46 patents
Utility
Compositions and Methods for Treating Glycogen Storage Disease Type 1A
30 Nov 23
Described and provided are adenosine base editors and compositions comprising adenosine base editors that have increased efficiency.
Brian CAFFERTY, Tanggis BOHNUUD, Lo-I CHENG, Michael PACKER, Yvonne ARATYN-SCHAUS
Filed: 14 Oct 21
Utility
Nanomaterials Comprising Carbonates
9 Nov 23
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Gregory Lawrence Hamilton, Neeraj Narendra Patwardhan, Cory Dane Sago, Mina Fawzy Shehata, Milloni Balwantkumar Chhabra
Filed: 20 Jun 23
Utility
Ionizable Lipids for Nanomaterials
2 Nov 23
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Mina Fawzy Shehata, Gregory Lawrence Hamilton, Neeraj Narendra Patwardhan, Cory Dane Sago, Milloni Balwantkumar Chhabra
Filed: 12 Jul 23
Utility
Uses of adenosine base editors
24 Oct 23
The disclosure provides methods and compositions for treating blood diseases/disorders, such as sickle cell disease, hemochromatosis, hemophilia, and beta-thalassemia.
David R. Liu, Nicole Marie Gaudelli, Michael S. Packer, Gregory Newby
Filed: 16 Oct 18
Utility
Nanomaterials Comprising Acetals
19 Oct 23
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Gregory Lawrence Hamilton, Neeraj Narendra Patwardhan, Cory Dane Sago, Mina Fawzy Shehata, Milloni Balwantkumar Chhabra
Filed: 20 Jun 23
Utility
Nanomaterials Comprising Ester-linked Acetals
19 Oct 23
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Neeraj Narendra Patwardhan, Gregory Lawrence Hamilton, Cory Dane Sago, Mina Fawzy Shehata, Milloni Balwantkumar Chhabra, Elizabeth Anne Campbell
Filed: 20 Jun 23
Utility
Compositions and methods for treating hemoglobinopathies
12 Sep 23
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD).
Ian Slaymaker, Nicole Gaudelli, Yi Yu, Bernd Zetsche, David A. Born, Seung-Joo Lee, Michael Packer
Filed: 13 Apr 22
Utility
Novel Crispr Enzymes, Methods, Systems and Uses Thereof
7 Sep 23
The present invention provides novel systems, methods and compositions for making and using a recombinantly engineered novel Cas9 optimized for human cells, for nucleic acid targeting and manipulation.
Bernd ZETSCHE, Luis BARRERA
Filed: 9 Sep 20
Utility
Viral Guide Rna Delivery
31 Aug 23
Provided herein are recombinant negative-strand RNA virus genomes (e.g., recombinant rabies virus genomes) and recombinant negative-strand RNA viruses (e.g., recombinant rabies viruses) and methods for their use in delivering a guide RNA and, optionally, a transgene, into a target cell.
Fei Ran, ChieYu Lin
Filed: 8 Sep 22
Utility
Compositions and Methods for Engraftment of Base Edited Cells
3 Aug 23
The invention provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence and methods of using same to treat genetical disorder or conditions, e.g. sickle cell disease, with engraftment.
Sarah SMITH, Dana LEVASSEUR, Jonathan YEN
Filed: 12 Feb 21
Utility
Methods of Editing Single Nucleotide Polymorphism Using Programmable Base Editor Systems
25 May 23
Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide.
David Bryson, John Evans, Michael Packer, Jason Michael Gehrke, Natalie Petrossian
Filed: 11 May 19
Utility
Methods of Editing a Disease-associated Gene Using Adenosine Deaminase Base Editors, Including for the Treatment of Genetic Disease
11 May 23
The invention provides compositions comprising novel programmable adenosine base editor systems (e.g., ABE8) that provide methods of treating a disease or disorder, (e.g., Parkinson's disease, Hurler syndrome, Rett syndrome, or Stargardt disease) in a subject by administering to the subject a programmable adenosine base editor system (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a disease-associated gene.
Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER, Jason Michael GEHRKE, Natalie PETROSSIAN, Angelica MESSANA, Shaunna BERKOVITCH
Filed: 13 Feb 20
Utility
Compositions and Methods for Treating Hemoglobinopathies
27 Apr 23
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD).
Ian SLAYMAKER, Nicole GAUDELLI, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Michael PACKER
Filed: 26 Jul 22
Utility
Pseudotyped Recombinant Lyssaviruses for Gene Therapy
13 Apr 23
Provided herein are pseudotyped recombinant lyssavirus particles and methods for their use in delivering a transgene into a target cell.
Fei Ran, ChieYu Lin
Filed: 26 Jul 22
Utility
Compositions and Methods for Treating ALPHA-1 Antitrypsin Deficiency
30 Mar 23
The present invention features compositions and methods for editing deleterious mutations associated with alpha-1 anti-trypsin (A1AT) deficiency.
Nicole GAUDELLI, Michael PACKER, Bernd ZETSCHE, Ian SLAYMAKER, Yi YU, David A. BORN, Seung-Joo LEE
Filed: 13 Feb 20
Utility
Modified Immune Cells Having Adenosine Deaminase Base Editors for Modifying a Nucleobase In a Target Sequence
16 Mar 23
The present invention features genetically modified immune cells comprising novel adenosine base editors (e.g., ABE8) having enhanced anti-neoplasia activity, resistance to immune suppression, and decreased risk of eliciting a graft-versus-host reaction or host-versus-graft reaction, or a combination thereof.
Nicole GAUDELLI, Michael PACKER, Ian SLAYMAKER, Yi YU, Bernd ZETSCHE, David A. BORN, Seung-Joo LEE, Jason M. GEHRKE
Filed: 13 Feb 20
Utility
Compositions and Methods for Treating Hepatitis B
9 Mar 23
The invention features compositions and methods for introducing mutations into the hepatitis B virus (HBV) genome.
Elena SMEKALOVA, Michael PACKER, Francine GREGOIRE, Luis BARRERA, Giuseppe CIARAMELLA
Filed: 8 May 20
Utility
Novel Nucleobase Editors and Methods of Using Same
9 Mar 23
The invention features novel programmable nucleobase editors comprising adenosine deaminase domains and methods of using the same for polynucleotide editing.
Nicole GAUDELLI, Michael PACKER
Filed: 9 Sep 20
Utility
Compositions and Methods for Treatment of Liquid Cancers
26 Jan 23
As described below, the present invention features genetically modified immune cells having enhanced anti-neoplasia activity, resistance to immune suppression, and decreased risk of eliciting a graft versus host reaction, or a combination thereof.
Jason Michael GEHRKE, Aaron D. EDWARDS, Ryan MURRAY, Waseem QASIM
Filed: 25 Sep 20
Utility
Compositions and Methods for Non-toxic Conditioning
19 Jan 23
The invention features compositions and methods for conditioning a patient (e.g., to facilitate transplantation and/or engraftment).
Adam HARTIGAN, Tanggis BOHNUUD
Filed: 28 Aug 20