Abstract: The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
Type:
Grant
Filed:
June 5, 2024
Date of Patent:
May 6, 2025
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Wencheng Liu, Todd Carter, Ishan Sanjeev Shah, Christopher Joseph Murray, James Bernard McClory, Dinah Wen-Yee Sah
Abstract: The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.
Abstract: Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
Type:
Grant
Filed:
September 13, 2022
Date of Patent:
November 19, 2024
Assignees:
VOYAGER THERAPEUTICS, INC., INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), UNIVERSITÉ DE STRASBOURG
Abstract: The present disclosure presents improved analytical tools, systems and methods related to AADC viral vectors, including AADC potency assays for measuring and analyzing AADC expression potency (i.e. enzymatic activity) related to AADC vectors such as adeno-associated virus (AAV) AADC vectors.
Type:
Grant
Filed:
April 26, 2019
Date of Patent:
October 29, 2024
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Lauren M. Drouin, Patrick Starremans, Eric D. Horowitz, Carlo Ciatto, Joseph Mombeleur
Abstract: 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.
Type:
Grant
Filed:
December 20, 2022
Date of Patent:
October 22, 2024
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Dinah Wen-Yee Sah, Jinzhao Hou, Mathieu E. Nonnenmacher, Pengcheng Zhou, Markus Hossbach, Jochen Deckert
Abstract: 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).
Type:
Grant
Filed:
August 4, 2022
Date of Patent:
October 15, 2024
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Dinah Wen-Yee Sah, Qingmin Chen, Joan R. Coates, Holger Patzke, Jinzhao Hou
Abstract: The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of non-structural AAV replication proteins, such as Rep78 and Rep52.
Type:
Grant
Filed:
June 13, 2019
Date of Patent:
August 13, 2024
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Luis Maranga, Sylvain Cecchini, David Dismuke
Abstract: 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.
Type:
Grant
Filed:
May 3, 2021
Date of Patent:
May 7, 2024
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Robert Kotin, Adrian Philip Kells, Bernard Ravina
Abstract: The present invention relates to small interfering RNA (siRNA) molecules against the HTT gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating Huntington's Disease (HD) using the siRNA molecules and AAV vectors.
Abstract: The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
Abstract: 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.
Type:
Grant
Filed:
October 26, 2021
Date of Patent:
January 2, 2024
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Mathieu E. Nonnenmacher, Jinzhao Hou, Wei Wang, Matthew Child, Shaoyong Li
Abstract: 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.
Abstract: The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
Abstract: 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.
Type:
Grant
Filed:
January 6, 2021
Date of Patent:
January 3, 2023
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Dinah Wen-Yee Sah, Jinzhao Hou, Mathieu E. Nonnenmacher, Pengcheng Zhou, Markus Hossbach, Jochen Deckert
Abstract: The invention 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 pol peptides, for the prevention and/or treatment of diseases and/or disorders.