors: C. Frank Bennett, Susan M. Freier, Robert A. MacLeod, Sanjay K. Pandey, Charles A. Thornton, Thurman Wheeler, Seng H. Cheng, Andrew Leger, Bruce M. Wentworth
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Publication number: 20230174988Abstract: The compositions and methods provided herein are related, in part, to the discovery of cholic acid 7-sulfate as a treatment for diabetes. Selective transport of the microbial metabolite lithocholic acid (LCA) from the gut to the liver after bariatric surgery activates hepatic vitamin D receptor (VDR), thereby inducing expression of bile acid sulfotransferase SULT2A, which produces the antidiabetic molecule CA7S. Provided herein is a method for treating diabetes, obesity, or an inflammatory disease in a subject, the method comprises administering to a subject in need thereof a compound of Formula I-XV or Formula F-XV? or an agent that increases levels or activity or cholic acid 7-sulfate or upstream targets in a subject.Type: ApplicationFiled: May 7, 2021Publication date: June 8, 2023Applicants: President and Fellows of Harvard College, The Brigham and Women's Hospital, IncInventors: Abigail Sloan Devlin, Snehal N. Chaudhari, Eric Garland Sheu, David A. Harris
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Publication number: 20230174989Abstract: The present invention relates to methods and compositions for editing an ABCA4 polynucleotide, e.g., an ABCA4 polynucleotide comprising a SNP associated with Stargardt Disease, type 1. The invention also relates to methods and compositions for treating or preventing Stargardt Disease, type 1, in a subject.Type: ApplicationFiled: May 14, 2021Publication date: June 8, 2023Applicant: Korro Bio, Inc.Inventors: Andrew Fraley, Mallikarjuna Reddy Putta, Matthew Jarpe
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Publication number: 20230174990Abstract: The present disclosure relates to immunostimulatory compositions that are effective in eliciting immune responses in avian species.Type: ApplicationFiled: August 8, 2022Publication date: June 8, 2023Inventor: Thomas ILG
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Publication number: 20230174991Abstract: This disclosure relates to the technical field of synthetic biology and metabolic engineering. More particularly, this disclosure relates to the technical field of fermentation of metabolically engineered microorganisms. This disclosure describes engineered micro-organisms that produce oligosaccharides that are free of KDO-lactose impurities and/or KDO-oligosaccharide impurities.Type: ApplicationFiled: February 12, 2021Publication date: June 8, 2023Inventors: Joeri Beauprez, Pieter Coussement, Gart Peters
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Publication number: 20230174992Abstract: The present disclosure provides ketoreductase enzymes having improved properties as compared to a naturally occurring wild-type ketoreductase enzyme including the capability of reducing N-(7-((2S,4R,5R)-4-fluoro-3,3-dihydroxy-5-(hydroxymethyl) tetrahydrofuran-2-yl)-4-oxo-4,7-dihydro-3H-pyrrolo[3,2-d]pyrimidin-2-yl)isobutyramide. Also provided are polynucleotides encoding the ketoreductase enzymes, host cells capable of expressing the ketoreductase enzymes, and methods of using the ketoreductase enzymes to synthesize N-(9-((2R,3S,4S,5R)-4-fluoro-3-hydroxy-5-(hydroxymethyl)tetrahydrofuran-2-yl)-6-oxo-6,9-dihydro-1H-purin-2-yl)isobutyramide.Type: ApplicationFiled: May 20, 2021Publication date: June 8, 2023Applicant: Merck Sharp & Dohme LLCInventors: Tamas Benkovics, Karla M. Camacho Soto, John McIntosh, Jeffrey C. Moore, Weilan Pan, Deeptak Verma, Li Xiao
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Publication number: 20230174993Abstract: Described in this application are UDP-glycosyltransferases (UGT) enzymes, host cells expressing the UGTs, and methods of producing mogrol precursors, mogrol, and/or mogrosides using such host cells.Type: ApplicationFiled: May 13, 2021Publication date: June 8, 2023Applicant: Ginkgo Bioworks, Inc.Inventors: Jeffrey Ian Boucher, Justin Michael Gardin, Scott Marr, Matthew McMahon, Krishnaben S. Patel, Jie Zhu
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Publication number: 20230174994Abstract: Parkin protein variants having activating mutations and/or fused to a mitochondrial targeting sequence are provided. The engineered Parkin may be a fusion protein including a mitochondrial targeting sequence (MTS); a transmembrane domain; and a Parkin protein or functional variant or fragment thereof, such as a Parkin having an N-terminal deletion. The MTS may be the MTS of PINK1 or a functional variant thereof. Alternatively or in addition, the engineered Parkin may have one or more activating mutations, such as single amino-acid substitutions. The engineered Parkin may be delivered in a vector, such as an adeno-associated virus (AAV) vector, and may be used to treat a disease or disorder, such as Parkinson’s disease or any of various neurodegenerative diseases.Type: ApplicationFiled: May 20, 2021Publication date: June 8, 2023Applicant: Spacecraft Seven, LLCInventors: Chester Bittencort SACRAMENTO, Christopher Dean HERZOG, Raj PRABHAKAR