31 patents
Utility
Methods and Compositions for Directed Genome Editing
9 Mar 23
Provided herein are compositions and methods for increasing editing efficiency of a target nucleic acid.
Schaked Omer HALPERIN
Filed: 3 May 22
Utility
Compositions and Methods Comprising a TTR Guide RNA and a Polynucleotide Encoding an RNA-Guided DNA Binding Agent
9 Feb 23
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided.
Yong Chang, Seth C. Alexander, Kristy M. Wood, Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Filed: 27 Sep 21
Utility
Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis Comprising a Corticosteroid or Use Thereof
2 Feb 23
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene in combination with administration of a corticosteroid are provided.
Yong CHANG, Seth C. ALEXANDER, Kristy M. WOOD, Arti Mahendra Prakash KANJOLIA, Shobu ODATE, Jessica Lynn SEITZER, Reynald Michael LESCARBEAU, Walter STRAPPS
Filed: 27 Sep 21
Utility
Polynucleotides, Compositions, and Methods for Polypeptide Expression
19 Jan 23
Compositions and methods for gene editing.
Bradley Andrew Murray, Christian Dombrowski, Seth C. Alexander
Filed: 27 Sep 21
Utility
Compositions and methods for treating alpha-1 antitrypsin deficiency
10 Jan 23
Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided.
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau
Filed: 20 Jun 19
Utility
Ionizable Amine Lipids and Lipid Nanoparticles
22 Dec 22
The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells.
Stephen S. Scully, Derek LaPlaca, Rachel Pelly, Rubina Giare Parmar, Micah Maatani
Filed: 24 Apr 20
Utility
Modified Guide RNAs for Gene Editing
24 Nov 22
This disclosure relates to modified guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Seth C. Alexander, Sabin Mulepati, Matthew Roy
Filed: 9 Jun 22
Utility
Compositions and Methods for Transgene Expression from an Albumin Locus
10 Nov 22
The present disclosure is directed to methods and compositions useful for inserting and expressing a heterologous (exogenous) gene within a genomic locus, such as a safe harbor site, of a host cell.
Jonathan Douglas Finn, Hon-Ren Huang, Moitri Roy, KehDih Lai, Rachel Sattler, Christos Kyratsous, Cheng Wang
Filed: 16 Apr 21
Utility
Modified guide RNAs
25 Oct 22
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: 7 Jun 19
Utility
Compositions and methods for the treatment of hemoglobinopathies
11 Oct 22
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: 5 Feb 18
Utility
Ionizable Amine Lipids
13 Jan 22
The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells.
Rubina Giare Parmar, Stephen S. Scully, Micah Maetani, Derek LaPlaca
Filed: 2 Oct 19
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
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
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
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
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
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
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
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
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