公开(公告)号：WO2023086928A2

公开(公告)日：2023-05-19

申请号：PCT/US2022/079701

申请日：2022-11-11

Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  AMICUS THERAPEUTICS, INC.

Inventor： HORDEAUX, Juliette ,  WILSON, James, M. ,  RASSOULI-TAYLOR, Leida ,  DO, Hung ,  TUSKE, Steven

IPC: C12N15/861 ,  A61K35/761 ,  A61K48/00 ,  A61K38/46 ,  C12N9/14 ,  C12N15/86 ,  A61K35/76

Abstract: Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5' inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional hSGSH, optionally comprising stabilizing amino acid changes, a regulatory sequence which direct expression of hSGSH in a target cell, and an AAV 3' ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIA.

公开(公告)号：WO2023028035A1

公开(公告)日：2023-03-02

申请号：PCT/US2022/041176

申请日：2022-08-23

Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED

Inventor： MCKENNA, Robert ,  MIETZSCH, Mario ,  HULL, Joshua

IPC: A61K35/761 ,  C12N15/861 ,  A61P35/00 ,  C07K14/005

Abstract: The disclosure relates to lipid enveloped rAAV particles, compositions comprising the same, and methods of treating a disease comprising the administration of such lipid enveloped rAAV peptides and compositions. Methods of preparing populations of lipid enveloped rAAV particles are also disclosed. Wherein the AAV can be AAV serotype 1 (AAV1), and the compositoin includes a pharmacuetically-accpetable carrier or excipient.

公开(公告)号：WO2023023256A1

公开(公告)日：2023-02-23

申请号：PCT/US2022/040766

申请日：2022-08-18

Applicant: OCULOGENEX INC.

Inventor： RAMKUMAR, Hema Lakshmi

IPC: A61P27/02 ,  A61K35/761 ,  C12N15/113 ,  C12N15/86

Abstract: The present invention relates generally to gene therapy for treating ailments that can affect vision such as retinal degeneration, retinal dystrophy, macular degeneration, macular dystrophy, ischemic retinopathies, and glaucoma. Embodiments include systems and treatments that use AAV-mediated gene therapy or non AAV- mediated DNA, mRNA, or protein therapy to target all retinal cells. An AAV virion can be introduced (e.g., via intravitreal or subretinal injection) into an eye of an individual, or systemically, to express a heterologous gene product such as BMI1 protein (B lymphoma Mo-MLV insertion region 1 homolog).

公开(公告)号：WO2022272296A2

公开(公告)日：2022-12-29

申请号：PCT/US2022/073136

申请日：2022-06-24

Applicant: HOMOLOGY MEDICINES, INC.

Inventor： VAN LIESHOUT, Laura ,  STANVICK, Marissa

IPC: C12N15/09 ,  A61K35/761 ,  A61K48/00 ,  C07K16/18 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2800/40 ,  C12N5/0601 ,  C12N9/0071 ,  C12N9/16 ,  C12Y114/16001 ,  C12Y301/06013

Abstract: Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.

公开(公告)号：WO2022234276A1

公开(公告)日：2022-11-10

申请号：PCT/GB2022/051137

申请日：2022-05-04

Applicant: SPYBIOTECH LIMITED

Inventor： DICKS, Matthew ,  BISWAS, Sumi

IPC: A61K35/761 ,  A61K39/00 ,  A61K39/015 ,  C12N15/861 ,  A61K39/145 ,  A61K39/215 ,  A61K39/385 ,  C07K14/005 ,  C07K14/05 ,  A61K2039/53 ,  A61K39/12 ,  C12N15/86 ,  C12N2710/10343 ,  C12N2710/16134 ,  C12N2760/16122 ,  C12N2760/16134 ,  C12N2770/20022 ,  C12N2770/20034

Abstract: The present disclosure relates to adenoviral vector comprising a transgene encoding an antigen having a T cell epitope. The vector capsid comprising a modified capsid protein having a first peptide partner. A second peptide partner is attached to the first peptide partner to provide a covalently linked peptide binding pair. The second peptide partner also being attached to an antigen, the antigen having a B cell epitope. In a preferred embodiment the transgene encoding one antigen is in the lumen of the viral capsid and the second peptide attached to the second antigen is displayed on the surface of the viral capsid. Further aspects of the invention relate to vaccines comprising said vector, its use in therapy and methods of manufacture and treatment thereof.

公开(公告)号：WO2022230924A1

公开(公告)日：2022-11-03

申请号：PCT/JP2022/019038

申请日：2022-04-27

Applicant: 国立大学法人大阪大学 ,  エディットフォース株式会社

Inventor： 中森　雅之 ,  八木　祐介 ,  今井　崇喜 ,  沖井　英里香 ,  玉井　喬之 ,  二宮　理紗

IPC: A61K48/00 ,  A61P21/00 ,  C07K16/18 ,  C12N5/10 ,  C12N7/00 ,  C12N1/15 ,  C12N1/19 ,  C12N1/21 ,  C12N15/12 ,  C12N15/86 ,  A61K38/16 ,  A61K35/76 ,  A61K35/761 ,  A61K35/763 ,  C07K14/47

Abstract: 【課題】　筋強直性ジストロフィー１型（ＤＭ１）の治療に有効な医薬組成物または方法を提供する。 【解決手段】　改変されたＰＰＲタンパク質を利用したＤＭ１の治療薬または治療方法を提供する。

公开(公告)号：WO2022208056A1

公开(公告)日：2022-10-06

申请号：PCT/GB2022/050745

申请日：2022-03-24

Applicant: UNIVERSITY COLLEGE CARDIFF CONSULTANTS LIMITED

Inventor： PARKER, Alan ,  DAVIES, James ,  BATES, Emily

IPC: A61K35/761 ,  C12N15/86 ,  C07K14/005 ,  C12N7/00 ,  C12N2710/10321 ,  C12N2710/10322 ,  C12N2710/10332 ,  C12N2710/10343 ,  C12N2710/10345

Abstract: The invention concerns a modified low seroprevalence adenovirus; a pharmaceutical composition comprising same; and a method of treating cancer using same.

公开(公告)号：WO2022132301A1

公开(公告)日：2022-06-23

申请号：PCT/US2021/055296

申请日：2021-10-15

Applicant: UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： GAO, Shou-Jiang

IPC: A61K35/76 ,  A61K35/761 ,  A61K39/00 ,  A61K39/235 ,  A61K39/395 ,  A61K48/00

Abstract: The present subject matter relates to the use of one or more inhibitors to treat a disease, e.g., cancer, in a subject. It is based, at least in part, on the discovery that protein kinase B (AKT) and ring finger protein 167 (RNF 167)-mediated CASTOR1 degradation activates the mammalian target of rapamycin complex 1 (mTORC1) independent of arginine and promotes cancer progression. Accordingly, the presently disclosed subject matter provides for compositions, methods, and kits for treating a subj ect using an RNF 167 inhibitor, an inhibitor that reduces phosphorylation of CASTOR1 at S14, ubiquitination and/or degradation of CASTOR1, or a combination thereof.

公开(公告)号：WO2022109503A1

公开(公告)日：2022-05-27

申请号：PCT/US2021/060669

申请日：2021-11-23

Applicant: DEVACELL, INC.

Inventor： ORTAC, Inanc

IPC: A61K35/761 ,  C07K14/705 ,  C12N15/86 ,  C12N7/04 ,  A61K35/768

Abstract: Provided herein are recombinant viruses and artificially coated delivery systems, and methods of use.

公开(公告)号：WO2022053587A1

公开(公告)日：2022-03-17

申请号：PCT/EP2021/074868

申请日：2021-09-09

Applicant: SORBONNE UNIVERSITE ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  ECOLE SUPERIEURE DE PHYSIQUE ET DE CHIMIE INDUSTRIELLES DE LA VILLE DE PARIS ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE)

Inventor： PICAUD, Serge ,  CADONI, Sara ,  SAHEL, José-Alain ,  TANTER, Mickaël ,  DEMENE, Charlie

IPC: A61N7/00 ,  A61K35/761 ,  C12N15/86 ,  A01K67/027

Abstract: The present invention relates to devices and methods for reversibly stimulating neuronal cells in a subject. Devices of the present invention comprise a module for generating ultrasounds at 4 MHz or more to stimulate neuronal cells expressing mechanosensitive channels with said ultrasounds. Methods of the present invention comprise expressing mechanosensitive channels into neuronal cells and exposing said cells to ultrasounds at 4 MHz or more. The present invention also relates to the use of said methods or devices for visual restoration in a subject.