20 patents
Utility
Liver De-targeted Recombinant Aav Capsid Proteins
14 Dec 23
Provided herein are recombinant adeno-associated virus (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, nucleic acids encoding the capsid proteins, and methods of making and using the capsid proteins.
Laura Jane SMITH, Laura Van Lieshout, Lindsay Schulman, Omar Francone
Filed: 17 May 23
Utility
Bidirectional Dual Promoter Expression Vectors and Uses Thereof
23 Nov 23
Provided herein are polynucleotides comprising novel bidirectional dual expression cassettes, recombination adeno-associated virus (rAAV) comprising these polynucleotides, and methods of making and using the polynucleotides and rAAV.
Carmen BARNES, Yogeshwar Sharma, Omar Francone, Hillard Rubin, Wei Wang, Gustavo Cerqueira, Serena Nicole Dollive, Andrew Pla
Filed: 10 Mar 23
Utility
Methods and Compositions for the Production of Adeno-associated Virus
12 Oct 23
Provided herein are methods for the production of recombinant adeno-associated virus (rAAV) particles.
Marissa Stanvick, Richard Gilmore, Divya Tsiros
Filed: 15 Dec 22
Utility
Non-naturally Occurring Polyadenylation Elements and Methods of Use Thereof
13 Apr 23
Provided herein are polynucleotides and vectors comprising non-naturally occurring polyadenylation (polyA) sequences, and methods of making and using these polynucleotides and vectors.
Serena Nicole DOLLIVE, Andrew Pla
Filed: 16 Sep 22
Utility
Adeno-associated Virus Formulations
23 Mar 23
Provided herein are pharmaceutical compositions (e.g., formulations) that can provide for the long-term stability of AAV vectors.
Lori KARPES, Michael MERCALDI, Eric FAULKNER
Filed: 24 Aug 22
Utility
Adeno-associated Virus Packaging Systems
9 Feb 23
Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV).
Laura VAN LIESHOUT, Marissa STANVICK
Filed: 24 Jun 22
Utility
Adeno-associated Virus Compositions and Methods of Use Thereof
9 Feb 23
Provided herein are novel adeno-associated virus (AAV) capsids, compositions (e.g., rAAV) comprising the capsids, and nucleic acids encoding the capsids.
Serena Nicole DOLLIVE
Filed: 10 Jun 22
Utility
Adeno-associated Virus Compositions for Restoring F8 Gene Function and Methods of Use Thereof
20 Oct 22
Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in cell.
Saswati CHATTERJEE, Laura Jane SMITH, Jeff Lynn ELLSWORTH, Hillard RUBIN, Jason Boke WRIGHT, James Anthony MCSWIGGEN
Filed: 10 Mar 22
Utility
Adeno-associated Virus Compositions for Restoring Pah Gene Function and Methods of Use Thereof
13 Oct 22
Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell.
Albert Barnes SEYMOUR, Seemin Seher AHMED, Jason Boke WRIGHT, Serena Nicole DOLLIVE, James Anthony MCSWIGGEN, Jaime Michelle PROUT, Danielle Lauren SOOKIASIAN
Filed: 25 Mar 22
Utility
Adeno-Associated Virus Compositions for ARSA Gene Transfer and Methods of Use Thereof
30 Jun 22
Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function.
Thia Baboval ST. MARTIN, Albert Barnes SEYMOUR, Hillard RUBIN
Filed: 10 Dec 21
Utility
Gene Therapy Methods
26 May 22
Provided are methods for reducing the risk of occurrence and/or the severity of transaminitis in a subject that is receiving a gene therapy.
Janet Nancy ILES-SOMARATNE, Gabriel M. Cohn, Steven Alan Glyman, Arthur O. Tzianabos
Filed: 5 Nov 21
Utility
Gene Therapy Methods
26 May 22
Provided are methods for reducing the risk of occurrence and/or the severity of pathogenic diseases (e.g., diseases associate with varicella zoster virus reactivation, influenza infection, and/or S. pneumoniae infection) in a subject that is receiving a gene therapy and an accompanying immunosuppressant regimen.
Janet Nancy ILES-SOMARATNE
Filed: 5 Nov 21
Utility
Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
19 Apr 22
Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in a cell without co-transducing or co-administering an exogenous nuclease or a nucleotide sequence that encodes an exogenous nuclease.
Saswati Chatterjee, Laura Jane Smith, Jeff Lynn Ellsworth, Hillard Rubin, Jason Boke Wright, James Anthony McSwiggen
Filed: 19 Feb 19
Utility
Adeno-associated Virus Compositions for Restoring Pah Gene Function and Methods of Use Thereof
23 Dec 21
Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods for using the same to treat diseases associated with reduction of PAH gene function (e.g., PKU).
Jason Boke WRIGHT, Danielle Lauren SOOKIASIAN, Thia Baboval ST. MARTIN, Omar FRANCONE, Albert Barnes SEYMOUR
Filed: 27 May 21
Utility
Adeno-associated Virus Compositions for Ids Gene Transfer and Methods of Use Thereof
25 Nov 21
Provided are adeno-associated virus (AAV) compositions that can restore IDS gene function in cells, and methods for using the these AAV compositions to treat disorders associated with reduction of IDS gene function (e.g., Hunter syndrome).
Jacinthe GINGRAS, Kruti PATEL, Laura Jane SMITH, Yvonne WHITE, Serena Nicole DOLLIVE, Laura VAN LIESHOUT, Brenda BURNHAM
Filed: 5 Apr 21
Utility
Methods of Treating Phenylketonuria
26 Aug 21
Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation.
Omar L. Francone, Deborah A. Kinch, Diana Lamppu, Albert Barnes Seymour, Teresa Leah Wright
Filed: 13 Jan 21
Utility
Adeno-associated Virus Compositions for Restoring Pah Gene Function and Methods of Use Thereof
6 May 21
Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell.
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, James Anthony McSwiggen, Jaime Michelle Prout, Danielle Lauren Sookiasian
Filed: 19 Oct 20
Utility
Adeno-associated Virus Compositions for Pah Gene Transfer and Methods of Use Thereof
7 Oct 20
Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function.
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
Filed: 18 Mar 20
Utility
Adeno-associated Virus Compositions for Pah Gene Transfer and Methods of Use Thereof
16 Sep 20
Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function.
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
Filed: 23 Feb 20
Utility
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
6 Apr 20
Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function.
Albert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
Filed: 28 Mar 19
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