David M Souza

US Patent:

7312324, Dec 25, 2007

Filed:

May 6, 2002

Appl. No.:

10/139763

Inventors:

David W. Souza - Waltham MA, US
Donna Armentano - Belmont MA, US
Samuel C. Wadsworth - Shrewsbury MA, US

Assignee:

Genzyme Corporation - Cambridge MA

International Classification:

C07H 21/04
C07H 21/02
A01N 63/00
A01N 65/00
A01N 43/04
A01N 31/70

US Classification:

536 241, 514 44, 424 931, 536 231

Abstract:

The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, α-1microglobulin or aldolase genes in single copies or in multimerized form linked to elements derived from the cytomegalovirus intermediate early (CMV), α-1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the β-fibrinogen gene.

US Patent:

20030100526, May 29, 2003

Filed:

May 24, 2002

Appl. No.:

10/156604

Inventors:

David Souza - Waltham MA, US
Donna Armentano - Belmont MA, US

International Classification:

A61K048/00
C12N015/85

US Classification:

514/044000, 435/455000, 435/320100

Abstract:

The invention is directed to novel combinations of muscle-specific enhancer and promoter elements useful for achieving persistent expression in the muscle or myocytes. The muscle-specific promoter elements are derived from a muscle creatine kinase promoter, a troponin I promoter, a skeletal alpha-actin promoter, or a desmin promoter. The muscle-specific enhancer elements are derived from either troponin I internal regulatory elements, muscle creatine kinase enhancers, or desmin enhancers.

US Patent:

20080070297, Mar 20, 2008

Filed:

Nov 13, 2007

Appl. No.:

11/938906

Inventors:

David SOUZA - Waltham MA, US
Donna ARMENTANO - Bedford MA, US
Samuel WADSWORTH - Shrewsbury MA, US

Assignee:

GENZYME CORPORATION - Framingham MA

International Classification:

C12N 15/861
C12N 15/11

US Classification:

435320100, 536024100

Abstract:

The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, α-1microglobulin or aldolase genes in single copies or in multimerized from linked to elements derived from the cytomegalovirus intermediate early (CMV), α-1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the β-fibrinogen gene.

US Patent:

20110212529, Sep 1, 2011

Filed:

Mar 12, 2007

Appl. No.:

11/716899

Inventors:

David Souza - Waltham MA, US
Donna Armentano - Belmont MA, US

International Classification:

C12N 15/85
C07H 21/04
C12N 1/00
C12N 5/10

US Classification:

435455, 4353201, 536 241, 435243, 435325

Abstract:

The invention is directed to novel combinations of muscle-specific enhancers and promoter elements useful for achieving persistent expression in the muscle or myocyctes. The muscle-specific promoter elements are derived from a muscle creatine kinase promoter, a troponin I promoter, a skeletal alpha-actin promoter, or a desmin promoter. The muscle-specific enhancer elements are derived from either troponin I internal regulatory elements, muscle creatine kinase enhancers, or desmin enhancers.

US Patent:

20190048362, Feb 14, 2019

Filed:

Apr 7, 2016

Appl. No.:

15/565020

Inventors:

- Cambridge MA, US
David SOUZA - Waltham MA, US
Karen VINCENT - Carlisle MA, US

International Classification:

C12N 15/86
A61P 11/00
A61K 38/17
A61P 21/00

Abstract:

Provided herein are methods for producing an adeno-associated virus (AAV) particle containing an oversized recombinant AAV genome (e.g., greater than 4.7 kb). In some aspects, the invention provides AAV particles and AAV vectors comprising oversized rAAV genomes. Producer cell lines to produce AAV particles comprising oversized genomes are also provided.