90 patents
Utility
Compositions and Methods for the Treatment of Tauopathy
4 Jan 24
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
Todd Carter, Wencheng Liu, Jerrah Holth, Jinzhao Hou, Yanqun Shu, Giridhar Murlidharan, Xiao-Qin Ren
Filed: 3 Nov 21
Utility
AAV capsids with increased tropism to brain tissue
2 Jan 24
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
Mathieu E. Nonnenmacher, Jinzhao Hou, Wei Wang, Matthew Child, Shaoyong Li
Filed: 26 Oct 21
Utility
Compositions and Methods of Treating Huntington's Disease
14 Dec 23
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
Jinzhao Hou, Xin Wang, Dinah Wen-Yee Sah, Pengcheng Zhou
Filed: 19 Oct 22
Utility
Compositions and Methods of Treating Amyotrophic Lateral Sclerosis (Als)
19 Oct 23
The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.
Dinah Wen-Yee SAH, Jinzhao HOU, Mathieu E. NONNENMACHER, Pengcheng ZHOU, Markus HOSSBACH, Jochen DECKERT
Filed: 20 Dec 22
Utility
Treatment of Amyotrophic Lateral Sclerosis (Als)
12 Oct 23
The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
Dinah Wen-Yee Sah, Qingmin CHEN, Joan R. COATES, Holger PATZKE, Jinzhao HOU
Filed: 4 Aug 22
Utility
Compositions and Methods for Delivery of Aav
28 Sep 23
The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
Dinah Wen-Yee SAH, Holger PATZKE, Jinzhao HOU, Mathieu E. NONNENMACHER, Martin GOULET, Todd CARTER
Filed: 20 Oct 22
Utility
Cell Culture Medium for Use In Producing Gene Therapy Products In Bioreactors
21 Sep 23
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions, and formulations, including recombinant adeno-associated viruses (rAAV).
Krishanu MATHUR, Peter SLADE, Anastasia NEUMAN, Charles LY, Dongyang YI, Zeynep GUILLEMIN
Filed: 6 Aug 21
Utility
Compositions and Methods of Treating Amyotrophic Lateral Sclerosis (Als)
21 Sep 23
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).
Dinah Wen-Yee SAH, Qingmin CHEN, Jinzhao HOU
Filed: 23 Feb 23
Utility
AADC polynucleotides for the treatment of Parkinson's disease
19 Sep 23
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
Maria Scheel, Bernard Ravina
Filed: 26 Jul 19
Utility
Compositions and Methods for the Treatment of Niemann-pick Type C1 Disease
14 Sep 23
The disclosure provides compositions and methods for altering, e.g., enhancing, the expression of NPC1 and/or NPC2 proteins, whether in vitro and/or in vivo.
Heather Yonutas, Jeffrey Brown, Jinzhao Hou, Yanqun Shu, Elisabeth Knoll, Priyantha Herath, Brett Hoffman
Filed: 26 Jul 21
Utility
Compositions and methods of treating Huntington's disease
12 Sep 23
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
Dinah Wen-Yee Sah, Fen Chen, Pengcheng Zhou, Xin Wang, Yanqun Shu, Jinzhao Hou, Jochen Deckert, Markus Hossbach
Filed: 4 May 18
Utility
Methods and Systems for Producing Aav Particles
3 Aug 23
The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof).
Krishanu Mathur, Andrade Hendricks, Matthew Luther, Jacob J. Cardinal, Daniel S. Hurwit, Lori B. Karpes, Christopher J. Morrison
Filed: 28 Jan 21
Utility
Compositions and Methods for the Treatment of Neurological Disorders Related to Glucosylceramidase Beta Deficiency
20 Jul 23
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo.
Giridhar MURLIDHARAN, Jeffrey BROWN, Elisabeth KNOLL, Yanqun SHU, Kelly BALES, Jinzhao HOU, Adewale ADELUYI, Brett HOFFMAN, Smita JAGTAP
Filed: 26 Jul 21
Utility
Compositions and methods for the production of scAAV
11 Jul 23
The present invention is directed to viral vectors and methods of their production and use.
James McLaughlin, Robert Kotin
Filed: 11 Dec 15
Utility
Redirection of Tropism of Aav Capsids
29 Jun 23
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts tor enhanced tropism to a target tissue.
Mathieu E. NONNENMACHER, Jinzhao HOU, Wei WANG, Matthew CHILD, Shaoyong LI
Filed: 31 Mar 21
Utility
Redirection of Tropism of Aav Capsids
27 Apr 23
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
Mathieu E. NONNENMACHER, Jinzhao HOU, Wei WANG, Matthew CHILD
Filed: 31 Mar 21
Utility
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
14 Mar 23
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).
Dinah Wen-Yee Sah, Qingmin Chen, Jinzhao Hou
Filed: 4 May 18
Utility
VECTORIZED ANTIBODIES (vAb) AND USES THEREOF
9 Mar 23
The present disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
Jinzhao Hou, Yanqun Shu, Todd Carter, Dinah Wen-Yee Sah, Po-Jen Yen, Donna T. Ward, Johanna L. Crimins
Filed: 29 Apr 20
Utility
Rescue of Central and Peripheral Neurological Phenotype of Friedreich's Ataxia by Intravenous Delivery
9 Feb 23
Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
Dinah Wen-Yee Sah, Martin Goulet, Holger Patzke, Yanqun Shu, Jinzhao Hou, Hélène Puccio
Filed: 13 Sep 22
Utility
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
3 Jan 23
The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.
Dinah Wen-Yee Sah, Jinzhao Hou, Mathieu E. Nonnenmacher, Pengcheng Zhou, Markus Hossbach, Jochen Deckert
Filed: 6 Jan 21