143 patents
Page 4 of 8
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Materials and Methods for Treatment of Spinocerebellar Ataxia Type 1 (SCA1) and Other Spinocerebellar Ataxia Type 1 Protein (ATXN1) Gene Related Conditions or Disorders
17 Feb 22
The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN1 whether ex vivo or in vivo.
Ante Sven LUNDBERG, Samarth KULKARNI, Lawrence KLEIN, Hari Kumar PADMANABHAN
Filed: 20 Feb 18
Utility
Materials and Methods for Treatment of Usher Syndrome Type 2A And/or Non-syndromic Autosomal Recessive Retinitis Pigmentosa (Arrp)
17 Feb 22
The present application provides materials and methods for treating a patient with one or more of Usher Syndrome Type 2A and ARRP, both ex vivo and in vivo; materials and methods for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299.
Mariacarmela Allocca, Akiko Noma, Abraham Scaria
Filed: 2 Aug 21
Utility
OPTIMIZED mRNA ENCODING CAS9 FOR USE IN LNPs
17 Feb 22
The present disclosure relates generally to novel lipid nanoparticle (LNP)-based compositions useful for, e.g., the delivery of a site-specific endonuclease or a nucleic acid molecule encoding same, into a target cell.
Christopher J. CHENG, Andrew SCHARENBERG, Kui WANG, Shailendra SANE
Filed: 26 Nov 19
Utility
Anti-idiotype Antibodies Targeting ANTI-CD70 Chimeric Antigen Receptor
10 Feb 22
High affinity antibodies capable of binding to a single-chain variable fragment (scFv) of anti-CD70 antibody, for example, the scFv expressed on cell surface as a portion of a chimeric antigen receptor (CAR).
Lalit KUMAR, Mary-Lee DEQUEANT
Filed: 3 Aug 21
Utility
Allogeneic Cell Therapy of Acute Lymphoblastic Leukemia Using Genetically Engineered T Cells Targeting CD19
27 Jan 22
Methods for treating a B cell malignancy comprising a population of genetically engineered immune cells (e.g., T cells), which express a chimeric antigen receptor (CAR) specific to CD19 and optionally an NK cell inhibitor (e.g., daratumumab).
Jonathan Alexander TERRETT, Demetrios KALAITZIDIS, Ewelina MORAWA, Mark BENTON
Filed: 25 Jun 21
Utility
Genetically Engineered T Cells with REGNASE-1 And/or Tgfbrii Disruption Have Improved Functionality and Persistence
20 Jan 22
A population of genetically engineered T cells, comprising a disrupted Reg1 gene and/or a disrupted TGFBRII gene.
Mary-Lee Dequeant, Demetrios Kalaitzidis, Mohammed Ghonime
Filed: 4 Oct 21
Utility
Universal Donor Cells
20 Jan 22
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein.
Alireza REZANIA, Rebeca RAMOS-ZAYAS
Filed: 23 Jul 21
Utility
Use of Chimeric Antigen Receptor T Cells and NK Cell Inhibitors for Treating Cancer
13 Jan 22
Methods for improving a clinical outcome in a subject comprising administering to a subject in need of the treatment a population of genetically engineered immune cells (e.g., T cells), which express a chimeric antigen receptor (CAR) and a natural killer (NK) cell inhibitor (e.g., daratumumab).
Jonathan Alexander Terrett, Lawrence Klein, Ewelina Morawa
Filed: 28 Sep 21
Utility
Materials and Methods for Treatment of Hereditary Haemochromatosis
13 Jan 22
Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
Roman Lvovitch Bogorad, Chad Albert Cowan, Ante Sven Lundberg
Filed: 9 Jul 21
Utility
Compositions and Methods for Nhej-mediated Genome Editing
6 Jan 22
The present application relates to compositions and methods for genome editing in cells by homology-independent mechanisms, in particular for genome editing in cells that lack the machinery necessary for repair by homology-dependent mechanisms.
Gregory J. Cost, Gene l. Uenishi
Filed: 30 Oct 19
Utility
Materials and Methods for Treatment of Usher Syndrome Type 2A
30 Dec 21
The present application provides materials and methods for treating a patient with Usher Syndrome Type 2A, both ex vivo and in vivo; materials and methods for editing a USH2A gene in a human cell; materials and methods for editing an USH2A gene containing an IVS40 mutation; materials and methods for treating a patient with an USH2A gene containing an IVS40 mutation; and a method for deleting a sequence comprising an IVS40 mutation within a USH2A gene of a cell.
Albena KANTARDZHIEVA, Akiko NOMA, Abraham SCARIA
Filed: 28 Jun 21
Utility
ANTI-CD70 Antibodies and Uses Thereof
9 Dec 21
High affinity and specificity antibodies capable of binding to human CD70.
Jason Sagert, Minh Thu PHAM
Filed: 3 Jun 21
Utility
Genetically Engineered Car T Cells That Secret INTERLEUKIN-12 and Therapeutic Uses Thereof
25 Nov 21
Genetically engineered immune cells such as T cells capable of secreting an interleukin-12 protein, for example, upon activation of the T cells.
Mohammed Ghonime, Demetrios Kalaitzidis, Jason Sagert, Jonathan Alexander Terrett
Filed: 21 May 21
Utility
Universal Donor Cells
25 Nov 21
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein.
Alireza REZANIA, Rebeca Ramos-Zayas
Filed: 23 Jul 21
Utility
Messenger Rna Encoding CAS9 for Use In Genome-editing Systems
18 Nov 21
The present disclosure provides optimized mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system.
Christian Cobaugh
Filed: 13 May 21
Utility
Masked Chimeric Antigen Receptor Specific to Tyrosine-protein Kinase Like 7 (PTK7) and Immune Cells Expressing Such
11 Nov 21
Masked chimeric antigen receptor (CAR) constructs comprising an extracellular antigen binding domain specific tyrosine-protein kinase-like 7 (PTK7), which is linked to a mask peptide that blocks binding of masked CAR from binding to PTK7.
Jason Sagert, Jui Dutta-Simmons, Jonathan Alexander Terrett
Filed: 6 May 21
Utility
Compositions and Methods for Delivering Transgenes
11 Nov 21
Provided herein are compositions, methods, and systems for targeted delivery of nucleic acids, including DNA and RNA, to a target cell.
Alan Richard Brooks, Karen Vo
Filed: 17 Oct 19
Utility
Universal Donor Cells
11 Nov 21
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetically modified cells are provided herein.
Alireza Rezania, Rebeca Ramos-Zayas
Filed: 26 Apr 21
Utility
ANTI-PTK7 Immune Cell Cancer Therapy
23 Sep 21
Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer, such as PTK7+ malignancies.
Jonathan Alexander Terrett, Jason Sagert
Filed: 7 Nov 19
Utility
ANTI-LIV1 Immune Cell Cancer Therapy
23 Sep 21
Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer, such as LIV1+ malignancies.
Jonathan Alexander Terrett, Jason Sagert
Filed: 7 Nov 19