Abstract: Aspects of the disclosure relate to a nucleic acid comprising a heterologous nucleic acid insert flanked by interrupted self-complementary sequences, wherein one self-complementary sequence is interrupted by a cross-arm sequence forming two opposing, lengthwise-symmetric stem-loops, and wherein the other of the self-complementary sequences is interrupted by a truncated cross-arm sequence. Methods of delivering the nucleic acid to a cell are also provided.
Type:
Application
Filed:
June 1, 2021
Publication date:
November 18, 2021
Applicants:
University of Massachusetts, Voyager Therapeutics, Inc.
Abstract: The present disclosure relates to compositions, methods, and processes for the design, preparation, manufacture, use, and/or formulation of adeno-associated virus (AAV) particles for improved biodistribution and/or expression to particular regions of the central nervous system (CNS).
Abstract: The present disclosure relates to methods, formulations and devices for the delivery and therapeutic administration of polynucleotides encoding AADC. The present disclosure relates to methods, formulations and devices for the delivery and therapeutic administration of AAV vectors which include polynucleotides encoding AADC. The present disclosure relates to methods, formulations and devices for the delivery and therapeutic administration of polynucleotides encoding AADC in the treatment of neurological diseases, disorders and conditions, including Parkinson's Disease.
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:
August 14, 2019
Date of Patent:
June 8, 2021
Assignee:
Voyager Therapeutics, Inc.
Inventors:
Robert Kotin, Adrian Philip Kells, Bernard Ravina
Abstract: The present invention relates to improved methods and assays for the detection of AAV neutralizing antibodies in sera. The present invention provides safe, sensitive and high throughput neutralization assays for antibody detection.
Type:
Grant
Filed:
December 2, 2016
Date of Patent:
April 20, 2021
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Tatiana I. Novobrantseva, Dinah Wen-Yee Sah
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 28, 2020
Date of Patent:
February 16, 2021
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 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:
November 13, 2015
Date of Patent:
March 24, 2020
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Dinah Wen-Yee Sah, Jinzhao Hou, Mathieu E. Nonnenmacher, Pengcheng Zhou, Markus Hossbach, Jochen Deckert
Abstract: The invention 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:
November 5, 2015
Date of Patent:
July 2, 2019
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Robert Kotin, Adrian Philip Kells, Bernard Ravina
Abstract: The invention relates to compositions and methods for the preparation, administration, manufacture and therapeutic use of viral particles for the treatment of CNS disorders.
Type:
Application
Filed:
October 28, 2016
Publication date:
February 21, 2019
Applicant:
Voyager Therapeutics, Inc.
Inventors:
Dinah Wen-Yee SAH, Jinzhao HOU, Martin GOULET, Adrian Philip KELLS, Pengcheng ZHOU, Gregory Robert STEWART
Abstract: The present invention relates to improved methods and assays for the detection of AAV neutralizing antibodies in sera. The present invention provides safe, sensitive and high throughput neutralization assays for antibody detection.
Type:
Application
Filed:
December 2, 2016
Publication date:
December 13, 2018
Applicant:
Voyager Therapeutics, Inc.
Inventors:
Tatiana I. NOVOBRANTSEVA, Dinah Wen-Yee SAH