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NTLA Intellia Therapeutics

APP
Utility
Compositions and Methods for Immunotherapy
4 Nov 21
Compositions and methods for editing, e.g., altering a DNA sequence, within the TRBC1, TRBC2 and/or TRAC genes are provided.
Amy Melissa Becker, Surbhi Goel, Sarah Beth Hesse, Troy Aaron Luster, Birgit Schultes, Stephanie A. Yazinski, Pooja Vinay
Filed: 15 Apr 21
APP
Utility
Nucleic Acid Constructs and Methods of Use
14 Oct 21
The present disclosure provides bidirectional nucleic acid constructs that allow enhanced insertion and expression of a nucleic acid sequence of interest, e.g., encoding a therapeutic agent such as a polypeptide.
Jonathan Douglas Finn, Hon-Ren Huang
Filed: 16 Apr 21
APP
Utility
Compositions and Methods for Lactate Dehydrogenase (LDHA) Gene Editing
22 Jul 21
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the LDHA gene are provided.
Zachary William Dymek, Shobu Odate, Anette Huebner, Srijani Sridhar, Bradley Andrew Murray, Walter Strapps
Filed: 25 Mar 21
APP
Utility
Compositions and Methods for Hydroxyacid Oxidase 1 (HAO1) Gene Editing for Treating Primary Hyperoxaluria Type 1 (PH1)
3 Jun 21
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided.
Zachary William Dymek, Shobu Odate, Bradley Andrew Murray, Reynald Michael Lescarbeau, Anette Huebner, Walter Strapps, Sarah Beth Hesse
Filed: 29 Jan 21
APP
Utility
Compositions and Methods for Immunooncology
29 Apr 21
The present disclosure is directed to genome editing systems, reagents and methods for immunooncology.
Ming-Wei CHEN, Glenn DRANOFF
Filed: 7 Jun 19
APP
Utility
Modified Guide RNAs for Gene Editing
25 Mar 21
This disclosure relates to modified guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Amy Madison Rhoden Smith, Seth C. Alexander, Duncan Brown, Robert Brown, Adhiraj Lanba, Rebecca Lescarbeau, Reynald Michael Lescarbeau, Rubina Parmar, Matthew Roy, Paige Salerno, Jessica Lynn Seitzer
Filed: 4 Dec 20
APP
Utility
Compositions and Methods for Immunooncology
11 Mar 21
The present disclosure is directed to genome editing systems, reagents and methods for immunooncology.
Jennifer BROGDON, Ming-Wei CHEN, Hyungwook LIM, Yi YANG, Morag STEWART, Sarah HESSE
Filed: 21 Mar 18
APP
Utility
Polynucleotides, Compositions, and Methods for Genome Editing
11 Nov 20
Compositions and methods for gene editing.
Christian Dombrowski, Jonathan Douglas Finn, Amy Madison Rhoden Smith, Seth C. Alexander
Filed: 23 Mar 20
APP
Utility
In Vitro Method of Mrna Delivery Using Lipid Nanoparticles
30 Sep 20
The present disclosure relates to compositions and methods for introducing an mRNA into stem cells, such as HSPCs, and for delivering gene editing components to such cells in vitro.
Morag H. Stewart, Markus P. Vallaster, Anthony Monti, Pooja K. Narendra, Quan Ho
Filed: 27 Sep 18
APP
Utility
Compositions and Methods for Expressing Factor IX
16 Sep 20
Compositions and methods for expressing Factor IX in a host cell or a population of host cells are provided.
John Finn, Hon-Ren Huang, Moitri Roy, KehDih Lai, Rachel Sattler, Christos Kyratsous, Cheng Wang
Filed: 17 Oct 19
APP
Utility
Nucleic Acid Constructs and Methods of Use
26 Aug 20
Nucleic acid constructs that allow insertion and/or expression of a sequence of interest, such as a transgene, are provided.
John Finn, Hon-Ren Huang
Filed: 17 Oct 19
APP
Utility
Compositions and Methods for Transgene Expression from an Albumin Locus
26 Aug 20
Methods for editing, e.g., introducing a heterologous transgene, within the human albumin gene (e.g., at intron 1) are provided.
John Finn, Hon-Ren Huang, Moitri Roy, KehDih Lai, Rachel Sattler, Christos Kyratsous, Cheng Wang
Filed: 17 Oct 19
APP
Utility
Compositions and Methods for Treating ALPHA-1 Antitrypsin Deficiency
26 Aug 20
Compositions and methods for expressing alpha 1 antitrypsin (AAT) in a host cell are provided.
John Finn, Hon-Ren Huang, Anthony Forget, Xin Xie
Filed: 17 Oct 19
APP
Utility
Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis
5 Aug 20
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided.
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Filed: 23 Mar 20
APP
Utility
Stabilized Nucleic Acids Encoding Messenger Ribonucleic Acid (mRNA)
3 Jun 20
This disclosure relates to the field of poly-adenylated (poly-A) tails.
Christian Dombrowski
Filed: 13 Feb 20
APP
Utility
Methods for the Treatment of Disease with Gene Editing Systems
6 May 20
Provided herein are methods of selectively treating a patient with a gene editing system on the basis of ascertaining the presence of a target sequence, at a target locus, that is fully complementary to a targeting domain of said gene editing system and/or on the basis of ascertaining the absence of a target sequence, at a locus other than the target locus, that is fully complementary to a targeting domain of said gene editing system.
Nicole RENAUD, Xiaojun ZHAO
Filed: 27 Jun 18
APP
Utility
Methods and Compositions for Ocular Cell Therapy
29 Apr 20
The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy.
Frada BERENSHTEYN, Bo HAN, Xueshi HAO, Jessica HEYDER, Timothy Z. HOFFMAN, Qihui JIN, Arnaud LACOSTE, Jun LIU, Yahu LIU, Tingting MO, Bradley Andrew MURRAY, Daniel Joseph O'CONNELL, Jianfeng PAN, Yun Feng XIE, Shanshan YAN, Yefen ZOU
Filed: 24 Oct 19
APP
Utility
Compositions and Methods for the Treatment of Hemoglobinopathies
1 Apr 20
The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.
Craig Stephen MICKANIN, Christian SCHMEDT, Jennifer SNEAD, Susan Stevenson, Yi YANG
Filed: 4 Feb 18
APP
Utility
Modified Guide RNAs
16 Oct 19
This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Amy Madison Rhoden Smith, David V. Morrissey, Walter Strapps
Filed: 6 Jun 19
APP
Utility
Compositions and Methods for Treating Alpha-1 Antitrypsin Deficiency
16 Oct 19
Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided.
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau
Filed: 19 Jun 19
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