36 patents
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
Utility
Methods of Substituting Pathogenic Amino Acids Using Programmable Base Editor Systems
22 Dec 22
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, Yanfang Fu, Nicole Gaudelli, Jason Michael Gehrke, J. Keith Joung
Filed: 11 May 19
Utility
Methods of Editing a Single Nucleotide Polymorphism Using Programmable Base Editor Systems
8 Dec 22
Described are compositions and methods for altering mutations associated with Rett Syndrome (RETT).
Jason Michael GEHRKE, Natalie PETROSSIAN
Filed: 20 May 20
Utility
Compositions and Methods for Treating Hemoglobinopathies
24 Nov 22
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
Compositions and Methods for Editing a Mutation to Permit Transcription or Expression
6 Oct 22
The present invention features compositions and methods for editing a gene associated with Shwachman Diamond Syndrome (SDS) using a programmable nucleobase editor, such that the gene is permissive for transcription and generates a functional gene product (e.g., providing a splice site and/or altering a nonsense mutation).
Jason Michael GEHRKE, Luis BARRERA, Angelica MESSANA
Filed: 28 Aug 20
Utility
Nanomaterials
11 Aug 22
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Cory Dane Sago, Gregory Lawrence Hamilton
Filed: 19 Jan 22
Utility
Nanomaterials Comprising a Biodegradable Feature
11 Aug 22
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Mina Fawzy Shehata, Neeraj Narendra Patwardhan, Gregory Lawrence Hamilton, Cory Dane Sago, Milloni Balwantkumar Chhabra
Filed: 19 Jan 22
Utility
Compositions and Methods for Treating Hemoglobinopathies
28 Jul 22
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 Feb 20
Utility
Adenosine Deaminase Base Editors and Methods of Using Same to Modify a Nucleobase In a Target Sequence
2 Jun 22
The disclosure 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.
Nicole GAUDELLI, Michael Michael PACKER, Ian SLAYMAKER, Yi YU, Bernd ZETSCHE, Jason Michael GEHRKE, Natalie PETROSSIAN, Angelica MESSANA, Yvonne ARATYN, Francine GREGOIRE, Genesis LUNG, Shaunna BERKOVITCH, David A. BORN, Seung-Joo LEE
Filed: 13 Feb 20
Utility
Compositions and methods for treating hemoglobinopathies
31 May 22
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 Mar 21
Utility
Modified Immune Cells Having Enhanced Anti-neoplasia Activity and Immunosuppression Resistance
5 May 22
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
Filed: 16 Jan 20