22 patents
Utility
Compositions and Methods for Increasing or Enhancing Transduction of Gene Therapy Vectors and for Removing or Reducing Immunoglobulins
3 Aug 23
Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering an agent that blocks, inhibits or reduces the interaction between immunoglobulin G (IgG) and the neonatal Fc receptor (FcRn), such as an anti-FcRn antibody, to reduce IgG recycling and enhance IgG clearance in vivo.
Sean ARMOUR
Filed: 22 Jan 21
Utility
Column-based fully scalable rAAV manufacturing process
18 Jul 23
In accordance with the invention, provided herein are methods for purifying recombinant adeno-associated (rAAV) vector particles.
Guang Qu, Younghoon Oh, Lin Lu, John Fraser Wright
Filed: 31 Mar 17
Utility
Compositions and Methods for Increasing or Enhancing Transduction of Gene Therapy Vectors and for Removing or Reducing Immunoglobulins
11 May 23
Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo.
Sébastien LACROIX-DESMAZES, Federico MINGOZZI, Jordan DIMITROV, Christian LEBORGNE, Sean ARMOUR
Filed: 12 Jul 22
Utility
Secretable Protein Induced Immune Tolerization and Treatment of Autoimmune, Allergic and Other Diseases and Disorders
9 Feb 23
Nucleic acids encoding fusion proteins that contain an unwanted antigen and a leader sequence for cell secretion are described.
David William ANDERSON, Brittney L. GURDA, Federico MINGOZZI, Mustafa N. YAZICIOGLU, William QUINN
Filed: 19 Nov 20
Utility
Adeno-Associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
8 Dec 22
Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system.
George Buchlis, Xavier Anguela, Katherine A. High
Filed: 29 Jul 22
Utility
Optimized Promoter Sequences, Intron-free Expression Constructs and Methods of Use
17 Nov 22
The invention provides expression cassettes.
Xavier ANGUELA, Liron ELKOUBY
Filed: 23 Aug 19
Utility
Aav Vector Treatment Methods for Late Infantile Neuronal Ceroid Lipofuscinosis Type 2
16 Jun 22
Disclosed herein are methods for treating a primate in need of tripeptidyl peptidase 1 (TPP1), comprising (a) providing a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding TPP1; and (b) administering an amount of the recombinant AAV vector to the central nervous system (CNS) of the primate, wherein the TPP1 is expressed in the primate.
David William ANDERSON, Maryann TOTO, Sue E.I. DASEN
Filed: 31 Jan 20
Utility
Plasmid Free Aav Vector Producing Cell Lines
27 Jan 22
Disclosed herein are packaging cell lines, in which adenovirus (Ad) E1A is constitutively expressed, that also contain integrated AAV rep and cap genes.
Guang QU, Denis PHICHITH, Jingmin ZHOU
Filed: 7 May 19
Utility
In Vitro Assay for Detecting Enhancers and Inhibitors of Adeno Associated Virus (Aav) Vector Transduction And/or Detecting or Quantitating Anti-aav Binding Antibodies
13 Jan 22
Disclosed herein are methods for analyzing for or detecting the presence of non-antibody inhibitors and/or enhancers of adeno-associated virus (AAV) vector cell transduction in a biological sample from a subject.
Klaudia KURANDA, Xavier ANGUELA, Federico MINGOZZI
Filed: 15 Nov 19
Utility
Adeno-associated Virus (Aav) Vector Lipid Nanoparticle Compositions and Methods of Use
2 Dec 21
A composition includes an adeno-associated vims (AAV) vector in a lipid nanoparticle (LNP).
Andrew William SHAW, Xavier ANGUELA
Filed: 18 Dec 18
Utility
Engineered Aav Capsids with Increased Tropism and Aav Vectors Comprising the Engineered Capsids and Methods of Making and Using Same
25 Nov 21
The invention provides modified adeno-associated virus (AAV) capsid proteins.
Xavier ANGUELA, Sean ARMOUR, Nicholas KEISER, Suryanarayan SOMANATHAN, Mustafa N. YAZICIOGLU
Filed: 26 Apr 19
Utility
Relative Potency Assay for Viral Vector Encoding Isomerohydrolases
11 Nov 21
Methods for assaying function and/or activity and/or potency of isomerohydrolase proteins are provided.
Linda COUTO
Filed: 28 Apr 17
Utility
CpG reduced factor VIII variants, compositions and methods and uses for treatment of hemostasis disorders
9 Nov 21
CpG reduced nucleic acid variants encoding FVIII protein and methods of use thereof are disclosed.
Xavier Anguela, Sam Hsien-I Shen
Filed: 17 Mar 17
Utility
Compositions and Methods for Increasing or Enhancing Transduction of Gene Therapy Vectors and for Removing or Reducing Immunoglobulins
12 Aug 21
Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo.
Sébastien LACROIX-DESMAZES, Federico MINGOZZI, Jordan DIMITROV, Christian LEBORGNE, Sean ARMOUR
Filed: 7 Apr 21
Utility
Compositions and Methods for Increasing or Enhancing Transduction of Gene Therapy Vectors and for Removing or Reducing Immunoglobulins
29 Jul 21
Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo.
Sébastien LACROIX-DESMAZES, Federico MINGOZZI, Jordan DIMITROV, Christian LEBORGNE, Sean ARMOUR
Filed: 17 Jul 17
Utility
Codon-optimized Acid Alpha-glucosidase Expression Cassettes and Methods of Using Same
22 Jul 21
The invention provides nucleic acids encoding acid a-glucosidase (GAA).
Xavier ANGUELA, Sean ARMOUR, Jayme NORDIN
Filed: 15 May 19
Utility
Aav Vector Column Purification Methods
18 Mar 21
Described and provided herein are purification, production and manufacturing methods for recombinant adeno-associated viral (rAAV) vector particles.
Younghoon OH, Guang QU
Filed: 29 Jun 18
Utility
COLUMN-BASED FULLY SCALABLE rAAV MANUFACTURING PROCESS
23 Sep 20
In accordance with the invention, provided herein are methods for purifying recombinant adeno-associated (rAAV) vector particles.
Guang QU, Younghoon OH, Lin LU, John Fraser WRIGHT
Filed: 30 Mar 17
Utility
Factor VIII (Fviii) Gene Therapy Methods
29 Jul 20
Methods of using vvectors comprising nucleic acid and nucleic acid variants encoding FVIII protein are disclosed.
Xavier ANGUELA
Filed: 31 Jul 18
Utility
ENHANCING AGENTS FOR IMPROVED CELL TRANSFECTION AND/OR rAAV VECTOR PRODUCTION
27 May 20
Provided are compositions and methods of transducing/transfecting cells with a molecule, such as a nucleic acid (e.g., plasmid), at high efficiency.
Guang QU, Lin LU, Jesusa JOSUE-ALMQVIST, John Fraser WRIGHT
Filed: 5 Jun 18