45 patents
Utility
Methods and Compositions for Treating Epilepsy
18 Jan 24
The present disclosure provides methods and compositions relating to gene therapy for treating epilepsy, such as a temporal lobe epilepsy, in a subject in need thereof by targeting Grik2 mRNA.
Valérie CREPEL, Christophe MULLE, Céline BOILEAU, Séverine DEFORGES, Olivier DANOS, Andrew MERCER, Richard PORTER, April R. TEPE
Filed: 9 Jul 21
Utility
Formulations for Suprachoroidal Administration Such As Gel Formulations
28 Dec 23
Provided herein are pharmaceutical compositions for administration to a suprachoroidal space of an eye of a subject.
Jared Bee, Tristan James Marshall, Sherri Van Everen, Stephen Joseph Pakola, Nicholas Alexander Piers Sascha Buss, Anthony Ray O'Berry, Jesse I. Yoo, Ewa Budzynski
Filed: 6 Oct 21
Utility
Vectorized Factor XII Antibodies and Administration Thereof
7 Dec 23
Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody that binds to factor XII to a human subject diagnosed with a disease or condition indicated for treatment with an anti-factor XII antibody.
Joseph Bruder, Ye Liu
Filed: 29 Oct 21
Utility
Vectorized Anti-tnf-alpha Antibodies for Ocular Indications
7 Dec 23
Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody, or an antigen binding fragment thereof, that binds to TNF-α, IL6 or IL6-R to a human subject for treatment of an ocular indication, particularly non-infectious uveitis.
Xu Wang, Devin McDougald, Joseph Bruder, Ye Liu, Olivier Danos, Wei-Hua Lee, Chunping Qiao, Ewa Budzynski, Mikayla Higgins, Mi Shi
Filed: 28 Oct 21
Utility
Adeno-associated Viruses for Ocular Delivery of Gene Therapy
30 Nov 23
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins that have a tropism for ocular tissue.
Joseph Bruder, Xu Wang, Wei-Hua Lee, Elad Firnberg, Samantha Yost, Andrew Mercer, Ye Liu, Olivier Danos, April R. Tepe
Filed: 7 Oct 21
Utility
Formulations for Suprachoroidal Administration Such As Formulations with Aggregate Formation
23 Nov 23
Provided herein are pharmaceutical compositions for administration to a suprachoroidal space of an eye of a subject.
Jared Bee, Tristan James Marshall, Sherri Van Everen, Stephen Joseph Pakola, Ewa Budzynski
Filed: 6 Oct 21
Utility
Recombinant Adeno-associated Viruses for CNS or Muscle Delivery
23 Nov 23
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences and/or amino acid substitutions that confer and/or enhance desired properties, particularly increased transduction in CNS or muscle cells relative to a rAAV having a reference capsid.
Olivier Danos, Samantha Yost, Andrew Mercer, Ye Liu, Joseph Bruder, Subha Karumuthil-Melethil, Elad Firnberg, Randolph Qian, April R. Tepe, Jennifer M. Egley
Filed: 7 Oct 21
Utility
Gene Therapy for Ocular Manifestations of CLN2 Disease
16 Nov 23
Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors.
Christina Maria Ohnsman, Stephen Joseph Pakola, Sherri Van Everen, Paulo Falabella, Alexander Marston Bailey, Nicholas Alexander Piers Sascha Buss, Kwi Hye Kim
Filed: 6 Oct 21
Utility
Compounds for Use In the Treatment of Epilepsy
12 Oct 23
The present disclosure relates to gene therapy targeting GluK2 subunit that can be used to inhibit epileptiform discharges.
Céline BOILEAU, Valérie CREPEL, Séverine DEFORGES, Julie MASANTE, Christophe NULLE, Angélique PERET, Olivier DANOS, Andrew MERCER
Filed: 9 Jul 21
Utility
Microdystrophin Gene Therapy Constructs and Uses Thereof
31 Aug 23
Provided is an invention based, in part, on novel gene constructs that encode a microdystrophin protein for use in gene therapy.
Chunping Qiao, Devin McDougald, Ye Liu, Olivier Danos
Filed: 27 Nov 20
Utility
Adeno-associated Virus for Therapeutic Delivery to Central Nervous System
20 Jul 23
A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.
R. Scott McIvor, Lalitha R. Belur, Karen Kozarsky
Filed: 5 Jan 23
Utility
Methods and Compositions for Treating Epilepsy
20 Jul 23
The present disclosure provides methods and compositions relating to gene therapy for treating epilepsy, such as a temporal lobe epilepsy, in a subject in need thereof by targeting Grik2 mRNA.
Valérie CREPEL, Christophe MULLE, Céline BOILEAU, Séverine DEFORGES, Olivier DANOS, Andrew MERCER, Richard PORTER, April R. TEPE
Filed: 13 Jan 23
Utility
Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
28 Mar 23
Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
Stephen Yoo, Rickey Robert Reinhardt, Curran Matthew Simpson, Zhuchun Wu
Filed: 13 Apr 18
Utility
Treatment of Mucopolysaccharidosis II with Recombinant Human IDURONATE-2-SULFATASE (Ids) Produced by Human Neural or Glial Cells
23 Mar 23
Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
Stephen Joseph PAKOLA, Paulo FALABELLA, Marie-Laure NEVORET
Filed: 28 Jan 21
Utility
Treatment of Mucopolysaccharidosis Iva
2 Mar 23
Provided herein are gene therapy methods for the treatment of mucopolysaccharidosis type IV A (MPS IV A) involving the use of recombinant adeno-associated viruses (rAAVs) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject diagnosed with MPS IVA.
Devin MCDOUGALD, Subha KARUMUTHIL-MELETHIL
Filed: 28 Jan 21
Utility
Treatment of Ocular Diseases with Fully-Human Post-Translationally Modified Anti-VEGF Fab
23 Feb 23
Compositions and methods are described for the delivery of a fully human post-translationally modified (HuPTM) monoclonal antibody (“mAb”) or the antigen-binding fragment of a mAb against human vascular endothelial growth factor (“hVEGF”)—such as, e.g., a fully human-glycosylated (HuGly) anti-hVEGF antigen-binding fragment—to the retina/vitreal humour in the eye(s) of human subjects diagnosed with ocular diseases caused by increased neovascularization, for example, neovascular age-related macular degeneration (“nAMD”), also known as “wet” age-related macular degeneration (“WAMD”), age-related macular degeneration (“AMD”), and diabetic retinopathy.
Curran Matthew Simpson, Stephen Yoo, Karen Fran Kozarsky, Rickey Robert Reinhardt, Laura A. Coruzzi
Filed: 25 Mar 22
Utility
Treatment of Mucopolysaccharidosis I with Fully-human Glycosylated Human Alpha-l-iduronidase (Idua)
23 Feb 23
Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) α-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).
Stephen Joseph PAKOLA, Paulo FALABELLA, Marie-Laure NEVORET
Filed: 20 Jan 21
Utility
Engineered Nucleic Acid Regulatory Element and Methods of Uses Thereof
9 Feb 23
The present invention relates to nucleic acid expression cassettes that are engineered to enhance gene expression.
Chunping Qiao, Devin McDougald
Filed: 24 Jul 20
Utility
Methods for the Treatment of Epilepsy
9 Feb 23
The present disclosure relates to gene therapy targeting GluK2 subunit that can be used to inhibit epileptiform discharges.
Valérie CREPEL, Christophe Mulle, Céline Boileau, Julie Masante, Séverine Deforges, Angélique Peret, Olivier Danos, Andrew Mercer
Filed: 30 Jun 22
Utility
Treatment of Diabetic Retinopathy with Fully-human Post-translationally Modified Anti-vegf Fab
8 Sep 22
Compositions and methods are described for the delivery of a fully human post-translaionally modified (HuPTM) monoclonal antibody (“mAh”) or the antigen-binding fragment of a mAh against human vascular endothelial growth factor (“hVEGF”)—such as, e.g., a fully human-glycosylated (HuGly) anti-hVEGF antigen-binding fragment—to the retina/vitreal humour in the eye(s) of human subjects diagnosed with diabetic retinopathy.
Stephen Joseph Pakola, Sherri Van Everen, Jesse I. Yoo, Samir Maganbhai Patel, Avanti Arvind Ghanekar, Anthony Ray O'Berry, Kim Rees Irwin-Pack, Darin Thomas Curtiss
Filed: 25 Aug 20