24 patents
Utility
Compositions and Methods for Reducing Ocular Neovascularization
12 Oct 23
The present disclosure provides pharmaceutical compositions and methods thereof for the prevention or treatment of ocular neovascularization, such as AMD, in a subject, by administering to the subject a pharmaceutical composition comprising a rAAV vector having a nucleic acid sequence that encodes an anti-VEGF agent.
Mark BLUMENKRANZ, Mehdi GASMI
Filed: 12 Oct 22
Utility
Compositions and methods for enhanced gene expression
3 Oct 23
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.
Annahita Keravala
Filed: 16 Mar 18
Utility
Compositions and Methods for Enhanced Gene Expression
17 Aug 23
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.
Annahita Keravala
Filed: 26 Apr 23
Utility
Intravitreal Dosing for Delivery of Polynucleotides to Retinal Cones
2 Mar 23
Provided is the intravitreal dosing of recombinant adeno-associated virus (rAAV)-based gene therapies for the treatment of color vision deficiencies such as Blue Cone Monochromacy (BCM) and Red-Green Color Blindness.
Claire GELFMAN, Diana CEPEDA, Ruslan GRISHANIN, Kristina BENDER, Pallavi SHARMA, Julio NIEVES
Filed: 25 Mar 22
Utility
Variant AAV capsids for intravitreal delivery
29 Nov 22
Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins.
Annahita Keravala, Diana Cepeda, Mehdi Gasmi
Filed: 24 Apr 20
Utility
Mutant viral capsid libraries and related systems and methods
30 Aug 22
Provided are mutant viral capsid cell libraries, individual cells of such libraries, systems, vectors, and methods for generating the cell libraries, and methods of use thereof to screen for mutant viral capsids with desired characteristics.
Christopher Chavez, Mehdi Gasmi, Annahita Keravala, Thomas W. Chalberg
Filed: 23 Jan 20
Utility
Treatment of Amd Using AAV2 Variant with Aflibercept
25 Aug 22
The present disclosure provides pharmaceutical compositions and methods thereof for the treatment of age-related macular degeneration (AMD) in a subject by administering intravitreally a pharmaceutical composition adapted for gene therapy, comprising a vector having a nucleic acid sequence that encodes aflibercept.
Mark BLUMENKRANZ, Mehdi GASMI
Filed: 20 Sep 21
Utility
Compositions and Methods for Enhanced Gene Expression In Cone Cells
18 Aug 22
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in cone cells.
Thomas W. CHALBERG, JR., Jay NEITZ, Maureen NEITZ
Filed: 20 Dec 21
Utility
Modified Aav Capsids and Uses Thereof
21 Jul 22
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection.
Annahita KERAVALA
Filed: 29 Oct 21
Utility
Anti-drug Antibody Assay
16 Jun 22
Provided is an immunoassay for detection of anti-drug antibodies (ADAs), such as anti-C1-Inhibitor antibodies (C1INH-ADA in a test sample.
Judith GREENGARD, Mark RENZ, Valerie THEOBALD
Filed: 3 Mar 22
Utility
Compositions and methods for enhanced gene expression
7 Jun 22
The present disclosure provides polynucleotide cassettes, expression vectors, and methods for the expression of a gene in mammalian cells.
Annahita Keravala
Filed: 20 Aug 20
Utility
Treatment of Ocular Neovascularization Using Anti-vegf Proteins
14 Apr 22
The present disclosure provides compositions and methods for the prevention or treatment of ocular neovascularization, such as AMD, in a human subject, by administering subretinally a pharmaceutical composition comprising a pharmaceutically effective amount of a vector comprising a nucleic acid encoding soluble Fms-related tyrosine kinase-1 (sFlt-1) protein to the human subject.
Ian J. CONSTABLE, P. Elizabeth RAKOCZY, Chooi-May LAI, Thomas W. CHALBERG, Jr.
Filed: 24 May 21
Utility
Compositions and methods for enhanced gene expression in cone cells
15 Feb 22
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in cone cells.
Thomas W. Chalberg, Jr., Jay Neitz, Maureen Neitz
Filed: 18 May 18
Utility
Compositions and Methods for Intravitreal Delivery of Polynucleotides to Retinal Cones
16 Dec 21
Methods and compositions are provided for intravitreally delivering a polynucleotide to cone photoreceptors.
Thomas W. CHALBERG, JR., Jay NEITZ, Maureen NEITZ
Filed: 5 May 21
Utility
Modified AAV capsids and uses thereof
7 Dec 21
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection.
Annahita Keravala
Filed: 19 Oct 17
Utility
Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
1 Jun 21
Methods and compositions are provided for intravitreally delivering a polynucleotide to cone photoreceptors.
Thomas W. Chalberg, Jr., Jay Neitz, Maureen Neitz
Filed: 2 Mar 16
Utility
Modified Aav Capsids and Uses Thereof
6 May 21
The present disclosure provides modified adeno-associated virus (AAV) virions with altered capsid proteins, where the modified AAV virions exhibit greater infectivity of retinal cells when administered to the eye or greater infectivity of liver cells when administered intravenously.
Annahita KERAVALA
Filed: 28 Feb 18
Utility
Methods of Treating Ocular Neovascular Diseases Using AAV2 Variants Encoding Aflibercept
8 Apr 21
Provided are methods for treating an ocular neovascular disease in an individual, comprising administering a unit dose of recombinant adeno-associated virus (rAAV) particles to an eye of the individual, wherein the rAAV particles comprise: a) a nucleic acid encoding a polypeptide comprising an amino acid sequence with at least about 95% identity to the amino acid sequence of SEQ ID NO: 35 and flanked by AAV2 inverted terminal repeats (ITRs), and b) an AAV2 capsid protein comprising an amino acid sequence LGETTRP (SEQ ID NO: 14) inserted between positions 587 and 588 of the capsid protein, wherein the amino acid residue numbering corresponds to an AAV2 VP1 capsid protein.
Mehdi GASMI, Szilard KISS, Aaron OSBORNE, Adam TURPCU
Filed: 10 Sep 20
Utility
Compositions and Methods for Enhanced Gene Expression
11 Feb 21
The present disclosure provides polynucleotide cassettes, expression vectors, and methods for the expression of a gene in mammalian cells.
Annahita KERAVALA
Filed: 20 Aug 20
Utility
Variant Aav Capsids for Intravitreal Delivery
28 Oct 20
Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins.
Annahita KERAVALA, Diana CEPEDA, Mehdi GASMI
Filed: 23 Apr 20